Condition: Ornithine Transcarbamylase Deficiency · Ornithine Transcarbamylase Deficiency Disease · Ornithine Carbamoyltransferase Deficiency (Disorder) · Sponsor: iECURE, Inc.
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.
This description comes directly from the study's public registry record.
George Diaz, M.D., Ph.D. · 1-877-694-3558 · medinfo@iecure.com
Trial Recruitment · clinicaltrials@iecure.com
Always discuss trial participation with your own doctor first.
| UCLA Mattel Children's Hospital | Los Angeles, California, United States | Recruiting |
| Children's Hospital of Colorado, Anshutz Medical Campus | Aurora, Colorado, United States | Recruiting |
| Emory University School of Medicine | Atlanta, Georgia, United States | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago, Illinois, United States | Recruiting |
| Icahn School of Medicine at Mount Sinai | New York, New York, United States | Recruiting |
| Oregon Health and Science University | Portland, Oregon, United States | Recruiting |
| The Children's Hospital at Westmead | Sydney, New South Wales, Australia | Active Not Recruiting |
| The Royal Children's Hospital | Melbourne, Victoria, Australia | Active Not Recruiting |
| Hopsital Sant Joan de Deu | Barcelona, Spain | Recruiting |
| Hospital Universitario 12 de Octubre | Madrid, Spain | Recruiting |
| Great Ormond Street Hospital | London, United Kingdom | Recruiting |
| The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital | Newcastle upon Tyne, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06255782