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

iECURE

Status: Enrolling

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

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT06805695

ECUR-LTFU

Details and patient eligibility

About

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.

Full 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.

Enrollment: 13 estimated patients

Sex: All

Ages: 7 to 15 months old

Volunteers: No 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.

Trial design

13 participants in 2 patient groups

Enrolled and Dosed

Description:

Previously dosed in a previous iECURE study.

Treatment:

Other: No Intervention

Enrolled but Not Dosed

Description:

Enrolled in a previous iECURE study, but not dosed.

Treatment:

Other: No Intervention