Effect of Salbutamol on Walking Capacity in Ambulatory ALS Patients

Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · May 5, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a medication called salbutamol on walking ability in patients with amyotrophic lateral sclerosis (ALS), a condition that affects muscle control. Researchers believe that salbutamol may help improve walking capacity by enhancing signals between nerves and muscles, which might also slow down the progression of the disease. The trial aims to see if salbutamol can help patients walk better and to assess its safety. Participants will also be monitored for changes in fatigue, muscle strength, respiratory function, and other health markers.

To be eligible for the study, participants must be adults aged 18 to 75 who have been diagnosed with probable or definite ALS and can still walk. They should be able to complete a simple walking test and have stable health for at least six months. It’s important that participants are not taking medications that could affect the study outcomes and do not have certain other health issues. If you qualify and choose to join, you can expect to receive the medication and undergo regular check-ups to monitor your health and progress.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Subjects who meet the revised El Escorial criteria for probable or definite sporadic ALS
• • Adult patients between 18 and 75 years of age
• • Patients who are ambulatory and able to perform the 6MWT and quantitative muscle testing at screening (ALSFRS-R-walking = 3)
• • Patients able and willing to travel to the site, and, in the investigator's opinion, who are likely to attend visits for at least 6 months
• • Patients who signed written informed consent
• • Stable dose of riluzole for a minimum of 4 weeks prior to baseline or has not taken it for 4 weeks prior to baseline
• • For child-bearing aged women, efficient contraception (cf protocol p32)
• • Forced vital capacity (fVC) in a sitting position \> 70 %
• Exclusion Criteria:
• • Patients with significant spasticity of the lower limbs interfering with walking capacity (Ashworth scale score \> 2)
• • Patients with fronto-temporal dementia associated with ALS
• • Patients presenting respiratory insufficiency causing dyspnea during walking
• • Patients taking drugs that could interfere with NMJ function (anticholinesterase ...) or muscle function (steroids, statins...)
• • Patients taking any forbidden drugs (see list in annex)
• • Hypersensitivity to salbutamol or to excipients of the drug and placebo
• • Known contraindication for the studied drug such as ischemic cardiomyopathy or risk of ischemic cardiomyopathy: history of ischemic heart disease or coronaropathy or/and significant ischemic ECG alterations at screening visit
• • Any clinically significant alterations in the following biological parameters glycemia, kalemia, creatinemia and hematology in the month prior to inclusion according to local laboratory threshold (cf protocol page 33)
• • Vulnerable persons defined in Articles L1121-5 to L 1121-8-1 and L1122-1-2 of the Code de la Santé Publique\* (\*CSP)
• • Participation in another interventional trial up to 3 months before inclusion
• • Patients having any relevant concomitant disease considered at risk of interfering with study procedures in the opinion of the investigator