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

• ClinicalTrials.gov Identifier: NCT06215755
• Recruitment Status: Recruiting
• First Posted: January 22, 2024
• Last Update Posted: March 15, 2024
• Sponsor: Verge Genomics
• Information provided by (Responsible Party): Verge Genomics

Tracking Information

• First Submitted Date: December 18, 2023
• First Posted Date: January 22, 2024
• Last Update Posted Date: March 15, 2024
• Actual Study Start Date: January 15, 2024
• Estimated Primary Completion Date: June 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 10, 2024)

• Number of Participants with Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to 80 weeks ]
• Number of Participants with Clinical Laboratory Evaluation Abnormalities [ Time Frame: Up to 80 weeks ]
• Number of Participants with Vital Sign Abnormalities [ Time Frame: Up to 80 weeks ]
• Number of Participants with Electrocardiogram (ECG) Abnormalities [ Time Frame: Up to 80 weeks ]
• Number of Participants with Physical Examination Abnormalities [ Time Frame: Up to 80 weeks ]
• Number of Participants with Neurological Examination Abnormalities [ Time Frame: Up to 80 weeks ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 10, 2024)

• Maximum Observed Concentration (Cmax) [ Time Frame: Up to 80 weeks ]
• Area Under the Concentration-time Curve (AUC) [ Time Frame: Up to 80 weeks ]
• Time to Maximum Observed Concentration (tmax) [ Time Frame: Up to 80 weeks ]
• Change from Baseline in Plasma Levels of Neurofilament Light Chain (NfL) as Measured by Immunoassay [ Time Frame: Baseline, up to 80 weeks ]
• Time to Disease Progression [ Time Frame: Up to 80 weeks ]
• Change in Harmonized ALS Functional Rating Scale-Revised (ALS-FRS-R) Score [ Time Frame: Up to 80 weeks ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)
• Official Title: 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
• Brief Summary: The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis

Intervention

Drug: VRG50635

Specified dose on specified days

Study Arms

Experimental: VRG50635

Intervention: Drug: VRG50635

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 10, 2024): 50
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 2026
• Estimated Primary Completion Date: June 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• 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 and < -2.
• Have slow vital capacity (SVC) ≥ 75% of the predicted value.
• Have a score of 3 or 4 on Item #3 (Swallowing) of the Harmonized 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/m^2.

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

Additional protocol-defined criteria may apply.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 74 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Verge Genomics; Please Email; clinicaltrials@vergegenomics.com

• Listed Location Countries: Canada, Netherlands

Administrative Information

• NCT Number: NCT06215755
• Other Study ID Numbers: VGCS-50635-002; 2023-506509-21 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Verge Genomics
• Original Responsible Party: Same as current
• Current Study Sponsor: Verge Genomics
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Verge Genomics
• Verification Date: March 2024