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A Phase 2b Study To Evaluate The Efficacy And Safety Profile Of PF-06651600 And PF-06700841 In Active Non-segmental Vitiligo Subjects

Primary Purpose

Active Non-segmental Vitiligo

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

PF-06651600

placebo

PF06700841

narrow-band UVB phototherapy

About this trial

This is an interventional treatment trial for Active Non-segmental Vitiligo

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male or female subjects between 18-65 years of age, inclusive, at time of informed consent.
Must have moderate to severe active non-segmental vitiligo.

Exclusion Criteria:

History of human immunodeficiency virus (HIV) or positive HIV serology at screening,
Infected with hepatitis B or hepatitis C viruses.
Have evidence of active or latent or inadequately treated infection with Mycobacterium tuberculosis (TB)

Sites / Locations

Marvel Research, LLC
University of California, Irvine, Dermatology Clinical Research Center
Vitiligo and Pigmentation Institute Of Southern California
Dermatology Specialist, Inc.
Dermatology Specialists, Inc.
UC Davis Health
Brookside Dermatology Associates
New England Research Associates, LLC
New Horizon Research Center
Park Avenue Dermatology
ForCare Clinical Research
Advanced Skin and MOHS Surgery Center, c/o TrialSpark, Inc.
Chevy Chase Dermatology Center (TrialSpark, Inc.)
TrialSpark - Samantha Toerge, MD
TrialSpark - Ronald Shore, MD
Tobias & Battite, Inc.
Tufts Medical Center
UMass Memorial Medical Center, Hahnemann Campus
Investigational Drug Service Pharmacy
UMass Memorial Medical Center Ear Nose and Throat
University of Massachusetts Medical School
Henry Ford Hospital Department of Dermatology
University of Minnesota Department of Dermatology
The Dermatology Group, P.C.
Icahn School of Medicine at Mount Sinai
Upper West Side Dermatology c/o TrialSpark, Inc
MDCS: Medical Dermatology & Cosmetic Surgery (TrialSpark, Inc.)
South Nassau Dermatology
TrialSpark, Inc. - Russell W. Cohen, MD
PMG Research of Wilmington, LLC
Remington-Davis, Inc. Clinical Research
ForeFront Dermatology
Vital Prospects Clinical Research Institute, PC
University Physicians Group
University of Texas Southwestern Medical Center
Pickens Academic Tower
The University of Texas Health Science Center at Houston
The University of Texas MD Anderson Cancer Center
Virginia Clinical Research, Inc
Tamjidi Skin Institute (TrialSpark, Inc.)
The Skin Hospital
Premier Specialists Pty Ltd
Veracity Clinical Research Pty Ltd
Skin Health Institute
Sinclair Dermatology
The Royal Melbourne Hospital
The Royal Melbourne Hospital
Hôpital Erasme Dermatology
UZ Brussel - Dermatology
UZ Gent - Dermatology
Dr. Chih-ho Hong Medical Inc.
Enverus Medical Research
University of British Columbia
Wiseman Dermatology Research Inc.
CCA Medical Research
Guenther Research Inc.
Lynderm Research Inc.
DermEdge Research
North York Research Inc.
The Centre for Clinical Trials
The Centre for Dermatology
K.Papp Clinical Research
Innovaderm Research Inc.
Diex Research Sherbrooke Inc.
Centre de Recherche Dermatologique du Quebec metropolitain (CRDQ)
Centre de Recherche Saint-Louis
Centre de Recherche Saint-Louis
Fachklinik Bad Bentheim, Fachbereich Dermatologie und Allergologie, Dermatologische Ambulanz
Universitaetsklinikum Erlangen Hautklinik Studienambulanz
Universitätsklinikum Frankfurt, Klinik für Dermatologie, Venerologie und Allergologie
Universitaetsklinikum Schleswig-Holstein, Campus Luebeck CCIM
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz
Universitaetsklinikum Muenster
Istituti Fisioterapici Ospitalieri, Istituto di Ricovero e Cura a Carattere Scientifico,
Nagoya City University Hospital - Dermatology
Tohoku University Hospital
Nippon Medical School Hospital
Tokyo Medical University Hospital
Yamanashi Prefectural Central Hospital
Dongguk University Ilsan Hospital
The Catholic University of Korea, St. Vincent's Hospital
Ajou University Hospital
Inha University Hospital
Severance Hospital, Yonsei University Health System
Hospital de la Santa Creu i Sant Pau
Hospital Universitario Reina Sofia
Hospital Universitario Ramón y Cajal
Hospital Universitario La Paz
Hospital Universitari i Politecnic La Fe
Chang Gung Medical Foundation - Kaohsiung Chang Gung Memorial Hospital
National Cheng Kung University Hospital
National Taiwan University Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm 8

Arm 9

Arm 10

Arm 11

Arm 12

Arm Type

Experimental

Placebo Comparator

Experimental

No Intervention

Arm Label

Cohort 1

Cohort 2

Cohort 3

Cohort 4

Cohort 5

Cohort 6

Extension Cohort 1

Extension Cohort 2

Extension Cohort 3

Extension Cohort 4

Extension Cohort 5

Extension Cohort 6

Arm Description

Induction dose 1 given once a day(QD) for 4 weeks followed by maintenance dose A given QD for 20 weeks

