Study Description
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). The main questions this trial aims to answer in comparing VHB937 to placebo are:
* How long will participants live without needing permanent help from a machine to breathe after starting the trial treatment?
* What is the change in the participant's ability to perform daily activities? This will be measured using a questionnaire called the amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R).
* What adverse events are reported during this trial? An adverse event is any sign or symptom that participants have during a trial. Adverse events may or may not be caused by treatments in the trial. The trial doctors will check participants' ALS and general health throughout the trial.
Interventions
Placebo
VHB937
Eligibility Criteria
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
Novartis Investigative Site
Recruiting
North Ryde,New South Wales,2109,Australia
Novartis Investigative Site
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Randwick,New South Wales,2031,Australia
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Herston,Queensland,4029,Australia
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SouthPort,4215,Australia
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Cauldfield,Victoria,3162,Australia
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Liege,4000,Belgium
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Leuven,3000,Belgium
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Hamilton,Ontario,L8n 3z5,Canada
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Montreal,Quebec,H4a 3t2,Canada
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Beijing,100191,China
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Aalborg,9000,Denmark
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Kobenhavn N V,2400,Denmark
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Nice,06001,France
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Tours,37044,France
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Munchen,81675,Germany
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Luebeck,23538,Germany
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Dublin,Dublin 9,Ireland
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Sendai city,Miyagi,980 8574,Japan
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Yangsan Si,Gyeongsangnam Do,50612,Korea, Republic of
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Seoul,04763,Korea, Republic of
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Seoul,05505,Korea, Republic of
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Utrecht,3584,Netherlands
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Bydgoszcz,85-163,Poland
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Krakow,30-721,Poland
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Warszawa,01-684,Poland
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Barcelona,Catalunya,08003,Spain
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Umea,Se-90185,Sweden
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Malmo,214 28,Sweden
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Basel,4031,Switzerland
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St Gallen,9007,Switzerland
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London,Wc1n 3bg,United Kingdom
Emory University School of Medicine
Recruiting
Atlanta,Georgia,30322,United States
Christina Fournier
AMR Knoxville
Recruiting
Knoxville,Tennessee,37920,United States
Randall Trudell
Nerve and Muscle Center of Texas
Recruiting
Houston,Texas,77030,United States
Aziz Shaibani
University Of Kansas Medical Center
Recruiting
Kansas City,Kansas,66160,United States
Jeffrey Statland
University of California San Diego
Recruiting
La Jolla,California,92037,United States
John Ravits
Massachusetts General Hospital
Recruiting
Boston,Massachusetts,02114,United States
James Berry
Keck Medical Center USC
Recruiting
Los Angeles,California,90033,United States
Said R Beydoun
Orlando Health Clinical Trials
Recruiting
Orlando,Florida,32806,United States
Vahid Tohidi
Neurology Associates P C
Recruiting
Lincoln,Nebraska,68506-2960,United States
Gary Pattee
UC San Francisco Medical Center
Recruiting
San Francisco,California,94143,United States
Jill Goslinga
Lange Neurology PC
Recruiting
New York,New York,10065,United States
Dale Lange
University of Cincinnati Medical Center
Recruiting
Cincinnati,Ohio,45219,United States
Hani Kushlaf
The Ohio State University
Recruiting
Columbus,Ohio,43210,United States
Stephen Kolb
Worldwide Contacts
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