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)
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).
• Fully understand the purpose and risks of the study and voluntarily provide a signed and dated informed consent form.
• Aged ≥18 years, male or female;
• A diagnosis of ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998 \[Brooks 2000\]), and confirmed through genetic diagnosis to exclude pathogenic mutations in the superoxide dismutase 1 (SOD1) and/or FUS genes;
• The duration of the disease from the first symptom (any ALS symptom) prior to the screening must be less than 2 years (inclusive);
• Forced Vital Capacity (FVC) adjusted for gender, age, and height (sitting position) ≥50% of the predicted value.
• Discontinued riluzole for more than five half-lives prior to screening (and is not expected to resume during the study) or has been on a stable dose of riluzole for ≥30 days and continue to maintain this dose during the study period.
• Discontinued edaravone for more than five half-lives prior to screening (and is not expected to resume edaravone during the study) or is receiving the standard edaravone treatment regimen at screening and has maintained a stable dose for ≥60 days (two treatment cycles) prior to administration, and continue to maintain this dose during the study period.