Induction dose 2 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks

Maintenance dose A given QD for 24 weeks

Maintenance dose B given QD for 24 weeks

Maintenance dose C given QD for 24 weeks

Placebo given QD for 24 weeks

4 week drug holiday (no drug given) followed by PF-06700841 oral tablet QD for 20 weeks

Induction dose 1 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks in conjunction with narrow band UVB phototherapy

Induction dose 1 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks

Maintenance dose A given QD for 24 weeks

Maintenance dose B given QD for 24 weeks

Observation period for 24 weeks

Outcomes

Primary Outcome Measures

Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period

Central read F-VASI was assessed based on the facial photographs taken at the site. Central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. The higher score of F-VASI signified severer symptoms of non-segmental vitiligo. Percent change from baseline in central read F-VASI = ((post-baseline central read F-VASI - baseline central read F-VASI)/baseline central read F-VASI)×100. A negative percent change from baseline in central read F-VASI signified an improvement.

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period

Adverse Event (AE) was defined as any untoward medical occurrence in a study participant administered a product or medical device; the event did not necessarily need to have a causal relationship with the treatment or usage. An AE was considered a TEAE if the event started during the effective duration of treatment. All events that started on or after the first dosing day and time/start time, if collected, but before the last dose plus the lag time were flagged as TEAEs. SAE was defined as any untoward medical occurrence at any dose that resulted in death; was life threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); resulted in congenital anomaly/birth defect; or that was considered to be an important medical event. Causality to study treatment was determined by the investigator.

Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period

An AE was any untoward medical occurrence in a study participant administered a product or medical device; the event did not necessarily need to have a causal relationship with the treatment or usage. The abnormal test findings, clinically significant signs and symptoms of anaemia, neutropenia, thrombocytopenia and lymphopenia were reported as AEs. The clinical significance was determined by the investigator. An AE was considered a TEAE if the event started during the effective duration of treatment. All events that started on or after the first dosing day and time/start time, if collected, but before the last dose plus the lag time were flagged as TEAEs. Baseline was defined as the last measurement prior to first dosing (Day 1).

Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period

Participants had to abstain from all food and drink (except water and non-investigational products) for an 8-hour overnight fast prior to fasting lipid profile panel collection. Fasting lipid assessment included total cholesterol, low-density lipoprotein (LDL) cholesterol, high-density lipoprotein (HDL) cholesterol, and triglycerides. The clinical meaningfulness was determined by the investigator. Baseline was defined as the last measurement prior to first dosing (Day 1).

Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period

Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin.

Number of Participants With TEAEs and SAEs - Extension (Ext) Period

AE was defined as any untoward medical occurrence in a study participant administered a product or medical device; the event did not necessarily need to have a causal relationship with the treatment or usage. An AE was considered a TEAE if the event started during the effective duration of treatment. All events that started on or after the first dosing day and time/start time, if collected, but before the last dose plus the lag time were flagged as TEAEs. SAE was defined as any untoward medical occurrence at any dose that resulted in death; was life threatening (immediate risk of death); requires inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); resulted in congenital anomaly/birth defect; or that was considered to be an important medical event. Causality to study treatment was determined by the investigator.

Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period

Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period

Participants had to abstain from all food and drink (except water and non-investigational products) for an 8-hour overnight fast prior to fasting lipid profile panel collection. Fasting lipid assessment included total cholesterol, LDL cholesterol, HDL cholesterol, and triglycerides. The clinical meaningfulness was determined by the investigator.

Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period

Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin.

Secondary Outcome Measures

Percentage of Participants Achieving Central F-VASI75 at Week 24 - DR Period

This outcome measure was the percentage of participants achieving at least 75% improvement from baseline in central read F-VASI (F-VASI75) at Week 24. A negative percent change from baseline in central read F-VASI signified an improvement. The central read F-VASI75 response rate was analyzed by first treating the missing data (non-COVID-19 related) as non responders and then applying Chan and Zhang exact confidence interval (CI) method at Week 24. Central read F-VASI75=1 if percent change from baseline ≥75; central read F-VASI75=0 if percent change from baseline <75. Percent change from baseline in F-VASI=((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. Baseline was defined as the last measurement prior to study Day 18.

Percentage of Participants Achieving T-VASI50 at Week 24 - DR Period

Total body VASI (T-VASI) was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: T-VASI= Ʃ[Hand Units]×[Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities (excluding hands), trunk, lower extremities (excluding feet), and feet. The extent of depigmentation was expressed by percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. The data below was the percentage of participants achieving at least 50% improvement from baseline in T-VASI (T-VASI50) at Week 24. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percent Change From Baseline in T-VASI at Designated Time Points - DR Period

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities (excluding hands), trunk, lower extremities (excluding the feet), and feet. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in T-VASI=((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period

The central read F-VASI was assessed based on the facial photographs taken at the site. The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in central read F-VASI = ((post-baseline central read F-VASI - baseline central read F-VASI)/baseline central read F-VASI)×100. Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0.00 4.00): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percent Change From Baseline in SA-VES at Designated Time Points - DR Period

The Self-Assessment Vitiligo Extent Score (SA-VES) was a validated patient report outcome measurement instrument to provide information about disease extent. Vitiligo Extent Score (VES) was a measure to express the overall vitiligo involvement of the body (extent). Clinical illustrations for 19 separate body areas that reflected different degrees of involvement (1%, 5%, 10%, 25%, 50% and 75% depigmentation) were chosen to represent the participant's skin lesions to get the total extent of the disease. VES was a sum of all surface measurement that was similar to VASI. Baseline was defined as the last measurement prior to first dosing (Day 1).

Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities (excluding hands), trunk, lower extremities (excluding the feet), and feet. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. The absolute change from baseline in T-VASI was analyzed using the ANCOVA analysis. Negative change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving T-VASI50 at Designated Time Points - DR Period

T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. The outcome measure was the percentage of participants achieving at least 50% improvement from baseline in T-VASI (T-VASI50). Percent change from baseline in T-VASI=((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving T-VASI75 at Designated Time Points - DR Period

T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. The outcome measure was the percentage of participants achieving at least 75% improvement from baseline in T-VASI (T-VASI75). Percent change from baseline in T-VASI=((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving T-VASI90 at Designated Time Points - DR Period

T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. The outcome measure was the percentage of participants achieving at least 90% improvement from baseline in T-VASI (T-VASI90). Percent change from baseline in T-VASI=((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving T-VASI100 at Designated Time Points - DR Period

T-VASI was calculated using a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. This outcome measure was the percentage of participants achieving 100% improvement from baseline in T-VASI (T-VASI100). Percent change from baseline in T-VASI = ((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving Central Read F-VASI50 at Designated Time Points - DR Period

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants achieving at least 50% improvement in central read F-VASI from baseline (F-VASI50). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Percentage of Participants Achieving Central Read F-VASI75 at Designated Time Points - DR Period

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants achieving at least 75% improvement in central read F-VASI from baseline (F-VASI75). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Percentage of Participants Achieving Central Read F-VASI90 at Designated Time Points - DR Period

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants achieving at least 90% improvement in central read F-VASI from baseline (F-VASI90). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Percentage of Participants Achieving Central Read F-VASI100 at Designated Time Points - DR Period

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants achieving at least 100% improvement in central read F-VASI from baseline (F-VASI100). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Percentage of Participants Achieving Local F-VASI50 at Designated Time Points - DR Period

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving at least 50% improvement in site assessment F-VASI from baseline (F-VASI50). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving Local F-VASI75 at Designated Time Points - DR Period

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving at least 75% improvement in site assessment F-VASI from baseline (F-VASI75). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving Local F-VASI90 at Designated Time Points - DR Period

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving at least 90% improvement in site assessment F-VASI from baseline (F-VASI90). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving Local F-VASI100 at Designated Time Points - DR Period

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving 100% improvement in site assessment F-VASI from baseline (F-VASI100). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Change From Baseline in Total VitiQoL Score at Designated Time Points - DR Period

The Vitiligo-Specific Quality of Life (VitiQoL) instrument was a reliable and validated vitiligo disease-specific health-related quality of life (HRQoL) instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from "not at all" (scored 0) to "most of the time" (scored 6) and gave a minimum and maximum score from 0 to 90, with higher scores representing greater burden. The VitiQoL total score was calculated as sum of items 1-15. The change from baseline in total VitiQoL score was analyzed using the mixed-effect models repeated measures (MMRM) analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).

Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period

The VitiQoL was a reliable and validated vitiligo disease specific HRQoL instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from "not at all" (scored 0) to "most of the time" (scored 6) and gave a minimum and maximum score from 0 to 90, with higher scores representing greater burden. The VitiQoL Participation Limitation domain score was the sum of items 3, 4, 6, 9, 10, 11, 14 and ranged from 0 to 42. The change from baseline in VitiQoL Participation Limitation Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).

Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period

The VitiQoL was a reliable and validated vitiligo disease specific HRQoL instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from "not at all" (scored 0) to "most of the time" (scored 6) and gave a minimum and maximum score from 0 to 90, with higher scores representing greater burden. The VitiQoL Stigma domain score was the sum of items 1, 2, 5, 7 and 15, and ranged from 0 to 30. The change from baseline in VitiQoL Stigma Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).

Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period

The VitiQoL was a reliable and validated vitiligo disease specific HRQoL instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from "not at all" (scored 0) to "most of the time" (scored 6) and gave a minimum and maximum score from 0 to 90, with higher scores representing greater burden. The VitiQoL Behaviors domain score was the sum of items 8, 12 and 13, and ranged from 0 to 18. The change from baseline in VitiQoL Behaviors Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).

Percentage of Participants Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period

The percentage of participants achieving a static Investigator Global Assessment (sIGA) Score 0/1 and sIGA ≥2-point improvement at Week 24 was presented in this outcome measure. The sIGA score ranged from 0 to 4. The sIGA Score 0 represented "Clear" with no signs of loss of pigmentation with natural light or with Woods lamp examination. The sIGA Score 1 represented "Almost Clear" with the following descriptors: Faint, barely detectable loss of pigmentation mainly located on dorsal hands, feet, bony prominences, and/or limited areas. Approximately 90% pigmentation within lesions. No or rare signs of Koebner phenomenon, confetti like or trichrome lesions could be present. The sIGA Scores 2, 3 and 4 represented "Mild Vitiligo", "Moderate Vitiligo" and "Severe Vitiligo", respectively.

