Rilzabrutinib for Autoimmune Hemolytic Anemia

(LUMINA 3 Trial)

Recruiting at 75 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must not be taking: BTK inhibitors

Disqualifiers: Lymphoma, Leukemia, Herpes zoster, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

90 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral medication, rilzabrutinib, to determine if it can help individuals with primary warm autoimmune hemolytic anemia (wAIHA) maintain healthy hemoglobin levels. Participants will receive either rilzabrutinib or a placebo for 24 weeks, with those continuing eventually receiving the actual medication. The trial seeks individuals who have had wAIHA for at least three months and have not found success with previous treatments like steroids. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially bringing a new treatment to market.

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

The trial information does not specify if you need to stop your current medications. However, you cannot be on other experimental drugs or have used BTK inhibitors for wAIHA before starting the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that rilzabrutinib is generally safe for participants in studies. In past trials, most patients taking rilzabrutinib did not experience serious side effects, indicating a good safety record over time. Some common side effects were mild to moderate, similar to those seen with many treatments.

The drug has been tested for its effectiveness in helping people with warm autoimmune hemolytic anemia (wAIHA), a condition where the immune system attacks red blood cells. Results demonstrated that the treatment can be both effective and safe for these patients.

Overall, the evidence suggests that rilzabrutinib is safe, but it is important to remember that all medications can have side effects. Researchers will closely monitor trial participants to ensure their safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autoimmune hemolytic anemia?

Rilzabrutinib is unique because it targets Bruton’s tyrosine kinase (BTK), a key player in the immune system that is not directly addressed by current treatments for autoimmune hemolytic anemia, such as corticosteroids or rituximab. By inhibiting BTK, rilzabrutinib potentially offers a more precise approach to managing the overactive immune response that characterizes this condition. Researchers are excited about this treatment because it promises to reduce the destruction of red blood cells in a more targeted manner, potentially leading to fewer side effects compared to broader immunosuppressive therapies.

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

Research has shown that rilzabrutinib might help treat warm autoimmune hemolytic anemia (wAIHA). In one study, 29% of patients who took rilzabrutinib maintained stable hemoglobin levels over time. Another study found that 80% of patients had stable hemoglobin levels when rilzabrutinib was used alone or with other medications. In this trial, participants will receive either rilzabrutinib or a placebo. Previous studies have demonstrated that rilzabrutinib outperformed a placebo in improving various health indicators related to wAIHA. These findings suggest that rilzabrutinib could be a promising treatment option for people with this condition. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

Adults over 18 with primary warm Autoimmune Hemolytic Anemia (wAIHA) who haven't responded to steroids, or can't take them due to side effects or other health issues. Participants must be able to perform daily activities with minimal assistance and agree to use contraception.

Inclusion Criteria

I have been diagnosed with primary wAIHA for at least 3 months.

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

My physical health allows me to do some light activities.

Exclusion Criteria

Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study

I have had symptoms of shingles within the last 3 months.

Participants with clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Primary Analysis Period (PAP)

Participants are randomized to receive rilzabrutinib or placebo to assess efficacy and safety

24 weeks

Open-label Period (OLP)

All participants receive rilzabrutinib to further assess efficacy and safety

28 weeks

Long-term Extension (LTE)

Participants who demonstrate Hb increase during the last 8 weeks of OLP continue to receive rilzabrutinib

52 weeks

Follow-up

Participants are monitored for safety after treatment completion or discontinuation

2 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rilzabrutinib

Trial Overview The trial is testing Rilzabrutinib, an oral medication, against a placebo in patients with wAIHA. It's a double-blind study where participants are randomly assigned in a 2:1 ratio to either the drug or placebo for up to 24 weeks, followed by an open-label period and long-term extension.

How Is the Trial Designed?

Group I: rilzabrutinib Experimental Treatment 1 Intervention

Oral rilzabrutinib BID

Group II: placebo Placebo Group 1 Intervention

Oral placebo 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. 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 ...

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 ... Rilzabrutinib monotherapy was given in 6 (40%) patients, and with concomitant CS in 9 (60%) patients. Durable Hb response was achieved in 12 (80 ...

4. 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).

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12427540/

Evaluating rilzabrutinib in the treatment of immune ... Rilzabrutinib treatment showed significant improvement over placebo for all secondary efficacy endpoints including longer least square mean number of weeks ...

6. 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 ...

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

Part a Efficacy and Safety of Oral Bruton Tyrosine Kinase ... Rilzabrutinib is an investigational therapy being evaluated in a clinical study for the treatment of patients with warm autoimmune hemolytic anemia.