Gene therapy boost for krabbe patients after transplant
NCT ID NCT05739643
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a single infusion of a gene therapy called FBX-101 in 9 people with Krabbe disease who have already received a stem cell transplant. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor function and safety. Researchers will compare results to patients who only had the transplant.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- FBX-101 (a gene therapy that delivers a working copy of the GALC gene via a harmless virus)
- What this could lead to
- If successful, this could improve motor function and slow disease progression for people with Krabbe disease who have already had a stem cell transplant.
- What could go wrong
- This is a very early, small trial (9 people) focused mainly on safety. The gene therapy may not provide additional benefit beyond the transplant, and there are unknown risks from the virus and gene insertion.
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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Children's Hospital of Orange County (CHOC)
Orange, California, 92868, United States
-
Duke University Medical Center
Durham, North Carolina, 27705, United States
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University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a repurposed drug slow rare childhood brain diseases?
- Global krabbe disease registry aims to decode early warning signs
- Newborn screening study aims to catch rare diseases at birth
- Gene therapy after stem cell transplant shows promise for rare brain disease
- Brain scan breakthrough could save babies from rare disease
- Major study tracks rare brain diseases to unlock their secrets