Full Information

NCT ID

NCT03715829

First Posted

October 19, 2018

Last Updated

March 1, 2022

Sponsor

Pfizer

1. Study Identification

Unique Protocol Identification Number

NCT03715829

Brief Title

A Phase 2b Study To Evaluate The Efficacy And Safety Profile Of PF-06651600 And PF-06700841 In Active Non-segmental Vitiligo Subjects

Official Title

A PHASE 2B RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER, DOSE-RANGING STUDY TO EVALUATE THE EFFICACY AND SAFETY PROFILE OF PF-06651600 WITH A PARTIALLY BLINDED EXTENSION PERIOD TO EVALUATE THE EFFICACY AND SAFETY OF PF-06651600 AND PF-06700841 IN SUBJECTS WITH ACTIVE NON-SEGMENTAL VITILIGO

Study Type

Interventional

2. Study Status

Record Verification Date

March 2022

Overall Recruitment Status

Completed

Study Start Date

November 26, 2018 (Actual)

Primary Completion Date

February 5, 2021 (Actual)

Study Completion Date

February 5, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. Study Description

Brief Summary

This is a Phase 2b, randomized, double blind, parallel group, multicenter study with an extension period. The study will have a maximum duration of approximately 60 weeks. This includes an up to 4 weeks Screening Period, a 24 week dose ranging period, an up to 24 week extension period and a 8 week Follow up Period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Active Non-segmental Vitiligo

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Model Description

Dose ranging period is a parallel design. Extension period is a sequential design.

Masking

ParticipantInvestigatorOutcomes Assessor

Masking Description

Dose ranging period is blinded. The partially blinded extension period includes open arms and blinded arms.

Allocation

Randomized

Enrollment

366 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cohort 1

Arm Type

Experimental

Arm Description

Induction dose 1 given once a day(QD) for 4 weeks followed by maintenance dose A given QD for 20 weeks

Arm Title

Cohort 2

Arm Type

Experimental

Arm Description

Induction dose 2 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks

Arm Title

Cohort 3

Arm Type

Experimental

Arm Description

Maintenance dose A given QD for 24 weeks

Arm Title

Cohort 4

Arm Type

Experimental

Arm Description

Maintenance dose B given QD for 24 weeks

Arm Title

Cohort 5

Arm Type

Experimental

Arm Description

Maintenance dose C given QD for 24 weeks

Arm Title

Cohort 6

Arm Type

Placebo Comparator

Arm Description

Placebo given QD for 24 weeks

Arm Title

Extension Cohort 1

Arm Type

Experimental

Arm Description

4 week drug holiday (no drug given) followed by PF-06700841 oral tablet QD for 20 weeks

Arm Title

Extension Cohort 2

Arm Type

Experimental

Arm Description

Induction dose 1 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks in conjunction with narrow band UVB phototherapy

Arm Title

Extension Cohort 3

Arm Type

Experimental

Arm Description

Induction dose 1 given QD for 4 weeks followed by maintenance dose A given QD for 20 weeks

Arm Title

Extension Cohort 4

Arm Type

Experimental

Arm Description

Maintenance dose A given QD for 24 weeks

Arm Title

Extension Cohort 5

Arm Type

Experimental

Arm Description

Maintenance dose B given QD for 24 weeks

Arm Title

Extension Cohort 6

Arm Type

No Intervention

Arm Description

Observation period for 24 weeks

Intervention Type

Drug

Intervention Name(s)

PF-06651600

Intervention Description

Induction dose 1. Oral tablet taken QD

Intervention Type

Drug

Intervention Name(s)

PF-06651600

Intervention Description

Induction dose 2. Oral tablet taken QD

Intervention Type

Drug

Intervention Name(s)

PF-06651600

Intervention Description

Maintenance dose A. Oral tablet taken QD

Intervention Type

Drug

Intervention Name(s)

PF-06651600

Intervention Description

Maintenance Dose B. Oral tablet taken QD

Intervention Type

Drug

Intervention Name(s)

PF-06651600

Intervention Description

Maintenance Dose C. Oral tablet taken QD

Intervention Type

Drug

Intervention Name(s)

placebo

Intervention Description

placebo

Intervention Type

Drug

Intervention Name(s)

PF06700841

Intervention Description

Oral tablet taken QD

Intervention Type

Device

Intervention Name(s)

narrow-band UVB phototherapy

Intervention Description

Phototherapy will be combined with PF-06651600

Primary Outcome Measure Information:

Title

Percent Change From Baseline in Central Read Facial-Vitiligo Area Scoring Index (F-VASI) at Week 24 - Dose Ranging (DR) Period

Description

Time Frame

Baseline, Week 24 (Baseline was defined as the last measurement prior to Study Day 18)

Title

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) up to Week 24 - DR Period

Description

Time Frame

24 weeks

Title

Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - DR Period

Description

Time Frame

Baseline up to Week 24

Title

Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile up to Week 24 - DR Period

Description

Time Frame

Baseline up to Week 24

Title

Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - DR Period

Description

Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin.

Time Frame

24 weeks

Title

Number of Participants With TEAEs and SAEs - Extension (Ext) Period

Description

Time Frame

24 weeks

Title

Number of Participants With the TEAEs of Anaemia, Neutropenia, Thrombocytopenia and Lymphopenia - Ext Period

Description

Time Frame

24 weeks

Title

Number of Participants With Clinically Meaningful Changes From Baseline in Lipid Profile - Ext Period

Description

Participants had to abstain from all food and drink (except water and non-investigational products) for an 8-hour overnight fast prior to fasting lipid profile panel collection. Fasting lipid assessment included total cholesterol, LDL cholesterol, HDL cholesterol, and triglycerides. The clinical meaningfulness was determined by the investigator.

