Small study paves way for gene therapy in rare childhood anemia
NCT ID NCT02678533
First seen Nov 01, 2025 · Last updated Apr 28, 2026 · Updated 20 times
Summary
This pilot study tested whether a combination of two drugs (G-CSF and plerixafor) could safely collect enough stem cells from the blood of children with Fanconi anemia for future gene therapy. Four patients aged 2 to 17 took part. The main goal was to see if the stem cell collection was feasible, and the study also tracked side effects. Results will help design a later gene therapy trial.
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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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Locations
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Hôpital necker-Enfants malades
Paris, PARIS, 75015, France
Conditions
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