drugset
drugset / Trial / NCT06288230

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

NCT06288230 ↗

Phase 1/2 Active not recruiting 20 enrolled Lantu Biopharma
RandomizedSequentialOpen-labelTreatment

Summary

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

Timeline

Start
2024-10-20
Primary completion
2027-10-30
Completion
2027-10-30

Drugs

EvaluationDrugModalityDoseRoute
Subject vesemnogene lantuparvovec Gene therapy (AAV / viral vector) — —

Indications

spinal muscular atrophy