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

NCT ID: NCT06805695

Last Updated: 2025-02-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

13 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-12-23

Study Completion Date

2041-07-31

Brief Summary

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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

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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.

Conditions

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Ornithine Transcarbamylase Deficiency Ornithine Transcarbamylase Deficiency Disease Urea Cycle Disorders, Inborn

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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Enrolled and Dosed

Previously dosed in a previous iECURE study.

No Intervention

Intervention Type OTHER

No Intervention

Enrolled but Not Dosed

Enrolled in a previous iECURE study, but not dosed.

No Intervention

Intervention Type OTHER

No Intervention

Interventions

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No Intervention

No Intervention

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

7 Months

Maximum Eligible Age

15 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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iECURE, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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George Diaz, M.D., Ph.D

Role: STUDY_DIRECTOR

iECURE, Inc.

Locations

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Great Ormond Street Hospital

London, , United Kingdom

Site Status RECRUITING

Countries

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United Kingdom

Central Contacts

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

Role: CONTACT

1-877-694-3558

Facility Contacts

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Christopher Jackson

Role: primary

Other Identifiers

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ECUR-LTFU

Identifier Type: -

Identifier Source: org_study_id

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