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

ID#: NCT06805695

Age: 7 - 15 months

Gender: All

Healthy Subjects: No

Recruitment Status: Recruiting

Start Date: December 23, 2024

End Date: July 01, 2041

Contact Information:
George Diaz, M.D., Ph.D.
1-877-694-3558
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:

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.
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