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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

NCT06255782 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-04-15

No results posted yet for this 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 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.

Conditions

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

Interventions

GENETIC

ECUR-506

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

Sponsors & Collaborators

  • iECURE, Inc.

    lead INDUSTRY

Principal Investigators

  • George Diaz, M.D., Ph.D · iECURE, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
24 Hours
Max Age
7 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-08
Primary Completion
2027-12-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06255782 on ClinicalTrials.gov