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Vesemnogene Lantuparvovec in Progressive Spinal Muscular Atrophy
NCT06288230Phase 2
Key facts
- Status
- Active, not recruiting
- Start
- Oct 2024
- Primary completion
- Oct 2027
- Countries
- 1
- Sponsor
- Lantu Biopharma
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Conditions
- spinal muscular atrophy (SMA)
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Overview
Phase 2, open-label trial of vesemnogene lantuparvovec in spinal muscular atrophy (SMA) (3 arms), sponsored by Lantu Biopharma. Primary endpoint: Adverse Events and Serious Adverse Events at 24 months. Started Oct 2024; status: active, not recruiting.
02
Design
3 arms- Study design
- Non-randomizedOpen-label
Stage 1: Dose A
Dose A of Vesemnogene Lantuparvovec in children < 24 months
Stage 1: Dose B
Dose B of Vesemnogene Lantuparvovec in children < 24 months
Stage 2: Selected Dose
Selected dose of Vesemnogene Lantuparvovec in children ≥ 24 months
03
Who it enrols
Patients with SMA based on bi-allelic SMN1 mutations, without active viral infections, anti-AAV9 antibodies >1:20, or need for invasive ventilatory support.
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Endpoints
Primary
Adverse Events and Serious Adverse Events
Secondary
World Health Organization Gross Motor Milestones
Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders
Hammersmith Functional Motor Scale - Expanded
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