Phase 1

VRG50635 study

Industry trial

Verge Genomics is conducting a phase 1b trial to evaluate the safety, tolerability, and potential efficacy of VRG50635 for people with Amyotrophic Lateral Sclerosis (ALS).

VRG50635

The exact cause of ALS is still unknown. However, piling up of toxic proteins seems to contribute to the damage of the nerve cells.  The oral drug VRG50635 is thought to induce the cleaning and recycling systems of the nerve cells. This way, the drug may help removing the toxic proteins and repairing the damage to the nerve cells.

Study design

This study is called a Phase 1b study. The main goal of a Phase 1 study is to see whether a new drug is safe; the b refers to administration in patients.

There is no placebo group, all participants receive VRG50635 in the study.

In total, the study will last for approximately 1.5 years. The study is split into different periods:

  • Screening: during this period, it will be checked if you are eligible to take part. This period takes 1 to 4 weeks.
  • Run-in: in this period, it is assessed how your disease is currently developing. In this period, you will not yet receive VRG50635. This period is needed to later see whether VRG50635 changes the course of the disease. This period takes 8 weeks.
  • Dose escalation: During dose escalation, you will receive increasing doses of VRG50635. The medication consists of capsules, taken orally, that contain 200 mg VRG50635.During the first 8 weeks you will take 2 capsules (400 mg) each morning. If all goes well, the dose will be increased to 4 capsules (800 mg) for the next 8 weeks, and to 6 capsules (1200 mg) for another 8 weeks. In the final 6 weeks of the dose escalation period, you will use the dose that fits best for you.

This period takes a total of 32 weeks.

  • Long-term treatment: you will continue using the dose of VRG50635 that was found best for you during dose escalation. This period takes 36 weeks.

During the study, you will visit the study centre every 8-12 weeks and every 2 weeks during the run-in and dose escalation period a study nurse visits you at home.

Can I participate in the study?

The main criteria to participate in this study are:

  • Age between 18 and 75 years
  • Diagnosis of ALS

Note: This list is not exhaustive, but these are the main criteria.

Registration and more information

If you are interested in participating in the VRG50635 study and fit the above profile, please contact one of the participating tricals centres below (contact details can be found by clicking on ‘read more’). They can provide you with more information and answer any questions you may have. If you experience difficulty contacting a centre, you may reach out directly to the TRICALS operational team at info@tricals.org for support in this process. More information about the study can be found at here.

The study aims to enroll 50 patients across European countries and Canada. Currently, the study is active in Belgium, Finland, the Netherlands and Spain.