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

Hangzhou Jiayin Biotech

Status: Invitation-only

Conditions: Spinal Muscular Atrophy

Treatments: Other: No intervention, only for observational studies

Study type: Observational

Funder types: Industry

Identifiers

NCT06588803

EXG001-307-LTFU

Details and patient eligibility

About

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)

Full 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 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.

Enrollment: 18 estimated patients

Sex: All

Ages: 1+ month old

Volunteers: No Healthy Volunteers

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.