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

Launched by NOVARTIS PHARMACEUTICALS · Oct 12, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called VHB937 for people with early-stage Amyotrophic Lateral Sclerosis (ALS), a condition that affects nerve cells and can lead to muscle weakness. The trial will involve participants who have been experiencing symptoms of ALS for no more than 24 months and have mild symptoms, as shown by a specific questionnaire. To be eligible, participants must be 18 years or older, and if they are women who could become pregnant, they will need to use effective birth control during the study and for a period afterward.

The trial will last for about 40 weeks, during which participants will either receive VHB937 or a placebo (a treatment that looks like the actual drug but has no active ingredients). This study is “double-blind,” meaning neither the participants nor the researchers will know who is receiving the actual treatment to ensure fair results. After the initial treatment period, participants may have the chance to continue receiving VHB937 in an open-label extension phase. Those interested in joining the trial should be aware of certain health criteria that could exclude them, such as serious heart or liver issues, or recent use of other investigational drugs.

Gender

ALL

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