Back to resultsSafety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome
Primary Purpose
Netherton Syndrome
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
QRX003-2% Lotion
QRX003-4% Lotion
Vehicle
Sponsored by
About this trial
This is an interventional treatment trial for Netherton Syndrome
Eligibility Criteria
Inclusion Criteria:
- Subject is a male or non-pregnant female at least 18 years of age.
- Females must be post-menopausal , surgically sterile , or use an effective method of birth control , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline.
- Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1/Screening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation.
- Subject has NS lesions in the Treatment Area (i.e., arms or lower legs).
- Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.
Exclusion Criteria:
- Subject is pregnant, lactating, or is planning to become pregnant during the study.
- Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy.
- Subject has Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females
- Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.
- Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.
- Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline.
- Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
- Subject has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
- Subject has used systemic prescription treatment for NS within 4 weeks prior to Visit 2/Baseline.
- Subject has used systemic biologic therapy for NS.
- Subject has used topical prescription treatment in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
- Subject has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
- Subject is currently enrolled in an investigational drug, biologic, or device study.
- Subject has used an investigational drug, biologic, or device treatment within 30 days prior to Visit 2/Baseline.
Sites / Locations
- Site #1Recruiting
- Site #2
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
QRX003-2%
QRX003-4%
Vehicle Lotion
Arm Description
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
Outcomes
Primary Outcome Measures
Efficacy Endpoints-1-point IGA
Proportion of subjects with 1-point reduction on the Investigator's Global Assessment (IGA) from Baseline. The IGA assesses overall severity of a subject's NS based on a 5-point scale where 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe.
Efficacy Endpoints-2-point IGA
Proportion of subjects with 2-point reduction in IGA from Baseline.
Efficacy Endpoints-NS Sign
Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale.
Efficacy Endpoints-BSA
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
Efficacy Endpoints-WI-NRS score
Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable".
Efficacy Endpoints-TSQM
Assessment of subject satisfaction with treatment based on the Treatment Satisfaction Questionnaire for Medication (TSQM). The TSQM measures a subject's level of satisfaction or dissatisfaction with the treatment. Composite scores can range from 0 to 100; higher scores indicate better satisfaction.
Efficacy Endpoints-Rescue Therapy
Proportion of subjects requiring rescue therapy
Safety Endpoints-AEs
Any local and systemic AEs (Adverse Events)/serious AEs
Safety Endpoints-LSR
Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05521438
Brief Title
Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome
Official Title
A Multicenter, Randomized, Vehicle-Controlled, Double-Blind, Parallel Comparison Study of QRX003 Lotion in Subjects With Netherton Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
August 2022
Overall Recruitment Status
Recruiting
Study Start Date
June 23, 2022 (Actual)
Primary Completion Date
June 2023 (Anticipated)
Study Completion Date
June 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Quoin Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study has been designed to determine the safety, tolerability and efficacy of QRX003 lotion 2%, 4% in subjects with Netherton Syndrome (NS) in comparison to vehicle
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Netherton Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Masking Description
Double-blind
Allocation
Randomized
Enrollment
18 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
QRX003-2%
Arm Type
Experimental
Arm Description
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
Arm Title
QRX003-4%
Arm Type
Experimental
Arm Description
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
Arm Title
Vehicle Lotion
Arm Type
Experimental
Arm Description
Subjects will apply test article once daily in the morning (QAM) for 12 weeks
Intervention Type
Drug
Intervention Name(s)
QRX003-2% Lotion
Intervention Description
QRX003Topical Lotion containing 2% active drug (serine protease inhibitor)
Intervention Type
Drug
Intervention Name(s)
QRX003-4% Lotion
Intervention Description
QRX003Topical Lotion containing 4% active drug (serine protease inhibitor)
Intervention Type
Drug
Intervention Name(s)
Vehicle
Intervention Description
Vehicle Lotion
Primary Outcome Measure Information:
Title
Efficacy Endpoints-1-point IGA
Description
Proportion of subjects with 1-point reduction on the Investigator's Global Assessment (IGA) from Baseline. The IGA assesses overall severity of a subject's NS based on a 5-point scale where 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe.
Time Frame
Up to week 16
Title
Efficacy Endpoints-2-point IGA
Description
Proportion of subjects with 2-point reduction in IGA from Baseline.
Time Frame
Up to week 16
Title
Efficacy Endpoints-NS Sign
Description
Change from Baseline in each individual NS Sign (erythema and scaling). Severity of each NS sign is graded on a 5-point scale.
Time Frame
Up to week 16
Title
Efficacy Endpoints-BSA
Description
Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area
Time Frame
Up to week 16
Title
Efficacy Endpoints-WI-NRS score
Description
Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable".
Time Frame
Up to week 16
Title
Efficacy Endpoints-TSQM
Description
Assessment of subject satisfaction with treatment based on the Treatment Satisfaction Questionnaire for Medication (TSQM). The TSQM measures a subject's level of satisfaction or dissatisfaction with the treatment. Composite scores can range from 0 to 100; higher scores indicate better satisfaction.
Time Frame
Up to week 16
Title
Efficacy Endpoints-Rescue Therapy
Description
Proportion of subjects requiring rescue therapy
Time Frame
Up to week 16
Title
Safety Endpoints-AEs
Description
Any local and systemic AEs (Adverse Events)/serious AEs
Time Frame
Up to week 16
Title
Safety Endpoints-LSR
Description
Number of subjects with presence of the following LSRs (Local Skin Reactions): edema, excoriations, oozing/vesiculation/crusting, and erosions at any study visit.
Time Frame
Up to week 16
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subject is a male or non-pregnant female at least 18 years of age.
Females must be post-menopausal , surgically sterile , or use an effective method of birth control , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline.
Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1/Screening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation.
Subject has NS lesions in the Treatment Area (i.e., arms or lower legs).
Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.
Exclusion Criteria:
Subject is pregnant, lactating, or is planning to become pregnant during the study.
Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy.
Subject has Fridericia-corrected QT ≥ 450 ms for males or ≥ 470 ms for females
Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.
Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.
Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2/Baseline.
Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
Subject has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
Subject has used systemic prescription treatment for NS within 4 weeks prior to Visit 2/Baseline.
Subject has used systemic biologic therapy for NS.
Subject has used topical prescription treatment in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
Subject has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
Subject is currently enrolled in an investigational drug, biologic, or device study.
Subject has used an investigational drug, biologic, or device treatment within 30 days prior to Visit 2/Baseline.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
TI Clinical Research
Phone
858-571-1800
Ext
147
Email
clinicalresearch@therapeuticsinc.com
First Name & Middle Initial & Last Name or Official Title & Degree
Oleg G Khatsenko
Phone
858-571-1800
Ext
166
Email
okhatsenko@therapeuticsinc.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Tony Andrasfay
Organizational Affiliation
Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Site #1
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Oleg G Khatsenko
Phone
858-571-1800
Ext
166
Email
okhatsenko@therapeuticsinc.com
Facility Name
Site #2
City
San Antonio
State/Province
Texas
ZIP/Postal Code
72218
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Oleg G Khatsenko
Phone
858-571-1800
Ext
166
Email
okhatsenko@therapeuticsinc.com
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome
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