New hope for CGD: stem cell transplant trial aims for a cure
NCT ID NCT05463133
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether adding certain drugs to a stem cell transplant can cure chronic granulomatous disease (CGD), a condition that weakens the immune system. About 50 people aged 4 to 65 with CGD will receive donor stem cells after a special drug and radiation regimen. The goal is to improve survival and reduce infections over 5 years of follow-up.
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 with drugs (alemtuzumab, busulfan, cyclophosphamide, sirolimus, and cytokine blockers)
What this could lead to
If successful, this could offer a cure for chronic granulomatous disease (CGD) by making stem cell transplants safer and more effective.
What could go wrong
This is an early-phase trial with only 50 participants, so results may not apply to everyone. The transplant and drugs carry risks like infection, graft-versus-host disease, and organ damage.
Disclaimer
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This is a summary of
the original study
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••