New stem cell approach aims to cure rare immune disease
NCT ID NCT02629120
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a high-dose stem cell transplant for people with chronic granulomatous disease (CGD), a condition that weakens the immune system. Participants receive donor stem cells along with drugs to prevent rejection and graft-versus-host disease. The goal is to improve transplant success while reducing complications.
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 (donor peripheral blood stem cells) with conditioning drugs (alemtuzumab, busulfan) and immunosuppressants (cyclophosphamide, sirolimus)
What this could lead to
If successful, this approach could offer a safer, more effective stem cell transplant option for people with chronic granulomatous disease, potentially controlling the disease long-term.
What could go wrong
This is an early-phase trial with only 45 participants, so results may not apply to everyone. There are serious risks like graft-versus-host disease, infection, and organ damage from the conditioning chemotherapy.
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
Bethesda, Maryland, 20892, United States