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

Last updated: November 11, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Not Recruiting

Phase

2

Condition

Amyotrophic Lateral Sclerosis (Als)

Myasthenia Gravis (Chronic Weakness)

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: 251
Treatment Group(s): 3
Primary Treatment: VHB837
Phase: 2
Study Start date:
October 17, 2024
Estimated Completion Date:
July 07, 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

    Site Not Available

  • Novartis Investigative Site

    Randwick, New South Wales 2031
    Australia

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  • Novartis Investigative Site

    North Ryde 7281782, New South Wales 2155400 2109
    Australia

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  • Novartis Investigative Site

    Randwick 2208285, New South Wales 2155400 2031
    Australia

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  • Novartis Investigative Site

    Herston, Queensland 4029
    Australia

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  • Novartis Investigative Site

    Herston 6931014, Queensland 2152274 4029
    Australia

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  • Novartis Investigative Site

    Cauldfield, Victoria 2145234 3162
    Australia

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    SouthPort, 4215
    Australia

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    Southport 2148928, 4215
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  • Novartis Investigative Site

    Leuven, 3000
    Belgium

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    Leuven 2792482, 3000
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    Liege, 4000
    Belgium

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    Liège 2792413, 4000
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    Calgary 5913490, Alberta 5883102 T2N 4N1
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    Hamilton, Ontario L8N 3Z5
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    Montreal, Quebec H4A 3T2
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    Beijing, 100191
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    Beijing 1816670, 100191
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    Aalborg, 9000
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    Kobenhavn N V, 2400
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    Ulm 2820256, 89081
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    La Jolla, California 92037
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    Los Angeles, California 90033
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    San Francisco, California 94143
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    Boston, Massachusetts 02114
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    Boston 4930956, Massachusetts 6254926 02114
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  • Neurology Associates P C

    Lincoln, Nebraska 68506-2960
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    Lincoln 5072006, Nebraska 5073708 68506-2960
    United States

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    New York, New York 10065
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    New York 5128581, New York 5128638 10065
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    Rochester 5134086, New York 5128638 14642
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    Charlotte 4460243, North Carolina 4482348 28207
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    Durham 4464368, North Carolina 4482348 27710
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    Cincinnati, Ohio 45219
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    Columbus, Ohio 43210
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    Cincinnati 4508722, Ohio 5165418 45219
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    Columbus 4509177, Ohio 5165418 43210
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  • Providence ALS Center

    Portland 5746545, Oregon 5744337 97213
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    Philadelphia 4560349, Pennsylvania 6254927 19140
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  • AMR Knoxville

    Knoxville, Tennessee 37920
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  • AMR Knoxville

    Knoxville 4634946, Tennessee 4662168 37920
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  • Nerve and Muscle Center of Texas

    Houston, Texas 77030
    United States

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  • Austin Neuromuscular Center

    Austin 4671654, Texas 4736286 78759
    United States

    Site Not Available

  • Nerve and Muscle Center of Texas

    Houston 4699066, Texas 4736286 77030
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  • University of Washington Medical Center

    Seattle 5809844, Washington 5815135 98195
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