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

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

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age7-15

SponsoriECURE, Inc.

About this trial

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

Qualifiers

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.

Disqualifiers

None

Trial design

Treatments tested in this trial

No Intervention

Treatment groups

13 Participants

are divided into 2 treatment groups

Sponsors and collaborators

iECURE, Inc.

Lead sponsor

Source ClinicalTrials.gov