Rilzabrutinib for Autoimmune Hemolytic Anemia

(LUMINA 2 Trial)

Not currently recruiting at 42 trial locations

Overseen By Trial Transparency email recommended (Toll free number for US & Canada)

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sanofi

Must not be taking: Experimental drugs

Disqualifiers: Lymphoma, Leukemia, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral medication, rilzabrutinib, for individuals with anemia caused by the immune system attacking red blood cells. It targets those with primary warm autoimmune hemolytic anemia (wAIHA) or wAIHA linked to lupus, who have not responded to steroid treatments. Participants will receive the treatment over varying periods to assess its effectiveness and safety. For those with wAIHA who have not found success with past treatments, this trial may be suitable. As a Phase 2 trial, the research aims to measure the treatment's effectiveness in an initial, smaller group.

Do I need to stop my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. However, you cannot participate if you are on other experimental drugs or in another clinical trial within 30 days before starting this trial.

Is there any evidence suggesting that rilzabrutinib is likely to be safe for humans?

Research has shown that rilzabrutinib is generally safe for use. Studies have found that most people do not experience serious side effects. In one study, researchers tested rilzabrutinib on patients with a different blood condition and found it safe for long-term use. Another study confirmed these results, noting only mild to moderate side effects in patients. Overall, the evidence suggests that rilzabrutinib is safe for humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Rilzabrutinib is unique because it targets the Bruton’s tyrosine kinase (BTK), which plays a key role in the immune system. Unlike traditional treatments for autoimmune hemolytic anemia, which often involve steroids or immunosuppressive drugs that can have significant side effects, rilzabrutinib offers a more targeted approach. This oral medication is designed to precisely interfere with the immune system's faulty attack on red blood cells without broadly suppressing immune function. Researchers are excited because this precision could mean fewer side effects and a better quality of life for patients.

What evidence suggests that rilzabrutinib might be an effective treatment for autoimmune hemolytic anemia?

Studies have shown that rilzabrutinib, the investigational treatment in this trial, can effectively treat autoimmune hemolytic anemia. In earlier research, patients taking rilzabrutinib experienced lasting symptom improvements. Another study found that rilzabrutinib raised hemoglobin levels, the protein in red blood cells that carries oxygen. This increase is crucial for reducing anemia symptoms. The treatment also lowered signs of red blood cell destruction, helping to prevent their breakdown. These findings suggest that rilzabrutinib may benefit people with autoimmune hemolytic anemia. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

My condition did not improve after steroid treatment.

I showed improvement with rilzabrutinib treatment in Part A.

Exclusion Criteria

I haven't had cancer, except for certain skin cancers, in the last 5 years.

I have been diagnosed with myelodysplastic syndrome.

I have a type of anemia caused by my immune system attacking my red blood cells, but I don't have a confirmed autoimmune disease.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment Part A

Participants receive rilzabrutinib orally for 24 weeks to evaluate efficacy, safety, and pharmacokinetics

24 weeks

Follow-up Part A

7-day safety follow-up period after receiving the last dose of study drug in Part A

1 week

Treatment Part B

Eligible participants continue to receive rilzabrutinib for 52 weeks following the Last Patient In (LPI-Part B)

52 weeks

Follow-up Part B

7-day safety follow-up period after receiving the last dose of study drug in Part B

1 week

Long-term Extension

Participants may continue in the Extended Part B until the study medication becomes commercially available or the program is stopped

Until commercial availability or program cessation

What Are the Treatments Tested in This Trial?

Interventions

• rilzabrutinib

Trial Overview 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.

How Is the Trial Designed?

Group I: Rilzabrutinib Experimental Treatment 1 Intervention

Oral rilzabrutinib 400 mg BID

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05002777

Study Details | NCT05002777 | Efficacy, Safety and ... Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A. Completion of Part A treatment period (24 weeks).

2. ashpublications.org ashpublications.org/thehematologist/article/doi/10.1182/hem.V22.4.202543/537937/Changing-Landscape-of-Management-of-Warm

Changing Landscape of Management of Warm Autoimmune ... This multicenter, phase II, open-label, single-arm study is designed to evaluate the efficacy, safety, and pharmacokinetics of rilzabrutinib in adult patients ...

3. library.ehaweb.org library.ehaweb.org/eha/2025/eha2025-congress/4159375/bruno.fattizzo.long-term.efficacy.and.safety.study.of.rilzabrutinib.oral.html?f=listing%3D3%2Abrowseby%3D8%2Asortby%3D1%2Amedia%3D1

LONG-TERM EFFICACY AND SAFETY STUDY OF ... Long-term rilzabrutinib showed sustained efficacy based on durable Hb response, decreased hemolytic markers, and clinically meaningful ...

4. sanofi.com sanofi.com/en/media-room/press-releases/2024/2024-12-07-16-30-00-2993342

Press Release: ASH: rilzabrutinib demonstrated significant ... For the combined double-blind and open-label periods, durable response was achieved in 29% (n=38) of rilzabrutinib-randomized patients as of the ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT07086976

NCT07086976 | A Study to Investigate the Efficacy, Safety, ... This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb ...

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124065947

Part a Efficacy and Safety of Oral Bruton Tyrosine Kinase ... Rilzabrutinib was well-tolerated with a favorable safety profile in patients with wAIHA.