VRG50635
Verge Genomics is conducting a Phase 1b study to assess the safety, tolerability and potential efficacy of VRG50635 in persons living with Amyotrophic lateral sclerosis (ALS).
Status: coming
The exact cause of ALS is as yet unknown. However, the accumulation of toxic proteins seems to contribute to nerve cell damage. The oral drug VRG50635 is a substance thought to be involved in the cleaning and recycling system of nerve cells. Thus, the drug may help remove the toxic proteins and repair nerve cell damage.
Study design
This study is called a Phase 1b study. The main purpose of a Phase 1 study is to test whether a new drug is safe. The ’b’ refers to administration in patients.
There is no placebo group; all patients will receive VRG50635 in the study.
In total, the study will last about 1.5 years. The study will be divided into different periods:
Screening: during this period, your eligibility to participate will be tested. That period lasts from 1 to 4 weeks.
Run-in period: during this period, we assess how your disease currently is progressing. During this period, you will not yet receive VRG50635. This period is needed to see later on whether VRG50635 has changed the course of your disease. That period lasts for 8 weeks.
Dosage increase: during the dose increase period, you will be given increasing doses of VRG50635. The medication consists of capsules taken orally that contain 200 mg of VRG50635. During the first 8 weeks, you will take 2 capsules (400 mg) every 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 the following 8 weeks. During the last 6 weeks of the dose increase period, you will take the dose that suits you best. That period lasts a total of 32 weeks.
Long-term treatment: you will continue to take the dose of VRG50635 that was most suitable for you during the dose increase period. This period lasts for 36 weeks.
During the study, you will visit the study centre every 8 to 12 weeks and a study nurse will visit you at home every 2 weeks during the walk-in and dose increase period.
