The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.
Who can participate
Age range18 Years – 74 Years
SexALL
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Inclusion criteria
✓. Provide written informed consent and be willing and able to comply with the requirements of the study protocol.
✓. Must be ≥ 18 and ≤ 75 years of age at the time of signing the informed consent form (ICF).
✓. Have a diagnosis of ALS according to the Gold Coast Diagnostic Criteria.
✓. Have either sporadic amyotrophic lateral sclerosis (sALS) or familial amyotrophic lateral sclerosis (fALS).
✓. Treatment Research Initiative to Cure ALS (TRICALS) risk profile \> -6.00 and \< -2.00.
✓. Have slow vital capacity (SVC) ≥ 60% of the predicted value.
✓. 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.
✓. Have a body weight ≥ 45 kg and body mass index (BMI) ≥ 18 kg/m2.
Exclusion criteria
✕. 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 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.
What they're measuring
1
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Timeframe: Up to 80 weeks
2
Number of Participants with Clinical Laboratory Evaluation Abnormalities
Timeframe: Up to 80 weeks
3
Number of Participants with Vital Sign Abnormalities
Timeframe: Up to 80 weeks
4
Number of Participants with Electrocardiogram (ECG) Abnormalities
Timeframe: Up to 80 weeks
5
Number of Participants with Physical Examination Abnormalities
Timeframe: Up to 80 weeks
6
Number of Participants with Neurological Examination Abnormalities
✕. 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).
✕. 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.
✕. Had major surgery ≤ 4 weeks before Screening.
✕. Be currently taking or planning to take strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers.
✕. Be currently taking or have discontinued treatment with riluzole \< 4 weeks before Screening. Participants who have been taking a stable dose of riluzole for ≥ 4 weeks are eligible if they remain on the same dose throughout the duration of the study.
✕. Be taking Radicava (administered orally or IV as approved in the participant's country), Relyvrio, any other approved standard of care treatment, or tauroursodeoxycholic acid (TUDCA) as a dietary supplement administered for \< 4 weeks prior to Screening or on a schedule of treatment different from the approved standard schedule of treatment. Participants who have completed ≥ 4 weeks of treatment before Screening are eligible if they plan to continue treatment at a stable dose throughout the duration of the study.