A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA

Launched by HANGZHOU JIAYIN BIOTECH LTD · Sep 6, 2024

Keywords

ClinConnect Summary

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 EXG001-302.

The first year of long-term follow-up will be conducted every 3 months ; In the second year, follow-up visits will be conducted every six months, with telephone follow ups at 15 and 21 months during this period; From 3 to 5 years after gene therapy, follow up by phone every six months and follow on on site once a year.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Previously received treatment with EXG001-307 in trial EXG001-307-102;
• • 2. 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 Criteria:
• • 1. Parents or guardians are unwilling or unable to participate in long-term follow-up studies.