NCT06215755

A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)

A Phase 1b, Open-Label, Multiple Ascending Dose, Multicenter Study of VRG50635 in Participants With Sporadic and Familial Amyotrophic Lateral Sclerosis Followed by Long-Term Treatment

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 54 (actual)
• Sponsor: Verge Genomics · Industry
• Sex: All
• Age: 18 Years – 74 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.

Detailed description

This is a Phase 1b, open-label, within-participant, multiple ascending dose, multicenter study of VRG50635 in participants with sporadic amyotrophic lateral sclerosis (sALS) and familial amyotrophic lateral sclerosis (fALS). Part 1 is a pre-treatment run-in period to establish the mean baseline based on repeated measurements of all biomarkers in eligible participants prior to initiating dosing with VRG50635. In Part 2, the safety, tolerability, PK, and efficacy of VRG50635 will be evaluated using a within-participant multiple ascending dose scheme. In Part 3, the long-term tolerability, safety, and efficacy of VRG50635 will be evaluated at the highest tolerated dose.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	VRG50635	Part 1, no study drug will be administered. Part 2, the starting dose is 400 mg for 8 weeks (Treatment Period 1) and doses will be escalated to 600 mg for 8 weeks (Treatment Period 2) and 800 mg for 8 weeks (Treatment Period 3). Part 3, each participant will continue receiving treatment with the highest tolerated dose achieved in Part 2 for up to 40 weeks.

Timeline

• Start date: 2024-01-15
• Primary completion: 2025-07-07
• Completion: 2025-07-07
• First posted: 2024-01-22
• Last updated: 2025-12-15

Locations

7 sites across 4 countries: Belgium, Canada, Finland, Netherlands