REFALS clinical trial
Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients with ALS
REFALS is a global clinical trial that will evaluate whether taking oral levosimendan for an extended period can preserve respiratory function better than placebo in people with MND (ALS). Oral levosimendan might be useful for improving the muscle strength of the diaphragm and might therefore improve breathing ability for patient with MND overall increasing well-being and function.
What does taking part involve?
After being assessed for eligibility, you would start receiving either oral levosimendan or placebo (ratio 2:1) for up to 48 weeks. The study is a double-blind study and therefore, you will not know if you are receiving oral levosimendan or Placebo. You will take the study treatment either once or twice a day depending on the study dose issued to you by the Investigator (1mg or 2mg per day). Throughout the 48-weeks period, you will be asked to visit the clinic at 2, 4, 8, 12, 24, 26 and 48 weeks, with telephone assessments conducted at weeks 18, 30 and 42. An end of study visit will be performed 14-25 days after the last study treatment administration. At the clinic visits, you will be asked to perform a Slow Vital Capacity (SVC) measurement to assess your breathing ability as well as provide blood samples and complete 3 patient questionnaires.
After you have completed the 48-week of treatment, there may be a possibility for you to participate in the open-label study where you would be receiving levosimendan (even if you received placebo during the double-blind study). The Investigator will inform you if you it is possible for you to take part in the open-label extension study (REFALS-ES).
London (King’s College Hospital and Barts Health)
Liverpool (The Walton Centre).
This trial is funded by Orion Corporation, Orion Pharma, Orionintie 1, FI-02200 Espoo, Finland.
Who can take part?
People living with ALS, who:
• are between 12-48 months since symptom onset, and
• are able to swallow study treatment capsules, and
• have a sitting SVC between 60-90% of the predicted value at the screening visit, and
• if taking riluzole and/or edaravone, have been on a stable dose for at least 4 weeks before screening visit
• are not currently on NIV
How do I take part?
If you think you may be eligible, you should speak to your neurologist to refer you to one of the recruitment centres.
You can also visit the Orion Pharma website and listen to a webinar given by Dr Merit Cudkowicz (Massachusetts General Hospital) and Prof Ammar Al-Chalabi (King’s College London) where the REFALS trial is discussed (organised by the Northeast Amyotrophic Lateral Sclerosis Consortium; NEALS).
Find this trial at clinicaltrials.gov (NCT03505021).
Expiry date: 19 May 2019