A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

NCTID	NCT05139316 (View at clinicaltrials.gov)
Description	The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control. (Show More)
Development Status	Active
Indication	Glycogen Storage Disease Type Ia
Disease Ontology Term	DOID:2749
Compound Name	DTX401
Compound Alias	Pariglasgene brecaparvovec
Sponsor	Ultragenyx Pharmaceutical Inc
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	49 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	G6PC
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	AAV8
Editor Type	none
Dose 1	Phase 1/2: 2E12 GC/kg
Dose 2	Phase 1/2: 6E12 GC/kg
Dose 3	Phase 3: 1.0E13 GC/kg
Dose 4
Dose 5

No.of Trial Sites	20
Locations	Canada,Netherlands,United States,Japan,Brazil,Denmark,Italy,Germany,Spain

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates	BLA rolling submission initiated, complete submission expected in 4Q25