Clinical Trial

Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

Recruiting Phase 3
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Summary
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA. The specific objectives are: * To determine clinical effectiveness of Vesemnogene lantuparvovec therapy for SMA as evaluated by developmental gross motor milestone and survival. * To describe the safety profile of Vesemnogene therapy for SMA as evaluated by adverse events reporting and laboratory tests, and monitoring of Adverse events of special interest.
Trial Details
NCT Number NCT07265232
Lead Sponsor Lantu Biopharma
Conditions Spinal Muscular Atrophy (SMA)
Enrollment 15 participants
Start Date 2025-10-15
Primary Completion 2030-10-15 (estimated)
Study Completion 2030-10-15 (estimated)
Updated on ClinicalTrials.gov 2025-12-04