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

NCT07086976 | Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: EFC17360U1111-1314-51122024-517972-39
• Study Type: interventional
• Target: 90
• Locations: 17 countries
• Sites: 92

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

Outcome Measures

Primary Outcomes (1)

• 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

  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 in the absence of rescue medication and transfusion.

  By Week 24

Secondary Outcomes (7)

• Proportion of participants achieving overall Hb response (response [R] or complete response [CR]) by Week 24 of treatment in the PAP

  By Week 24

• Change from baseline in fatigue total score as measured by Functional Assessment of Chronic Illness Therapy (FACIT) - fatigue at Week 24

  Baseline to Week 24

• The time taken (days) to achieve the first Hb increase by ≥2 g/dL from baseline during the PAP in the previous 14 days and in the absence of rescue medication in the previous 6 weeks

  Until Week 24

• Change from baseline in levels of LDH at Week 24

  Baseline to Week 24

• Proportion of participants requiring use of rescue therapy after Week 4 of treatment during the PAP

  Until Week 24

Study Arms (2)

rilzabrutinib

EXPERIMENTAL

Oral rilzabrutinib BID

Drug: rilzabrutinib

placebo

PLACEBO COMPARATOR

Oral placebo BID

Drug: placebo

Interventions

placebo DRUG

Pharmaceutical form:tablet-Route of administration:oral

placebo

rilzabrutinib DRUG

Pharmaceutical form:tablet-Route of administration:oral

Also known as: SAR444671

rilzabrutinib

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (92)

