/A
Phase 1
- Conditions
- Spinal Muscular AtrophyMedDRA version: 20.0Level: LLTClassification code: 10079413Term: Spinal muscular atrophy type I Class: 10010331MedDRA version: 20.0Level: LLTClassification code: 10079415Term: Spinal muscular atrophy type III Class: 10010331MedDRA version: 20.0Level: LLTClassification code: 10079416Term: Spinal muscular atrophy type II Class: 10010331Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2024-513086-39-00
- Lead Sponsor
- ovartis Pharma AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 131
Inclusion Criteria
Any patient with SMA who received AVXS-101 gene replacement therapy in a Novartis Pharma AG -sponsored clinical study, Patient/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Exclusion Criteria
Patient/parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method