Actively Recruiting
A Phase 1/2/3 Open-Label Dose-Escalation Study to Evaluate Safety and Efficacy of Single IV ECUR-506 in Male Babies Under 9 Months with Neonatal-Onset OTC Deficiency
Led by iECURE, Inc. · Updated on 2026-04-15
20
Participants Needed
12
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Ornithine Transcarbamylase (OTC) deficiency is a genetic metabolic disorder affecting the liver's ability to detoxify ammonia, often causing severe neurological damage, coma, or death shortly after birth. This condition is more common and severe in male babies. Researchers are studying ECUR-506, an investigational gene editing therapy, to evaluate its safety, tolerability, and effectiveness in treating male infants under 9 months old with neonatal-onset OTC deficiency in a Phase 1/2/3 open-label trial. ECUR-506 delivers a functional OTC gene and a gene for an editing enzyme using a viral vector through a single intravenous infusion. The study tests three dose levels: low, intermediate, and high, each given once by IV infusion. Participants receive only one dose and are monitored over time to assess the impact of this gene editing treatment on their condition. During the study, babies will be closely monitored for safety and response through physical exams, neurologic assessments, vital signs, blood tests, urinalysis, and ECGs at scheduled times up to 24 weeks after infusion. Researchers will track adverse events, ammonia levels, hospitalizations, and other clinical outcomes such as survival and need for liver transplant. The total participation period covers about 24 weeks of follow-up to evaluate ECUR-506's effects and safety.
CONDITIONS
Brief 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 37 weeks or more
- Age between 24 hours and 7 months at screening
- Weight between 3.5 kg and 13.5 kg at screening
- Received age-appropriate vaccinations
- Genetically confirmed OTC deficiency or same OTC gene variant as a family member with severe neonatal OTC deficiency
- Severe neonatal OTC deficiency defined by high ammonia levels (>560 µmol/L) and symptoms within the first week of life
- Currently receiving dietary protein restriction and nitrogen scavenger therapy
- Parent or legal representative able to understand and sign informed consent
You will not qualify if you...
- Severe to profound Hypoxic Ischemic Encephalopathy due to birth injury
- Urgent need for liver transplant due to liver failure
- Contiguous gene deletion involving OTC and specific adjacent genes (CYBB or TSPAN7)
- Known or suspected major organ injury, dysfunction, or anomalies
- Vital signs or lab tests outside normal ranges
- Previous treatment with gene therapy or gene editing therapy
- Participation in other clinical studies without sponsor approval
- Any condition deemed unsafe by the investigator
- Documented vertical transmission of Hepatitis A, B, or C
- Documented in-utero exposure to teratogens, substances, or alcohol increasing risk of developmental or medical complications
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single administration with follow-up lasting up to 24 weeks
Participants receive a single intravenous infusion of ECUR-506, a gene editing therapy delivering a functional OTC gene.
Multiple visits over 24 weeks post infusion for monitoring and assessments
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.
T
Trial Recruitment
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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