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

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ClinicalTrials.gov Identifier: NCT05073458
Recruitment Status : Recruiting
First Posted : October 11, 2021
Last Update Posted : November 17, 2022
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation

Study Description
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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),

Condition or disease Intervention/treatment Phase
Warm Autoimmune Hemolytic Anemia (wAIHA) Drug: parsaclisinib Drug: placebo 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 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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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 period, followed by a long term 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 : March 15, 2022
Estimated Primary Completion Date : August 15, 2024
Estimated Study Completion Date : April 22, 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arms and Interventions
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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 period for an additional 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
 Drug: parsaclisinib
parsaclisib will be administered QD orally
Other Name: 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 period. Participants may then continue to receive parsaclisib in a long-term extension period.
 Drug: placebo
placebo will be administered QD orally follwed by Parsaclisinib in the open label period


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of participants attaining a durable hemoglobin response [ Time Frame: Up to Week 24 ]
    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 [ Time Frame: Up 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 [ Time Frame: Up to Week 24 ]
    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 [ Time Frame: Up to 3 years ]
    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. Percent Change in FACIT-F [ Time Frame: Up to 3 years ]
    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.

  5. Change in hemoglobin [ Time Frame: Up to 3 years ]
    Changes will be measured and compared in the hematology panel.

  6. Percentage change in hemoglobin [ Time Frame: Up to 3 years ]
    Percentage change will be measured and compared in the hematology panel.

  7. Proportion of participants who received transfusions [ Time Frame: Up to 48 weeks ]
    Proportion of participants who received transfusions.

  8. Change in corticosteroid dose from baseline [ Time Frame: Up to Week 24 ]
    Change from baseline of daily corticosteroids dose

  9. Percentage change from baseline in daily corticosteroid dose [ Time Frame: Up to Week 24 ]
    Percentage change from baseline of daily corticosteroids dose

  10. Proportion of participants who required rescue therapy at any visit [ Time Frame: Up to 48 weeks ]
    Rescue therapy will include new/increased dose of corticosteroids, transfusions, intravenous immunoglobulin (IVIG), and Erythropoietin.

  11. Number of Participants with Treatment Emergent Adverse Events (TEAE) [ Time Frame: Up to 3 years ]
    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
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Ages Eligible for Study:   18 Years to 99 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05073458


Contacts
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Contact: Incyte Corporation Call Center (US) 1.855.463.3463 medinfo@incyte.com
Contact: Incyte Corporation Call Center (ex-US) +800 00027423 eumedinfo@incyte.com

Locations
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Sponsors and Collaborators
Incyte Corporation
Investigators
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Study Director: Kathleen Butler, MD Incyte Corporation
More Information
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Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT05073458    
Other Study ID Numbers: INCB 50465-309
First Posted: October 11, 2021    Key Record Dates
Last Update Posted: November 17, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
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
URL: https://www.incyte.com/our-company/compliance-and-transparency

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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:
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Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hemolysis
 Hematologic Diseases
Pathologic Processes
Autoimmune Diseases
Immune System Diseases