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NCT07086976PHASE3Recruiting

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

Sanofi

Start Date

8/18/2025

Completion Date

12/26/2029

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.

Eligibility Criteria

Age Range: 18 years to No maximum

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.

Interventions

DRUG

rilzabrutinib

DRUG

placebo

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Conditions

Autoimmune Haemolytic Anaemia

Locations

Mayo Clinic in Arizona - Phoenix- Site Number : 8400032

Phoenix, Arizona 85054

United States

Noble Clinical Research- Site Number : 8400003

Tucson, Arizona 85704

United States

City of Hope National Medical Center- Site Number : 8400023

Duarte, California 91010

United States

USC Norris Comprehensive Cancer Center- Site Number : 8400007

Los Angeles, California 90033

United States

Stanford University Medical Center- Site Number : 8400026

Stanford, California 94305

United States

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

Torrance, California 90502

United States

Yale University School of Medicine- Site Number : 8400027

New Haven, Connecticut 06510

United States

MedStar Georgetown University Hospital- Site Number : 8400004

Washington D.C., District of Columbia 20007

United States

Hialeah Hospital- Site Number : 8400009

Hialeah, Florida 33013

United States

Sylvester Comprehensive Cancer Center- Site Number : 8400031

Miami, Florida 33136

United States

University Hospital and Medical Center- Site Number : 8400005

Tamarac, Florida 33321

United States

Massachusetts General Hospital- Site Number : 8400001

Boston, Massachusetts 02114

United States

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

Ann Arbor, Michigan 48109

United States

Henry Ford Hospital- Site Number : 8400012

Detroit, Michigan 48202

United States

Mayo Clinic in Rochester - Minnesota- Site Number : 8400008

Rochester, Minnesota 55905

United States

Montefiore Medical Center - Einstein Campus- Site Number : 8400011

The Bronx, New York 10461

United States

Ohio State University Hospital East- Site Number : 8400020

Columbus, Ohio 43203

United States

Fox Chase Cancer Center - Philadelphia- Site Number : 8400019

Philadelphia, Pennsylvania 19111

United States

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

Providence, Rhode Island 02903

United States

Baptist Memorial Hospital- Site Number : 8400018

Memphis, Tennessee 38120

United States

UT Health - San Antonio- Site Number : 8400015

San Antonio, Texas 78229

United States

Fred Hutchinson Cancer Center- Site Number : 8400016

Seattle, Washington 98109

United States

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

La Crosse, Wisconsin 54601

United States

Investigational Site Number : 0320001

Rosario, Santa Fe Province 2000

Argentina

Investigational Site Number : 0320003

Buenos Aires, 1181

Argentina

Investigational Site Number : 0320002

Buenos Aires, 1431

Argentina

Investigational Site Number : 0400001

Vienna, 1090

Austria

Investigational Site Number : 0400002

Vienna, 1140

Austria

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

Vitória, Espírito Santo 29047-105

Brazil

Universidade Federal de Goias- Site Number : 0760001

Goiânia, Goiás 74605-020

Brazil

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

São Paulo, 05403-000

Brazil

Investigational Site Number : 1560001

Beijing, 100730

China

Investigational Site Number : 1560007

Chengde, 067020

China

Investigational Site Number : 1560011

Guangzhou, 510000

China

Investigational Site Number : 1560005

Guangzhou, 510180

China

Investigational Site Number : 1560009

Hohhot, 010050

China

Investigational Site Number : 1560003

Nanchang, 330006

China

Investigational Site Number : 1560012

Nanchang, 330006