Time Frame

24 Weeks

Title

Number of Participants With Liver Function Test Values Meeting the Protocol-Specified Discontinuation Criteria - Ext Period

Description

Liver function tests included tests of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin.

Time Frame

24 weeks

Secondary Outcome Measure Information:

Title

Percentage of Participants Achieving Central F-VASI75 at Week 24 - DR Period

Description

Time Frame

Week 24

Title

Percentage of Participants Achieving T-VASI50 at Week 24 - DR Period

Description

Time Frame

Week 24

Title

Percent Change From Baseline in T-VASI at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percent Change From Baseline in Central Read F-VASI at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 8, 16 and 24

Title

Percent Change From Baseline in Local F-VASI at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percent Change From Baseline in SA-VES at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 16 and 24

Title

Absolute Change From Baseline in T-VASI at Designated Time Points - DR Period

Description

The T-VASI was calculated using a formula that included contribution from 6 different body regions (possible range, 0-100) with a modified method: VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 separate and mutually exclusive regions: face/neck, hands, upper extremities (excluding hands), trunk, lower extremities (excluding the feet), and feet. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. The absolute change from baseline in T-VASI was analyzed using the ANCOVA analysis. Negative change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving T-VASI50 at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving T-VASI75 at Designated Time Points - DR Period

Description

T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. The outcome measure was the percentage of participants achieving at least 75% improvement from baseline in T-VASI (T-VASI75). Percent change from baseline in T-VASI=((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving T-VASI90 at Designated Time Points - DR Period

Description

T-VASI was calculated by a formula that included contribution from 6 body regions (possible range, 0-100): T-VASI = Ʃ [Hand Units] × [Depigmentation]. One hand unit, which encompassed the palm plus the volar surface of all the digits, was approximately 1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of each body region. The body was divided into 6 mutually exclusive regions: face/neck, hands, upper extremities, trunk, lower extremities, and feet. The extent of depigmentation was expressed by the percentages: 0, 10%, 25%, 50%, 75%, 90% or 100%. The outcome measure was the percentage of participants achieving at least 90% improvement from baseline in T-VASI (T-VASI90). Percent change from baseline in T-VASI=((post-baseline T-VASI - baseline T-VASI)/baseline T-VASI)×100. Negative percent change from baseline in T-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving T-VASI100 at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving Central Read F-VASI50 at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 8, 16 and 24

Title

Percentage of Participants Achieving Central Read F-VASI75 at Designated Time Points - DR Period

Description

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants achieving at least 75% improvement in central read F-VASI from baseline (F-VASI75). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Time Frame

Baseline, Weeks 4, 8, 16 and 24

Title

Percentage of Participants Achieving Central Read F-VASI90 at Designated Time Points - DR Period

Description

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants achieving at least 90% improvement in central read F-VASI from baseline (F-VASI90). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Time Frame

Baseline, Weeks 4, 8, 16 and 24

Title

Percentage of Participants Achieving Central Read F-VASI100 at Designated Time Points - DR Period

Description

The central read F-VASI was calculated using a formula that included contribution of affected facial surface areas showing all 6 different depigmentation rates (0.1, 0.25, 0.5, 0.75, 0.9 and 1) with a modified method: F-VASI (central read)=Ʃ [Affected Facial Surface Area] × 4 × [Depigmentation Rates]. Face was defined as the area from the hairline on top of the forehead to the jawline at the bottom of the cheeks. F-VASI (central read) ranged from 0.000 to 4.000 by defining the affected Facial Surface Area (expressed as the value between 0.0 to 1.0) being 4% of total Body Surface Area. Percent change from baseline in F-VASI = ((post-baseline F-VASI - baseline F-VASI)/baseline F-VASI)×100. This outcome measure was the percentage of participants achieving at least 100% improvement in central read F-VASI from baseline (F-VASI100). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to Study Day 18.

Time Frame

Baseline, Weeks 4, 8, 16 and 24

Title

Percentage of Participants Achieving Local F-VASI50 at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving Local F-VASI75 at Designated Time Points - DR Period

Description

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving at least 75% improvement in site assessment F-VASI from baseline (F-VASI75). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving Local F-VASI90 at Designated Time Points - DR Period

Description

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving at least 90% improvement in site assessment F-VASI from baseline (F-VASI90). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Percentage of Participants Achieving Local F-VASI100 at Designated Time Points - DR Period

Description

The site assessment of the F-VASI was calculated using a formula that included contribution from face (possible range, 0-4): Local F-VASI = [Digit Units] × [Depigmentation] × 0.1. Scalp, neck, eyebrows, eyelashes, and vermilion were excluded from this calculation. The volar surface of 1 digit (the participant's thumb) was approximately 0.1% of the total body surface area and was used as a guide to estimate the baseline percentage of vitiligo involvement of face. The extent of depigmentation was expressed by the following percentages: 0, 10%, 25%, 50%, 75%, 90%, or 100%. Percent change from baseline in Local F-VASI = ((post baseline Local F-VASI - baseline Local F-VASI)/baseline Local F-VASI)×100. This outcome measure was the percentage of participants achieving 100% improvement in site assessment F-VASI from baseline (F-VASI100). Negative percent change from baseline in F-VASI signified an improvement. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 8, 12, 16, 20 and 24

Title

Change From Baseline in Total VitiQoL Score at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 16 and 24

Title

Change From Baseline in VitiQoL Participation Limitation Domain Score at Designated Time Points - DR Period

