This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale.
A long-term extension study will be available for patients completing the study.
Patient safety is monitored using conventional methods including adverse events, safety laboratory tests, vital signs and 12-lead EKG. The study will be monitored by an independent data and safety monitoring board.
The primary endpoint is slow vital capacity (SVC) at 12 weeks, with the impact on patient function assessed through 48 weeks, adjusted for patient outcome, using ALSFRS-R (combined assessment of function and survival, CAFS).
Other important efficacy measures include time to respiratory events, clinical global impression (CGI), assessment of dyspnea using the Borg scale and sleep scales (Pittsburgh sleep quality index and Epworth sleepiness scale).
- Male or female subjects with diagnosis of laboratory supported probable, probable or definite ALS
- Sitting SVC between 60-90% of the predicted value for age, height and sex at screening visit
- Disease duration from symptom onset (defined by first muscle weakness or dysarthria) 12-48 months at the time of visit 1 (baseline)
- Subjects with or without riluzole and/or edaravone.
- Subject with a diagnosis of another neurodegenerative disease (e.g. Parkinson's or Alzheimer's disease)
- Assisted ventilation of any type within 3 months before the screening visit or at screening
In this randomized, double-blind, placebo-controlled, parallel-group, multicenter study, subjects are allocated in a 2:1 ratio to receive either levosimendan (1 -2 mg daily) or placebo for 48 weeks.
Following screening and baseline visits, patients attend the clinic at 2, 4, 8, 12, 24, 36 and 48 weeks, with telephone assessments conducted at weeks 18, 30 and 42. An end of study visit is performed 14-25 days after the last study treatment administration.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. If you have any questions, use the form to contact a member of our patient support team.