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RECRUITING

NCT07086976

PHASE3

A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia

Sponsor: Sanofi

View on ClinicalTrials.gov

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.

Official title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study With an Open-label Period and Long-term Extension to Assess the Efficacy and Safety of Rilzabrutinib in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2025-08-18

Completion Date

2029-12-26

Last Updated

2026-03-31

Healthy Volunteers

Conditions

Autoimmune Haemolytic Anaemia

Interventions

DRUG

rilzabrutinib

Pharmaceutical form:tablet-Route of administration:oral

DRUG

placebo

Pharmaceutical form:tablet-Route of administration:oral

Inclusion Criteria: * Male and female participants with a documented (confirmed) diagnosis of primary wAIHA for at least 3 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 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). * 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.

Locations (91)

Mayo Clinic in Arizona - Phoenix- Site Number : 8400032

Phoenix, Arizona, United States

Noble Clinical Research- Site Number : 8400003

Tucson, Arizona, United States

City of Hope National Medical Center- Site Number : 8400023

Duarte, California, United States

USC Norris Comprehensive Cancer Center- Site Number : 8400007

Los Angeles, California, United States

Stanford University Medical Center- Site Number : 8400026

Stanford, California, United States

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

Torrance, California, United States

Yale University School of Medicine- Site Number : 8400027

New Haven, Connecticut, United States

Hialeah Hospital- Site Number : 8400009

Hialeah, Florida, United States

Sylvester Comprehensive Cancer Center- Site Number : 8400031

Miami, Florida, United States

University Hospital and Medical Center- Site Number : 8400005

Tamarac, Florida, United States

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

Ann Arbor, Michigan, United States

Henry Ford Hospital- Site Number : 8400012

Detroit, Michigan, United States

Mayo Clinic in Rochester - Minnesota- Site Number : 8400008

Rochester, Minnesota, United States

Montefiore Medical Center - Einstein Campus- Site Number : 8400011

The Bronx, New York, United States

Ohio State University Hospital East- Site Number : 8400020

Columbus, Ohio, United States

Fox Chase Cancer Center - Philadelphia- Site Number : 8400019

Philadelphia, Pennsylvania, United States

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

Providence, Rhode Island, United States

Baptist Memorial Hospital- Site Number : 8400018

Memphis, Tennessee, United States

UT Health - San Antonio- Site Number : 8400015

San Antonio, Texas, United States

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

La Crosse, Wisconsin, United States

Investigational Site Number : 0320001

Rosario, Santa Fe Province, Argentina

Investigational Site Number : 0320003

Buenos Aires, Argentina

Investigational Site Number : 0320002

Buenos Aires, Argentina

Investigational Site Number : 0400001

Vienna, Austria

Investigational Site Number : 0400002

Vienna, Austria

Hemoes - Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos- Site Number : 0760002

Vitória, Espírito Santo, Brazil

Universidade Federal de Goias- Site Number : 0760001

Goiânia, Goiás, Brazil

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760003

São Paulo, Brazil

Investigational Site Number : 1560001

Beijing, China

Investigational Site Number : 1560007

Chengde, China

Investigational Site Number : 1560011

Guangzhou, China

Investigational Site Number : 1560005

Guangzhou, China

Investigational Site Number : 1560009

Hohhot, China

Investigational Site Number : 1560003

Nanchang, China

Investigational Site Number : 1560012

Nanchang, China

Investigational Site Number : 1560004

Suzhou, China

Investigational Site Number : 1560002

Tianjin, China

Investigational Site Number : 1560010

Wuhan, China

Investigational Site Number : 1560006

Xi'an, China

Investigational Site Number : 1560008

Zhengzhou, China

Investigational Site Number : 2030002

Prague, Cardiff [Caerdydd Gb-crd], Czechia

Investigational Site Number : 2030001

Brno, Czechia

Investigational Site Number : 2080003

Aarhus, Denmark

Investigational Site Number : 2080002

Copenhagen, Denmark

Investigational Site Number : 2080001

Odense, Denmark

Investigational Site Number : 2760003

Berlin, Germany

Investigational Site Number : 2760001

Essen, Germany

Investigational Site Number : 3000002

Athens, Greece

Investigational Site Number : 3000005

Larissa, Greece

Investigational Site Number : 3000004

Pátrai, Greece

Investigational Site Number : 3000003

Pátrai, Greece

Investigational Site Number : 3000001

Thessaloniki, Greece

Investigational Site Number : 3480002

Debrecen, Hungary

Investigational Site Number : 3760001

Afula, Israel

Investigational Site Number : 3760007

Haifa, Israel

Investigational Site Number : 3760005

Kefar Sava, Israel

Investigational Site Number : 3760002

Tel Aviv, Israel

Investigational Site Number : 3800004

Florence, Firenze, Italy

Investigational Site Number : 3800010

Genoa, Genova, Italy

Investigational Site Number : 3800001

Milan, Milano, Italy

Investigational Site Number : 3800005

Naples, Napoli, Italy

Investigational Site Number : 3800002

Meldola, Reggio Emilia, Italy

Investigational Site Number : 3800009

Novara, Italy

Investigational Site Number : 3800006

Palermo, Italy

Investigational Site Number : 3800007

Trieste, Italy

Investigational Site Number : 3800008

Varese, Italy

Investigational Site Number : 3800003

Vicenza, Italy

Investigational Site Number : 3920007

Nagoya, Aichi-ken, Japan

Investigational Site Number : 3920013

Tsuchiura, Ibaraki, Japan

Investigational Site Number : 3920006

Kanazawa, Ishikawa-ken, Japan

Investigational Site Number : 3920012

Fujisawa, Kanagawa, Japan

Investigational Site Number : 3920003

Suita, Osaka, Japan

Investigational Site Number : 3920001

Iruma, Saitama, Japan

Investigational Site Number : 3920010

Kofu, Yamanashi, Japan

Investigational Site Number : 3920015

Fukuoka, Japan

Investigational Site Number : 3920002

Fukushima, Japan

Investigational Site Number : 3920014

Kyoto, Japan

Investigational Site Number : 3920008

Okayama, Japan

Investigational Site Number : 3920004

Tokushima, Japan

Investigational Site Number : 3920005

Yamagata, Japan

Investigational Site Number : 5280002

Amsterdam, Netherlands

Investigational Site Number : 5280003

Leiden, Netherlands

Investigational Site Number : 6160005

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland

Investigational Site Number : 6160006

Lodz, Lódzkie, Poland

Investigational Site Number : 7240005

Barakaldo, Basque Country, Spain

Investigational Site Number : 7240001

Barcelona, Catalunya [Cataluña], Spain

Investigational Site Number : 7240007

Majadahonda, Madrid, Spain

Investigational Site Number : 7240003

Seville, Sevilla, Spain

Investigational Site Number : 7240004

Madrid, Spain

Investigational Site Number : 7520001

Huddinge, Sweden

Investigational Site Number : 7520003

Malmo, Sweden