Back to resultsStudy of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia (PATHWAY)
Primary Purpose
Warm Autoimmune Hemolytic Anemia (wAIHA)
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
parsaclisinib
placebo
Sponsored by
About this trial
This is an interventional treatment trial for Warm Autoimmune Hemolytic Anemia (wAIHA) focused on measuring Warm Autoimmune Hemolytic Anemia (wAIHA)
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of primary warm AIHA.
- Participants who have at least 1 unsuccessful prior therapy for warm AIHA or unable to receive or tolerate other therapies.
- Hemoglobin ≥ 6.5 to < 10 g/dL with symptoms of anemia at screening.
- FACIT-F score ≤ 43 at screening.
- Willingness to avoid pregnancy or fathering children.
- Willingness to receive PJP prophylaxis.
- Further inclusion criteria apply.
Exclusion Criteria:
- Women who are pregnant, breastfeeding or who are planning a pregnancy.
- Diagnosis of other types of AIHA (CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria).
- Secondary warm AIHA from any cause or diagnosis of Evans syndrome.
- Splenectomy less than 3 months before randomization.
- Participants with a history or ongoing significant illness as assessed by the investigator.
- Participants with a current of medical history of a malignancy within the past 5 years except basal or squamous cell skin cancer that has been removed and considered cured, or superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy.
- Participants know to be infected with HIV, Hepatitis B, or hepatitis C.
- Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine.
- Participants with laboratory values outside of the protocol defined ranges.
- Further exclusion criteria apply.
Sites / Locations
- Investigative Site US005
- Investigative Site US004
- Investigative Site US006
- Investigative Site US012
- Investigative Site US007
- Investigative Site US002
- Investigative Site US003
- Investigative Site US009
- Investigative Site US010
- Investigative Site US001
- Investigative Site AT002
- Investigative Site AT001
- Investigative Site BE001
- Investigative Site BE002
- Investigative Site CA001
- Investigative Site FR002
- Investigative Site FR003
- Investigative Site FR001
- Investigative Site DE001
- Investigative Site DE002
- Investigative Site IL002
- Investigative Site IL001
- Investigative Site IT003
- Investigative Site IT002
- Investigative Site IT001
- Investigative Site IT004
- Investigative Site IT006
- Investigative Site IT005
- Investigative Site JP008
- Investigative Site JP004
- Investigative Site JP006
- Investigative Site JP009
- Investigative Site JP002
- Investigative Site JP010
- Investigative Site JP005
- Investigative Site JP007
- Investigative Site JP001
- Investigative Site JP003
- Investigative Site NL001
- Investigative Site PL001
- Investigative Site PL006
- Investigative Site PL003
- Investigative Site PL005
- Investigative Site PL004
- Investigative Site PL002
- Investigative Site ES006
- Investigative Site ES001
- Investigative Site ES003
- Investigative Site ES005
- Investigative Site ES004
- Investigative Site ES002
- Investigative Site GB002
- Investigative Site GB006
- Investigative Site GB003
- Investigative Site GB004
- Investigative Site GB005
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Group A: Parsaclisib
Group B: Placebo followed by Parsaclisib
Arm Description
Participants will receive parsaclisib for 24 weeks (double-blind period). Participant who completed the double-blind period and tolerating the study treatment upon investigator's opinion will continue into open-label period for an additional 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive parsaclisib in the 24 week open-label period. Participants may then continue to receive parsaclisib in a long-term extension period.
Outcomes
Primary Outcome Measures
Proportion of participants attaining a durable hemoglobin response
Proportion of participants attaining a durable hemoglobin response, defined as hemoglobin ≥ 10 g/dL with an increase from baseline of ≥ 2 g/dL not attributed to rescue therapy at ≥ 3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.
Secondary Outcome Measures
Proportion of participants with a ≥ 3-point increase in FACIT-F score
Increase is measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.
Proportion of participants with a 50 m increase in a 6MWT
Defined as an increase of 50 m using the Six-minute walk test, a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes.
Change in FACIT-F score
Change will be measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.
Percent Change in FACIT-F
will be measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.
Change in hemoglobin
Changes will be measured and compared in the hematology panel.
Percentage change in hemoglobin
Percentage change will be measured and compared in the hematology panel.
Proportion of participants who received transfusions
Proportion of participants who received transfusions.
Change in corticosteroid dose from baseline
Change from baseline of daily corticosteroids dose
Percentage change from baseline in daily corticosteroid dose
Percentage change from baseline of daily corticosteroids dose
Proportion of participants who required rescue therapy at any visit
Rescue therapy will include new/increased dose of corticosteroids, transfusions, intravenous immunoglobulin (IVIG), and Erythropoietin.
