New hope for CGD: stem cell transplant trial aims for a cure

NCT ID NCT05463133

Recruiting now ⭐️ CURE ⭐️ Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

chronic granulomatous disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CHRONIC GRANULOMATOUS DISEASE

Contacts and locations

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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