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Study of rilzabrutinib tablets compared to placebo in adults with warm autoimmune hemolytic anemia

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical study focuses on warm autoimmune hemolytic anemia (wAIHA), a condition where the body's immune system mistakenly destroys its own red blood cells. The study will test a medication called rilzabrutinib, given as film-coated tablets taken by mouth, compared to a placebo. This is a new treatment approach for people whose disease has not responded well to standard treatments or who cannot take conventional medications.

The research aims to determine if rilzabrutinib can help maintain stable blood levels in people with warm autoimmune hemolytic anemia. During the study, participants will receive either rilzabrutinib tablets or placebo tablets. The maximum daily dose of rilzabrutinib will be 800 mg, and the treatment period will last for 224 weeks.

The study will measure several aspects of the treatment's effects, including changes in hemoglobin levels (a protein in red blood cells that carries oxygen throughout the body), changes in fatigue levels, and how quickly the treatment works. The study will also track how well participants feel, including their ability to breathe comfortably, and monitor any side effects that may occur during treatment.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial participation

    After confirming your eligibility for the study, you will be diagnosed with warm autoimmune hemolytic anemia (wAIHA) for at least 3 months

    Your physical condition will be evaluated using the Eastern Cooperative Oncology Group (ECOG) scale, which must be Grade 2 or lower

    You must have previously tried corticosteroid treatment without maintaining a sustained response, or be unable to take corticosteroids

  2. Step 2

    Treatment assignment

    You will be randomly assigned to receive either rilzabrutinib tablets or placebo tablets

    The tablets are to be taken by mouth

    Neither you nor your doctor will know which treatment you are receiving during this phase

  3. Step 3

    Primary assessment period

    Your hemoglobin levels will be regularly monitored to track your response to treatment

    Your fatigue levels will be measured using a special questionnaire called FACIT-fatigue

    Your breathing difficulties will be assessed using FACIT-Dyspnea questionnaire

    Blood tests will check your LDH (lactate dehydrogenase) levels

  4. Step 4

    Ongoing monitoring

    Regular physical examinations will be conducted

    Your vital signs will be checked periodically

    You will undergo heart monitoring through electrocardiograms (ECG)

    Any side effects or health changes will be recorded and monitored

  5. Step 5

    Study duration

    The study is scheduled to run from June 2025 to October 2029

    After the initial double-blind period, there will be an open-label period and long-term extension phase

Who can join the trial?

6 criteria

  • Must be diagnosed with primary warm autoimmune hemolytic anemia (wAIHA) for at least 3 months (a condition where the body's immune system destroys red blood cells)
  • Must have experienced one of these situations with corticosteroid treatment (medications like prednisone that reduce inflammation): - Failed to respond to treatment within 3 weeks while taking at least 1 mg/kg or 60 mg daily - Need more than 10 mg daily to maintain improvement - Cannot tolerate the medication due to side effects - Have medical conditions that make corticosteroid use unsafe
  • Must have an ECOG performance status of 2 or lower (ability to carry out daily activities - Grade 2 means able to walk and handle self-care but unable to work)
  • Both men and women can participate
  • Must use appropriate contraception methods during the study according to local guidelines
  • Must be an adult (18 years or older)

Who cannot join the trial?

14 criteria

  • Current diagnosis of Evans syndrome (a condition where the body makes antibodies against both red blood cells and platelets)
  • History of blood clotting disorders or significant blood clots in the past 6 months
  • Recent major surgery within 4 weeks before starting the study
  • Active or chronic hepatitis B or C infection (liver infections)
  • Current HIV infection
  • Active tuberculosis infection
  • Presence of any active cancer or history of cancer within the past 3 years (except for successfully treated skin cancer)
  • Severe heart conditions or uncontrolled high blood pressure
  • Severe kidney disease requiring dialysis
  • Current pregnancy or breastfeeding
  • Use of certain medications that affect blood clotting within 7 days before starting the study
  • Participation in another clinical trial within 30 days before this study
  • Allergic reactions to similar medications in the past
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
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Investigated drugs

Rilzabrutinib is a medication being studied for treating warm autoimmune hemolytic anemia (wAIHA), a condition where the body's immune system destroys its own red blood cells. This medication works by targeting and inhibiting an enzyme called BTK (Bruton's tyrosine kinase), which plays a role in the immune system response. The goal is to reduce the destruction of red blood cells and help maintain healthy hemoglobin levels in patients with this condition.

What is already known about the treatment

Rilzabrutinib - An oral Bruton's tyrosine kinase (BTK) inhibitor that is currently being investigated in clinical trials for the treatment of warm autoimmune hemolytic anemia (wAIHA), a condition where the immune system attacks and destroys red blood cells. The medication works by blocking the BTK enzyme, which plays a crucial role in B-cell development and autoimmune responses, thereby potentially reducing the autoimmune destruction of red blood cells. This investigational drug is administered in pill form and represents a novel approach to treating wAIHA, as it targets the underlying mechanism of the disease rather than just managing symptoms, though it is still undergoing Phase 3 clinical trials to establish its safety and efficacy.

Investigated diseases

Autoimmune Hemolytic Anemia - A rare condition where the immune system mistakenly attacks and destroys red blood cells. The destruction of red blood cells occurs faster than the body can replace them, leading to anemia. The condition causes the body to produce antibodies that attach to red blood cells, marking them for destruction by the immune system. This process takes place primarily in the spleen and liver, where damaged red blood cells are broken down. The condition can develop gradually or suddenly, and can occur at any age.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-517972-39-00Protocol codeEFC17360 - LUMINA 3Estimated enrolment136 patientsSponsorSanofi-Aventis Recherche & Developpement

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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