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
NCT ID: NCT07086976
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
90 participants
INTERVENTIONAL
2025-08-18
2029-12-26
Brief Summary
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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.
Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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rilzabrutinib
Oral rilzabrutinib BID
rilzabrutinib
Pharmaceutical form:tablet-Route of administration:oral
placebo
Oral placebo BID
placebo
Pharmaceutical form:tablet-Route of administration:oral
Interventions
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rilzabrutinib
Pharmaceutical form:tablet-Route of administration:oral
placebo
Pharmaceutical form:tablet-Route of administration:oral
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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.
18 Years
ALL
No
Sponsors
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Sanofi
INDUSTRY
Responsible Party
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Locations
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Mayo Clinic in Arizona - Phoenix- Site Number : 8400032
Phoenix, Arizona, United States
Noble Clinical Research- Site Number : 8400003
Tucson, Arizona, United States
Hialeah Hospital- Site Number : 8400009
Hialeah, Florida, United States
University of Michigan Health System - Ann Arbor- Site Number : 8400014
Ann Arbor, Michigan, United States
Mayo Clinic in Rochester - Minnesota- Site Number : 8400008
Rochester, Minnesota, United States
Ohio State University Hospital East- Site Number : 8400020
Columbus, Ohio, United States
Investigational Site Number : 0320001
Rosario, Santa Fe Province, Argentina
Investigational Site Number : 0320003
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 : 1560011
Guangzhou, , China
Investigational Site Number : 1560005
Guangzhou, , China
Investigational Site Number : 1560003
Nanchang, , China
Investigational Site Number : 1560012
Nanchang, , 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 : 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 : 3800007
Trieste, , Italy
Investigational Site Number : 3800003
Vicenza, , Italy
Investigational Site Number : 3920007
Nagoya, Aichi-ken, Japan
Investigational Site Number : 3920006
Kanazawa, Ishikawa-ken, Japan
Investigational Site Number : 3920012
Fujisawa, Kanagawa, Japan
Investigational Site Number : 3920001
Iruma, Saitama, Japan
Investigational Site Number : 3920002
Fukushima, , Japan
Investigational Site Number : 5280002
Amsterdam, , Netherlands
Investigational Site Number : 5280003
Leiden, , Netherlands
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
Countries
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Central Contacts
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Trial Transparency email recommended (Toll free for US & Canada)
Role: CONTACT
Phone: 800-633-1610
Email: [email protected]
Related Links
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EFC17360 Plain Language Results Summary
Other Identifiers
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U1111-1314-5112
Identifier Type: REGISTRY
Identifier Source: secondary_id
2024-517972-39
Identifier Type: REGISTRY
Identifier Source: secondary_id
EFC17360
Identifier Type: -
Identifier Source: org_study_id