Description

Time Frame

Baseline, Weeks 4, 16 and 24

Title

Change From Baseline in VitiQoL Stigma Domain Score at Designated Time Points - DR Period

Description

The VitiQoL was a reliable and validated vitiligo disease specific HRQoL instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from "not at all" (scored 0) to "most of the time" (scored 6) and gave a minimum and maximum score from 0 to 90, with higher scores representing greater burden. The VitiQoL Stigma domain score was the sum of items 1, 2, 5, 7 and 15, and ranged from 0 to 30. The change from baseline in VitiQoL Stigma Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 16 and 24

Title

Change From Baseline in VitiQoL Behaviors Domain Score at Designated Time Points - DR Period

Description

The VitiQoL was a reliable and validated vitiligo disease specific HRQoL instrument which measured concepts relevant to vitiligo participants. The VitiQoL was a 15-item PRO measure which measured concepts of symptoms, daily activities, leisure activities, work, personal relationships and treatment. Responses ranged from "not at all" (scored 0) to "most of the time" (scored 6) and gave a minimum and maximum score from 0 to 90, with higher scores representing greater burden. The VitiQoL Behaviors domain score was the sum of items 8, 12 and 13, and ranged from 0 to 18. The change from baseline in VitiQoL Behaviors Domain Score was analyzed using the MMRM analysis. Baseline was defined as the last measurement prior to first dosing (Day 1).

Time Frame

Baseline, Weeks 4, 16 and 24

Title

Percentage of Participants Achieving sIGA 0 or 1 and at Least a 2-Point Improvement at Week 24 - DR Period

Description

Time Frame

Week 24

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male or female subjects between 18-65 years of age, inclusive, at time of informed consent. Must have moderate to severe active non-segmental vitiligo. Exclusion Criteria: History of human immunodeficiency virus (HIV) or positive HIV serology at screening, Infected with hepatitis B or hepatitis C viruses. Have evidence of active or latent or inadequately treated infection with Mycobacterium tuberculosis (TB)

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Pfizer CT.gov Call Center

Organizational Affiliation

Pfizer

Official's Role

Study Director

Facility Information:

Facility Name

Marvel Research, LLC

City

Huntington Beach

State/Province

California

ZIP/Postal Code

92647

Country

United States

Facility Name

University of California, Irvine, Dermatology Clinical Research Center

City

Irvine

State/Province

California

ZIP/Postal Code

92697

Country

United States

Facility Name

Vitiligo and Pigmentation Institute Of Southern California

City

Los Angeles

State/Province

California

ZIP/Postal Code

90036

Country

United States

Facility Name

Dermatology Specialist, Inc.

City

Murrieta

State/Province

California

ZIP/Postal Code

92562

Country

United States

Facility Name

Dermatology Specialists, Inc.

City

Oceanside

State/Province

California

ZIP/Postal Code

92056

Country

United States

Facility Name

UC Davis Health

City

Sacramento

State/Province

California

ZIP/Postal Code

95816

Country

United States

Facility Name

Brookside Dermatology Associates

City

Bridgeport

State/Province

Connecticut

ZIP/Postal Code

06606

Country

United States

Facility Name

New England Research Associates, LLC

City

Bridgeport

State/Province

Connecticut

ZIP/Postal Code

06606

Country

United States

Facility Name

New Horizon Research Center

City

Miami

State/Province

Florida

ZIP/Postal Code

33165

Country

United States

Facility Name

Park Avenue Dermatology

City

Orange Park

State/Province

Florida

ZIP/Postal Code

32073

Country

United States

Facility Name

ForCare Clinical Research

City

Tampa

State/Province

Florida

ZIP/Postal Code

33613

Country

United States

Facility Name

Advanced Skin and MOHS Surgery Center, c/o TrialSpark, Inc.

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60657

Country

United States

Facility Name

Chevy Chase Dermatology Center (TrialSpark, Inc.)

City

Chevy Chase

State/Province

Maryland

ZIP/Postal Code

20815

Country

United States

Facility Name

TrialSpark - Samantha Toerge, MD

City

Chevy Chase

State/Province

Maryland

ZIP/Postal Code

20815

Country

United States

Facility Name

TrialSpark - Ronald Shore, MD

City

Rockville

State/Province

Maryland

ZIP/Postal Code

20852

Country

United States

Facility Name

Tobias & Battite, Inc.

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02111

Country

United States

Facility Name

Tufts Medical Center

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02111

Country

United States

Facility Name

UMass Memorial Medical Center, Hahnemann Campus

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01605

Country

United States

Facility Name

Investigational Drug Service Pharmacy

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01655

Country

United States

Facility Name

UMass Memorial Medical Center Ear Nose and Throat

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01655

Country

United States

Facility Name

University of Massachusetts Medical School

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01655

Country

United States

Facility Name

Henry Ford Hospital Department of Dermatology

City

Detroit

State/Province

Michigan

ZIP/Postal Code

48202

Country

United States

Facility Name

University of Minnesota Department of Dermatology

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

Facility Name

The Dermatology Group, P.C.

City

Verona

State/Province

New Jersey

ZIP/Postal Code

07044

Country

United States

Facility Name

Icahn School of Medicine at Mount Sinai

City

New York

State/Province

New York

ZIP/Postal Code

10003

Country

United States

Facility Name

Upper West Side Dermatology c/o TrialSpark, Inc

City

New York

State/Province

New York

ZIP/Postal Code

10023

Country

United States

Facility Name

MDCS: Medical Dermatology & Cosmetic Surgery (TrialSpark, Inc.)

