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

Recruiting now

Conditions studied: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn

In brief

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.

Key facts

Study ID

NCT06805695

Run by

iECURE, Inc.

People needed

13

Starts

2024-12-23

Expected to finish

2041-07-01

Last updated by the study team

2025-02-03

Who can join

Age: 1 and older, up to 1. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• 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.

Where it is running

• Great Ormond Street Hospital — London, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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