Oral rilzabrutinib versus placebo for adults with warm autoimmune hemolytic anemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Mayo Clinic in Arizona - Phoenix- Site Number : 8400032
  Phoenix, Arizona, United States Recruiting
• Noble Clinical Research- Site Number : 8400003
  Tucson, Arizona, United States Recruiting
• City of Hope Comprehensive Cancer Center- Site Number : 8400023
  Duarte, California, United States Recruiting
• Cancer and Blood Specialty Clinic - Los Alamitos- Site Number : 8400038
  Los Alamitos, California, United States Recruiting
• USC Norris Comprehensive Cancer Center- Site Number : 8400007
  Los Angeles, California, United States Recruiting
• Stanford University Medical Center- Site Number : 8400026
  Stanford, California, United States Recruiting

+55 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Trial Transparency email recommended (Toll free for US & Canada)
• Email: contact-us@sanofi.com
• Phone: 800-633-1610

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Rilzabrutinib in adults with severe kidney impairment vs. normal kidney function

  This study tests how rilzabrutinib behaves in the body and its safety in adults with severe kidney impairment compared to healthy adults.

  Recruiting Phase 1 Sanofi · Hialeah, Florida · NCT07853131

• Rilzabrutinib versus placebo for adults with IgG4-related disease (52-week treatment)

  This 52-week study will try rilzabrutinib tablets to see if they prevent disease flares and better control symptoms in adults with active IgG4-related disease compared with placebo.

  Recruiting at 91 sites Phase 3 Sanofi · Fontana, California and 94 other locations · NCT07190196

• Oral rilzabrutinib for adults with immune thrombocytopenia after first-line treatment fails.

  This study will test whether the oral drug rilzabrutinib can raise and keep platelet counts up in adults with ITP who did not have lasting benefit from first-line therapy.

  Recruiting at 36 sites Phase 3 Sanofi · Clovis, California and 35 other locations · NCT07007962

• Rilzabrutinib treatment for adults with Graves' disease

  This trial will test two doses of the oral drug rilzabrutinib in adults with Graves' disease, with or without Graves' orbitopathy, to see which dose better controls hyperthyroidism and is safe.

  Recruiting at 19 sites Phase 2 Sanofi · Buenos Aires and 23 other locations · NCT06984627

• Evaluating Rilzabrutinib for Sickle Cell Disease in Patients Aged 10 to 65

  This study is testing a new medication called rilzabrutinib to see if it can help people aged 10 to 65 with sickle cell disease have fewer pain crises and improve their overall health.

  Recruiting at 63 sites Phase 3 Sanofi · Birmingham, Alabama and 62 other locations · NCT06975865

• Evaluating treatments for kidney diseases in young to middle-aged adults

  This study is testing three different treatments for kidney diseases in young to middle-aged adults to see if they can reduce protein levels in urine and help with symptoms over 24 weeks.

  Recruiting at 78 sites Phase 2 Sanofi · Birmingham, Alabama and 78 other locations · NCT06500702

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.