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An Open Label Study of Gene Therapy Product in Spinal Muscular Atrophy Patients

Spinal Muscular Atrophy Clinical Trial

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

Clinical Trial Summary

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

Clinical Trial 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 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06288230
Study type Interventional
Source Lantu Biopharma
Contact
Status Not yet recruiting
Phase Phase 1/Phase 2
Start date March 1, 2024
Completion date March 1, 2027
View Details
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