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

Last updated: January 28, 2025
Sponsor: iECURE, Inc.
Overall Status: Active - Recruiting

Phase

N/A

Condition

Hyperargininemia

Urea Cycle Disorder

Arginase Deficiency

Treatment

No Intervention

Clinical Study ID

NCT06805695
ECUR-LTFU
  • Ages 7-15
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Enrolled in an iECURE parent protocol and have either completed or discontinued thatprotocol

  2. Participant parent(s)/LAR is willing and able to adhere to the protocolrequirements.

  3. Consent was obtained by the participants parent(s)/LAR (and participant assent,where applicable), prior to any study-related data being collected.

Exclusion

Exclusion Criteria:

  1. Participants who enroll into an interventional drug or gene therapy clinical trialutilizing an IP other than the IP provided in the parent protocol will be excludedfrom this protocol.

Study Design

Total Participants: 13
Treatment Group(s): 1
Primary Treatment: No Intervention
Phase:
Study Start date:
December 23, 2024
Estimated Completion Date:
July 31, 2041

Study 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.

Connect with a study center

  • Great Ormond Street Hospital

    London,
    United Kingdom

    Active - Recruiting

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