New drug combo aims to make bone marrow transplants safer for kids
NCT ID NCT02928991
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study includes 25 children up to age 22 with acquired aplastic anemia or inherited bone marrow failure.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Fludarabine, cyclophosphamide, and thymoglobulin (conditioning drugs) followed by bone marrow transplant
- What this could lead to
- If successful, this approach could improve donor cell engraftment and reduce transplant-related complications for children with bone marrow failure.
- What could go wrong
- This is a very early pilot study with only 25 participants, so results may not apply broadly. Risks include graft failure, infection, and transplant-related mortality.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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