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.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for KRABBE DISEASE are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Children's Hospital of Orange County (CHOC)
Orange, California, 92868, United States
-
Duke University Medical Center
Durham, North Carolina, 27705, United States
-
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109, United States