999AS003 - Methodology Study in ALS
Research type
Research Study
Full title
Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis
IRAS ID
192679
Contact name
Pamela Shaw
Contact email
Sponsor organisation
Biogen
Duration of Study in the UK
4 years, 0 months, 12 days
Research summary
Amyotrophic lateral sclerosis (ALS), is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles (muscle action we are able to control, such as those in the arms, legs, and face).
The purpose of this study is to help improve the understanding of the characteristics and progression of amyotrophic lateral sclerosis (ALS) and explore ways to measure potential future therapies. The methods that are found to be the most valuable in this study will be considered for use in future ALS clinical studies.
The methods for measuring ALS symptoms that will be used in this study include electrophysiological (i.e. the electrical properties of cells and tissues) tests, muscle strength tests, lung function tests, questionnaires about the participants ALS status. Spinal cord magnetic resonance imaging (MRI) will also be used for measuring ALS symptoms at some sites.
In addition, this study will try to find biomarkers linked to certain aspects of ALS that differ between individuals in order to help target future therapies to the patients most likely to benefit. Biomarkers are naturally occurring substances in the body. Examples of biomarkers are proteins, parts of proteins, or ribonucleic acid ([RNA], which tells the cells in the body which proteins to produce). Biomarkers can be found in the blood, spinal fluid, and tissue. Testing if biomarkers are present and the amount of biomarkers present may help diagnose a certain disease, determine the severity of the disease, or evaluate how well and how effectively a medicine may treat the disease. Biomarkers may also determine if certain patients are more likely than others to benefit from treatment with a specific medicine. Very few biomarkers for ALS have been identified, this study may help contribute to new biomarker discoveries. Peripheral blood mononuclear cells (PBMC) will also be collected. These blood cells are a critical component in the immune system to fight infection and adapt to intruders. A blood sample for deoxyribonucleic acid [DNA] testing will also be collected as part of this study. Genetic testing may help find reasons why patients respond differently to medicines, find genes that cause a disease or change the outcome of a disease, or develop better medicines for preventing or treating a disease.
As an additional optional part of this study a spinal tap for cerebrospinal fluid (CSF) may be done. CSF will be used to learn more about the disease and to identify biomarkers.
Approximately 250 people will participate in this study, at approximately 25 sites in the United States, Canada, and Europe. Participation will last approximately 2 years.
Participants will not receive any study treatment from the Sponsor. Participants will continue to be treated for their ALS by their regular physician.
If participants currently receive treatment with riluzole they will be encouraged to continue this treatment regime. If they do not currently receive treatment with riluzole, then they will be encouraged not to start this treatment during the study.
This study is sponsored by Biogen Idec Research LimitedREC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
15/YH/0528
Date of REC Opinion
14 Dec 2015
REC opinion
Favourable Opinion