Number of Participants with Treatment Emergent Adverse Events (TEAE)
Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05073458
Brief Title
Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia
Acronym
PATHWAY
Official Title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
March 15, 2022 (Actual)
Primary Completion Date
April 17, 2023 (Actual)
Study Completion Date
April 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Incyte Corporation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),
Detailed Description
Prospective participants must have primary wAIHA as well as other protocol-defined criteria. After participants have been determined to be eligible for the study, they will be randomized to 2:1, with stratification factor of corticosteroid dose and hemoglobin (Hgb <9 g/dL or ≥ 9 g/dL). Once a participant has completed the week 24 assessments in the double-blind period, the participant will have the opportunity to receive parsaclisib in the open-label treatment which will last up to another 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Warm Autoimmune Hemolytic Anemia (wAIHA)
Keywords
Warm Autoimmune Hemolytic Anemia (wAIHA)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Masking Description
Study will be a 24 week double-blind period followed by a 24 week open-label period, followed by a long term extension period.
Allocation
Randomized
Enrollment
13 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group A: Parsaclisib
Arm Type
Experimental
Arm Description
Participants will receive parsaclisib for 24 weeks (double-blind period). Participant who completed the double-blind period and tolerating the study treatment upon investigator's opinion will continue into open-label period for an additional 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
Arm Title
Group B: Placebo followed by Parsaclisib
Arm Type
Placebo Comparator
Arm Description
Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive parsaclisib in the 24 week open-label period. Participants may then continue to receive parsaclisib in a long-term extension period.
Intervention Type
Drug
Intervention Name(s)
parsaclisinib
Other Intervention Name(s)
INCB050465
Intervention Description
parsaclisib will be administered QD orally
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
placebo will be administered QD orally follwed by Parsaclisinib in the open label period
Primary Outcome Measure Information:
Title
Proportion of participants attaining a durable hemoglobin response
Description
Proportion of participants attaining a durable hemoglobin response, defined as hemoglobin ≥ 10 g/dL with an increase from baseline of ≥ 2 g/dL not attributed to rescue therapy at ≥ 3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.
Time Frame
Up to Week 24
Secondary Outcome Measure Information:
Title
Proportion of participants with a ≥ 3-point increase in FACIT-F score
Description
Increase is measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.
Time Frame
Up to Week 24
Title
Proportion of participants with a 50 m increase in a 6MWT
Description
Defined as an increase of 50 m using the Six-minute walk test, a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes.
Time Frame
Up to Week 24
Title
Change in FACIT-F score
Description
Change will be measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.
Time Frame
Up to 3 years
Title
Percent Change in FACIT-F
Description
will be measured by Functional Assessment of Chronic Illness Therapy - Fatigue questionnaire. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days.
Time Frame
Up to 3 years
Title
Change in hemoglobin
Description
Changes will be measured and compared in the hematology panel.
Time Frame
Up to 3 years
Title
Percentage change in hemoglobin
Description
Percentage change will be measured and compared in the hematology panel.
Time Frame
Up to 3 years
Title
Proportion of participants who received transfusions
Description
Proportion of participants who received transfusions.
Time Frame
Up to 48 weeks
Title
Change in corticosteroid dose from baseline
Description
Change from baseline of daily corticosteroids dose
Time Frame
Up to Week 24
Title
Percentage change from baseline in daily corticosteroid dose
Description
Percentage change from baseline of daily corticosteroids dose
Time Frame
Up to Week 24
Title
Proportion of participants who required rescue therapy at any visit
Description
Rescue therapy will include new/increased dose of corticosteroids, transfusions, intravenous immunoglobulin (IVIG), and Erythropoietin.
Time Frame
Up to 48 weeks
Title
Number of Participants with Treatment Emergent Adverse Events (TEAE)
Description
Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.
Time Frame
Up to 3 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
99 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of primary warm AIHA.
Participants who have at least 1 unsuccessful prior therapy for warm AIHA or unable to receive or tolerate other therapies.
Hemoglobin ≥ 6.5 to < 10 g/dL with symptoms of anemia at screening.
FACIT-F score ≤ 43 at screening.
Willingness to avoid pregnancy or fathering children.
Willingness to receive PJP prophylaxis.
Further inclusion criteria apply.
Exclusion Criteria:
Women who are pregnant, breastfeeding or who are planning a pregnancy.
Diagnosis of other types of AIHA (CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria).
Secondary warm AIHA from any cause or diagnosis of Evans syndrome.
Splenectomy less than 3 months before randomization.
Participants with a history or ongoing significant illness as assessed by the investigator.
Participants with a current of medical history of a malignancy within the past 5 years except basal or squamous cell skin cancer that has been removed and considered cured, or superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy.
Participants know to be infected with HIV, Hepatitis B, or hepatitis C.
Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine.
Participants with laboratory values outside of the protocol defined ranges.
