NCT06588803

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

A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 Spinal Muscular Atrophy (SMA)

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 18 (estimated)
• Sponsor: Guangzhou Jiayin Biotech Ltd · Industry
• Sex: All
• Age: 1 Month
• Healthy volunteers: Not accepted

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 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.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
OTHER	No intervention, only for observational studies	No intervention, only for observational studies

Timeline

• Start date: 2025-03-01
• Primary completion: 2030-09-10
• Completion: 2030-12-31
• First posted: 2024-09-19
• Last updated: 2025-03-05

Locations

1 site across 1 country: China