NCT07265232

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Lantu Biopharma · Industry
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

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.

Detailed description

This is an observational study designed to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec therapy for SMA. Potential patients with genetic diagnosis of SMA will be evaluated for eligibility to undergo available gene therapies. Following the administration of Vesemnogene therapy, patient will be monitored for toxicity and response to treatment. No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.

Conditions

• Spinal Muscular Atrophy (SMA)

Interventions

Type	Name	Description
BIOLOGICAL	vesemnogene lantuparvovec	Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA.

Timeline

• Start date: 2025-10-15
• Primary completion: 2030-10-15
• Completion: 2030-10-15
• First posted: 2025-12-04
• Last updated: 2025-12-04

Locations

1 site across 1 country: Indonesia