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Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn — Stella

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Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(1 site)

United Kingdom

Great Ormond Street Hospital, London

Key details

Phase

Early testing / Safety & dosing

Sponsor

University College, London

Enrollment target

~12 participants

Started

November 2023

Primary completion

June 2026

Age range

0 Days – 16 Years

Last updated November 2023

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