NCT06288230

An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy

Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Lantu Biopharma · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.

Detailed description

Study duration per participant is approximately 25 months including an approximately 30-day screening/baseline period, an approximately 24-month study observation period including 1 treatment day, and an approximately 24-month follow-up period. Patients will be stratified in two groups, those \< 24 months of age at time of dosing and those ≥ 24 months of age at time of dosing. This study will be conducted in 2 stages: Stage 1: dose escalting study in children \<24 months of age. Stage 2: the selecetd dose from Stage 1 in children ≥ 24 months of age. Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose, and followed by visits at months 2, 3, 6 12, 18 and 24 post infusion. Unscheduled visits may occur if the investigator determines that they are necessary. For patients enrolled from overseas, follow-up visits with patient's own paediatrician together with remote virtual visits are allowed.

Conditions

• Spinal Muscular Atrophy

Interventions

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

Timeline

• Start date: 2024-10-20
• Primary completion: 2027-10-30
• Completion: 2027-10-30
• First posted: 2024-03-01
• Last updated: 2025-07-22

Locations

1 site across 1 country: China