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Study on the Effects and Safety of Rilzabrutinib for Adults with Warm Autoimmune Hemolytic Anemia

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What is this trial about?

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

This clinical trial is focused on studying the effects of a medication called Rilzabrutinib in patients with a condition known as warm autoimmune hemolytic anemia (wAIHA). This condition occurs when the body's immune system mistakenly attacks and destroys its own red blood cells, leading to anemia. The medication being tested, Rilzabrutinib, is taken in the form of a tablet and is designed to help manage this condition by potentially reducing the immune system's attack on red blood cells.

The purpose of the study is to evaluate how effective and safe Rilzabrutinib is for patients with wAIHA. The study is divided into two parts. In the first part, participants will receive the medication to see how well it works in improving their condition. If the medication shows positive results, participants may continue to the second part, which looks at the long-term effects of the treatment. Throughout the study, participants will be monitored to assess their response to the medication and any side effects they may experience.

Participants in the study will take the medication orally, and their progress will be closely observed by healthcare professionals. The study aims to provide valuable information on the potential benefits of Rilzabrutinib for individuals with wAIHA, contributing to the understanding and management of this condition. The trial will also gather data on the safety of the medication, ensuring that any risks are identified and managed appropriately.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will be informed about the trial's purpose, which is to evaluate the effectiveness and safety of rilzabrutinib in treating warm autoimmune hemolytic anemia (wAIHA).

    The patient must have a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA, without other SLE-related symptoms apart from skin and joint issues.

  2. Step 2

    Initial assessment

    The patient will undergo an initial assessment to confirm eligibility, including a review of medical history and current health status.

    Eligibility criteria include a body mass index (BMI) between 17.5 and 40 kg/m² and an Eastern Cooperative Oncology Group (ECOG) performance status of grade 2 or lower.

  3. Step 3

    Part A treatment

    The patient will begin Part A of the study, which involves taking rilzabrutinib in tablet form orally.

    The treatment period for Part A lasts for 24 weeks, during which the patient will be monitored for hemoglobin response and overall health.

  4. Step 4

    Evaluation of treatment efficacy

    Throughout Part A, the patient's response to the treatment will be evaluated based on hemoglobin levels and other health indicators.

    The primary goal is to assess the proportion of participants who achieve an overall hemoglobin response.

  5. Step 5

    Transition to part B

    If the patient shows evidence of treatment efficacy during Part A, they may transition to Part B of the study.

    Part B focuses on evaluating the long-term effectiveness of rilzabrutinib in maintaining hemoglobin response.

  6. Step 6

    Part B treatment

    During Part B, the patient will continue taking rilzabrutinib and will be monitored for sustained hemoglobin response and overall health.

    The study aims to determine the proportion of participants who maintain a durable response achieved during Part A or achieve a durable response during Part B.

  7. Step 7

    Ongoing monitoring and assessments

    Throughout the study, the patient will undergo regular assessments to monitor health, including safety evaluations and changes in fatigue levels using the FACIT-Fatigue scale.

    The frequency of any additional treatments for wAIHA, other than prednisolone or transfusion, will also be recorded.

  8. Step 8

    Completion of the study

    The study is estimated to conclude by May 28, 2025.

    Upon completion, the patient's overall response to the treatment will be evaluated, and the results will contribute to understanding the long-term efficacy and safety of rilzabrutinib for wAIHA.

Who can join the trial?

8 criteria

  • Male and female patients with a confirmed diagnosis of primary **warm autoimmune hemolytic anemia (wAIHA)** or **systemic lupus erythematosus (SLE)**-associated wAIHA. SLE is a condition where the immune system attacks the body, and wAIHA is a type of anemia where the immune system destroys red blood cells.
  • Participants who have previously not been able to maintain a lasting improvement after treatment with **corticosteroids**, which are medications used to reduce inflammation.
  • Have an **Eastern Cooperative Oncology Group (ECOG) performance status** of grade 2 or lower. This is a scale used to assess how a disease affects a patient's daily living abilities, with lower numbers indicating better functioning.
  • Must have an up-to-date vaccination status according to local guidelines.
  • Body mass index (BMI) must be greater than 17.5 and less than 40 kg/m². BMI is a measure of body fat based on height and weight.
  • All contraceptive use by men and women should follow local regulations regarding methods of contraception for those participating in clinical studies.
  • For Part B only: Must show evidence of treatment effectiveness with **rilzabrutinib** by achieving an overall response during Part A. Rilzabrutinib is a medication being studied for its effects on wAIHA.
  • For Part B only: Must have completed the Part A treatment period, which lasts 24 weeks.

Who cannot join the trial?

10 criteria

  • Patients who have any other serious health condition that might interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial recently.
  • Patients who have a history of allergic reactions to similar medications.
  • Patients who are unable to follow the study procedures or attend the required visits.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who have an active infection that requires treatment.
  • Patients who have received certain treatments for their condition recently.
  • Patients who have a condition that affects their immune system.
  • Patients who have a history of cancer, except for certain types that have been treated successfully.
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Investigated drugs

Rilzabrutinib is a medication being studied for its effectiveness in treating warm autoimmune hemolytic anemia (wAIHA). This condition causes the immune system to mistakenly attack and destroy red blood cells, leading to anemia. Rilzabrutinib is designed to help manage this condition by potentially reducing the immune system's attack on red blood cells, thereby improving symptoms and overall health in patients with wAIHA. The study aims to assess how well this medication works and its safety for long-term use in individuals with this type of anemia.

What is already known about the treatment

Rilzabrutinib – Rilzabrutinib is administered orally and is currently being studied in clinical trials for its effectiveness in treating warm autoimmune hemolytic anemia (wAIHA). It is in the Phase IIb stage of clinical research, focusing on its safety, efficacy, and pharmacokinetics. The medication works by inhibiting Bruton's tyrosine kinase, a protein involved in the immune response, which helps reduce the destruction of red blood cells in wAIHA. Rilzabrutinib is classified as a kinase inhibitor, a type of drug that blocks specific enzymes involved in disease processes.

Investigated diseases

Warm autoimmune hemolytic anemia – This is a condition where the immune system mistakenly attacks and destroys the body's own red blood cells, leading to anemia. It is characterized by the presence of antibodies that react at body temperature, causing red blood cells to break down prematurely. Symptoms may include fatigue, shortness of breath, and a pale or yellowish skin tone. The disease progresses as the destruction of red blood cells continues, leading to a decrease in hemoglobin levels. Over time, this can result in an increased workload for the bone marrow to produce new red blood cells. The condition can vary in severity, with some individuals experiencing mild symptoms and others having more significant health impacts.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-509441-13-00Protocol codeACT17209Estimated enrolment20 patientsSponsorSanofi-Aventis Recherche & Developpement

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