NCT06805695

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 13 (estimated)
• Sponsor: iECURE, Inc. · Industry
• Sex: All
• Age: 7 Months – 15 Months
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

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

Interventions

Type	Name	Description
OTHER	No Intervention	No Intervention

Timeline

• Start date: 2024-12-23
• Primary completion: 2041-07-01
• Completion: 2041-07-01
• First posted: 2025-02-03
• Last updated: 2025-02-03

Locations

1 site across 1 country: United Kingdom

Regulatory

• FDA-regulated drug study