Rilzabrutinib for Autoimmune Hemolytic Anemia

(LUMINA 2 Trial)

Recruiting at 39 trial locations

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

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Sanofi

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

22 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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