ClinicalTrials.gov record NCT07086976
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
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)
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 3 randomized, double-blind study compares oral rilzabrutinib with placebo in adults (≥18) who have primary warm autoimmune hemolytic anemia (wAIHA). Participants are randomized 2:1 to rilzabrutinib or placebo for up to 24 weeks, then all who complete that period enter a 28-week open-label period receiving rilzabrutinib; responders may continue into a long-term extension. The study collects data on hemoglobin response, safety, and drug pharmacokinetics, with a 2-week safety follow-up after treatment stops.
What it is studying. The study is testing the efficacy, safety, and pharmacokinetics of oral rilzabrutinib compared with placebo for achieving a durable increase in hemoglobin in adults with primary wAIHA.
What the researchers hope to learn. To determine if rilzabrutinib leads to a durable hemoglobin response by Week 24. Secondary goals include measuring overall hemoglobin response, fatigue (FACIT-fatigue), time to first hemoglobin response, changes in markers of hemolysis (for example LDH), use of rescue therapy, dyspnea scores, safety and adverse events up to Week 104, and pharmacokinetic/pharmacodynamic profiles.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- rilzabrutinib
- placebo
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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 description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After a screening period, participants are randomized 2:1 to take oral study tablets twice daily (rilzabrutinib or matching placebo) for up to 24 weeks (primary analysis period). Those who complete that period enter an open‑label 28‑week period receiving rilzabrutinib; some may then enter a long‑term extension where they can be followed for up to 52 weeks in that extension if they meet the protocol's Hb‑increase criteria. Study involvement includes regular clinic visits for blood tests (including hemoglobin and safety labs), safety monitoring, and assessments such as fatigue and breathing questionnaires; there is a 2‑week safety follow‑up after stopping or completing treatment.
How the study is structured. Phase 3, randomized (2:1), double-blind, placebo-controlled, parallel-group trial. During the double-blind period, participants receive rilzabrutinib or matching placebo twice daily for up to 24 weeks; that is followed by a 28-week open-label period in which all participants receive rilzabrutinib, and a later long-term extension for eligible responders. The double-blind phase is quadruple-masked so participants and study staff do not know treatment assignments.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Autoimmune Haemolytic Anaemia. Does it apply to my diagnosis?
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The listed sites are in Argentina, Austria, Brazil, China, Czechia, Denmark, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, Poland, Saudi Arabia, Spain, Sweden, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on autoimmune hemolytic anemia
Important dates
- Study start
- Aug 18, 2025
- Primary completion
- May 31, 2028 (estimated)
- Study completion
- Dec 26, 2029 (estimated)
- Target enrollment
- 90 participants (estimated)
- Last updated on registry
- Sep 22, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Sanofi
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Trial Transparency email recommended (Toll free for US & Canada) 📞 800-633-1610 ext. option 6 ✉ contact-us@sanofi.com
Participating sites (99)
- Mayo Clinic in Arizona - Phoenix- Site Number : 8400032Recruiting
Phoenix, Arizona, 85054 · United States
- Noble Clinical Research- Site Number : 8400003Recruiting
Tucson, Arizona, 85704 · United States
- City of Hope Comprehensive Cancer Center- Site Number : 8400023Recruiting
Duarte, California, 91010 · United States
- Cancer and Blood Specialty Clinic - Los Alamitos- Site Number : 8400038Recruiting
Los Alamitos, California, 90720 · United States
- USC Norris Comprehensive Cancer Center- Site Number : 8400007Recruiting
Los Angeles, California, 90033 · United States
- Stanford University Medical Center- Site Number : 8400026Recruiting
Stanford, California, 94305 · United States
- Lundquist Institute for Biomedical Innovation at Harbor - UCLA Medical Center- Site Number : 8400006Recruiting
Torrance, California, 90502 · United States
- Yale University School of Medicine- Site Number : 8400027Recruiting
New Haven, Connecticut, 06510 · United States
- MedStar Georgetown University Hospital- Site Number : 8400004Recruiting
Washington D.C., District of Columbia, 20007 · United States
- Hialeah Hospital- Site Number : 8400009Recruiting
Hialeah, Florida, 33013 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- parallel-group
- Participants are divided into two or more groups, and each group receives a different intervention at the same time, so the groups can be compared.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- PK
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- screening period
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- 2:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- safety follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- prednisone
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- contraindications
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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