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PATHWAY: Study of the Efficacy and Safety of Parsaclisib in Participants With Primary Warm Autoimmune Hemolytic Anemia

Sponsor
Incyte Corporation (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05073458
Collaborator
(none)
100
Enrollment
53
Locations
2
Arms
26.7
Anticipated Duration (Months)
1.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of parsclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),

Condition or Disease Intervention/Treatment Phase
Phase 3

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 1 of 2 treatment groups, with stratification for hemoglobin (Hgb <9 g/dL or ≥ 9 g/dL). Once a participant has completed the week 24 assessments, the participant will have the opportunity to crossover to begin receiving parsaclisib in the open-label treatment which will last up to another 24 weeks in duration.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
100 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Care Provider, Investigator)
Masking Description:
Study will be a 24 week double-blind period followed by a 24 week open-label extension period,.
Primary Purpose:
Treatment
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
Actual Study Start Date :
Mar 15, 2022
Anticipated Primary Completion Date :
Mar 4, 2024
Anticipated Study Completion Date :
Jun 4, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Group A: Parsaclisib

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 extension period for an additional 24 weeks. starting from Day 1 for 24 weeks.

Drug: parsaclisinib
parsaclisib will be administered QD orally
Other Names:
  • INCB050465

    		•
    Placebo Comparator: Group B: Placebo followed by Parsaclisib

    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 extension period.

    Drug: placebo
    placebo will be administered QD orally

    Outcome Measures

    Primary Outcome Measures

    1. Proportion of participants attaining a durable hemoglobin response [Baseline up to 24 weeks]

      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

    1. Proportion of participants with a ≥ 3-point increase in FACIT-F score [Baseline to Week 24]

      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.

    2. Proportion of participants with a 50 m increase in a 6MWT [Baseline to 24 weeks]

      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.

    3. Change in FACIT-F score [Up to 15 months]

      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.

    4. Change in hemoglobin [Up to 15 months]

      Changes will be measured and compared in the hematology panel.

    5. Percentage change in hemoglobin [Up to 15 months]

      Percentage change will be measured and compared in the hematology panel.

    6. Proportion of participants who received transfusions [Up to 48 weeks]

      Proportion of participants who received transfusions.

    7. Change in corticosteroid dose from baseline [Week 24]

      Change from baseline of daily corticosteroids dose

    8. Percentage change from baseline in daily corticosteroid dose [Up to 24 weeks]

      Percentage change from baseline of daily corticosteroids dose

    9. Proportion of participants who required rescue therapy at any visit [Up to 48 weeks]

      Rescue therapy will include new/increased dose of corticosteroids, transfusions, intravenous immunoglobulin (IVIG), and Epotein alfa.

    10. Number of Participants with Treatment Emergent Adverse Events (TEAE) [Up to 15 months]

      Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 99 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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 ≥ 7 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.

    • 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Investigative Site US005 Los Angeles California United States 90089
    2 Investigative Site US004 Whittier California United States 90603
    3 Investigative Site US006 Miami Florida United States 33165
    4 Investigative Site US001 Knoxville Kentucky United States 37920
    5 Investigative Site US007 Bronx New York United States 10461
    6 Investigative Site US002 Bronx New York United States 10467
    7 Investigative Site US003 Greenville North Carolina United States 27834
    8 Investigative Site AT002 Salzburg Austria 5020
    9 Investigative Site AT001 Wien Austria 1090
    10 Investigative Site BE001 La Louviere Belgium 7100
    11 Investigative Site BE002 Liege Belgium 4000
    12 Investigative Site CA001 Edmonton Canada T6G 2P4
    13 Investigative Site FR002 Lille France 59037
    14 Investigative Site FR003 Marseille France 13285
    15 Investigative Site FR001 Paris France 75015
    16 Investigative Site DE001 Essen Germany 45147
    17 Investigative Site DE002 Ulm Germany 89081
    18 Investigative Site IL002 Haifa Israel 31096
    19 Investigative Site IL001 Nahariya Israel 2210001
    20 Investigative Site IT003 Firenze Italy 50134
    21 Investigative Site IT002 Milan Italy 20122
    22 Investigative Site IT001 Novara Italy 28100
    23 Investigative Site IT004 Pavia Italy 27100
    24 Investigative Site IT006 Rome Italy 00168
    25 Investigative Site IT005 Trieste Italy 34125
    26 Investigative Site JP008 Fukuoka Japan 807-8556
    27 Investigative Site JP004 Isehara Japan 259-1193
    28 Investigative Site JP006 Nagoya Japan 467-8602
    29 Investigative Site JP009 Okayama Japan 700-8557
    30 Investigative Site JP002 Okayama Japan 701-0192
    31 Investigative Site JP010 Osaka Japan 589-8511
    32 Investigative Site JP005 Saitama Japan 350-0495
    33 Investigative Site 007 Sendai-shi Japan 980-8574
    34 Investigative Site JP001 Suita-shi Japan 565-0871
    35 Investigative Site JP003 Tokyo Japan 141-8625
    36 Investigative Site NL 001 Rotterdam Netherlands 3015CA
    37 Investigative Site PL001 Legnica Poland 59220
    38 Investigative Site PL006 Lodz Poland 93-510
    39 Investigative Site PL003 Nowy Sacz Poland 33-300
    40 Investigative Site PL005 Opole Poland 45-372
    41 Investigative Site PL004 Walbrzych Poland 58-309
    42 Investigative Site PL002 Wroclaw Poland 53-439
    43 Investigative Site ES006 Badalona Spain 08916
    44 Investigative Site ES001 Barcelona Spain 08036
    45 Investigative Site ES003 Madrid Spain 28006
    46 Investigative Site ES005 Murcia Spain 30008
    47 Investigative Site ES004 Tarragona Spain 43005
    48 Investigative Site ES002 Valencia Spain 46026
    49 Investigative Site GB001 Carlisle United Kingdom CA 144LS
    50 Investigative Site GB002 Glasgow United Kingdom G4 0SF
    51 Investigative Site GB003 Norwich United Kingdom NR4 7UY
    52 Investigative Site GB004 Plymouth United Kingdom PL68DH
    53 Investigative Site GB005 Reading United Kingdom RG1 5AN

    Sponsors and Collaborators

    • Incyte Corporation

    Investigators

    • Study Director: Kathleen Butler, MD, Incyte Corporation

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Incyte Corporation
    ClinicalTrials.gov Identifier:
    NCT05073458
    Other Study ID Numbers:
    • INCB 50465-309
    First Posted:
    Oct 11, 2021
    Last Update Posted:
    May 3, 2022
    Last Verified:
    May 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Incyte Corporation
    Warm Autoimmune Hemolytic Anemia (wAIHA)
    Additional relevant MeSH terms:
    Anemia
    Anemia, Hemolytic
    Anemia, Hemolytic, Autoimmune
    Hemolysis

    Study Results

    No Results Posted as of May 3, 2022