NCT06588803Enrolling by invitation
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)
Source: ClinicalTrials.gov · Last updated 5 Mar 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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)
- Status (registry)
- Enrolling by invitation
- Study type
- Observational
- Enrollment
- 18 (estimated)
- First submitted
- 21 Aug 2024
- Start date
- Mar 2025
- Primary completion date
- 10 Sept 2030
- Lead sponsor
- Guangzhou Jiayin Biotech Ltd
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT06588803
What you commit to
- Study type: Observational
- Planned participants: 18
- From Mar 2025 to the primary completion date, 10 Sept 2030
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- 1\. Parents or guardians are unwilling or unable to participate in long-term follow-up studies.
Who the study is looking for
- 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.
Age in the registry: 1 month to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The Children's Hospital of Fudan University Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.