Trial Profile

Phase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for Paediatric Patients With Ornithine Transcarbamylase Deficiency

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 11 Mar 2026

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At a glance

Drugs BGT OTCD (Primary)
Indications Ornithine carbamoyltransferase deficiency disease
Focus Adverse reactions; First in man
Acronyms HORACE

Most Recent Events

11 Mar 2026 New trial record
16 Nov 2023 Status changed from not yet recruiting to recruiting, according to a Bloomsbury Genetic Therapies Limited media release.

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