Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCTID	NCT02716246 (View at clinicaltrials.gov)
Description	The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA. (Show More)
Development Status	Active
Indication	Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Disease Ontology Term	DOID:0111395
Compound Name	UX111
Compound Alias	Rebisufligene etisparvovec, ABO-102
Compound Description	scAAV9.U1a.hSGSH
Sponsor	Ultragenyx Pharmaceutical Inc
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	36 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	SGSH
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	0.5E13 vg/kg
Dose 2	1.0E13 vg/kg
Dose 3	3.0E13 vg/kg (n=22)

No.of Trial Sites	5
Locations	United States,Australia,Spain

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	BLA filed under Accelerated Approval, CRL issued 7/11/25 requesting additional information and improvements related to CMC and manufacturing inspections