Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)

NCT ID: NCT05002777

Last Updated: 2025-05-21

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 22 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-07
• Study Completion Date: 2029-12-31

Brief Summary

All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B can continue in the Core Part B period followed by an Extended Part B period for up to 253 weeks.

There will be a 7-day safety follow-up period after receiving the last dose of study medication either in Part A (for those not eligible for Part B or early terminated) or Part B. In addition, each participant will be asked to attend an EOT-Core Part B visit when the last participant completes 52 weeks in Core Part B. The Extended Part B period will last for up to 253 weeks.

Conditions

Warm Autoimmune Hemolytic Anemia (wAIHA)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Rilzabrutinib

Oral rilzabrutinib 400 mg BID

Group Type: EXPERIMENTAL

rilzabrutinib

Intervention Type: DRUG

Pharmaceutical form: Tablet Route of administration: Oral

Interventions

rilzabrutinib

Pharmaceutical form: Tablet Route of administration: Oral

Intervention Type: DRUG

Other Intervention Names

PRN1008/SAR444671

Eligibility Criteria

Inclusion Criteria

• Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)
• Participants who have previously failed to maintain a sustained response after treatment with corticosteroids.
• Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
• Up-to-date vaccination status as per local guidelines.
• Body mass index (BMI) >17.5 and <40 kg/m2
• All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Core Part B

• Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A.
• Completion of Part A treatment period (24 weeks). Extended Part B
• Completion of Core Part B period.

Exclusion Criteria

• Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator.
• Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
• Secondary wAIHA from any cause including drugs, lymphoproliferative disorders (low-count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
• Myelodysplastic syndrome.
• Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA.
• HIV infection.
• Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half-lives, whichever is greater, prior to treatment start.
• Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent.

Part B only

• Participants who receive any therapy during Part A known to be active in wAIHA.
• Presence of unacceptable side effect(s) or toxicity associated with rilzabrutinib such that there is an unfavorable risk-benefit assessment for continued treatment with rilzabrutinib in the opinion of the Investigator and/or Sponsor.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

The Oncology Institute of Hope & Innovation Site Number : 8400006

Cerritos, California, United States

TOI Clinical Research LLC_ Cerritos_Investigational Site Number: 8400006

Cerritos, California, United States

TOI Clinical Research LLC_Glendale_Investigational Site Number: 8400006

Glendale, California, United States

TOI Clinical Research LLC_Long Beach_Investigational Site Number: 8400006

Long Beach, California, United States

University of Southern California_Investigational Site Number: 8400009

Los Angeles, California, United States

TOI Clinical Research LLC_Santa Ana_Investigational Site Number: 8400006

Santa Ana, California, United States

The Lundquist Institute_Investigational Site Number: 8400005

Torrance, California, United States

TOI Clinical Research LLC_ Whittier_Investigational Site Number: 8400006

Whittier, California, United States

Georgetown University Hospital_Investigational Site Number: 8400003

Washington D.C., District of Columbia, United States

Oncology & Hematology Associates of West Broward_Investigational Site Number: 8400002

Tamarac, Florida, United States

Massachusetts General Hospital_Investigational Site Number: 8400001

Boston, Massachusetts, United States

Hanush-Krankenhaus_Investigational Site Number: 0400001

Vienna, , Austria

Peking Union Medical College Hospital_Investigational Site Number: 1560002

Beijing, , China

Institute of hematology&blood diseases hospital_Investigational Site Number: 1560003

Tianjin, , China

Odense Universitetshospital Hæmatologisk Forskningsenhed_Investigational Site Number: 2080001

Odense, , Denmark

Klinik für Hämatologie und Stammzellentransplantation_Investigational Site Number: 2760001

Essen, , Germany

Fejer Varmegyei Szent Gyorgy Egyetemi Oktato Korhaz_Investigational Site Number: 3480001

Székesfehérvár, , Hungary

Ospedale Giuseppe Moscati_Investigational Site Number: 3800002

Avellino, , Italy

Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori"_Investigational Site Number: 3800003

Meldola, , Italy

Ospedale Maggiore Policlinico_Investigational Site Number: 3800001

Milan, , Italy

Hospital Universitario de Cruces_Investigational Site Number: 7240004

Barakaldo, , Spain

Hospital Clinic de Barcelona_Investigational Site Number: 7240001

Barcelona, , Spain

Hospital Universitario La Paz_Investigational Site Number: 7240003

Madrid, , Spain

Hospital Universitario Virgen del Rocío_Investigational Site Number: 7240002

Seville, , Spain

Leeds Teaching Hospitals NHS Trust_Investigational Site Number: 8260001

Leeds, , United Kingdom

Barts Health NHS Trust_Investigational Site Number: 8260005

London, , United Kingdom

Imperial College Healthcare NHS Trust_Investigational Site Number: 8260002

London, , United Kingdom

Countries

United States; Austria; China; Denmark; Germany; Hungary; Italy; Spain; United Kingdom

Other Identifiers

2021-001671-16

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1262-2929

Identifier Type: REGISTRY

Identifier Source: secondary_id

2023-509441-13

Identifier Type: REGISTRY

Identifier Source: secondary_id

ACT17209

Identifier Type: -

Identifier Source: org_study_id