Recruiting
Phase 3

Rilzabrutinib

Sponsor:

Sanofi

Code:

NCT07086976

Conditions

Autoimmune Haemolytic Anaemia

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

rilzabrutinib

placebo

Study Details

Brief summary:

This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb response (DHR) compared with placebo in approximately 90 male and female participants ≥ 18 years of age with a confirmed diagnosis of primary wAIHA.

Following a 4-week screening period, eligible participants will be randomized in a 2:1 ratio to receive rilzabrutinib or placebo in primary analysis period (PAP) for a duration of up to 24 weeks. All participants who completed PAP will then continue in open-label period (OLP) to receive rilzabrutinib for a duration of 28 weeks. Upon the completion of OLP, only participants who demonstrate Hb increase during the last 8 weeks of OLP per specified criteria in the protocol will be eligible to continue in long-term extension (LTE) of the study. The duration of the LTE period will be from the first-participant-in (FPI)-LTE until the last participant completes 52 weeks in LTE. The safety follow-up period of this study following treatment completion or discontinuation will be 2 weeks.

Conditions

Autoimmune Haemolytic Anaemia

Study ID

NCT07086976

Start date

Aug 18, 2025

Status verified date

Aug, 2026

Completion date

Dec 26, 2029

Anticipated

Primary completion date

May 31, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Male and female participants with a documented (confirmed) diagnosis of primary wAIHA for at least 6 months.
  • Participants who have previously failed to maintain a sustained response after treatment with CS (CS-resistance \[defined as failure to obtain hemoglobin response within 3 weeks on at least 1 mg/kg or 60 mg prednisone or equivalent per day\], CS-dependent wAIHA \[defined as need to continue on prednisone or equivalent at a dose of >10 mg/day to maintain a response\]), or are intolerant or ineligible to CS (defined as with contraindications, pre-existing medical conditions or CS-related complications that may render CS intolerant or ineligible per the best clinical judgement of the investigators).
  • Participants with Eastern Cooperative Oncology Group (ECOG) performance status Grade 2 or lower.
  • Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion Criteria:

  • Participants with 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.
  • Participants with symptomatic herpes zoster within 3 months prior to screening.
  • Participants with mixed wAIHA, or secondary wAIHA from any cause including drugs, Evans Syndrome, 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.
  • Participants with history of myelodysplastic syndrome.
  • Participants with uncontrolled or active HBV infection or Active HCV infection.
  • HIV infection.
  • Participants with history of solid organ transplant.
  • Participants with a history of active or latent tuberculosis (TB).
  • Splenectomy within 12 weeks before screening and planned surgery during the PAP.
  • Concurrent treatment with other experimental/investigational drugs within 30 days or 5 half-lives, whichever is longer, prior to treatment start. Participants who previously received treatment with BTK inhibitors for wAIHA before Day 1 (randomization) are not eligible.
  • Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.

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

Study Design

Enrollment

90 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: rilzabrutinib

Oral rilzabrutinib BID

placebo comparator: placebo

Oral placebo BID

Interventions

rilzabrutinib

Pharmaceutical form:tablet-Route of administration:oral

placebo

Pharmaceutical form:tablet-Route of administration:oral

Primary outcome measure

  • Proportion of participants achieving DHR. DHR is defined as an increase of Hb by ≥2 g/dL from baseline on at least two thirds of evaluable scheduled visits between Week 12 and Week 24 (inclusive) in the PAP [ Time Frame: By Week 24 ]

Central Contacts and Locations

Central contacts

Trial Transparency email recommended (Toll free for US & Canada)

800-633-1610contact-us@sanofi.com

Locations

Mayo Clinic in Arizona - Phoenix- Site Number : 8400032

Recruiting

Phoenix, Arizona, United States, 85054

Noble Clinical Research- Site Number : 8400003

Recruiting

Tucson, Arizona, United States, 85704

City of Hope National Medical Center- Site Number : 8400023

Recruiting

Duarte, California, United States, 91010

USC Norris Comprehensive Cancer Center- Site Number : 8400007

Recruiting

Los Angeles, California, United States, 90033

Stanford University Medical Center- Site Number : 8400026

Recruiting

Stanford, California, United States, 94305

Lundquist Institute for Biomedical Innovation at Harbor - UCLA Medical Center- Site Number : 8400006

Recruiting

Torrance, California, United States, 90502

Yale University School of Medicine- Site Number : 8400027

Recruiting

New Haven, Connecticut, United States, 06510

MedStar Georgetown University Hospital- Site Number : 8400004

Recruiting

Washington D.C., District of Columbia, United States, 20007

Hialeah Hospital- Site Number : 8400009

Recruiting

Hialeah, Florida, United States, 33013

Sylvester Comprehensive Cancer Center- Site Number : 8400031

Recruiting

Miami, Florida, United States, 33136

University Hospital and Medical Center- Site Number : 8400005

Recruiting

Tamarac, Florida, United States, 33321

Massachusetts General Hospital- Site Number : 8400001

Recruiting

Boston, Massachusetts, United States, 02114

University of Michigan Health System - Ann Arbor- Site Number : 8400014

Recruiting

Ann Arbor, Michigan, United States, 48109

Henry Ford Hospital- Site Number : 8400012

Recruiting

Detroit, Michigan, United States, 48202

Mayo Clinic in Rochester - Minnesota- Site Number : 8400008

Recruiting

Rochester, Minnesota, United States, 55905

Montefiore Medical Center - Einstein Campus- Site Number : 8400011

Recruiting

The Bronx, New York, United States, 10461

Ohio State University Hospital East- Site Number : 8400020

Recruiting

Columbus, Ohio, United States, 43203

Fox Chase Cancer Center - Philadelphia- Site Number : 8400019

Recruiting

Philadelphia, Pennsylvania, United States, 19111

Brown University Health - Rhode Island Hospital- Site Number : 8400028

Recruiting

Providence, Rhode Island, United States, 02903

Baptist Memorial Hospital- Site Number : 8400018

Recruiting

Memphis, Tennessee, United States, 38120

UT Health - San Antonio- Site Number : 8400015

Recruiting

San Antonio, Texas, United States, 78229

Fred Hutchinson Cancer Center- Site Number : 8400016

Recruiting

Seattle, Washington, United States, 98109

Gundersen Health System - La Crosse Medical Center- Site Number : 8400033

Recruiting

La Crosse, Wisconsin, United States, 54601

More Information

Sponsor

Sanofi

Last update posted

Aug 21, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Sanofi on 2026-08-21.