Could a Half-Matched donor cure a rare immune disease?
NCT ID NCT03910452
First seen Jun 26, 2026 · Last updated Aug 05, 2026 · Updated 5 times
Summary
This early-phase trial tests whether a bone marrow transplant from a partially matched relative can safely cure chronic granulomatous disease (CGD), a condition that causes severe infections and inflammation. Four people aged 4 to 65 with CGD and no perfect donor will receive chemotherapy, radiation, and immune-suppressing drugs before and after the transplant to help their body accept the new cells. The goal is to see if the transplant can restore normal immune function without causing severe graft-versus-host disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- bone marrow transplant from a partially matched relative, with chemotherapy (busulfan), immune-suppressing drug (alemtuzumab), radiation, and post-transplant cyclophosphamide
- What this could lead to
- If it works, this could offer a cure for people with chronic granulomatous disease who don't have a perfectly matched donor.
- What could go wrong
- This is a very early, small trial (only 4 people) testing a high-risk procedure. The transplant may fail, cause severe graft-versus-host disease, or lead to serious infections or other complications.
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
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Newborn screening study aims to catch rare diseases at birth
- Gene fix for rare immune disease: can it stop deadly infections?
- Groundbreaking gene therapy aims to fix immune system in rare disease
- New hope for CGD: stem cell transplant trial aims for a cure
- Milder stem cell transplant shows promise for kids with immune disorders
- Scientists dig into immune cells to unlock lung disease mysteries