NCT06805695Recruiting

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

iECURE, Inc.

Start Date

12/23/2024

Completion Date

7/1/2041

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.

Detailed Description

This is a 14.5 year LTFU protocol to assess long term safety and clinical response to IP as well as to capture natural disease history of participants not dosed with IP in a parent protocol. No IP is provided as part of this protocol. Participants will remain on standard of care medication as indicated and prescribed by their physicians.

Eligibility Criteria

Age Range: No minimum to No maximum

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.

Interventions

OTHER

No Intervention

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Conditions

Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseUrea Cycle Disorders, Inborn

Locations

Children's Hospital of Colorado, Anshutz Medical Campus

Aurora, Colorado 80045

United States

Icahn School of Medicine at Mount Sinai

New York, New York 10029

United States

Great Ormond Street Hospital

London,

United Kingdom