City

New York

State/Province

New York

ZIP/Postal Code

10065

Country

United States

Facility Name

South Nassau Dermatology

City

Oceanside

State/Province

New York

ZIP/Postal Code

11572

Country

United States

Facility Name

TrialSpark, Inc. - Russell W. Cohen, MD

City

Oceanside

State/Province

New York

ZIP/Postal Code

11572

Country

United States

Facility Name

PMG Research of Wilmington, LLC

City

Wilmington

State/Province

North Carolina

ZIP/Postal Code

28411

Country

United States

Facility Name

Remington-Davis, Inc. Clinical Research

City

Columbus

State/Province

Ohio

ZIP/Postal Code

43215

Country

United States

Facility Name

ForeFront Dermatology

City

Columbus

State/Province

Ohio

ZIP/Postal Code

43220

Country

United States

Facility Name

Vital Prospects Clinical Research Institute, PC

City

Tulsa

State/Province

Oklahoma

ZIP/Postal Code

74136

Country

United States

Facility Name

University Physicians Group

City

Austin

State/Province

Texas

ZIP/Postal Code

78701

Country

United States

Facility Name

University of Texas Southwestern Medical Center

City

Dallas

State/Province

Texas

ZIP/Postal Code

75390-9191

Country

United States

Facility Name

Pickens Academic Tower

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

The University of Texas Health Science Center at Houston

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

The University of Texas MD Anderson Cancer Center

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

Virginia Clinical Research, Inc

City

Norfolk

State/Province

Virginia

ZIP/Postal Code

23502

Country

United States

Facility Name

Tamjidi Skin Institute (TrialSpark, Inc.)

City

Vienna

State/Province

Virginia

ZIP/Postal Code

22182

Country

United States

Facility Name

The Skin Hospital

City

Darlinghurst

State/Province

New South Wales

ZIP/Postal Code

2010

Country

Australia

Facility Name

Premier Specialists Pty Ltd

City

Kogarah

State/Province

New South Wales

ZIP/Postal Code

2217

Country

Australia

Facility Name

Veracity Clinical Research Pty Ltd

City

Woolloongabba

State/Province

Queensland

ZIP/Postal Code

4102

Country

Australia

Facility Name

Skin Health Institute

City

Carlton

State/Province

Victoria

ZIP/Postal Code

3053

Country

Australia

Facility Name

Sinclair Dermatology

City

East Melbourne

State/Province

Victoria

ZIP/Postal Code

3002

Country

Australia

Facility Name

The Royal Melbourne Hospital

City

Parkville

State/Province

Victoria

ZIP/Postal Code

3050

Country

Australia

Facility Name

The Royal Melbourne Hospital

City

Parkville

State/Province

Victoria

ZIP/Postal Code

3052

Country

Australia

Facility Name

Hôpital Erasme Dermatology

City

Brussels

ZIP/Postal Code

1070

Country

Belgium

Facility Name

UZ Brussel - Dermatology

City

Brussel

ZIP/Postal Code

1090

Country

Belgium

Facility Name

UZ Gent - Dermatology

City

Gent

ZIP/Postal Code

9000

Country

Belgium

Facility Name

Dr. Chih-ho Hong Medical Inc.

City

Surrey

State/Province

British Columbia

ZIP/Postal Code

V3R 6A7

Country

Canada

Facility Name

Enverus Medical Research

City

Surrey

State/Province

British Columbia

ZIP/Postal Code

V3V 0C6

Country

Canada

Facility Name

University of British Columbia

City

Vancouver

State/Province

British Columbia

ZIP/Postal Code

V5Z 4E8

Country

Canada

Facility Name

Wiseman Dermatology Research Inc.

City

Winnipeg

State/Province

Manitoba

ZIP/Postal Code

R3M 3Z4

Country

Canada

Facility Name

CCA Medical Research

City

Ajax

State/Province

Ontario

ZIP/Postal Code

L1S 7K8

Country

Canada

Facility Name

Guenther Research Inc.

City

London

State/Province

Ontario

ZIP/Postal Code

N6A 3H7

Country

Canada

Facility Name

Lynderm Research Inc.

City

Markham

State/Province

Ontario

ZIP/Postal Code

L3P 1X3

Country

Canada

Facility Name

DermEdge Research

City

Mississauga

State/Province

Ontario

ZIP/Postal Code

L5H 1G9

Country

Canada

Facility Name

North York Research Inc.

City

North York

State/Province

Ontario

ZIP/Postal Code

M2M 4J5

Country

Canada

Facility Name

The Centre for Clinical Trials

City

Oakville

State/Province

Ontario

ZIP/Postal Code

L6J 7W5

Country

Canada

Facility Name

The Centre for Dermatology

City

Richmond Hill

State/Province

Ontario

ZIP/Postal Code

L4B 1A5

Country

Canada

Facility Name

K.Papp Clinical Research

City

Waterloo

State/Province

Ontario

ZIP/Postal Code

N2J 1C4

Country

Canada

Facility Name

Innovaderm Research Inc.

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H2X 2V1

Country

Canada

Facility Name

Diex Research Sherbrooke Inc.

