Home › Ornithine Carbamoyltransferase Deficiency Disease › NCT06805695

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

Who can join

7 Months – 15 Months · All sexes

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.

About the study

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.

What is being tested

• No Intervention (other)

Sponsor: iECURE, Inc. · Participants: 13 · Started: Dec 23, 2024

Contact the study team

• George Diaz, M.D., Ph.D. · Phone: 1-877-694-3558

Official record on ClinicalTrials.gov — NCT06805695

Locations in the U.S.

Conditions

• Ornithine Carbamoyltransferase Deficiency Disease
• Inborn Urea Cycle Disorders
• Brain Diseases
• Genetic Disorders
• X-Linked Genetic Diseases
• Liver Diseases
• Metabolic Disorders
• Inborn Errors Metabolism
• Neurologic Diseases

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.