Actively Recruiting
Early Phase Study of Intrathecal VGN-R13 Gene Therapy for Treating Amyotrophic Lateral Sclerosis ALS
Led by Hong Chen · Updated on 2025-03-17
6
Participants Needed
1
Research Sites
4 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of intrathecal delivery of VGN-R13, a gene therapy, for treating Amyotrophic Lateral Sclerosis ALS, a fatal neurodegenerative disease with no current effective treatments beyond supportive care. This early phase clinical trial aims to assess how well patients tolerate the therapy and its potential benefits in ALS patients. Participants will receive a one-time intrathecal injection of VGN-R13, with up to two different dose levels planned. The study includes a screening period, a treatment day D1 when the gene therapy and prophylactic immunosuppressive therapy start, followed by a follow-up period lasting up to 52 weeks. During follow-up, patients will undergo regular evaluations to monitor safety and treatment effects, with unscheduled visits possible if needed. Throughout the study, participants will have scheduled assessments including functional rating scales, lung function tests, muscle strength measurements, and antibody or immunogenicity tests. Researchers will closely track adverse events, disease progression, and survival outcomes over one year. This comprehensive monitoring aims to understand the therapys safety profile and impact on ALS progression.
CONDITIONS
Brief Title
A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS
Research Team
H
Hong Chen
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