Investigating a new treatment for pediatric aplastic anemia
Official title: Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
Funded at Sloan-Kettering Inst Can Research · NIH-11105281 · Data updated 2026-11-12
This study is looking at how well a new treatment called emapalumab works for kids with acquired aplastic anemia, a rare blood disorder where the body stops making enough blood cells, to see if it can help them produce more blood cells and feel better.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Sloan-Kettering Inst Can Research NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | September 5, 2024 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $650,000 |
| NIH project number | 1R01FD008175-01A1 |
| Project ID | NIH-11105281 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with acquired aplastic anemia who are in need of new treatment options.
✕Less likely to help
Patients with other forms of anemia or those who are not diagnosed with aplastic anemia may not benefit from this research.
What this research studies
This research focuses on a rare blood disorder called acquired aplastic anemia (AA) in children, where the immune system mistakenly attacks blood-producing stem cells. The study aims to evaluate the effectiveness of a treatment called emapalumab, which may help restore blood cell production. Participants will be closely monitored to assess their response to the treatment and any potential side effects. The research seeks to provide a more effective therapy option for children diagnosed with this life-threatening condition.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options and outcomes for children suffering from aplastic anemia.
How similar studies have gone: While this specific approach is novel, similar therapies targeting immune-mediated conditions have shown promise in other studies.
Where this research is happening
-
Sloan-Kettering Inst Can Research
New York, United States Active
Researchers
- Principal investigator: Oved, Joseph H — Sloan-Kettering Inst Can Research
- Study coordinator: Oved, Joseph H
Related research
Other NIH-funded projects on the same topics.
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Emapalumab for children with aplastic anemia
Doctors will give emapalumab to children with acquired aplastic anemia to try to stop the immune attack on the bone marrow and help it make blood cells.
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Clinical and immunologic study of pediatric severe aplastic anemia to personalize care
A pediatric-focused research effort will collect and study immune and clinical data to tailor treatments for children with severe aplastic anemia.
-
Aplastic Anemia Registry to Understand How Immune and Genetic Discoveries Affect Disease
A collaborative registry study to learn how genetic background and immune factors influence aplastic anemia and related bone marrow failure conditions in patients.
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Eltombopag: a new way to boost blood stem cells in aplastic anemia
A new drug approach aims to help people with idiopathic aplastic anemia by stimulating blood-forming stem cells through mechanisms that may not depend on traditional pathways.
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Understanding how Eltrombopag helps with aplastic anemia
This research explores how the medication Eltrombopag helps patients with aplastic anemia by looking at its effects on blood-forming cells.
-
Comparing bone marrow transplantation with immune therapy for severe aplastic anemia in young patients
This study is looking at whether bone marrow transplants from unrelated donors or immune suppression therapy works better for kids and young adults with newly diagnosed severe aplastic anemia, especially since not everyone has a sibling who can donate bone marrow.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.