Trial Profile

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 24 Jun 2025

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At a glance

Drugs Avalotcagene ontaparvovec (Primary) ; Sodium acetate (Primary) ; Prednisolone
Indications Ornithine carbamoyltransferase deficiency disease
Focus Pharmacokinetics; Registrational; Therapeutic Use
Acronyms Enh3ance
Sponsors Ultragenyx Pharmaceutical

15 Jun 2025 Planned number of patients changed from 50 to 32.
15 Jun 2025 Planned End Date changed from 1 Dec 2028 to 1 Mar 2031.
13 Feb 2025 Status changed from recruiting to active, no longer recruiting, according to an Ultragenyx Pharmaceutical media release.

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