Actively Recruiting
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Led by iECURE, Inc. · Updated on 2026-04-15
20
Participants Needed
12
Research Sites
190 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male sex
- Gestational or adjusted gestational age of at least 37 weeks
- Age at screening between 24 hours and 7 months
- Weight between 3.5 kg and 13.5 kg at screening
- Has received age-appropriate vaccinations
- Genetically confirmed OTC deficiency or same OTC variant as a family member with severe neonatal OTC deficiency
- Severe neonatal OTC deficiency with hyperammonemic crisis and elevated ammonia over 560 µmol/L within first week of life, treated with dietary protein restriction and nitrogen scavenger therapy
- Current or past biochemical profile consistent with OTC deficiency
- Parent or legal representative able to understand and sign informed consent
You will not qualify if you...
- Severe to profound hypoxic ischemic encephalopathy diagnosed at birth due to injury
- Urgent liver transplant needed due to liver failure
- Contiguous gene deletion involving OTC and CYBB or TSPAN7 genes
- Known or suspected major organ injury, dysfunction, or anomalies
- Vital signs or lab tests outside normal ranges
- Treatment with other gene or gene editing therapies
- Participation in another study without sponsor approval
- Any condition that may affect safety or study data as judged by the investigator
- Documented transmission of Hepatitis A, B, or C
- Documented prenatal exposure to teratogens, substances, or alcohol that may increase risk of developmental or medical issues
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 12 locations
1
UCLA Mattel Children's Hospital
Los Angeles, California, United States, 90095
Actively Recruiting
2
Children's Hospital of Colorado, Anshutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
4
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
5
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Actively Recruiting
6
Oregon Health and Science University
Portland, Oregon, United States, 97239
Actively Recruiting
7
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
Active, Not Recruiting
8
The Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
Active, Not Recruiting
9
Hopsital Sant Joan de Deu
Barcelona, Spain, 08950
Actively Recruiting
10
Hospital Universitario 12 de Octubre
Madrid, Spain, 28041
Actively Recruiting
11
Great Ormond Street Hospital
London, United Kingdom
Actively Recruiting
12
The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital
Newcastle upon Tyne, United Kingdom
Actively Recruiting
Research Team
G
George Diaz, M.D., Ph.D.
CONTACT
T
Trial Recruitment
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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