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Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

NCT05092685 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2023-11-07

No results posted yet for this study

Summary

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).

Conditions

  • Ornithine Transcarbamylase Deficiency

Interventions

GENETIC

AAVLK03hOTC

Peripheral intravenous infusion of AAVLK03hOTC.

Sponsors & Collaborators

  • University College, London

    lead OTHER

Principal Investigators

  • Federicco Mingozzi · Genethon

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Days
Max Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-11-01
Primary Completion
2026-06-30
Completion
2027-06-30

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05092685 on ClinicalTrials.gov