An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Recruiting
Phase 3
Interventional Study
Ornithine Transcarbamylase Deficiency
Ornithine Transcarbamylase Deficiency Disease
Ornithine Carbamoyltransferase Deficiency (Disorder)
Urea Cycle Disorders, Inborn
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 24 Hours – 7 Months
- Sex
- Male
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 20 (estimated)
- Sponsor
- iECURE, Inc. · Industry
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About This Trial
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, an…
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
1. Male sex
2. Gestational or adjusted (corrected) gestational age ≥ 37 weeks
3. Age at screening is 24 hours to 7 months
4. Weight ≥ 3.5 kg and ≤ 13.5 kg at screening
5. Has received age-appropriate vaccinations
6. Genetically confirmed OTCD defined by genetic confirmation …
Contacts