Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCTID	NCT05345171 (View at clinicaltrials.gov)
Description	The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels. (Show More)
Development Status	Active
Indication	Ornithine Transcarbamylase (OTC) Deficiency
Disease Ontology Term	DOID:9271
Compound Name	DTX301
Compound Alias	Avalotcagene ontaparvovec
Sponsor	Ultragenyx Pharmaceutical Inc
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	32 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	OTC
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: 3.4E12 GC/kg
Dose 2	Phase 1/2: 1.0E13 GC/kg
Dose 3	Phase 1/2: 1.7E13 GC/kg
Dose 4	Phase 3: 1.0E13 GC/kg
Dose 5

No.of Trial Sites	16
Locations	Canada,Argentina,Netherlands,United States,Japan,Brazil,France,Portugal,Germany,Spain

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	Enrollment of Phase 3 study complete in February 2025