Clinical Trials

Clinical trials are currently underway in the UK and around the world to explore new drugs capable of treating MND.

A clinical trial is a scientifically controlled study of the safety and effectiveness of a therapeutic agent (such as a drug) using consenting people.

There are different types of clinical trials but the randomized clinical trial (RCT) is often considered to be the gold standard. In an RCT, the effects of the experimental treatment will be compared between the groups who receive the treatment and the group who receive no treatment (the control, or placebo group). It is important to remember, therefore, that if you are asked, or volunteer, to participate in a trial, you may not be allocated to the treatment group : instead, you may be allocated to the control group.

Unfortunately, the results from previous clinical trials for novel drug therapies have been disappointing : the positive results seen in pre-clinical studies have, sadly, failed to live up to expectations when tested in humans.

To date, Riluzole is still the only proven treatment for MND. However, research into finding a successful treatment for MND is on-going. Details of clinical trials (for new drugs) which are taking place in the United Kingdom at present, are listed below.


Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year (VITALITY-ALS) Identifier: NCT02496767

Drug Name: Tirasemtiv or CK-2017357

Sponsor: Cytokinetics

United Kingdom Locations:

  • Derriford Hospital, Plymouth, Devon.
  • Walton Centre for Neurology and Neurosurgery, Liverpool
  • Clinical Research Centre, Royal London Hospital, London
  • Kings College Hospital, London

 Status: This study is ongoing, but not recruiting participants.


Single and Multiple Dose Study of BIIB067 in Adults With Amyotrophic Lateral Sclerosis (ALS) Identifier: NCT02623699

Sponsors: Biogen  and Ionis Pharmaceuticals, Inc.

Drug name: BIIB067

United Kingdom Locations:

  • Sheffield Institute for Translational Neuroscience, Sheffield

 Status: This study is currently recruiting participants. 

Note: Restricted to people with familial MND caused by mistakes in the SOD1 gene.


Long-Term Evaluation of BIIB067 Identifier:  NCT03070119

Sponsors: Biogen  and Ionis Pharmaceuticals, Inc.

Drug name: BIIB067

United Kingdom Locations:

  • Research Site, Sheffield S10 2HQ

Status: This study is enrolling participants by invitation only. Patients must have diagnosis of SOD1-ALS, and must have completed Part A and/or Part B of Study 233AS101 (NCT02623699)

MIROCALS: Modifying Immune Response and OutComes in ALS (MIROCALS) Identifier: NCT03039673

Drug name: Interleukin 2 or IL-2

Sponsor: Centre Hospitalier Universitaire de Nīmes

United Kingdom Locations:

  • Trafford Centre for Biomedical Research, Brighton BN1 9RY - Recruiting
  • North-East London and Essex MND Regional Care Centre, London - Not yet recruiting
  • King's MND Care and Research Centre, London -Not yet recruiting
  • Centre for Neuromuscular Diseases - National Hospital of Neurology, London - Not yet recruiting
  • Sheffield Care and Research Centre, Sheffield - Not yet recruiting

Status: This study is open for participant recruitment at the Trafford Centre in Brighton - June 2017


Effects of ODM-109 on Respiratory Function in Patients With Amyotrophic Lateral Sclerosis (ALS) Identifier:   NCT02487407

Drug  name: ODM-109 Other Name: Levosimendan

Sponsor: Orion Corporation, Orion Pharma

 United Kingdom Locations

  • Royal Sussex County Hospital, Brighton
  • The Walton Centre, Liverpool
  • London Kings College Hospital, London
  • Royal London Hospital, London
  • University of Sheffield, Sheffield

 Status: Active, but not recruiting


World-Wide Clinical Trials and Research 

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