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NCT06255782 · ClinicalTrials.gov registry record · Phase 1

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 study of Ornithine Transcarbamylase Deficiency and Urea Cycle Disorders, Inborn, sponsored by iECURE.

Recruiting
Registry status
Phase 1
Development phase
8
Enrollment target
10
Study locations

NCT06255782: Recruiting Phase 1 study of Ornithine Transcarbamylase Deficiency and Urea Cycle Disorders, Inborn, sponsored by iECURE.

NCT06255782 is a Phase 1 study of Ornithine Transcarbamylase Deficiency and Urea Cycle Disorders, Inborn that is actively recruiting participants, run by iECURE. The registered enrollment target is 8 participants, below the 65-participant average among 4 other Ornithine Transcarbamylase Deficiency trials with a reported enrollment target (88% lower). The trial reports 10 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT06255782, a Phase 1 study of Ornithine Transcarbamylase Deficiency and Urea Cycle Disorders, Inborn, is actively recruiting participants, sponsored by iECURE.

RECRUITING
Registry status
Phase 1
Development phase
8 participants
Enrollment target
10
Study locations

Study 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 1/2/3, open-label, multicenter, safety, efficacy, 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, tolerability, and efficacy of up to three dose levels of ECUR-506 following intravenous (IV) administration of a single dose.

Primary Outcome

Toxicity is graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Adverse events (AEs) are reported based on clinical laboratory tests, vital signs, physical examinations, Pediatric Neurologist exam parameters, electrocardiograms, and any other medically indicated assessments from the time informed consent is signed through the end of the safety follow-up period. AEs are considered to be treatment emergent (TEAE) if th

Conditions Studied

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

Interventions

  • GENETIC ECUR-506

Study Locations (10)

Other

  • Hopsital Sant Joan de Deu - Barcelona
  • Hospital Universitario 12 de Octubre - Madrid
  • Great Ormond Street Hospital - London
  • The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital - Newcastle upon Tyne

California

  • UCLA Mattel Children's Hospital - Los Angeles

Colorado

  • Children's Hospital of Colorado, Anshutz Medical Campus - Aurora

Illinois

  • Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago

New York

  • Icahn School of Medicine at Mount Sinai - New York

New South Wales

  • The Children's Hospital at Westmead - Sydney

Victoria

  • The Royal Children's Hospital - Melbourne

Trial Details

FieldValue
Enrollment Target 8 participants
Start Date 2024-04-08
Est. Completion 2026-09
Phase Phase 1
iECURE

1 total trials

What NCT06255782 shows while recruiting

NCT06255782 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 65-participant average among 4 other Ornithine Transcarbamylase Deficiency trials with a reported enrollment target (88% lower).

The record links to 4 conditions, with Ornithine Transcarbamylase Deficiency appearing as the primary indexed condition, and to 1 intervention - of which ECUR-506 is the first listed.

NCT06255782 lists 10 locations in 7 states (Other, California, Colorado).

Frequently Asked Questions

What is clinical trial NCT06255782 about?

NCT06255782 is a clinical study titled "An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency". 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, potential...

What is the current status of trial NCT06255782?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 8 participants. The study started on 2024-04-08. Estimated completion is 2026-09.

What conditions does trial NCT06255782 study?

This clinical trial studies the following conditions: Ornithine Transcarbamylase Deficiency, Urea Cycle Disorders, Inborn, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder).

What interventions are being tested in trial NCT06255782?

The interventions under investigation include: ECUR-506 (GENETIC).

Who is sponsoring clinical trial NCT06255782?

This trial is sponsored by iECURE, which has 1 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT06255782 being conducted?

This trial has 10 study locations across California, Colorado, Illinois, New York, New South Wales. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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More recruiting context for NCT06255782

Source: ClinicalTrials.gov NCT06255782, the US trial registry maintained by the National Library of Medicine. NCT06255782 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.