China

Investigational Site Number : 1560004

Suzhou, 215006

China

Investigational Site Number : 1560002

Tianjin, 300020

China

Investigational Site Number : 1560010

Wuhan, 430022

China

Investigational Site Number : 1560006

Xi'an, 710054

China

Investigational Site Number : 1560008

Zhengzhou, 450008

China

Investigational Site Number : 2030002

Prague, Cardiff [Caerdydd Gb-crd] 128 20

Czechia

Investigational Site Number : 2030001

Brno, 625 00

Czechia

Investigational Site Number : 2080003

Aarhus, 8200

Denmark

Investigational Site Number : 2080002

Copenhagen, 2100

Denmark

Investigational Site Number : 2080001

Odense, 5000

Denmark

Investigational Site Number : 2760003

Berlin, 10707

Germany

Investigational Site Number : 2760001

Essen, 45147

Germany

Investigational Site Number : 3000002

Athens, 115 27

Greece

Investigational Site Number : 3000005

Larissa, 411 10

Greece

Investigational Site Number : 3000004

Pátrai, 254 43

Greece

Investigational Site Number : 3000003

Pátrai, 265 04

Greece

Investigational Site Number : 3000001

Thessaloniki, 570 10

Greece

Investigational Site Number : 3480004

Budapest, 1085

Hungary

Investigational Site Number : 3480002

Debrecen, 4032

Hungary

Investigational Site Number : 3760001

Afula, 1834111

Israel

Investigational Site Number : 3760007

Haifa, 3104802

Israel

Investigational Site Number : 3760005

Kefar Sava, 4428164

Israel

Investigational Site Number : 3760002

Tel Aviv, 6423906

Israel

Investigational Site Number : 3800004

Florence, Firenze 50134

Italy

Investigational Site Number : 3800010

Genoa, Genova 16132

Italy

Investigational Site Number : 3800001

Milan, Milano 20122

Italy

Investigational Site Number : 3800005

Naples, Napoli 80131

Italy

Investigational Site Number : 3800002

Meldola, Reggio Emilia 47014

Italy

Investigational Site Number : 3800009

Novara, 28100

Italy

Investigational Site Number : 3800006

Palermo, 90146

Italy

Investigational Site Number : 3800007

Trieste, 34125

Italy

Investigational Site Number : 3800008

Varese, 21100

Italy

Investigational Site Number : 3800003

Vicenza, 36100

Italy

Investigational Site Number : 3920007

Nagoya, Aichi-ken 453-8511

Japan

Investigational Site Number : 3920013

Tsuchiura, Ibaraki 300-0028

Japan

Investigational Site Number : 3920006

Kanazawa, Ishikawa-ken 920-8201

Japan

Investigational Site Number : 3920012

Fujisawa, Kanagawa 251-0052

Japan

Investigational Site Number : 3920003

Suita, Osaka 565-0871

Japan

Investigational Site Number : 3920001

Iruma, Saitama 350-0495

Japan

Investigational Site Number : 3920010

Kofu, Yamanashi 400-0027

Japan

Investigational Site Number : 3920015

Fukuoka, 815-8555

Japan

Investigational Site Number : 3920002

Fukushima, 960-1295

Japan

Investigational Site Number : 3920014

Kyoto, 605-0981

Japan

Investigational Site Number : 3920008

Okayama, 700-8557

Japan

Investigational Site Number : 3920004

Tokushima, 770-8503

Japan

Investigational Site Number : 3920005

Yamagata, 990-9585

Japan

Investigational Site Number : 5280002

Amsterdam, 1081 HV

Netherlands

Investigational Site Number : 5280003

Leiden, 2333 ZA

Netherlands

Investigational Site Number : 6160005

Bydgoszcz, Kuyavian-Pomeranian Voivodeship 85-090

Poland

Investigational Site Number : 6160006

Lodz, Lódzkie 90-302

Poland

Investigational Site Number : 7240005

Barakaldo, Basque Country 48903

Spain

Investigational Site Number : 7240001

Barcelona, Catalunya [Cataluña] 08036

Spain

Investigational Site Number : 7240007

Majadahonda, Madrid 28222

Spain

Investigational Site Number : 7240003

Seville, Sevilla 41013

Spain

Investigational Site Number : 7240004

Madrid, 28007

Spain

Investigational Site Number : 7520001

Huddinge, 141 57

Sweden

Investigational Site Number : 7520003

Malmö, 205 02

Sweden