Gene therapy for krabbe disease: did it last?
NCT ID NCT06308718
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.
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 (gene therapy)
- What this could lead to
- If successful, this follow-up could show whether a single gene therapy infusion provides lasting safety and motor benefits for children with Krabbe disease.
- What could go wrong
- This is a tiny observational follow-up (only 2 participants) of an already terminated trial. It cannot prove effectiveness and may not apply to other patients.
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
-
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.
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