Investigating a new treatment for pediatric aplastic anemia
Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
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 ID | NIH-11105281 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with acquired aplastic anemia who are in need of new treatment options.
Not a fit: Patients with other forms of anemia or those who are not diagnosed with aplastic anemia may not benefit from this research.
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 performed: While this specific approach is novel, similar therapies targeting immune-mediated conditions have shown promise in other studies.
Where this research is happening
New York, United States
- 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
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 below.