This is a multicenter, randomized, double-blind, placebo-controlled, parallel group Phase II study to evaluate the efficacy and safety of VHB937 in participants with early-stage ALS (within 2 years of ALS symptoms onset). The study comprises a core double-blind (DB) 40-week treatment period followed by an open label extension (OLE).
Eligibility
Sex
ALL
Min age
18 Years
Max age
100 Years
Healthy volunteers
No
Inclusion Criteria:
* are 18 years of age or older
* male or female, if of childbearing potential, strict contraception required
* have ALS confirmed by the trial doctors using different tests.
* have mild symptoms of ALS as measured by the ALSFRS-R questionnaire (total score \>=30).
* have had symptoms of ALS (weakness) within 24 months of taking part in this trial.
* have not received treatment for ALS or are currently on a stable dose of an approved treatment for ALS.
* have the ability to slowly exhale a volume of air at least 60% of what is expected for the participant's sex, height and age.
Exclusion Criteria:
* Use of other investigational drugs within 5 half-lives of screening, or within 30 days (e.g., small molecules) / or until the expected pharmacodynamic effect has returned to baseline (e.g., biologics), whichever is longer; or longer if required by local regulations.
* Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for 24 weeks after stopping study medication.
* History or current diagnosis of cardiac conditions or ECG abnormalities indicating significant risk of safety for participants in the study.
* Clinical evidence of liver or renal disease/injury.
* Laboratory evidence of hematological abnormalities
* Presence of unstable psychiatric disease, cognitive impairment, neurological disease other than ALS, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the investigator's opinion.
* Participants that reported 'yes' on any suicidal ideation section except for the "Non-Suicidal Self-Injurious Behavior" in the past 2 years as per C-SSRS.
* Presence of cancer, HIV, Hep B, Hep C, tuberculosis, uncontrolled diabetes
* History of active severe respiratory disease, including Chronic Obstructive Pulmonary Disease, interstitial lung disease or pulmonary fibrosis.
* Taking any prohibited medications
Primary outcome measure(s)
The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS) — Baseline to DB Week 40 To compare the efficacy of VHB937 vs. placebo on a composite of permanent assisted ventilation (PAV) free survival and function in DB epoch
Trial sites (74)
Facility
City
Region
Status
University of California San Diego
La Jolla
California
Loma Linda University Health
Loma Linda
California
Keck Medical Center USC
Los Angeles
California
UC San Francisco Medical Center
San Francisco
California
University of Miami
Miami
Florida
Orlando Health Clinical Trials
Orlando
Florida
Emory University School of Medicine
Atlanta
Georgia
University Of Kansas Medical Center
Kansas City
Kansas
Massachusetts General Hospital
Boston
Massachusetts
University of Nebraska Medical Center
Omaha
Nebraska
Lange Neurology PC
New York
New York
University of Rochester Medical Center
Rochester
New York
Atrium Health
Charlotte
North Carolina
Duke University Health System
Durham
North Carolina
Univ of Cincinnati Medical Center
Cincinnati
Ohio
The Ohio State University
Columbus
Ohio
Temple University
Philadelphia
Pennsylvania
AMR Knoxville
Knoxville
Tennessee
Austin Neuromuscular Center
Austin
Texas
Nerve and Muscle Center of Texas
Houston
Texas
University of Washington Medical Center
Seattle
Washington
Novartis Investigative Site
North Ryde
New South Wales
Novartis Investigative Site
Herston
Queensland
Novartis Investigative Site
Caulfield South
Victoria
Novartis Investigative Site
Southport
Australia
Novartis Investigative Site
Leuven
Vlaams Brabant
Novartis Investigative Site
Liège
Belgium
Novartis Investigative Site
Calgary
Alberta
Novartis Investigative Site
Montreal
Quebec
Novartis Investigative Site
Beijing
China
Novartis Investigative Site
Aalborg
Denmark
Novartis Investigative Site
Kobenhavn N V
Denmark
Novartis Investigative Site
Bron
France
Novartis Investigative Site
Lille
France
Novartis Investigative Site
Nice
France
Novartis Investigative Site
Paris
France
Novartis Investigative Site
Tours
France
Novartis Investigative Site
Mannheim
Baden-Wurttemberg
Novartis Investigative Site
Munich
Bavaria
Novartis Investigative Site
Würzburg
Bavaria
+ 34 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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