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.
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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
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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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- A common amino acid may calm sickle cell pain crises — a trial puts it to the test
- A drug that protects the liver may make stem cell transplants safer for children with sickle cell disease