NA
Company
iECURE
Disease: Long-term Follow-up Study of Participants in Any iECURE Protocol Using an Investigational Product, (NCT06805695)
Disease info:
Frequency:
NA
Official title:
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Who:
Study contact:
Name: George Diaz, M.D., Ph.D.
Phone Number: 1-877-694-3558
Email: medinfo@iecure.com
Study Director: George Diaz, M.D., Ph.D,iECURE, Inc.
Sponsor:
Partners:
Locations:
United Kingdom, England London, United Kingdom Recruiting Great Ormond Street Hospital Contact: Christopher Jackson christopher.jackson@gosh.nhs.uk
Study start:
Dec. 23, 2024
Enrollment:
13
Gene editing method:
NA
Type of edit:
NA
Gene:
NA
Delivery method:
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy
Status: Active recruiting
Description
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.
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.
Last updated: Jun. 23, 2025