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

May 25, 2026 updated by: Novartis Pharmaceuticals

A Phase 2, Randomized, Double-blind, Placebo-controlled Parallel Group Study of VHB937 in Amyotrophic Lateral Sclerosis (ALS) Over 40 Weeks Followed by an Open Label Extension (ASTRALS)

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed 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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
251

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

  • Name: Novartis Pharmaceuticals
  • Phone Number: +41613241111

Study Locations

  • Australia
      • Southport, Australia, 4215
        • Novartis Investigative Site
    • New South Wales
      • North Ryde, New South Wales, Australia, 2109
        • Novartis Investigative Site
    • Queensland
      • Herston, Queensland, Australia, 4029
        • Novartis Investigative Site
    • Victoria
      • Caulfield South, Victoria, Australia, 3162
        • Novartis Investigative Site
  • Belgium
      • Liège, Belgium, 4000
        • Novartis Investigative Site
    • Vlaams Brabant
      • Leuven, Vlaams Brabant, Belgium, 3000
        • Novartis Investigative Site
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 4N1
        • Novartis Investigative Site
    • Quebec
      • Montreal, Quebec, Canada, H4A 3T2
        • Novartis Investigative Site
  • China
      • Beijing, China, 100191
        • Novartis Investigative Site
  • Denmark
      • Aalborg, Denmark, 9000
        • Novartis Investigative Site
      • Kobenhavn N V, Denmark, 2400
        • Novartis Investigative Site
  • France
      • Bron, France, 69677
        • Novartis Investigative Site
      • Lille, France, 59037
        • Novartis Investigative Site
      • Nice, France, 06001
        • Novartis Investigative Site
      • Paris, France, 75013
        • Novartis Investigative Site
      • Tours, France, 37044
        • Novartis Investigative Site
  • Germany
      • Berlin, Germany, 13353
        • Novartis Investigative Site
      • Hanover, Germany, 30559
        • Novartis Investigative Site
      • Lübeck, Germany, 23538
        • Novartis Investigative Site
      • Münster, Germany, 48149
        • Novartis Investigative Site
      • Ulm, Germany, 89081
        • Novartis Investigative Site
    • Baden-Wurttemberg
      • Mannheim, Baden-Wurttemberg, Germany, 68167
        • Novartis Investigative Site
    • Bavaria
      • Munich, Bavaria, Germany, 81675
        • Novartis Investigative Site
      • Würzburg, Bavaria, Germany, 97080
        • Novartis Investigative Site
    • Mecklenburg-Vorpommern
      • Rostock, Mecklenburg-Vorpommern, Germany, 18057
        • Novartis Investigative Site
  • Ireland
      • Dublin, Ireland, DUBLIN 9
        • Novartis Investigative Site
  • Italy
    • MI
      • Milan, MI, Italy, 20138
        • Novartis Investigative Site
    • MO
      • Modena, MO, Italy, 41126
        • Novartis Investigative Site
    • PI
      • Pisa, PI, Italy, 56126
        • Novartis Investigative Site
    • TO
      • Torino, TO, Italy, 10126
        • Novartis Investigative Site
  • Netherlands
      • Utrecht, Netherlands, 3584 CX
        • Novartis Investigative Site
  • Poland
      • Bydgoszcz, Poland, 85-163
        • Novartis Investigative Site
      • Krakow, Poland, 31 531
        • Novartis Investigative Site
      • Krakow, Poland, 30-721
        • Novartis Investigative Site
      • Warsaw, Poland, 01-684
        • Novartis Investigative Site
      • Warsaw, Poland, 02-473
        • Novartis Investigative Site
  • South Korea
      • Seoul, South Korea, 05505
        • Novartis Investigative Site
      • Seoul, South Korea, 04763
        • Novartis Investigative Site
    • Gyeongsangnam-do
      • Yangsan, Gyeongsangnam-do, South Korea, 50612
        • Novartis Investigative Site
  • Spain
      • Barcelona, Spain, 08035
        • Novartis Investigative Site
      • Valencia, Spain, 46026
        • Novartis Investigative Site
    • A Coruna
      • Santiago Compostela, A Coruna, Spain, 15706
        • Novartis Investigative Site
    • Barcelona
      • L'Hospitalet de Llobregat, Barcelona, Spain, 08907
        • Novartis Investigative Site
  • Sweden
      • Malmö, Sweden, 214 28
        • Novartis Investigative Site
      • Stockholm, Sweden, 113 61
        • Novartis Investigative Site
      • Umeå, Sweden, SE-90185
        • Novartis Investigative Site
  • Switzerland
      • Basel, Switzerland, 4031
        • Novartis Investigative Site
      • Sankt Gallen, Switzerland, 9007
        • Novartis Investigative Site
  • United Kingdom
      • Farnborough, United Kingdom, BR6 8ND
        • Novartis Investigative Site
      • London, United Kingdom, SW17 0QT
        • Novartis Investigative Site
      • London, United Kingdom, WC1N 3BG
        • Novartis Investigative Site
      • Stoke-on-Trent, United Kingdom, ST4 6QG
        • Novartis Investigative Site
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, S10 2JF
        • Novartis Investigative Site
  • United States
    • California
      • La Jolla, California, United States, 92037
        • University of California San Diego
      • Loma Linda, California, United States, 92354
        • Loma Linda University Health
      • Los Angeles, California, United States, 90033
        • Keck Medical Center USC
      • San Francisco, California, United States, 94143-0348
        • UC San Francisco Medical Center
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami
      • Orlando, Florida, United States, 32806
        • Orlando Health Clinical Trials
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University School of Medicine
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska Medical Center
    • New York
      • New York, New York, United States, 10065
        • Lange Neurology PC
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28207
        • Atrium Health
      • Durham, North Carolina, United States, 27710
        • Duke University Health System
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Univ of Cincinnati Medical Center
      • Columbus, Ohio, United States, 43210
        • The Ohio State University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19140
        • Temple University
    • Tennessee
      • Knoxville, Tennessee, United States, 37920
        • AMR Knoxville
    • Texas
      • Austin, Texas, United States, 78759
        • Austin Neuromuscular Center
      • Houston, Texas, United States, 77030
        • Nerve And Muscle Center Of Texas
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Arm 1
I.V. infusions
Biological: VHB937
VHB937 solution for infusion
Placebo Comparator: Arm 2
I.V. infusions
Other: Placebo
Solution for infusion

