臨床研究等提出・公開システム

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) (CVHB937B12201)

Study of efficacy and safety of VHB937 in participants with ALS (CVHB937B12201)

Maruyama Hideki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku@novartis.com

Maruyama Hideki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku@novartis.com

Pending

Mar. 21, 2025

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

- 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.

- 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

Both

Amyotrophic Lateral Sclerosis

Experimental: VHB937 solution for infusion. I.V. infusions every 4 weeks.
Placebo Comparater: Placebo solution for infusion. I.V. infusions every 4 weeks.

The composite of PAV-free survival and change in ALSFRS-R.

+81-88-633-9294

Dec. 19, 2024

Yes

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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.

United States of America/Canada/Austraria/Belgium/Denmark/China/Finland/France/Germany/Ireland/Italy/Republic of Korea/Netherlands/Poland/Spain/Sweden/Switzerland/United Kingdom