Status:
ENROLLING_BY_INVITATION
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
Lead Sponsor:
Hangzhou Jiayin Biotech Ltd
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
1+ years
Brief Summary
An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)
Detailed Description
This study plans to enroll type 1 SMA patients who have previously received EXG001-307 treatment in the parent trial EXG001-307-102 to observe the long-term safety and efficacy of gene therapy with EX...
Eligibility Criteria
Inclusion
- Previously received treatment with EXG001-307 in trial EXG001-307-102;
- Parents or guardians understand the research procedure and sign the ICF; Good compliance and willingness to follow research procedures. Voluntarily participate in this clinical trial.
Exclusion
- 1\. Parents or guardians are unwilling or unable to participate in long-term follow-up studies.
Key Trial Info
Start Date :
March 1 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2030
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT06588803
Start Date
March 1 2025
End Date
December 31 2030
Last Update
March 5 2025
Active Locations (1)
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1
The Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China