← Eichor
Study identifier: NCT05092685 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

Condition: Ornithine Transcarbamylase Deficiency  ·  Sponsor: University College, London

PhasePhase 1/Phase 2
Planned participants12
Who can joinAll sexes, 0 Days to 16 Years
Healthy volunteersNo

About this study

Ornithine transcarbamylase deficiency (OTCD) is an inherited metabolic liver disease which means that the body cannot maintain normal levels of ammonia. Ammonia levels can rise (called hyperammonaemic decompensations) which can be life-threatening and may result in impaired neurological development in children. OTCD is a rare genetic disorder characterised by complete or partial lack of the enzyme ornithine transcarbamylase (OTC).

This description comes directly from the study's public registry record.

Talk to the study team

Trial Manager  ·  +44 (0) 20 7907 4669  ·  cctu.horace@ucl.ac.uk

Always discuss trial participation with your own doctor first.

Locations (1)

Great Ormond Street HospitalLondon, United KingdomRecruiting

Follow this study

Get one email when the public record changes — results posted, or the study's status changes. Nothing else, ever.

We email about this public record only. Unsubscribe anytime with one click. Never medical advice.

Is this your study? This page was generated automatically from the public registry record. Sponsors can claim it — free — to add branding and verified contact routing. Claim this page →

This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.

Source record: clinicaltrials.gov/study/NCT05092685