Further exclusion criteria apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kathleen Butler, MD
Organizational Affiliation
Incyte Corporation
Official's Role
Study Director
Facility Information:
Facility Name
Investigative Site US005
City
Los Angeles
State/Province
California
ZIP/Postal Code
90089
Country
United States
Facility Name
Investigative Site US004
City
Whittier
State/Province
California
ZIP/Postal Code
90603
Country
United States
Facility Name
Investigative Site US006
City
Miami
State/Province
Florida
ZIP/Postal Code
33165
Country
United States
Facility Name
Investigative Site US012
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
Investigative Site US007
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Facility Name
Investigative Site US002
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Investigative Site US003
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27858
Country
United States
Facility Name
Investigative Site US009
City
Canton
State/Province
Ohio
ZIP/Postal Code
44718
Country
United States
Facility Name
Investigative Site US010
City
Easton
State/Province
Pennsylvania
ZIP/Postal Code
18045
Country
United States
Facility Name
Investigative Site US001
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37920
Country
United States
Facility Name
Investigative Site AT002
City
Salzburg CET
ZIP/Postal Code
A-5020
Country
Austria
Facility Name
Investigative Site AT001
City
Vienna
ZIP/Postal Code
01090
Country
Austria
Facility Name
Investigative Site BE001
City
La Louviere
ZIP/Postal Code
07100
Country
Belgium
Facility Name
Investigative Site BE002
City
Liege
ZIP/Postal Code
04000
Country
Belgium
Facility Name
Investigative Site CA001
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2P4
Country
Canada
Facility Name
Investigative Site FR002
City
Lille Cedex
ZIP/Postal Code
59037
Country
France
Facility Name
Investigative Site FR003
City
Marseille
ZIP/Postal Code
13285
Country
France
Facility Name
Investigative Site FR001
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Investigative Site DE001
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Investigative Site DE002
City
ULM
ZIP/Postal Code
89081
Country
Germany
Facility Name
Investigative Site IL002
City
Haifa
ZIP/Postal Code
31096
Country
Israel
Facility Name
Investigative Site IL001
City
Nahariya
ZIP/Postal Code
2210001
Country
Israel
Facility Name
Investigative Site IT003
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Investigative Site IT002
City
Milan
ZIP/Postal Code
20122
Country
Italy
Facility Name
Investigative Site IT001
City
Novara
ZIP/Postal Code
28100
Country
Italy
Facility Name
Investigative Site IT004
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Investigative Site IT006
City
Rome
ZIP/Postal Code
00168
Country
Italy
Facility Name
Investigative Site IT005
City
Trieste
ZIP/Postal Code
34125
Country
Italy
Facility Name
Investigative Site JP008
City
Fukuoka
ZIP/Postal Code
807-8556
Country
Japan
Facility Name
Investigative Site JP004
City
Isehara
ZIP/Postal Code
259-1193
Country
Japan
Facility Name
Investigative Site JP006
City
Nagoya
ZIP/Postal Code
467-8602
Country
Japan
Facility Name
Investigative Site JP009
City
Okayama
ZIP/Postal Code
700-8557
Country
Japan
Facility Name
Investigative Site JP002
City
Okayama
ZIP/Postal Code
701-0192
Country
Japan
Facility Name
Investigative Site JP010
City
Osakasayama-shi
ZIP/Postal Code
589-8511
Country
Japan
Facility Name
Investigative Site JP005
City
Saitama
ZIP/Postal Code
350-0495
Country
Japan
Facility Name
Investigative Site JP007
City
Sendai-shi
ZIP/Postal Code
980-8574
Country
Japan
Facility Name
Investigative Site JP001
City
Suita-shi
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Investigative Site JP003
City
Tokyo
ZIP/Postal Code
141-8625
Country
Japan
Facility Name
Investigative Site NL001
City
Rotterdam
ZIP/Postal Code
3015CA
Country
Netherlands
Facility Name
Investigative Site PL001
City
Legnica
ZIP/Postal Code
59220
Country
Poland
Facility Name
Investigative Site PL006
City
Lodz
ZIP/Postal Code
93-510
Country
Poland
Facility Name
Investigative Site PL003
City
Nowy Sacz
ZIP/Postal Code
33-300
Country
Poland
Facility Name
Investigative Site PL005
City
Opole
ZIP/Postal Code
45-372
Country
Poland
Facility Name
Investigative Site PL004
City
Walbrzych
ZIP/Postal Code
58-309
Country
Poland
Facility Name
Investigative Site PL002
City
Wroclaw
ZIP/Postal Code
53-439
Country
Poland
Facility Name
Investigative Site ES006
City
Badalona
ZIP/Postal Code
08916
Country
Spain
Facility Name
Investigative Site ES001
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Investigative Site ES003
City
Madrid
ZIP/Postal Code
28006
Country
Spain
Facility Name
Investigative Site ES005
City
Murcia
ZIP/Postal Code
30008
Country
Spain
Facility Name
Investigative Site ES004
City
Tarragona
ZIP/Postal Code
43005
Country
Spain
Facility Name
Investigative Site ES002
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Investigative Site GB002
City
Glasgow
ZIP/Postal Code
G4 0SF
Country
United Kingdom
Facility Name
Investigative Site GB006
City
London
ZIP/Postal Code
W12 0HS
Country
United Kingdom
Facility Name
Investigative Site GB003
City
Norwich
ZIP/Postal Code
NR4 7UY
Country
United Kingdom
Facility Name
Investigative Site GB004
City
Plymouth
ZIP/Postal Code
PL6 8DH
Country
United Kingdom
Facility Name
Investigative Site GB005
City
Reading
ZIP/Postal Code
RG1 5AN
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency
IPD Sharing Time Frame
Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
IPD Sharing Access Criteria
Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement
IPD Sharing URL
https://www.incyte.com/our-company/compliance-and-transparency
Learn more about this trial
Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia
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