NCT06255782

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: iECURE, Inc. · Industry
• Sex: Male
• Age: 24 Hours – 7 Months
• Healthy volunteers: Not accepted

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 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.

Detailed description

The study drug, ECUR-506, is an investigational gene editing therapy. Gene editing is an approach used to repair, replace, or introduce functional copies of genes that are not working properly. ECUR-506 contains a functional copy of the OTC gene, along with a gene to encode an editing enzyme that enables insertion of the OTC gene into the genome. The study drug is administered as a single IV infusion. Because genes cannot enter cells on their own, ECUR-506 uses a delivery system based on adeno-associated virus (AAV), a commonly used viral vector, to transport the genetic material into cells.

Conditions

• Ornithine Transcarbamylase Deficiency
• Ornithine Transcarbamylase Deficiency Disease
• Ornithine Carbamoyltransferase Deficiency (Disorder)
• Urea Cycle Disorders, Inborn

Interventions

Type	Name	Description
GENETIC	ECUR-506	ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene.

Timeline

• Start date: 2024-04-08
• Primary completion: 2027-12-01
• Completion: 2027-12-01
• First posted: 2024-02-13
• Last updated: 2026-04-15

Locations

12 sites across 4 countries: United States, Australia, Spain, United Kingdom

Regulatory

• FDA-regulated drug study