New drug combo aims to make bone marrow transplants safer for kids
NCT ID NCT02928991
First seen May 31, 2026 · Last updated Jun 23, 2026 · Updated 3 times
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.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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.
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.