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Oral rilzabrutinib versus placebo for adults 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)

Phase 3 Interventional Sanofi · NCT07086976

This trial tests whether the oral drug rilzabrutinib can raise and sustain hemoglobin better than placebo in adults with primary warm autoimmune hemolytic anemia who have not had lasting benefit from steroids.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment90 (estimated)
Ages18 Years and up
SexAll
SponsorSanofi Industry-sponsored
Drugs / interventionsprednisone, rilzabrutinib
Locations92 sites (Phoenix, Arizona and 91 other locations)
Trial IDNCT07086976 on ClinicalTrials.gov

What this trial studies

This is a randomized, double-blind Phase 3 trial enrolling about 90 adults with confirmed primary warm autoimmune hemolytic anemia who are steroid-refractory, steroid-dependent, or steroid-intolerant. Participants are randomized 2:1 to rilzabrutinib or placebo for a primary analysis period of up to 24 weeks, followed by a 28-week open-label period in which all participants receive rilzabrutinib. Only participants who meet hemoglobin response criteria during the open-label period may continue into a long-term extension for additional follow-up. The study measures durable hemoglobin response as the primary efficacy endpoint and also collects safety, pharmacokinetic, and pharmacodynamic data.

Who should consider this trial

Good fit: Adults aged 18 or older with a confirmed diagnosis of primary warm autoimmune hemolytic anemia for at least 6 months who are steroid-refractory, steroid-dependent, or steroid-intolerant and have ECOG performance status ≤2 are the intended participants.

Not a fit: Patients with secondary autoimmune hemolytic anemia, those who respond adequately to standard steroid therapy, children under 18, or those with major contraindications to study procedures are unlikely to benefit from joining this trial.

Why it matters

Potential benefit: If successful, rilzabrutinib could provide a steroid-sparing option that produces a durable increase in hemoglobin for adults with refractory warm autoimmune hemolytic anemia.

How similar studies have performed: Early-phase studies of rilzabrutinib and other BTK inhibitors have shown activity in autoimmune cytopenias, but large randomized Phase 3 confirmation in warm AIHA is limited.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Phoenix, Arizona and 91 other locations

+42 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Autoimmune Haemolytic Anaemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.