An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Public ClinicalTrials.gov record NCT06255782. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2/3 First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT06255782
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 20 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 24 Hours to 7 Months
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 7, 2024
- Primary completion
- Nov 30, 2027
- Completion
- Nov 30, 2027
- Last update posted
- Aug 11, 2026
2024 – 2027
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCLA Mattel Children's Hospital | Los Angeles | California | 90095 | Recruiting |
| Children's Hospital of Colorado, Anshutz Medical Campus | Aurora | Colorado | 80045 | Recruiting |
| Emory University School of Medicine | Atlanta | Georgia | 30322 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | Recruiting |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | Recruiting |
| Oregon Health and Science University | Portland | Oregon | 97239 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 6 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06255782, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 11, 2026 · Synced Sep 18, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06255782 live on ClinicalTrials.gov.