New transplant approach offers hope for severe sickle cell disease
NCT ID NCT03240731
First seen Jun 26, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This phase 2 trial tested a bone marrow transplant from a half-matched (haploidentical) donor in 25 people with severe sickle cell disease. The goal was to see if a less intense conditioning regimen and a drug called cyclophosphamide could prevent graft-versus-host disease while allowing the donor cells to take over. The main measure of success was survival without sickle cell symptoms and with low levels of abnormal hemoglobin.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
bone marrow transplant from a half-matched donor
What this could lead to
If successful, this approach could offer a way to control severe sickle cell disease with a less intense transplant procedure, potentially reducing long-term complications.
What could go wrong
This is a small, early-phase trial with only 25 participants, so results may not apply to everyone. The transplant carries risks like graft-versus-host disease and infection, and lifelong monitoring may still be needed.
Disclaimer
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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.
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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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
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Locations
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CHU Henri-Mondor
Créteil, 94000, France
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CHU La Timone
Marseille, France
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CHU Strasbourg
Strasbourg, France
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Hospital Necker
Paris, France
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Hospital Robert-Debré
Paris, France
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Saint-Louis hospital
Paris, France
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intercommunal hospital of Créteil
Créteil, 94000, France