An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Recruiting now · Phase 1/Phase 2
Conditions studied: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
In brief
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia. Individuals with OTC deficiency can develop elevated levels of ammonia in the blood, potentially resulting in severe consequences, including cumulative and irreversible neurological damage, coma, and death. The most severe form presents shortly after birth and occurs more commonly in boys than girls. This is a Phase 1/2/3, open-label, multicenter study evaluating the safety, efficacy, and dose of ECUR-506 in male babies with neonatal-onset OTC deficiency. The primary objective is to evaluate the safety, tolerability, and efficacy of up to three dose levels of ECUR-506 following intravenous (IV) administration of a single dose.
Key facts
- Study ID
- NCT06255782
- Run by
- iECURE, Inc.
- People needed
- 20
- Starts
- 2024-04-08
- Expected to finish
- 2027-12-01
- Last updated by the study team
- 2026-04-15
Who can join
Age: 0 and older, up to 1. Sex: male. Healthy volunteers: not accepted.
Where it is running
- UCLA Mattel Children's Hospital — Los Angeles, California, United States (enrolling)
- Children's Hospital of Colorado, Anshutz Medical Campus — Aurora, Colorado, United States (enrolling)
- Emory University School of Medicine — Atlanta, Georgia, United States (enrolling)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
- Icahn School of Medicine at Mount Sinai — New York, New York, United States (enrolling)
- Oregon Health and Science University — Portland, Oregon, United States (enrolling)
- Great Ormond Street Hospital — London, United Kingdom (enrolling)
- The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital — Newcastle upon Tyne, United Kingdom (enrolling)
- Hopsital Sant Joan de Deu — Barcelona, Spain (enrolling)
- Hospital Universitario 12 de Octubre — Madrid, Spain (enrolling)
- The Children's Hospital at Westmead — Sydney, New South Wales, Australia
- The Royal Children's Hospital — Melbourne, Victoria, Australia
Full record on ClinicalTrials.gov
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