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Bruton's Tyrosine Kinase (BTK) Inhibitor

Rilzabrutinib for Autoimmune Hemolytic Anemia

Phase 2
Waitlist Available
Research Sponsored by Sanofi
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower
Participants who have previously failed to maintain a sustained response after treatment with corticosteroids
Must not have
Myelodysplastic syndrome
HIV infection
Timeline
Screening 3 weeks
Treatment Varies
Follow Up until week 24 in part a and week 75 in part b
Awards & highlights
No Placebo-Only Group

Summary

This trial tests a medication called rilzabrutinib, taken by mouth, in adults with a type of anemia where their immune system attacks their red blood cells. The goal is to see if this medication can safely and effectively stop this immune attack.

Who is the study for?
Adults with warm autoimmune hemolytic anemia (wAIHA) who haven't responded well to steroids can join this trial. They should be relatively healthy, have a BMI between 17.5 and 40, and up-to-date vaccinations. Participants must use approved contraception methods and not have other serious illnesses or recent treatments with experimental drugs.
What is being tested?
The trial is testing rilzabrutinib's effectiveness for wAIHA patients over two parts: initial treatment for 24 weeks (Part A), then potentially continuing for another year if they respond well (Part B). It's an open-label study, meaning everyone knows they're taking the drug, which is given orally.
What are the potential side effects?
Specific side effects of rilzabrutinib aren't listed here, but generally could include reactions at the medication site, digestive issues, fatigue or allergic reactions. Any severe or unacceptable side effects may lead to discontinuation from the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can do most of my daily activities but not heavy physical work.
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My condition did not improve after steroid treatment.
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I have been diagnosed with wAIHA or SLE-related wAIHA, with only skin and muscle symptoms.
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I did not improve long-term after steroid treatment.
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I have completed a 24-week treatment period.
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I can do most of my daily activities but not heavy physical work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have been diagnosed with myelodysplastic syndrome.
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I am HIV positive.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~until week 24 in part a and week 75 in part b
This trial's timeline: 3 weeks for screening, Varies for treatment, and until week 24 in part a and week 75 in part b for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Hemoglobin
Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response
Secondary study objectives
Change from baseline in FACIT-Fatigue scale score
Incidence of treatment emergent adverse events (TEAEs), serious TEAEs, adverse events of special interest (AESIs)
Proportion of participants with durable hemoglobin response

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: RilzabrutinibExperimental Treatment1 Intervention
Oral rilzabrutinib 400 mg BID
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
rilzabrutinib
2021
Completed Phase 2
~170

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Autoimmune Hemolytic Anemia (AIHA) include Bruton's Tyrosine Kinase (BTK) inhibitors like rilzabrutinib, corticosteroids, and immunosuppressive drugs such as rituximab. BTK inhibitors block the BTK enzyme, reducing B cell activation and autoantibody production, thereby decreasing the immune attack on red blood cells. Corticosteroids suppress the overall immune response, while rituximab targets and depletes B cells. Understanding these mechanisms helps in selecting the most appropriate treatment, leading to more effective management and fewer side effects for AIHA patients.

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor
2,197 Previous Clinical Trials
4,035,291 Total Patients Enrolled

Media Library

rilzabrutinib (Bruton's Tyrosine Kinase (BTK) Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05002777 — Phase 2
Autoimmune Hemolytic Anemia Research Study Groups: Rilzabrutinib
Autoimmune Hemolytic Anemia Clinical Trial 2023: rilzabrutinib Highlights & Side Effects. Trial Name: NCT05002777 — Phase 2
rilzabrutinib (Bruton's Tyrosine Kinase (BTK) Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05002777 — Phase 2
~6 spots leftby Nov 2025