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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT06215755
Recruitment Status : Recruiting
First Posted : January 22, 2024
Last Update Posted : March 15, 2024
Sponsor:
Information provided by (Responsible Party):
Verge Genomics

Study Description
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Brief Summary:
The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: VRG50635 Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : January 15, 2024
Estimated Primary Completion Date : June 2026
Estimated Study Completion Date : June 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: VRG50635 Drug: VRG50635
Specified dose on specified days


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to 80 weeks ]
  2. Number of Participants with Clinical Laboratory Evaluation Abnormalities [ Time Frame: Up to 80 weeks ]
  3. Number of Participants with Vital Sign Abnormalities [ Time Frame: Up to 80 weeks ]
  4. Number of Participants with Electrocardiogram (ECG) Abnormalities [ Time Frame: Up to 80 weeks ]
  5. Number of Participants with Physical Examination Abnormalities [ Time Frame: Up to 80 weeks ]
  6. Number of Participants with Neurological Examination Abnormalities [ Time Frame: Up to 80 weeks ]

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

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 74 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06215755


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

Locations
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Canada, Quebec
CAN001 Recruiting
Montreal, Quebec, Canada, H3A 2B4
Contact       als-cru.neuro@mcgill.ca   
Netherlands
NLD001 Recruiting
Utrecht, Netherlands, 3584 CX
Sponsors and Collaborators
Verge Genomics
More Information
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Responsible Party: Verge Genomics
ClinicalTrials.gov Identifier: NCT06215755    
Other Study ID Numbers: VGCS-50635-002
2023-506509-21 ( EudraCT Number )
First Posted: January 22, 2024    Key Record Dates
Last Update Posted: March 15, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Verge Genomics:
VRG50635
Familial Amyotrophic Lateral Sclerosis
Sporadic Amyotrophic Lateral Sclerosis
ALS
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases