Gene therapy after stem cell transplant shows promise for rare brain disease
NCT ID NCT04693598
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor skills like sitting independently. The main goals are safety and checking whether the treatment helps children achieve better movement compared to those who only had a stem cell 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 using a harmless virus to deliver a working copy of the GALC gene)
What this could lead to
If successful, this could improve motor function and quality of life for children with Krabbe disease, offering a better outcome than stem cell transplant alone.
What could go wrong
This is an early-phase trial with only 6 participants, so results may not apply to all patients. Gene therapy carries risks like immune reactions or the treatment not working as expected.
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
-
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109, United States