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: 14 Mar 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

13 Feb 2025 Status changed from recruiting to active, no longer recruiting, according to an Ultragenyx Pharmaceutical media release.
04 Feb 2025 Planned primary completion date changed from 1 Dec 2024 to 1 Mar 2025.
12 Jan 2025 According to an Ultragenyx Pharmaceutical media release, company expect enrollment to be completed in the early 2025.

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