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An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
NCT06255782 · View on ClinicalTrials.gov ↗
Study Summary
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 detoxification of ammonia. Individuals with OTC deficiency can build-up excess levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. This is a Phase 1/2/3, open-label, multicenter, safety, efficacy, and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency. The primary objective of this study 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 Studied
Interventions
- GENETIC ECUR-506
Study Locations (10)
Other
- Hopsital Sant Joan de Deu — Barcelona
- Hospital Universitario 12 de Octubre — Madrid
- Great Ormond Street Hospital — London
- The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital — Newcastle upon Tyne
California
- UCLA Mattel Children's Hospital — Los Angeles
Colorado
- Children's Hospital of Colorado, Anshutz Medical Campus — Aurora
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
New York
- Icahn School of Medicine at Mount Sinai — New York
New South Wales
- The Children's Hospital at Westmead — Sydney
Victoria
- The Royal Children's Hospital — Melbourne
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2024-04-08 |
| Est. Completion | 2026-09 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06255782
The ClinicalTrials.gov registry entry for NCT06255782 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 8 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is iECURE, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 4 conditions, with Ornithine Transcarbamylase Deficiency appearing as the primary indexed condition, and to 1 intervention — of which ECUR-506 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06255782 reports 10 study locations spanning 7 distinct geographic areas — top geographies include Other, California, Colorado. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06255782 about?
NCT06255782 is a clinical study titled "An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency". 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 detoxification of ammonia. Individuals with OTC deficiency can build-up excess levels of ammonia in their blood, potential...
What is the current status of trial NCT06255782?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 8 participants. The study started on 2024-04-08. Estimated completion is 2026-09.
What conditions does trial NCT06255782 study?
This clinical trial studies the following conditions: Ornithine Transcarbamylase Deficiency, Urea Cycle Disorders, Inborn, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06255782?
The interventions under investigation include: ECUR-506 (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06255782?
This trial is sponsored by iECURE, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06255782 being conducted?
This trial has 10 study locations across California, Colorado, Illinois, New York, New South Wales. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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