REGN7257 for Severe Aplastic Anemia

Recruiting in Palo Alto (17 mi)

+14 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Regeneron Pharmaceuticals

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial is testing a new drug called REGN7257 for patients with severe aplastic anemia who haven't responded to other treatments. The drug aims to help the bone marrow produce more blood cells and reduce the need for transfusions. The study will check if the drug is safe and how well it works.

Eligibility Criteria

This trial is for adults with severe aplastic anemia who haven't improved or have relapsed after immunosuppressive therapy. They can't be candidates for stem cell transplant, either because it's not suitable or they've refused it. Participants need to have good liver and kidney function.

Inclusion Criteria

My liver and kidney functions are within normal ranges.

I cannot or do not want to undergo a stem cell transplant.

My aplastic anemia has returned after immune-suppressive therapy.

+1 more

Exclusion Criteria

I haven't had T cell-depleting therapy like ATG or alemtuzumab in the last 6 months.

I have been diagnosed with Fanconi anemia or another similar bone marrow failure condition.

I have PNH with significant red blood cell breakdown or a history of blood clots due to PNH.

+6 more

Participant Groups

The study tests REGN7257's safety and how well patients tolerate it. For part of the study, they'll also check if the drug effectively treats severe aplastic anemia by observing clinical responses, changes in transfusion needs, blood counts, and immune reactions over time.

2Treatment groups

Experimental Treatment

Group I: Part BExperimental Treatment1 Intervention

Part B: Multiple REGN7257 dosages.

Group II: Part AExperimental Treatment1 Intervention

Part A: Single ascending dose (SAD) escalation cohorts