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
The composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Survival (CAFS)
Time Frame: Baseline to DB Week 40
To compare the efficacy of VHB937 vs. placebo on a composite of permanent assisted ventilation (PAV) free survival and function in DB epoch
Baseline to DB Week 40

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
ALS Functional Rating Scale Revised (ALSFRS-R) total score
Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first) and Baseline to OLE Week 100 or until death or PAV (whichever occurs first
To assess the efficacy of VHB937 on functional decline in DB and OLE epochs.
Baseline to DB Week 40 or until death or PAV (whichever occurs first) and Baseline to OLE Week 100 or until death or PAV (whichever occurs first
Slow Vital Capacity (SVC) (% of predicted normal value)
Time Frame: Baseline to DB Week 40 or until death or PAV (whichever occurs first) and Baseline to OLE Week 100 or until death or PAV (whichever occurs first)
To assess the efficacy of VHB937 in delaying decline in respiratory function in DB and OLE epochs.
Baseline to DB Week 40 or until death or PAV (whichever occurs first) and Baseline to OLE Week 100 or until death or PAV (whichever occurs first)
Ratio to baseline in Neurofilament Light (NfL) concentration in serum
Time Frame: DB up to Week 40; DB and OLE up to Week 100]
To assess the effect of VHB937 on a biomarker of neurodegeneration in DB and OLE epochs.
DB up to Week 40; DB and OLE up to Week 100]
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Baseline to end of study
To assess the safety and tolerability of VHB937 in DB and OLE epochs.
Baseline to end of study
Time to death and Time to event (death or PAV, whichever comes first).
Time Frame: Baseline to DB Week 40
To assess the efficacy of VHB937 vs. placebo on survival endpoints in DB epoch.
Baseline to DB Week 40
Time to death and Time to event (death or PAV, whichever comes first) - endpoints referring to treatment policy estimand
Time Frame: Baseline to OLE Week 100, and Baseline to end of study
To assess the efficacy of early vs. delayed VHB937 administration on survival endpoints (DB VHB937 followed by OLE VHB937 vs. DB placebo followed by OLE VHB937)
Baseline to OLE Week 100, and Baseline to end of study
Patient Global Impression of change in functional ability and ALS symptom severity (PGI-C)
Time Frame: DB up to Week 40; DB and OLE up to Week 100
To assess change in ALS condition in DB and OLE epochs.
DB up to Week 40; DB and OLE up to Week 100
Change in QoL from baseline as measured with Amyotrophic Lateral Sclerosis Assessment Questionnaire -5 (ALSAQ-5)
Time Frame: DB up to Week 40; DB and OLE up to Week 100
To assess Quality of Life (QoL) with VHB937 in DB and OLE epochs.
DB up to Week 40; DB and OLE up to Week 100
Change in Clinician Global Impression of change in functional ability and ALS symptom severity (CGI-C)
Time Frame: DB up to Week 40; DB and OLE up to Week 100
To assess change in ALS condition in DB and OLE epochs.
DB up to Week 40; DB and OLE up to Week 100
Change in QoL from baseline as measured with EuroQoL 5 Dimension 5 Level (EQ-5D-5L)
Time Frame: DB up to Week 40; DB and OLE up to Week 100
To assess Quality of Life (QoL) with VHB937 in DB and OLE epochs.
DB up to Week 40; DB and OLE up to Week 100
Change in QoL from baseline as measured with 12-item Short form health survey (SF-12)
Time Frame: DB up to Week 40; DB and OLE up to Week 100
To assess Quality of Life (QoL) with VHB937 in DB and OLE epochs.
DB up to Week 40; DB and OLE up to Week 100
Pharmacokinetics (PK) of VHB937-CMAX
Time Frame: Day 1 to end of study
CMAX - The maximum concentration of VHB937 in serum
Day 1 to end of study
Pharmacokinetics (PK) of VHB937-TMAX
Time Frame: Day 1 to end of study
TMAX - The time to reach the maximum concentration of VHB937 in serum
Day 1 to end of study
Pharmacokinetics (PK) of VHB937-CTROUGH
Time Frame: Day 1 to end of study
CTROUGH - Minimum observed concentration of VHB937 in serum
Day 1 to end of study
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CMAX
Time Frame: Screening to Week 12
CMAX - The maximum concentration of VHB937 in CSF
Screening to Week 12
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-TMAX
Time Frame: Screening to Week 12
TMAX - The time to reach the maximum concentration of VHB937 in CSF
Screening to Week 12
Cerebralspinal Spinal Fluid Pharmacokinetics (PK) of VHB937-CTROUGH
Time Frame: Screening to Week 12
CTROUGH - Minimum observed concentration of VHB937 in CSF
Screening to Week 12
To assess immunogenicity (IG) of VHB937
Time Frame: Day 1 up to end of study
To assess immunogenicity of Anti-VHB937 antibodies in serum
Day 1 up to end of study

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 17, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 21, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 10, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 30, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 12, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 16, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 27, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 25, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CVHB937B12201
  • 2024-512536-29-00 (Other Identifier: EUCTIS)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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