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

NCT05073458 | Terminated Phase 3 Results DMC FDA Drug

• 2 other identifiers: INCB 50465-3092021-002844-66
• Study Type: interventional
• Target: 13
• Locations: 13 countries
• Sites: 56

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),

Conditions

Warm Autoimmune Hemolytic Anemia (wAIHA)

Keywords

Warm Autoimmune Hemolytic Anemia (wAIHA)

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Attaining a Durable Hemoglobin Response

  A durable hemoglobin response was defined as hemoglobin ≥10 grams per deciliter (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.

  up to Week 24

Secondary Outcomes (12)

• Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24

  Baseline; Week 24

• Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT)

  Baseline; Week 24

• Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit

  Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56

• Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit

  Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56

• Change From Baseline in Hemoglobin at Each Post-Baseline Visit

  Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56

Study Arms (2)

Group A: Parsaclisib

EXPERIMENTAL

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

Group B: Placebo followed by Parsaclisib

PLACEBO COMPARATOR

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

Interventions

parsaclisinib DRUG

parsaclisib will be administered QD orally

Also known as: INCB050465

Group A: Parsaclisib

placebo DRUG

placebo will be administered QD orally follwed by Parsaclisinib in the open label period

Group B: Placebo followed by Parsaclisib

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (56)

Investigative Site US005

Los Angeles, California, 90089, United States

Location

Investigative Site US004

Whittier, California, 90603, United States

Location

Investigative Site US006

Miami, Florida, 33165, United States

Location

Investigative Site US012

Indianapolis, Indiana, 46260, United States

Location

Investigative Site US007

The Bronx, New York, 10461, United States

Location

Investigative Site US002

The Bronx, New York, 10467, United States

Location

Investigative Site US003

Greenville, North Carolina, 27858, United States

Location

Investigative Site US009

Canton, Ohio, 44718, United States

Location

Investigative Site US010

Easton, Pennsylvania, 18045, United States

Location

Investigative Site US001

Knoxville, Tennessee, 37920, United States

Location

Investigative Site AT002

Salzburg CET, A-5020, Austria

Location

Investigative Site AT001

Vienna, 01090, Austria

Location

Investigative Site BE001

La Louvière, 07100, Belgium

Location

Investigative Site BE002

Liège, 04000, Belgium

Location

Investigative Site CA001

Edmonton, Alberta, T6G 2P4, Canada

Location

Investigative Site FR002

Lille, 59037, France

Location

Investigative Site FR003

Marseille, 13285, France

Location

Investigative Site FR001

Paris, 75015, France

Location

Investigative Site DE001

Essen, 45147, Germany

Location

Investigative Site DE002

Ulm, 89081, Germany

Location

Investigative Site IL002

Haifa, 31096, Israel

Location

Investigative Site IL001

Nahariya, 2210001, Israel

Location

Investigative Site IT003

Florence, 50134, Italy

Location

Investigative Site IT002

Milan, 20122, Italy

Location

Investigative Site IT001

Novara, 28100, Italy

Location

Investigative Site IT004

Pavia, 27100, Italy

Location

Investigative Site IT006

Rome, 00168, Italy

Location

Investigative Site IT005

Trieste, 34125, Italy

Location

Investigative Site JP008

Fukuoka, 807-8556, Japan

Location

Investigative Site JP004

Isehara, 259-1193, Japan

Location

Investigative Site JP006

Nagoya, 467-8602, Japan

Location

Investigative Site JP009

Okayama, 700-8557, Japan

Location

Investigative Site JP002

Okayama, 701-0192, Japan

Location

Investigative Site JP010

Osakasayama-shi, 589-8511, Japan

Location

Investigative Site JP005

Saitama, 350-0495, Japan

Location

Investigative Site JP007

Sendai, 980-8574, Japan

Location

Investigative Site JP001

Suita-shi, 565-0871, Japan

Location

Investigative Site JP003

Tokyo, 141-8625, Japan

Location

Investigative Site NL001

Rotterdam, 3015CA, Netherlands

Location

Investigative Site PL001

Legnica, 59220, Poland

Location

Investigative Site PL006

Lodz, 93-510, Poland

Location

Investigative Site PL003

Nowy Sącz, 33-300, Poland

Location

Investigative Site PL005

Opole, 45-372, Poland

Location

Investigative Site PL004

Wałbrzych, 58-309, Poland

Location

Investigative Site PL002

Wroclaw, 53-439, Poland

Location

Investigative Site ES006

Badalona, 08916, Spain

Location

Investigative Site ES001

Barcelona, 08036, Spain

Location

Investigative Site ES003

Madrid, 28006, Spain

Location

Investigative Site ES005

Murcia, 30008, Spain

Location

Investigative Site ES004

Tarragona, 43005, Spain

Location

Investigative Site ES002

Valencia, 46026, Spain

Location

Investigative Site GB002

Glasgow, G4 0SF, United Kingdom

Location

Investigative Site GB006

London, W12 0HS, United Kingdom

Location

Investigative Site GB003

Norwich, NR4 7UY, United Kingdom

Location

Investigative Site GB004

Plymouth, PL6 8DH, United Kingdom

Location

Investigative Site GB005

Reading, RG1 5AN, United Kingdom

Location

MeSH Terms

Interventions

parsaclisib

Results Point of Contact

• Title: Study Director
• Organization: Incyte Corporation

medinfo@incyte.com

1-855-463-3463

Study Officials

• Kathleen Butler, MD

  Incyte Corporation

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Study will be a 24 week double-blind period followed by a 24 week open-label period, followed by a long term extension period.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 28, 2021
• First Posted: October 11, 2021
• Study Start: March 15, 2022
• Primary Completion: October 17, 2023
• Study Completion: April 29, 2024
• Last Updated: November 3, 2025
• Results First Posted: January 15, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, 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

Locations

FR (3); NL (1); AU (2); IL (2); BE (2); PL (6); DE (2); ES (6); GB (5); US (10); IT (6); JP (10); CA (1)