High-Dose stem cell transplant aims to cure rare immune disease
NCT ID NCT02629120
First seen Nov 01, 2025 · Last updated May 25, 2026 · Updated 32 times
Summary
This study tests a high-dose stem cell transplant for people with chronic granulomatous disease (CGD), a condition that weakens the immune system. The goal is to replace faulty stem cells with healthy donor cells to cure the disease. To prevent the donor cells from attacking the patient's body, two drugs (cyclophosphamide and sirolimus) are given after the transplant. The study includes 45 participants aged 4 to 65 who have a matched donor.
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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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Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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