NCT06849609

A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS

An Open, Dose-Escalation Early Phase Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of Intrathecal of VGN-R13 in Patients with Amyotrophic Lateral Sclerosis (ALS)

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Hong Chen · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of VGN-R13 as a treatment of Amyotrophic Lateral Sclerosis (ALS).

Detailed description

The study will evaluate safety and efficacy of gene therapy in Amyotrophic Lateral Sclerosis (ALS) patients. ALS is a fatal central nervous system neurodegenerative disease. There is no effective treatment for ALS and current drug therapy has been unsuccessful in stabilizing or reversing this disease. Only supportive care is currently possible. This study consists of screening period, treatment period and follow-up period. Eligible subjects will be enrolled. The day of administration set to be D1. Prophylactic immunosuppressive therapy will be initiated on D1. During the follow-up period (up to 52 weeks after administration), all the examinations will be completed based on the evaluation time point specified in the Schedule of Assessments table for efficacy and safety assessments until the End of Trial. Unscheduled visits may occur if the PI determines that they are necessary.

Conditions

• Amyotrophic Lateral Sclerosis (ALS)

Interventions

Type	Name	Description
BIOLOGICAL	VGN-R13	An adeno-associated viral vector 9 gene therapy product.

Timeline

• Start date: 2025-01-16
• Primary completion: 2026-05-01
• Completion: 2026-06-01
• First posted: 2025-02-27
• Last updated: 2025-03-17

Locations

1 site across 1 country: China