Actively Recruiting
Long-term Follow-up Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Led by iECURE, Inc. · Updated on 2025-02-03
13
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a long-term follow-up study lasting 14.5 years to assess the safety and lasting effects of an investigational product (IP) used in previous iECURE studies. The study also aims to gather ongoing natural disease information from participants who enrolled but were not treated with the IP. Participants continue their usual medical care prescribed by their doctors throughout the study. This observational study does not provide any investigational treatment. Instead, it follows two groups: those who received the IP in earlier iECURE studies and those who were enrolled but not dosed. No new drug or therapy is given as part of this follow-up. Participants maintain their prescribed standard care while researchers collect data over time. Participants will be monitored regularly for safety, growth changes, and other health measures over 14.5 years. The study collects information on adverse events, changes in height and weight, urinalysis results, and detailed laboratory tests including vector clearance in blood, saliva, urine, and feces. Researchers also track serious health events related to the disease, hospitalizations, and survival outcomes. This comprehensive follow-up aims to provide valuable long-term data on participants' health and treatment effects.
CONDITIONS
Brief Title
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 participant's parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected
You will not qualify if you...
- Participants who enroll into an interventional drug or gene therapy clinical trial using an investigational product other than the one provided in the parent protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 14.5 years
Participants are observed over 14.5 years to assess long-term safety and clinical response, as well as natural disease history. Participants continue their standard of care medication prescribed by their physicians.
Periodic visits over 14.5 years
Trial Site Locations
Total: 1 location
1
Great Ormond Street Hospital
London, United Kingdom
Actively Recruiting
Research Team
G
George Diaz, M.D., Ph.D.
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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