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

RECRUITING

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

NCT06805695•iECURE, Inc.

View on ClinicalTrials.gov

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

Age

0 - 1 years

Sex

ALL

Healthy Volunteers

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.

Locations (1)

Great Ormond Street Hospital

London, United Kingdom

Key Dates

Start Date

December 23, 2024

Primary Completion Date

July 1, 2041

Completion Date

July 1, 2041

Last Updated

February 3, 2025

Enrollment

ESTIMATED participants

Conditions

Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseUrea Cycle Disorders, Inborn

Interventions

No Intervention

OTHER

Contacts

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

CONTACT

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

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 3, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI

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