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

NCTID	NCT05739643 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Krabbe Disease
Disease Ontology Term	DOID:10587
Compound Name	FBX-101
Sponsor	Forge Biologics, Inc
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	9
Results Posted	Not Available

Target Gene/Variant	GALC
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh10
Editor Type	none
Dose 1	1.6E13 gc/kg
Dose 2	Undisclosed dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Dose escalation to highest dose approved, will start 2Q24