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)

ClinicalTrials.gov 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: In a press release of November 21st, 2017, Cytokinetics announced that they will not continue to develop this drug. No benefit to lung function was observed at 24 weeks or any other benefit at 48 weeks. Read more.

 

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

ClinicalTrials.gov 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

ClinicalTrials.gov 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)

ClinicalTrials.gov Identifier: NCT03039673

Drug name: Interleukin 2 or IL-2

Sponsor: Centre Hospitalier Universitaire de Nīmes

United Kingdom - Recruiting Centres include

Trafford Centre for Biomedical Research

  • Brighton, United Kingdom, BN1 9RY
  • Principal Investigator: P Nigel Leigh, MD, PhD     

North-East London and Essex MND Regional Care Centre

  • London, United Kingdom, E1 4NS
  • Principal Investigator: Andrea Malaspina, MD, PhD

King's MND Care and Research Centre

  • London, United Kingdom, SE5 8AF
  • Principal Investigator: Ammar Al-Chalabi, MD, PhD

Centre for Neuromuscular Diseases - National Hospital of Neurology

  • London, United Kingdom, WC1N 3BG
  • Principal Investigator: Dr Nikhil SHARMA, MD, PhD

Sheffield Care and Research Centre

  • Sheffield, United Kingdom, S10 2JF
  • Principal Investigator: Pamela J Shaw, MD, PhD

 

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

ClinicalTrials.gov 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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