Long-term follow-up of gene therapy EXG001-307 in type 1 spinal muscular atrophy
Follows people with type 1 spinal muscular atrophy who received gene therapy EXG001-307 earlier.
Plain-language title generated from the registry record
Registry title: A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
Key facts
- Where
- Shanghai, ChinaSee the site
Where in China
- The Children's Hospital of Fudan UniversityShanghai, ChinaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Guangzhou Jiayin Biotech Ltd
- Trial code
- NCT06588803
- Record updated
- 5 March 2025
- Original description
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)Read full original descriptionShow less
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 Spinal Muscular Atrophy (SMA)
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)
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: Parents or guardians are unwilling or unable to participate in long-term follow-up studies.