Effects of VHB937 on Amyotrophic Lateral Sclerosis

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 study is testing a new treatment called VHB937 to see if it can help people with early-stage ALS live longer without needing breathing support and improve their daily activities.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 225 (estimated)
• Ages: 18 Years to 100 Years
• Sex: All
• Sponsor: Novartis (industry)
• Locations: 1 site (Houston, Texas)
• Trial ID: NCT06643481 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy and safety of VHB937, a monoclonal antibody, in individuals with early-stage Amyotrophic Lateral Sclerosis (ALS). It is a multicenter, randomized, double-blind, placebo-controlled study that lasts 40 weeks, followed by an open-label extension. The primary goals are to assess how long participants can live without needing permanent breathing assistance and to measure changes in their ability to perform daily activities using the ALS functional rating scale. Participants will also be monitored for any adverse events throughout the trial.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with mild ALS symptoms who have experienced symptoms within the last 24 months.

Not a fit: Patients with advanced ALS symptoms or those currently receiving other investigational treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the quality of life and prolong independence for patients with ALS.

How similar studies have performed: Other studies involving monoclonal antibodies for ALS have shown promise, but this specific approach is relatively novel.

Eligibility criteria

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

Where this trial is running

Houston, Texas

• Nerve and Muscle Center of Texas — Houston, Texas, United States (RECRUITING)

Study contacts

• Study coordinator: Novartis Pharmaceuticals

Conditions: Amyotrophic Lateral Sclerosis, ALS, monoclonal antibody, motor neuron disease, MND