Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT (NCT05739643)
A Phase 1/2 Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)
🩺 Plain-English Summary (8th-Grade Level)
This is a non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital of Orange County (CHOC) — Orange, California
Age range: Not specified to 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (3 Sites)
- Children's Hospital of Orange County (CHOC) — Orange, California
- University of Michigan Hospitals - Michigan Medicine — Ann Arbor, Michigan
- Duke University Medical Center — Durham, North Carolina
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Krabbe Disease
- Gene Transfer Clinical Trial for Krabbe Disease — Ann Arbor, Michigan
- Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease — Pittsburgh, Pennsylvania
- Krabbe Disease Global Patient Registry — Buffalo, New York
Explore by condition: All Krabbe Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.