What side effects are associated with this type of intervention?

Common treatments for Autoimmune Hemolytic Anemia (AIHA) include corticosteroids, immunosuppressive drugs, and monoclonal antibodies. Corticosteroids can cause side effects such as weight gain, hypertension, diabetes, and increased risk of infections. Immunosuppressive drugs like azathioprine and cyclophosphamide may lead to bone marrow suppression, increased infection risk, and liver toxicity. Monoclonal antibodies, such as rituximab, can cause infusion reactions, infections, and cytopenias. Bruton's Tyrosine Kinase (BTK) inhibitors, like Rilzabrutinib, are being studied for AIHA and are known to potentially cause side effects such as bleeding, infections, and gastrointestinal disturbances.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved Bruton's Tyrosine Kinase (BTK) inhibitors for the treatment of Autoimmune Hemolytic Anemia (AIHA). However, treatments for AIHA generally include corticosteroids, immunosuppressive agents, and monoclonal antibodies like rituximab. Rilzabrutinib, a BTK inhibitor, is currently being studied for its efficacy and safety in treating warm AIHA, which represents a novel approach in targeting the underlying mechanisms of the disease.

What other types of research exist?

Promising novel alternatives to Rilzabrutinib for Autoimmune Hemolytic Anemia patients include other BTK inhibitors such as Acalabrutinib and Zanubrutinib, which are being explored for their efficacy and safety in various autoimmune conditions. Additionally, Syk inhibitors like Fostamatinib and complement inhibitors such as Eculizumab are also being investigated for their potential in treating AIHA. These alternatives are in various stages of clinical trials and have shown encouraging results in terms of efficacy and safety.

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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ until week 24 in part a and week 75 in part b

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

2020

Completed Phase 2

~190

0 / 8Eligibility criteria met

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.