New stem cell transplant could help sickle cell patients without a perfect donor match
NCT ID NCT00977691
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a stem cell transplant using cells from a half-matched family donor, combined with low-dose radiation and drugs to prevent rejection, for adults with severe sickle cell disease or beta-thalassemia. The goal is to see if this gentler approach can replace the diseased cells without the high toxicity of standard chemotherapy. Twenty-three participants will be followed for five years to check if the donor cells survive and reduce disease symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
stem cell transplant (from a half-matched donor) with alemtuzumab, sirolimus, cyclophosphamide, and low-dose radiation
What this could lead to
If successful, this approach could offer a treatment option for sickle cell disease and beta-thalassemia patients who lack a fully matched donor, potentially reducing disease complications.
What could go wrong
This is an early-phase trial with only 23 participants, so results may not apply broadly. The transplant carries risks like graft rejection, infection, and side effects from immunosuppressant drugs.
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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National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892, United States