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

Last updated: February 6, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Recruiting

Phase

2

Condition

Amyotrophic Lateral Sclerosis (Als)

Neurologic Disorders

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

  • Nerve and Muscle Center of Texas

    Houston, Texas 77030
    United States

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.