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

Ornithine Transcarbamylase Deficiency Clinical Trial

Official title:
A Phase I/II 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

Clinical Trial 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, open-label, multicenter, safety 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 and tolerability of multiple dose levels of ECUR-506 following intravenous (IV) administration of a single dose.

Clinical Trial Description

The study drug, ECUR-506, is an investigational gene editing therapy. Gene editing is a way to repair, replace, or introduce new copies of genes that don't work. The study drug contains a working copy of the OTC gene that will be delivered by an IV infusion. It also contains a gene to encode the editing enzyme which is the part of the study drug that can cut DNA so that the OTC gene can be inserted. The study drug was designed to introduce a working copy of the OTC gene and a gene to encode the editing enzyme. A gene cannot enter cells by itself, it needs a delivery mechanism to move the gene into the cells. In this study, a commonly used virus called adeno-associated virus (AAV) is used to enter the cells and deliver the genes. ;

Study Design

Related Conditions & MeSH terms

Deficiency Diseases
Ornithine Transcarbamylase Deficiency
Ornithine Transcarbamylase Deficiency Disease
Urea Cycle Disorders, Inborn

Clinical Trial Details

NCT number	NCT06255782
Study type	Interventional
Source	iECURE, Inc.
Contact	George Diaz, M.D., Ph.D.
Phone	1-877-694-3558
Email	medinfo@iecure.com
Status	Recruiting
Phase	Phase 1/Phase 2
Start date	April 8, 2024
Completion date	September 2026

View Details

See also
Status	Clinical Trial	Phase
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Active, not recruiting	`NCT04442347` - Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency	Phase 1
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Recruiting	`NCT05092685` - Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn	Phase 1/Phase 2
Completed	`NCT00472732` - Neurologic Injuries in Adults With Urea Cycle Disorders	N/A
Terminated	`NCT04909346` - Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Completed	`NCT04416126` - Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects	Phase 1
Recruiting	`NCT05526066` - Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810	Phase 2
Completed	`NCT00718627` - Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders	Phase 2
Recruiting	`NCT04908319` - Hepatic Histopathology in Urea Cycle Disorders
Withdrawn	`NCT03767270` - Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency	Phase 1/Phase 2
Completed	`NCT04269122` - A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.