VRG50635 for ALS

Not currently recruiting at 9 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral medication, VRG50635, to determine its safety and tolerability for people with ALS (amyotrophic lateral sclerosis), a disease affecting nerve cells in the brain and spinal cord. Participants will take a daily capsule after a low-fat meal to help researchers understand how the body processes the drug. Individuals diagnosed with ALS who can safely swallow capsules might be suitable candidates for this study. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

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.

Is there any evidence suggesting that VRG50635 is likely to be safe for humans?

Research has shown that VRG50635 is safe and generally well-tolerated. Studies with healthy adults found no serious side effects, whether they took the drug once or multiple times. The early results on the drug's safety are promising. This suggests that VRG50635 might be safe for people with ALS, but more research is needed to confirm this in those patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ALS?

Most treatments for ALS, like riluzole and edaravone, aim to slow the progression of the disease by reducing nerve damage, but they often have limited effectiveness and cumbersome administration. VRG50635 is unique because it offers a new oral capsule form, which is more convenient and potentially more consistent for patients than infusions. This treatment is also exciting because it involves a novel mechanism that might offer a fresh way to protect nerve cells from damage, potentially providing better outcomes for those with ALS. Researchers are hopeful that VRG50635 could lead to improved quality of life for patients by offering a more straightforward and potentially more effective treatment option.

What evidence suggests that VRG50635 might be an effective treatment for ALS?

Research has shown that VRG50635, the treatment under study in this trial, could help treat ALS (amyotrophic lateral sclerosis). In early studies, this treatment extended the lifespan of ALS models, suggesting it might also slow the disease in people. Previous trials found that VRG50635 was safe and well-tolerated in healthy adults. This treatment aims to address the root causes of ALS, potentially offering a new way to manage the disease. While more research is needed, these early results provide hope that it could slow down ALS. ^{1 2 5 6 7}

Who Is on the Research Team?

Diego Cadavid, MD

Principal Investigator

Verge Genomics

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06215755

A Study of VRG50635 in Participants With Amyotrophic ... This is a Phase 1b, open-label, within-participant, multiple ascending dose, multicenter study of VRG50635 in participants with sporadic amyotrophic lateral ...

2. vergegenomics.com vergegenomics.com/news-blog/verge-genomics-announces-positive-safety-and-tolerability-data-from-the-phase-1-clinical-trial-of-vrg50635-a-potential-best-in-class-therapeutic-for-all-forms-of-als

VRG50635 was safe and well tolerated in ... VRG50635 was safe and well tolerated in healthy adult volunteers after both single and repeated administration, with favorable pharmacokinetics showing ...

3. firstwordpharma.com firstwordpharma.com/story/5948022

Verge Genomics' AI-enabled biomarkers accurately track ... Verge dosed the first patient in its Phase I study of VRG50635 in October 2022 and subsequently released favourable safety and tolerability data ...

4. alsnewstoday.com alsnewstoday.com/news/digital-biomarkers-gauging-short-term-als-progression-trial/

Trial to use digital biomarkers to assess short-term ALS ... VRG50635 is therefore expected to slow ALS progression and potentially extend patient survival. In preclinical studies, the therapy extended ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11155303/

Advancements and challenges in amyotrophic lateral sclerosis The prolonged delay in ALS diagnosis, which in some cases can take up to 14 months, poses substantial challenges for patient outcomes. An ...

6. isrctn.com isrctn.com/ISRCTN14792372

Study to investigate the safety of VRG50635 in healthy ... VRG50635 is hypothesized in improving motor neuron health and survival, has exposures in the central nervous system (CNS), has effects on the relevant target, ...

7. alsnewstoday.com alsnewstoday.com/news/potential-oral-therapy-vrg50635-found-safe-healthy-adults/

Potential oral ALS therapy, VRG50635, found to be safe in ... VRG50635, an experimental therapy for all ALS types, was found to be safe and well-tolerated when given to healthy adults in a Phase 1 ...