City

Sherbrooke

State/Province

Quebec

ZIP/Postal Code

J1L 0H8

Country

Canada

Facility Name

Centre de Recherche Dermatologique du Quebec metropolitain (CRDQ)

City

Quebec

ZIP/Postal Code

G1V 4X7

Country

Canada

Facility Name

Centre de Recherche Saint-Louis

City

Quebec

ZIP/Postal Code

G1W 4R4

Country

Canada

Facility Name

Centre de Recherche Saint-Louis

City

Quebec

ZIP/Postal Code

G1W1S2

Country

Canada

Facility Name

Fachklinik Bad Bentheim, Fachbereich Dermatologie und Allergologie, Dermatologische Ambulanz

City

Bad Bentheim

ZIP/Postal Code

48455

Country

Germany

Facility Name

Universitaetsklinikum Erlangen Hautklinik Studienambulanz

City

Erlangen

ZIP/Postal Code

91054

Country

Germany

Facility Name

Universitätsklinikum Frankfurt, Klinik für Dermatologie, Venerologie und Allergologie

City

Frankfurt am Main

ZIP/Postal Code

60590

Country

Germany

Facility Name

Universitaetsklinikum Schleswig-Holstein, Campus Luebeck CCIM

City

Luebeck

ZIP/Postal Code

23538

Country

Germany

Facility Name

Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz

City

Mainz

ZIP/Postal Code

55131

Country

Germany

Facility Name

Universitaetsklinikum Muenster

City

Muenster

ZIP/Postal Code

48149

Country

Germany

Facility Name

Istituti Fisioterapici Ospitalieri, Istituto di Ricovero e Cura a Carattere Scientifico,

City

Roma

State/Province

ZIP/Postal Code

00144

Country

Italy

Facility Name

Nagoya City University Hospital - Dermatology

City

Nagoya

State/Province

Aichi

ZIP/Postal Code

467-8602

Country

Japan

Facility Name

Tohoku University Hospital

City

Sendai

State/Province

Miyagi

ZIP/Postal Code

980-8574

Country

Japan

Facility Name

Nippon Medical School Hospital

City

Bunkyo-ku

State/Province

Tokyo

ZIP/Postal Code

113-8603

Country

Japan

Facility Name

Tokyo Medical University Hospital

City

Shinjuku-ku

State/Province

Tokyo

ZIP/Postal Code

160-0023

Country

Japan

Facility Name

Yamanashi Prefectural Central Hospital

City

Kofu

State/Province

Yamanashi

ZIP/Postal Code

400-8506

Country

Japan

Facility Name

Dongguk University Ilsan Hospital

City

Goyang-si

State/Province

Gyeonggi-do

ZIP/Postal Code

10326

Country

Korea, Republic of

Facility Name

The Catholic University of Korea, St. Vincent's Hospital

City

Suwon-si

State/Province

Gyeonggi-do

ZIP/Postal Code

16247

Country

Korea, Republic of

Facility Name

Ajou University Hospital

City

Suwon

State/Province

Gyeonggi-do

ZIP/Postal Code

16499

Country

Korea, Republic of

Facility Name

Inha University Hospital

City

Incheon

ZIP/Postal Code

22332

Country

Korea, Republic of

Facility Name

Severance Hospital, Yonsei University Health System

City

Seoul

ZIP/Postal Code

03722

Country

Korea, Republic of

Facility Name

Hospital de la Santa Creu i Sant Pau

City

Barcelona

ZIP/Postal Code

08041

Country

Spain

Facility Name

Hospital Universitario Reina Sofia

City

Cordoba

ZIP/Postal Code

14004

Country

Spain

Facility Name

Hospital Universitario Ramón y Cajal

City

Madrid

ZIP/Postal Code

28034

Country

Spain

Facility Name

Hospital Universitario La Paz

City

Madrid

ZIP/Postal Code

28046

Country

Spain

Facility Name

Hospital Universitari i Politecnic La Fe

City

Valencia

ZIP/Postal Code

46026

Country

Spain

Facility Name

Chang Gung Medical Foundation - Kaohsiung Chang Gung Memorial Hospital

City

Kaohsiung

ZIP/Postal Code

83301

Country

Taiwan

Facility Name

National Cheng Kung University Hospital

City

Tainan

ZIP/Postal Code

70403

Country

Taiwan

Facility Name

National Taiwan University Hospital

City

Taipei

ZIP/Postal Code

10002

Country

Taiwan

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

IPD Sharing URL

https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

Citations:

PubMed Identifier

36370907

Citation

Ezzedine K, Peeva E, Yamaguchi Y, Cox LA, Banerjee A, Han G, Hamzavi I, Ganesan AK, Picardo M, Thaci D, Harris JE, Bae JM, Tsukamoto K, Sinclair R, Pandya AG, Sloan A, Yu D, Gandhi K, Vincent MS, King B. Efficacy and safety of oral ritlecitinib for the treatment of active nonsegmental vitiligo: A randomized phase 2b clinical trial. J Am Acad Dermatol. 2023 Feb;88(2):395-403. doi: 10.1016/j.jaad.2022.11.005. Epub 2022 Nov 9. Erratum In: J Am Acad Dermatol. 2023 Sep;89(3):639.

Results Reference

derived

Links:

URL

https://pmiform.com/clinical-trial-info-request?StudyID=B7981019

Description

To obtain contact information for a study center near you, click here.

Learn more about this trial

A Phase 2b Study To Evaluate The Efficacy And Safety Profile Of PF-06651600 And PF-06700841 In Active Non-segmental Vitiligo Subjects

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