New stem cell approach aims to cure rare immune disease
NCT ID NCT02629120
First seen Jun 26, 2026 · Last updated Aug 07, 2026 · Updated 7 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. 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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States