Actively Recruiting
Phase 2 Study of Emapalumab Treatment for Children and Young Adults with Severe Aplastic Anemia
Led by Memorial Sloan Kettering Cancer Center · Updated on 2025-12-17
35
Participants Needed
6
Research Sites
N/A
Total Duration
AI-Summary
What this Trial Is About
This research aims to evaluate whether starting treatment with emapalumab can improve treatment planning and increase the effectiveness of standard therapies for pediatric patients with severe aplastic anemia sAA. The study focuses on children and young adults under 25 years old who have been newly diagnosed with sAA characterized by severe cytopenias and hypocellular bone marrow. The trial is sponsored by Memorial Sloan Kettering Cancer Center and funded by the FDAs Office of Orphan Products Development. Participants will initially receive emapalumab, an antibody that blocks interferon gamma, for six weeks. After this initial treatment, they will either receive standard immunosuppressive therapy IST with equine anti-thymocyte globulin and cyclosporin along with a reduced dose of emapalumab, or they will proceed to a standard hematopoietic stem cell transplant HCT. These two treatment paths are both experimental arms within the study. During the study, participants will be closely monitored to assess their response to treatment at six weeks. Evaluations include clinical assessments and laboratory tests to measure blood counts and marrow function. Researchers will track the best response to therapy and monitor safety throughout the process. The total study duration and follow-up extend up to May 2029, allowing for long-term observation of outcomes.
CONDITIONS
Brief Title
A Study of Emapalumab for Pediatric Aplastic Anemia
Research Team
A
Andromachi Scaradavou, MD
J
Jaap Jan Boelens, MD, PhD
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