REFALS

  • Research type

    Research Study

  • Full title

    Effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS

  • IRAS ID

    240367

  • Contact name

    Ammar Al-Chalabi

  • Contact email

    ammar.al-chalabi@kcl.ac.uk

  • Sponsor organisation

    Orion Corporation

  • Eudract number

    2017-002754-36

  • Clinicaltrials.gov Identifier

    134169, IND Number

  • Duration of Study in the UK

    1 years, 10 months, 28 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. There are 3 medicines currently indicated for ALS, only one of which is licensed in Europe. There is an unmet need for new treatments in ALS.

    Levosimendan (Simdax®) is currently approved in 60 countries, excluding the United Kingdom, as a medication given through a vein for heart failure. In this study the same substance will be tested for ALS given as a capsule (taken via the mouth). Levosimendan (ODM-109) is an investigational medicine (study medicine).

    Studies have shown that levosimendan (ODM-109) may be useful to improve 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 purpose of this research study is to measure how well and how safe levosimendan (ODM-109) is in treating patients with ALS.

    Approximately 450 participants will take part in this research study. Participation will last approximately 1 year (51-52 weeks) including 10 study visits at the clinic (including screening and end of study visit) and 3 phone calls.

    In this study, each group will receive either the study medicine levosimendan (ODM-109) or placebo (dummy medication). The chances of the participant receiving levosimendan (ODM-109) are 2:1 (67% chance to receive study medicine and 33% chance to receive placebo).

    Procedures include: Physical examination, weight, height and body mass index, blood pressure, heart rate, electrocardiogram (recording of the heart), blood and urine samples, breathing function, swallow test, questionnaires, evaluations, questions about health status, diary including assistive devices and home care/health care support.

  • REC name

    London - Riverside Research Ethics Committee

  • REC reference

    18/LO/0355

  • Date of REC Opinion

    4 May 2018

  • REC opinion

    Further Information Favourable Opinion