Gene therapy could end pain crises for sickle cell patients
NCT ID NCT05353647
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy that modifies a patient's own blood stem cells to produce more fetal hemoglobin, a healthy type that prevents sickling. The goal is to reduce or eliminate severe pain crises in people with sickle cell disease. The treatment involves chemotherapy to prepare the body, then infusion of the modified cells. It is for 25 patients aged 13-40 with severe disease who have had at least 4 pain crises in the last 2 years.
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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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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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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, 30322, United States
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Dana-Farber Cancer Institute/Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
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Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States