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

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06215755
• Other study ID #: VGCS-50635-002
• Secondary ID: 2023-506509-21
• Status: Recruiting
• Phase: Phase 1
• Start date: January 15, 2024
• Est. completion date: June 2026

Study information

• Verified date: March 2024
• Source: Verge Genomics
• Contact: Verge Genomics
• Phone: Please Email
• Email: clinicaltrials[@]vergegenomics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: June 2026
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 74 Years

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• VRG50635
Specified dose on specified days

Locations

Country	Name	City	State
Canada	CAN001	Montreal	Quebec
Netherlands	NLD001	Utrecht

Sponsors (1)

Lead Sponsor	Collaborator
Verge Genomics

Countries where clinical trial is conducted

Canada,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Treatment-emergent Adverse Events (TEAEs)		Up to 80 weeks
Primary	Number of Participants with Clinical Laboratory Evaluation Abnormalities		Up to 80 weeks
Primary	Number of Participants with Vital Sign Abnormalities		Up to 80 weeks
Primary	Number of Participants with Electrocardiogram (ECG) Abnormalities		Up to 80 weeks
Primary	Number of Participants with Physical Examination Abnormalities		Up to 80 weeks
Primary	Number of Participants with Neurological Examination Abnormalities		Up to 80 weeks
Secondary	Maximum Observed Concentration (Cmax)		Up to 80 weeks
Secondary	Area Under the Concentration-time Curve (AUC)		Up to 80 weeks
Secondary	Time to Maximum Observed Concentration (tmax)		Up to 80 weeks
Secondary	Change from Baseline in Plasma Levels of Neurofilament Light Chain (NfL) as Measured by Immunoassay		Baseline, up to 80 weeks
Secondary	Time to Disease Progression		Up to 80 weeks
Secondary	Change in Harmonized ALS Functional Rating Scale-Revised (ALS-FRS-R) Score		Up to 80 weeks