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 with removal of dietary protein restriction and alternative pathway medication. (Show More)
Indication	Ornithine Transcarbamylase Deficiency, Late Onset
Compound Name	Avalotcagene ontaparvovec (DTX301)
Sponsor	Ultragenyx Pharmaceutical Inc
Funder Type	Industry
Status	Recruiting
Enrollment Count	50

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
Dose 1	1.0 x 10^13 GC/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	25
Locations	Canada,Argentina,Netherlands,United States,Japan,Brazil,Italy,United Kingdom,France,Portugal,Germany,Spain

Has US IND	True
Recent Updates	Enrollment expected to be completed H2 2024