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.
ClinicalTrials.gov Identifier: NCT02496767
Drug Name: Tirasemtiv or CK-2017357
Sponsor: Cytokinetics
United Kingdom Locations:
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:
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:
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
North-East London and Essex MND Regional Care Centre
King's MND Care and Research Centre
Centre for Neuromuscular Diseases - National Hospital of Neurology
Sheffield Care and Research Centre
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
Status: Active, but not recruiting
