Recruiting

Observational Study

Sponsor:

iECURE, Inc.

Code:

NCT06805695

Conditions

Ornithine Transcarbamylase Deficiency

Ornithine Transcarbamylase Deficiency Disease

Urea Cycle Disorders, Inborn

Eligibility Criteria

Sex: All

Age: 0 - 1

Healthy Volunteers: Not accepted

Interventions

No Intervention

Study Details

Brief summary:

This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.

Conditions

Ornithine Transcarbamylase Deficiency

Ornithine Transcarbamylase Deficiency Disease

Urea Cycle Disorders, Inborn

Study ID

NCT06805695

Start date

Dec 23, 2024

Status verified date

Aug, 2026

Completion date

Jul, 2041

Anticipated

Primary completion date

Jul, 2041

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 1

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol
2. Participant parent(s)/LAR is willing and able to adhere to the protocol requirements.
3. Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.

Exclusion Criteria:

1\. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.

Study Design

Enrollment

13 participants

Anticipated

Interventions and Outcome Measures

Arms

Enrolled and Dosed

Previously dosed in a previous iECURE study.

Enrolled but Not Dosed

Enrolled in a previous iECURE study, but not dosed.

Interventions

No Intervention

No Intervention

Primary outcome measure

  • AE and SAE (incidence, severity, seriousness, and relatedness) [ Time Frame: Over 14.5 years post dosing with ECUR-506 ]
  • Change from baseline over 14.5 years post infusion in length [ Time Frame: Over 14.5 years post dosing with ECUR-506 ]
  • Change from baseline over 14.5 years post infusion in weight [ Time Frame: Over 14.5 years post dosing with ECUR-506 ]
  • Urinalysis (Dip Stick) Evaluations [ Time Frame: Over 14.5 years post dosing with ECUR-506 ]

Central Contacts and Locations

Central contacts

George Diaz, M.D., Ph.D.

1-877-694-3558medinfo@iecure.com

Locations

Children's Hospital of Colorado, Anshutz Medical Campus

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Lanie Dougherty

Lanie.Dougherty@childrenscolorado.org

Icahn School of Medicine at Mount Sinai

Recruiting

New York, New York, United States, 10029

Contacts

Silvia Gunderson

silvia.gunderson@mssm.edu

More Information

Sponsor

iECURE, Inc.

Last update posted

Aug 31, 2026

Last verified

Aug, 2026

Keywords

  • Amino Acid Metabolism, Inborn Errors
  • Ammonia
  • Brain Diseases
  • Brain Diseases, Metabolic
  • Brain Diseases, Metabolic, Inborn
  • Central Nervous System Diseases
  • Genetic Diseases, Inborn
  • Genetic Diseases, X-Linked
  • High Ammonia
  • Hyperammonemia
  • Inborn
  • Inborn Errors
  • Liver Disease
  • Liver Transplant
  • Metabolism
  • Metabolic Diseases
  • Metabolism, Inborn Errors
  • Neonatal
  • Nervous System Diseases
  • NH4
  • Ornithine
  • Ornithine Transcarbamylase Deficiency
  • OTC
  • OTC Deficiency
  • OTCD
  • Transcarbamylase
  • UCD
  • Urea Cycle Disorders
  • Long Term Follow Up
  • Long-Term Follow-Up
  • LTFU

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by iECURE, Inc. on 2026-08-31.