A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)

Last updated: June 23, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Recruiting

Phase

2

Condition

Neurologic Disorders

Amyotrophic Lateral Sclerosis (Als)

Scar Tissue

Treatment

VHB837

Placebo

VHB937

Clinical Study ID

NCT06643481
CVHB937B12201
2024-512536-29-00
  • Ages 18-100
  • All Genders

Study Summary

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 Criteria

Inclusion

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 approvedtreatment for ALS.

  • have the ability to slowly exhale a volume of air at least 60% of what is expectedfor the participant's sex, height and age.

Exclusion

Exclusion Criteria:

  • Use of other investigational drugs within 5 half-lives of screening, or within 30days (e.g., small molecules) / or until the expected pharmacodynamic effect hasreturned to baseline (e.g., biologics), whichever is longer; or longer if requiredby local regulations.

  • Women of child-bearing potential, defined as all women physiologically capable ofbecoming pregnant, unless they are using highly effective methods of contraceptionwhile taking study treatment and for 24 weeks after stopping study medication.

  • History or current diagnosis of cardiac conditions or ECG abnormalities indicatingsignificant 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 diseaseother than ALS, dementia or substance abuse that would impair ability of theparticipant 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 ObstructivePulmonary Disease, interstitial lung disease or pulmonary fibrosis.

  • Taking any prohibited medications

Study Design

Total Participants: 225
Treatment Group(s): 3
Primary Treatment: VHB837
Phase: 2
Study Start date:
October 17, 2024
Estimated Completion Date:
June 20, 2028

Study Description

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.

Connect with a study center

  • Novartis Investigative Site

    North Ryde, New South Wales 2109
    Australia

    Active - Recruiting

  • Novartis Investigative Site

    Randwick, New South Wales 2031
    Australia

    Active - Recruiting

  • Novartis Investigative Site

    Herston, Queensland 4029
    Australia

    Active - Recruiting

  • Novartis Investigative Site

    Cauldfield, Victoria 3162
    Australia

    Active - Recruiting

  • Novartis Investigative Site

    SouthPort, 4215
    Australia

    Active - Recruiting

  • Novartis Investigative Site

    Leuven, 3000
    Belgium

    Active - Recruiting

  • Novartis Investigative Site

    Liege, 4000
    Belgium

    Active - Recruiting

  • Novartis Investigative Site

    Hamilton, Ontario L8N 3Z5
    Canada

    Active - Recruiting

  • Novartis Investigative Site

    Montreal, Quebec H4A 3T2
    Canada

    Active - Recruiting

  • Novartis Investigative Site

    Beijing, 100191
    China

    Active - Recruiting

  • Novartis Investigative Site

    Aalborg, 9000
    Denmark

    Active - Recruiting

  • Novartis Investigative Site

    Kobenhavn N V, 2400
    Denmark

    Active - Recruiting

  • Novartis Investigative Site

    Nice, 06001
    France

    Active - Recruiting

  • Novartis Investigative Site

    Tours, 37044
    France

    Active - Recruiting

  • Novartis Investigative Site

    Luebeck, 23538
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Munchen, 81675
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Dublin, DUBLIN 9
    Ireland

    Active - Recruiting

  • Novartis Investigative Site

    Sendai city, Miyagi 980 8574
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Yangsan Si, Gyeongsangnam Do 50612
    Korea, Republic of

    Active - Recruiting

  • Novartis Investigative Site

    Seoul, 05505
    Korea, Republic of

    Active - Recruiting

  • Novartis Investigative Site

    Utrecht, 3584
    Netherlands

    Active - Recruiting

  • Novartis Investigative Site

    Bydgoszcz, 85-163
    Poland

    Active - Recruiting

  • Novartis Investigative Site

    Krakow, 30-721
    Poland

    Active - Recruiting

  • Novartis Investigative Site

    Warszawa, 01-684
    Poland

    Active - Recruiting

  • Novartis Investigative Site

    Barcelona, Catalunya 08003
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Malmo, 214 28
    Sweden

    Active - Recruiting

  • Novartis Investigative Site

    Umea, SE-90185
    Sweden

    Active - Recruiting

  • Novartis Investigative Site

    Basel, 4031
    Switzerland

    Active - Recruiting

  • Novartis Investigative Site

    St Gallen, 9007
    Switzerland

    Active - Recruiting

  • Novartis Investigative Site

    London, WC1N 3BG
    United Kingdom

    Active - Recruiting

  • University of California San Diego

    La Jolla, California 92037
    United States

    Active - Recruiting

  • Keck Medical Center USC

    Los Angeles, California 90033
    United States

    Active - Recruiting

  • UC San Francisco Medical Center

    San Francisco, California 94143
    United States

    Active - Recruiting

  • Orlando Health Clinical Trials

    Orlando, Florida 32806
    United States

    Active - Recruiting

  • Emory University School of Medicine

    Atlanta, Georgia 30322
    United States

    Active - Recruiting

  • University Of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Active - Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • Neurology Associates P C

    Lincoln, Nebraska 68506-2960
    United States

    Active - Recruiting

  • Lange Neurology PC

    New York, New York 10065
    United States

    Active - Recruiting

  • University of Cincinnati Medical Center

    Cincinnati, Ohio 45219
    United States

    Active - Recruiting

  • The Ohio State University

    Columbus, Ohio 43210
    United States

    Active - Recruiting

  • AMR Knoxville

    Knoxville, Tennessee 37920
    United States

    Active - Recruiting

  • Nerve and Muscle Center of Texas

    Houston, Texas 77030
    United States

    Active - Recruiting

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