Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Other
Study Type: Observational
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 7 months
Maximum Age: 1
Healthy Volunteers: f
View:
• 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.
Locations
United States
Colorado
Children's Hospital of Colorado, Anshutz Medical Campus
RECRUITING
Aurora
New York
Icahn School of Medicine at Mount Sinai
RECRUITING
New York
Other Locations
United Kingdom
Great Ormond Street Hospital
RECRUITING
London
Contact Information
Primary
George Diaz, M.D., Ph.D.
medinfo@iecure.com
1-877-694-3558
Time Frame
Start Date: 2024-12-23
Estimated Completion Date: 2041-07
Participants
Target number of participants: 13
Treatments
Enrolled and Dosed
Previously dosed in a previous iECURE study.
Enrolled but Not Dosed
Enrolled in a previous iECURE study, but not dosed.
Related Therapeutic Areas
Sponsors
Leads: iECURE, Inc.