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A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS) | Clinical Trials | Clareo Health

TERMINATEDPHASE1

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

NCT06215755•Verge Genomics

View on ClinicalTrials.gov

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.

Eligibility

Age

18 - 74 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Provide written informed consent and be willing and able to comply with the requirements of the study protocol.
•2. Must be ≥ 18 and ≤ 75 years of age at the time of signing the informed consent form (ICF).
•3. Have a diagnosis of ALS according to the Gold Coast Diagnostic Criteria.
•4. Have either sporadic amyotrophic lateral sclerosis (sALS) or familial amyotrophic lateral sclerosis (fALS).
•5. Treatment Research Initiative to Cure ALS (TRICALS) risk profile \> -6.00 and \< -2.00.
•6. Have slow vital capacity (SVC) ≥ 60% of the predicted value.
•7. Have a score of 3 or 4 on Item #3 (Swallowing) of the Harmonized ALS Functional Rating Scale-Revised (ALS-FRS-R). Participants with a score of 3 can be enrolled with the Sponsor's approval only if they are able to safely swallow capsules.
•8. Have a body weight ≥ 45 kg and body mass index (BMI) ≥ 18 kg/m2.
•9. Participants of childbearing potential are eligible to participate if they are not pregnant or breastfeeding and agree to use one highly effective method of contraception, if sexually active, for the duration of the study through 90 days after the last study drug administration. Participants must not donate eggs for the duration of study through 90 days after the last dose of study drug.
•10. Participants capable of producing sperm and their partners of childbearing potential must agree to use condoms and one highly effective method of contraception, respectively, for the duration of the study through 90 days after the last study drug administration. Participants must not donate sperm for the duration of study through 90 days after the last dose of study drug.
+19 more criteria

Exclusion Criteria

•1. 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.
•2. Have a history of unstable or severe cardiac, pulmonary, neurological, oncological, hepatic, or renal disease or another medically significant illness other than ALS precluding their safe participation in this study.
•3. Have a history of substance use disorder or illicit drug use in the last year (medically prescribed or over-the-counter cannabis use is allowed, if legal in the country).
•4. Have a history of serious infection (e.g., pneumonia, septicemia) ≤ 4 weeks of Screening; infection requiring hospitalization or treatment with intravenous (IV) antibiotics, antivirals, or antifungals within 4 weeks of Screening; or chronic bacterial infection (e.g., tuberculosis) deemed unacceptable as per the Investigator's judgment.
•5. Had major surgery ≤ 4 weeks before Screening.
•6. Be currently taking or planning to take strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers.

Locations (7)

UZ Leuven

Leuven, Flemish Brabant, Belgium

Stan Cassidy Centre for Rehabilitation (Horizon NB)

Montreal, Quebec, Canada

The Neuro - Montréal Neurological Institute-Hospital

Montreal, Quebec, Canada

University of Eastern Finland, Brain Research Unit

Kuopio, Eastern Finland, Finland

Helsinki University Hospital

Helsinki, Uusimaa, Finland

Turku University Hospital

Turku, Western Finland, Finland

University Medical Center Utrecht

Utrecht, Netherlands

Key Dates

Start Date

January 15, 2024

Primary Completion Date

July 7, 2025

Completion Date

July 7, 2025

Last Updated

December 15, 2025

Enrollment

ACTUAL participants

Conditions

Amyotrophic Lateral Sclerosis

Interventions

VRG50635

DRUG

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

NCT07322003

Amyotrophic Lateral Sclerosis

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RECRUITING

Intermuscular Coherence as a Biomarker for ALS

NCT05104710

Amyotrophic Lateral Sclerosis

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RECRUITING

UPenn Observational Research Repository on Neurodegenerative Disease

NCT04715399

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 15, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

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