Mayo Clinic in Arizona - Phoenix- Site Number : 8400032

Phoenix, Arizona, 85054, United States

RECRUITING

Noble Clinical Research- Site Number : 8400003

Tucson, Arizona, 85704, United States

RECRUITING

City of Hope National Medical Center- Site Number : 8400023

Duarte, California, 91010, United States

RECRUITING

USC Norris Comprehensive Cancer Center- Site Number : 8400007

Los Angeles, California, 90033, United States

RECRUITING

Stanford University Medical Center- Site Number : 8400026

Stanford, California, 94305, United States

RECRUITING

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

Torrance, California, 90502, United States

RECRUITING

Yale University School of Medicine- Site Number : 8400027

New Haven, Connecticut, 06510, United States

RECRUITING

Hialeah Hospital- Site Number : 8400009

Hialeah, Florida, 33013, United States

RECRUITING

Sylvester Comprehensive Cancer Center- Site Number : 8400031

Miami, Florida, 33136, United States

RECRUITING

University Hospital and Medical Center- Site Number : 8400005

Tamarac, Florida, 33321, United States

RECRUITING

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

Ann Arbor, Michigan, 48109, United States

RECRUITING

Henry Ford Hospital- Site Number : 8400012

Detroit, Michigan, 48202, United States

RECRUITING

Mayo Clinic in Rochester - Minnesota- Site Number : 8400008

Rochester, Minnesota, 55905, United States

RECRUITING

Montefiore Medical Center - Einstein Campus- Site Number : 8400011

The Bronx, New York, 10461, United States

RECRUITING

Ohio State University Hospital East- Site Number : 8400020

Columbus, Ohio, 43203, United States

RECRUITING

Fox Chase Cancer Center - Philadelphia- Site Number : 8400019

Philadelphia, Pennsylvania, 19111, United States

RECRUITING

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

Providence, Rhode Island, 02903, United States

RECRUITING

Baptist Memorial Hospital- Site Number : 8400018

Memphis, Tennessee, 38120, United States

RECRUITING

UT Health - San Antonio- Site Number : 8400015

San Antonio, Texas, 78229, United States

RECRUITING

Fred Hutchinson Cancer Center- Site Number : 8400016

Seattle, Washington, 98109, United States

RECRUITING

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

La Crosse, Wisconsin, 54601, United States

RECRUITING

Investigational Site Number : 0320001

Rosario, Santa Fe Province, 2000, Argentina

RECRUITING

Investigational Site Number : 0320003

Buenos Aires, 1181, Argentina

RECRUITING

Investigational Site Number : 0320002

Buenos Aires, 1431, Argentina

RECRUITING

Investigational Site Number : 0400001

Vienna, 1090, Austria

RECRUITING

Investigational Site Number : 0400002

Vienna, 1140, Austria

RECRUITING

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

Vitória, Espírito Santo, 29047-105, Brazil

RECRUITING

Universidade Federal de Goias- Site Number : 0760001

Goiânia, Goiás, 74605-020, Brazil

RECRUITING

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

São Paulo, 05403-000, Brazil

RECRUITING

Investigational Site Number : 1560001

Beijing, 100730, China

RECRUITING

Investigational Site Number : 1560007

Chengde, 067020, China

RECRUITING

Investigational Site Number : 1560011

Guangzhou, 510000, China

RECRUITING

Investigational Site Number : 1560005

Guangzhou, 510180, China

RECRUITING

Investigational Site Number : 1560009

Hohhot, 010050, China

RECRUITING

Investigational Site Number : 1560003

Nanchang, 330006, China

RECRUITING

Investigational Site Number : 1560012

Nanchang, 330006, China

RECRUITING

Investigational Site Number : 1560004

Suzhou, 215006, China

RECRUITING

Investigational Site Number : 1560002

Tianjin, 300020, China

RECRUITING

Investigational Site Number : 1560010

Wuhan, 430022, China

RECRUITING

Investigational Site Number : 1560006

Xi'an, 710054, China

RECRUITING

Investigational Site Number : 1560008

Zhengzhou, 450008, China

RECRUITING

Investigational Site Number : 2030002

Prague, Cardiff [Caerdydd Gb-crd], 128 20, Czechia

RECRUITING

Investigational Site Number : 2030001

Brno, 625 00, Czechia

RECRUITING

Investigational Site Number : 2080003

Aarhus, 8200, Denmark

RECRUITING

Investigational Site Number : 2080002

Copenhagen, 2100, Denmark

RECRUITING

Investigational Site Number : 2080001

Odense, 5000, Denmark

RECRUITING

Investigational Site Number : 2760003

Berlin, 10707, Germany

RECRUITING

Investigational Site Number : 2760001

Essen, 45147, Germany

RECRUITING

Investigational Site Number : 3000002

Athens, 115 27, Greece

RECRUITING

Investigational Site Number : 3000005

Larissa, 411 10, Greece

RECRUITING

Investigational Site Number : 3000004

Pátrai, 254 43, Greece

RECRUITING

Investigational Site Number : 3000003

Pátrai, 265 04, Greece

RECRUITING

Investigational Site Number : 3000001

Thessaloniki, 570 10, Greece

RECRUITING

Investigational Site Number : 3480002

Debrecen, 4032, Hungary

RECRUITING

Investigational Site Number : 3760001

Afula, 1834111, Israel

RECRUITING

Investigational Site Number : 3760007

Haifa, 3104802, Israel

RECRUITING

Investigational Site Number : 3760005

Kefar Sava, 4428164, Israel

RECRUITING

Investigational Site Number : 3760002

Tel Aviv, 6423906, Israel

RECRUITING

Investigational Site Number : 3800004

Florence, Firenze, 50134, Italy

RECRUITING

Investigational Site Number : 3800010

Genoa, Genova, 16132, Italy

RECRUITING

Investigational Site Number : 3800001

Milan, Milano, 20122, Italy

RECRUITING

Investigational Site Number : 3800005

Naples, Napoli, 80131, Italy

RECRUITING

Investigational Site Number : 3800002

Meldola, Reggio Emilia, 47014, Italy

RECRUITING

Investigational Site Number : 3800009

Novara, 28100, Italy

RECRUITING

Investigational Site Number : 3800006

Palermo, 90146, Italy

RECRUITING

Investigational Site Number : 3800007

Trieste, 34125, Italy

RECRUITING

Investigational Site Number : 3800008

Varese, 21100, Italy

RECRUITING

Investigational Site Number : 3800003

Vicenza, 36100, Italy

RECRUITING

Investigational Site Number : 3920007

Nagoya, Aichi-ken, 453-8511, Japan

RECRUITING

Investigational Site Number : 3920013

Tsuchiura, Ibaraki, 300-0028, Japan

RECRUITING

Investigational Site Number : 3920006

Kanazawa, Ishikawa-ken, 920-8201, Japan

RECRUITING

Investigational Site Number : 3920012

Fujisawa, Kanagawa, 251-0052, Japan

RECRUITING

Investigational Site Number : 3920003

Suita, Osaka, 565-0871, Japan

RECRUITING

Investigational Site Number : 3920001

Iruma, Saitama, 350-0495, Japan

RECRUITING

Investigational Site Number : 3920010

Kofu, Yamanashi, 400-0027, Japan

RECRUITING

Investigational Site Number : 3920015

Fukuoka, 815-8555, Japan

RECRUITING

Investigational Site Number : 3920002

Fukushima, 960-1295, Japan

RECRUITING

Investigational Site Number : 3920014

Kyoto, 605-0981, Japan

RECRUITING

Investigational Site Number : 3920008

Okayama, 700-8557, Japan

RECRUITING

Investigational Site Number : 3920004

Tokushima, 770-8503, Japan

RECRUITING

Investigational Site Number : 3920005

Yamagata, 990-9585, Japan

RECRUITING

Investigational Site Number : 5280002

Amsterdam, 1081 HV, Netherlands

RECRUITING

Investigational Site Number : 5280003

Leiden, 2333 ZA, Netherlands

RECRUITING

Investigational Site Number : 6160005

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-090, Poland

RECRUITING

Investigational Site Number : 6160006

Lodz, Lódzkie, 90-302, Poland

RECRUITING

Investigational Site Number : 7240005

Barakaldo, Basque Country, 48903, Spain

RECRUITING

Investigational Site Number : 7240001

Barcelona, Catalunya [Cataluña], 08036, Spain

RECRUITING

Investigational Site Number : 7240007

Majadahonda, Madrid, 28222, Spain

RECRUITING

Investigational Site Number : 7240003

Seville, Sevilla, 41013, Spain

RECRUITING

Investigational Site Number : 7240004

Madrid, 28007, Spain

RECRUITING

Investigational Site Number : 7520001

Huddinge, 141 57, Sweden

RECRUITING

Investigational Site Number : 7520003

Malmö, 205 02, Sweden

RECRUITING

Related Links

• Warm Autoimmune Hemolytic Anemia Website
• EFC17360 Plain Language Results Summary

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

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

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 17, 2025
• First Posted: July 25, 2025
• Study Start: August 18, 2025
• Primary Completion (Estimated): May 31, 2028
• Study Completion (Estimated): December 26, 2029
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

AU (2); DE (2); CN (12); GR (5); SE (2); NL (2); DK (3); ES (5); AR (3); IL (4); US (21); CZ (2); HU (1); IT (10); JP (13); PL (2); BR (3)