Gene therapy trial offers hope for babies with rare, deadly metabolic disease

NCT ID NCT06255782

Recruiting now Disease control Sponsor: iECURE, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 2 times

Summary

This study tests a one-time gene therapy called ECUR-506 in baby boys under 9 months old with a severe form of OTC deficiency, a genetic disorder that prevents the body from breaking down ammonia. The goal is to see if the treatment is safe and can reduce dangerous ammonia levels, potentially allowing babies to stop taking daily medications. The trial involves up to 20 participants and will monitor for side effects and effectiveness over time.

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Conditions

The condition(s) this trial relates to.

Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn brain disorder central nervous system disorder hereditary disease Hyperammonemia inborn disorder of amino acid metabolism inborn errors of metabolism liver disorder metabolic disease nervous system disorder ornithine carbamoyltransferase deficiency urea cycle disorder X-linked disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ORNITHINE CARBAMOYLTRANSFERASE DEFICIENCY (DISORDER) ORNITHINE TRANSCARBAMYLASE DEFICIENCY ORNITHINE TRANSCARBAMYLASE DEFICIENCY DISEASE UREA CYCLE DISORDERS, INBORN

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Children's Hospital of Colorado, Anshutz Medical Campus
RECRUITING

Aurora, Colorado, 80045, United States
Emory University School of Medicine
RECRUITING

Atlanta, Georgia, 30322, United States
Great Ormond Street Hospital
RECRUITING

London, United Kingdom
Hopsital Sant Joan de Deu
RECRUITING

Barcelona, 08950, Spain
Hospital Universitario 12 de Octubre
RECRUITING

Madrid, 28041, Spain
Icahn School of Medicine at Mount Sinai
RECRUITING

New York, New York, 10029, United States
Oregon Health and Science University
RECRUITING

Portland, Oregon, 97239, United States
The Children's Hospital at Westmead
ACTIVE_NOT_RECRUITING

Sydney, New South Wales, Australia
The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital
RECRUITING

Newcastle upon Tyne, United Kingdom
The Royal Children's Hospital
ACTIVE_NOT_RECRUITING

Melbourne, Victoria, 3052, Australia
UCLA Mattel Children's Hospital
RECRUITING

Los Angeles, California, 90095, United States

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