REFALS-ES
Research type
Research Study
Full title
Effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS: open label extension for patients completing study 3119002
IRAS ID
260865
Contact name
Ammar Al-Chalabi
Contact email
Sponsor organisation
Orion Corporation
Eudract number
2018-004180-31
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
134169, IND Number
Duration of Study in the UK
2 years, 5 months, 24 days
Research summary
Amyotrophic lateral sclerosis (ALS) is a rare, rapidly progressive neurological disease characterised by degeneration of upper and lower motor neurons with subsequent muscle atrophy (decrease in muscle mass) and weakness and loss of respiratory function. Median survival time from symptom onset is approximately 3 years. No cure currently exists for ALS, the mainstay of treatment is symptom management and palliative care. There are 3 medicines currently indicated for ALS, only one of which is licensed in Europe.
Levosimendan is a calcium sensitising medicine that improves force generation in both cardiac (heart) and skeletal muscles without increasing oxygen consumption. Levosimendan (Simdax®) is currently approved in approximately 60 countries, excluding the United Kingdom, as a medication given through a vein for heart failure. Levosimendan (ODM-109) is an investigational medicine (study medicine).
Studies have shown that levosimendan (ODM-109) improved the muscle strength of the diaphragm and thereby the breathing function for patients with ALS. If ALS patients have increased breathing function, their overall well-being and function may also be increased.
The sponsor Orion Corporation Orion Pharma, is running an open-label extension study for participants with ALS who have completed the REFALS, 3119002, study to continue their treatment with oral Levosimendan and to evaluate long-term safety of oral levosimendan in participants with ALS.
A maximum of 450 participants (depending on how many complete the REFALS study) will take part in this open-label extension research study. The study aim is to provide levosimendan until either patients don’t benefit any longer or until they no longer need to be part of the trial to access it, to a maximum of 3 years total. Orion will undertake to run the study for up to a total of 3 years, or until levosimendan is available for prescription if that occurs sooner. Study site visits will be at a minimum every 3 months. The only fixed visits in the study are Week 2 at the study site and Week 6 over the phone after start of study treatment.
In this study, all participants will receive the study medicine Oral levosimendan (ODM-109).REC name
North East - York Research Ethics Committee
REC reference
19/NE/0223
Date of REC Opinion
5 Sep 2019
REC opinion
Further Information Favourable Opinion