Clinical Trial: Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

Study Status: RECRUITING
Recruit Status: RECRUITING
Study Type: INTERVENTIONAL

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Official Title: A Phase 1b Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM)

Brief Summary: 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.
Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group.