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

NCT ID: NCT06643481

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

251 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-17

Study Completion Date

2028-07-07

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Amyotrophic Lateral Sclerosis (ALS)

Keywords

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Amyotrophic Lateral Sclerosis ALS monoclonal antibody motor neuron disease MND

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double-blind, Randomized 2:1 ratio
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors
Blinded placebo for infusion

Study Groups

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Arm 1

I.V. infusions

Group Type EXPERIMENTAL

VHB937

Intervention Type BIOLOGICAL

VHB937 solution for infusion

Arm 2

I.V. infusions

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Solution for infusion

Interventions

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VHB937

VHB937 solution for infusion

Intervention Type BIOLOGICAL

Placebo

Solution for infusion

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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University of California San Diego

La Jolla, California, United States

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Loma Linda University Health

Loma Linda, California, United States

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Keck Medical Center USC

Los Angeles, California, United States

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UC San Francisco Medical Center

San Francisco, California, United States

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University of Miami

Miami, Florida, United States

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Orlando Health Clinical Trials

Orlando, Florida, United States

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Emory University School of Medicine

Atlanta, Georgia, United States

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University Of Kansas Medical Center

Kansas City, Kansas, United States

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Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

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Massachusetts General Hospital

Boston, Massachusetts, United States

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Neurology Associates P C

Lincoln, Nebraska, United States

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University of Nebraska Medical Center

Omaha, Nebraska, United States

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Lange Neurology PC

New York, New York, United States

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University of Rochester Medical Center

Rochester, New York, United States

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Atrium Health

Charlotte, North Carolina, United States

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Duke University Health System

Durham, North Carolina, United States

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Univ of Cincinnati Medical Center

Cincinnati, Ohio, United States

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The Ohio State University

Columbus, Ohio, United States

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Providence ALS Center

Portland, Oregon, United States

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Temple University

Philadelphia, Pennsylvania, United States

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AMR Knoxville

Knoxville, Tennessee, United States

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Austin Neuromuscular Center

Austin, Texas, United States

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Nerve and Muscle Center of Texas

Houston, Texas, United States

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University of Washington Medical Center

Seattle, Washington, United States

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Novartis Investigative Site

North Ryde, New South Wales, Australia

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Randwick, New South Wales, Australia

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Herston, Queensland, Australia

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Cauldfield, Victoria, Australia

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Southport, , Australia

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Leuven, , Belgium

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Liège, , Belgium

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Calgary, Alberta, Canada

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Hamilton, Ontario, Canada

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Montreal, Quebec, Canada

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Montreal, Quebec, Canada

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Beijing, , China

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Aalborg, , Denmark

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Kobenhavn N V, , Denmark

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Turku, , Finland

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Limoges, Haute Vienne, France

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Bron, , France

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Lille, , France

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Montpellier, , France

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Nice, , France

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Paris, , France

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Tours, , France

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Mannheim, Baden-Wurttemberg, Germany

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Munich, Bavaria, Germany

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Würzburg, Bavaria, Germany

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Rostock, Mecklenburg-Vorpommern, Germany

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Berlin, , Germany

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Hanover, , Germany

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Lübeck, , Germany

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Münster, , Germany

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Ulm, , Germany

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Dublin, , Ireland

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Milan, MI, Italy

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Modena, MO, Italy

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Pisa, PI, Italy

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Torino, TO, Italy

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Nagoya, Aichi-ken, Japan

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Sendai, Miyagi, Japan

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Ohtsu, Shiga, Japan

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Tokushima, Tokushima, Japan

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Ōta-ku, Tokyo, Japan

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Utrecht, , Netherlands

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Krakow, Poland, Poland

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Bydgoszcz, , Poland

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Krakow, , Poland

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Warsaw, , Poland

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Warsaw, , Poland

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Yangsan, Gyeongsangnam-do, South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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L'Hospitalet de Llobregat, Barcelona, Spain

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Donostia / San Sebastian, Basque Country, Spain

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Barcelona, Catalonia, Spain

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Barcelona, Catalonia, Spain

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Santiago, Corunja, Spain

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Madrid, , Spain

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Málaga, , Spain

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Valencia, , Spain

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Malmo, , Sweden

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Stockholm, , Sweden

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Umeå, , Sweden

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Basel, , Switzerland

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Sankt Gallen, , Switzerland

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Sheffield, South Yorkshire, United Kingdom

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Farnborough, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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Stoke-on-Trent, , United Kingdom

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Countries

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United States Australia Belgium Canada China Denmark Finland France Germany Ireland Italy Japan Netherlands Poland South Korea Spain Sweden Switzerland United Kingdom

Other Identifiers

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2024-512536-29-00

Identifier Type: OTHER

Identifier Source: secondary_id

CVHB937B12201

Identifier Type: -

Identifier Source: org_study_id