VRG50635 for ALS

Recruiting at 8 trial locations

Overseen By Verge Genomics

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Verge Genomics

Must not be taking: CYP3A4 inhibitors, Riluzole, Radicava, Relyvrio

Disqualifiers: Active psychiatric disease, Unstable cardiac, Active malignancy, Uncontrolled seizures, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

54 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called VRG50635 to see if it is safe for people with ALS, a disease that affects nerve cells. The main goal is to check if patients can tolerate the drug without serious side effects.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, like strong CYP3A4 inhibitors or inducers, and discontinue riluzole if taken for less than 4 weeks before screening. If you have been on a stable dose of riluzole or other ALS treatments for at least 4 weeks, you can continue them during the study.

Research Team

Diego Cadavid, MD

Principal Investigator

Verge Genomics

Eligibility Criteria

This trial is for people with ALS who meet the Gold Coast Diagnostic Criteria, have either sporadic or familial ALS, a certain risk profile score, can swallow safely, and have specific breathing capacity and body weight measurements. It's not for those with severe psychiatric issues or other medical conditions that could affect study results.

Inclusion Criteria

I have ALS, either sporadic or inherited from my family.

Have slow vital capacity (SVC) ≥ 75% of the predicted value

I have been diagnosed with ALS.

Exclusion Criteria

I do not have severe health issues that would make it unsafe for me to join this study.

Have active psychiatric disease, substance abuse, neuromuscular weakness other than ALS, or any other medical condition that, in the opinion of the Investigator, might confound the results of the study or interfere with the intake or absorption of the study drug or participation for the full duration of the study

Have a history of substance use disorder or illicit drug use in the last year

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-treatment Run-in

Establish the mean baseline based on repeated measurements of all biomarkers in eligible participants prior to initiating dosing with VRG50635

4 weeks

Treatment

Safety, tolerability, PK, and efficacy of VRG50635 evaluated using a within-participant multiple ascending dose scheme

8 weeks

Long-term Treatment

Long-term tolerability, safety, and efficacy of VRG50635 evaluated at the highest tolerated dose

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• VRG50635

Trial Overview The study tests VRG50635's safety and tolerability in ALS patients. The main goal is to see how well participants handle the drug and what effects it has on their condition over time.

Participant Groups

Group I: VRG50635 Experimental Treatment 1 Intervention

The study drug is VRG50635 200 mg oral capsules. VRG50635 will be administered as oral capsules once daily in the morning after a low-fat meal, approximately 30 minutes prior to VRG50635 administration. Following administration there is a 5 to 6-hour restriction period where participants should consume only low-fat food.

Who Is Running the Clinical Trial?

Verge Genomics

Lead Sponsor

Trials

2

Recruited

80+