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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

Study on ECUR-506 for male babies with OTC deficiency.

Recruiting
24-7 years
Male
Phase 3
**Study on OTC Deficiency in Male Babies** **Ornithine Transcarbamylase (OTC) deficiency** is a genetic disorder affecting the liver's ability to remove ammonia, a harmful substance, from the body. Too much ammonia can lead to serious health issues like brain damage, coma, or even death. This study focuses on boys because they are more commonly affected shortly after birth. **ECUR-506** is a medication being tested to help treat this disorder. It uses **gene editing**, which is a technique to fix or replace faulty genes. Here, ECUR-506 delivers a healthy copy of the OTC gene to the liver using a virus that safely carries the gene into cells. This is done through a single **intravenous (IV) infusion** – a method where the medicine is given directly into the vein. **Key Points:** - The study involves a single IV dose and checks its safety and effectiveness. - Participants are male babies under 9 months with confirmed severe OTC deficiency. - Parents must be willing to sign consent forms for participation. This study offers a chance to explore new treatments for this serious condition, but parents should consider the study requirements and potential risks.
Study details
    Ornithine Transcarbamylase Deficiency
    Ornithine Transcarbamylase Deficiency Disease
    Ornithine Carbamoyltransferase Deficiency (Disorder)
    Urea Cycle Disorders
    Inborn

NCT06255782

iECURE, Inc.

26 September 2026

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