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

Part of paid clinical trials in Los Angeles, California.

Sponsor
iECURE, Inc.
Study ID
NCT06255782
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

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

Eligibility Criteria

Sex
MALE
Age
24 Hours - 7 Months
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Apr 8, 2024
Status verified
Apr 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
20 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Low Dose Level
    Participants will receive the Low Dose of ECUR-506 delivered one time via IV Infusion.
  • Experimental: Intermediate Dose Level
    Participants will receive an intermediate dose of ECUR-506 delivered on time via IV infusion
  • Experimental: High Dose Level
    Participants will receive a higher dose of ECUR-506 delivered one time via IV infusion.

Primary Outcome Measure

Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness) [ Time Frame: Over 24 weeks post infusion ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
UCLA Mattel Children's HospitalLos AngelesCalifornia90095
Monserrath Campos
[email protected]
Children's Hospital of Colorado, Anshutz Medical CampusAuroraColorado80045
Ruth Fisseha
[email protected]
Emory University School of MedicineAtlantaGeorgia30322
Eleanor Geller Botha
[email protected]
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Michael Sawin
[email protected]
Icahn School of Medicine at Mount SinaiNew YorkNew York10029
Silvia Gunderson
[email protected]
Oregon Health and Science UniversityPortlandOregon97239
Hadley Morotti, MS
[email protected]

Find similar trials in Los Angeles, CA

By research site
UCLA Mattel Children's Hospital· Los Angeles, CAChildren's Hospital of Colorado, Anshutz Medical Campus· Aurora, COEmory University School of Medicine· Atlanta, GAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILIcahn School of Medicine at Mount Sinai· New York, NYOregon Health and Science University· Portland, OR

Related Studies