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| [ Print-friendly version ] |
| The Natural History of Inclusion Body Myositis |
| ISRCTN | ISRCTN96803614 |
| DOI | 10.1186/ISRCTN96803614 |
| ClinicalTrials.gov identifier | |
| EudraCT number | |
| Public title | The Natural History of Inclusion Body Myositis |
| Scientific title | The Natural History of Inclusion Body Myositis: an observational cohort study |
| Acronym | N/A |
| Serial number at source | 11688 |
| Study hypothesis |
Inclusion body myositis (IBM) is the commonest muscle disease beginning in those aged over 50. However it is still poorly understood and its cause is unknown. Furthermore, IBM has no treatment and leads to progressive disability.
To date, the largest published study of the illness followed only eleven patients for six months. Thus, information on the pattern and prognosis of IBM is based on anecdote from clinical experience, rather than firm fact. Our project seeks to better characterise the condition by gathering data from as many cases of IBM as possible. This will build a crucial resource and form the starting-point for future studies of the illness. Participants in the project will be asked to volunteer to be seen at least annually and undergo a standardised assessment. This will consist of information on the history of the illness, their medical background, how IBM affects everyday tasks and findings on physical examination. All data will be recorded on a secure central computer database. To allow as many people as possible to participate in the study we will make data entry available (over a secure internet link) to other medical specialists around the UK, so people can be seen nearer to home and information entered locally. Alternatively, the necessary clinical data can be sent to our hospital for us to enter into the database. By repeating our assessments over five years, we will be able to give a much more reliable and accurate prediction of the course of the disease. As well as the clinical data, we also wish to ask participants to donate a small blood sample for storage and extraction of DNA. The blood and DNA samples will be stored and can be used in future studies of the disease. More details can be found at: http://public.ukcrn.org.uk/search/StudyDetail.aspx?StudyID=11688 |
| Lay summary |
Background and study aims
Inclusion body myositis (IBM) is the commonest muscle disease beginning in those aged over 50. It leads to progressive disability with, classically, a characteristic pattern of muscle involvement. IBM is still poorly understood and its cause unknown. At present, there is no conclusive diagnostic test and it has no treatment. Furthermore, information on the pattern and prognosis of IBM is based more on anecdote (account of/story) from clinical experience, rather than on firm fact. The largest published series of data on the natural history of the illness followed only eleven patients for six months. This study seeks to better characterise the condition by gathering data from as many cases of IBM as possible. This will build an important resource and so form the starting point for future studies of the illness. Who can participate? Any person who meets the established diagnostic criteria for inclusion body myositis. What does the study involve? Participants in the project will be asked to volunteer to undergo a standardised assessment at least annually for a five year period. We will record background information on the history of the illness and any other medical conditions, plus how IBM currently affects everyday tasks and the findings of a physical examination. As well as this clinical data, we will also ask participants to donate a small blood sample for storage and extraction of DNA and serum. The serum and DNA samples will be stored for used in future studies of the disease. All data will be recorded on a secure central computer database. This is a multi-centre study and to allow as many people as possible to participate we will make data entry possible by other muscle disease specialists around the UK (over a secure internet link). This will allow people can be seen near to home. Alternatively, the necessary clinical data can be sent to our hospital for us to enter into the database. By repeating our assessments over five years, we will be able to give a much more reliable and accurate prediction of the course of the disease. What are the possible benefits and risks of participating? This study will therefore improve knowledge of IBM and so first allow us and other doctors to give more accurate prediction of the likely progression of the disease to our IBM patients. In time, analysis of the clinical data plus studies of the DNA or serum gathered for the study may contribute further. Such future follow-on studies offer the possibility of identifying risks for developing IBM and could help generate interventions to reduce the disability IBM causes. The only burden we foresee to participants is the inconvenience posed by an additional annual trip to hospital for the assessment, and the discomfort of undergoing a single blood test. Where is the study run from? The study is run from The National Hospital for Neurology and Neurosurgery in London, with the inclusion of other participating centres around the UK. When is the study starting and how long is it expected to run for? The study started in January 2012 and will run till 2014. Who is funding the study? The study is partly funded by the Muscular Dystrophy Campaign. Who is the main contact? Dr. Pedro Machado , p.machado@ion.ucl.ac.uk Gisela Barreto , Gisela.barreto@uclh.nhs.uk |
| Ethics approval | East Central London Research Ethics Committee, 07 June /2010, ref: 10HO72128 |
| Study design | Non-randomised observational cohort study |
| Countries of recruitment | United Kingdom |
| Disease/condition/study domain | Inclusion body myositis |
| Participants - inclusion criteria |
Any person who meets the established diagnostic criteria for inclusion body myositis. The age criteria set above in section A16 are to allow for one criterion being age at onset of over 30, while the upper limit is set so as not to exclude any participant on the grounds of being too old.
Griggs' diagnostic criteria for IBM: Clinical features: 1. Illness duration of more than six months 2. Male and female, age at onset greater than 30 years 3. Proximal and distal weakness of arms and legs, with finger flexion weakness, wrist flexion more than extension weakness, and quadriceps weakness. Laboratory features: 1. Creatine kinase (CK) less than 12 times normal 2. Neurophysiology consistent with myopathy Muscle biopsy features: 1. Inflammation with mononuclear cell invasion of non-necrotic fibres 2. Vacuolated fibres 3. Intracellular amyloid and/or 15–18 nm filaments on electron microscopy If all the muscle biopsy features above are present, then the disease is labelled as ""definite"" (regardless of the presence or absence of other criteria). To meet the ""probable"" standard of disease, then the muscle biopsy must show at least inflammation and vacuolation of fibres, plus all the clinical and laboratory features above. |
| Participants - exclusion criteria | Does not meet inclusion criteria |
| Anticipated start date | 27/01/2012 |
| Anticipated end date | 01/11/2014 |
| Status of trial | Ongoing |
| Patient information material | Not available in web format, please contact Gisela Barreto (Gisela.barreto@uclh.nhs.uk) to request a patient information sheet |
| Target number of participants | UK Sample Size: 120 |
| Interventions |
Our project seeks to better characterise the condition by gathering data from as many cases of IBM as possible. This will build a crucial resource and so form the starting point for future studies of the illness.
Participants in the project will be asked to volunteer to undergo a standardised assessment at least annually for a five year period. We will record background information on the history of the illness and any other medical conditions, plus how IBM currently affects everyday tasks and the findings of a physical examination. As well as this clinical data, we will also ask participants to donate a small blood sample for storage and extraction of DNA and serum. The serum and DNA samples will be stored for used in future studies of the disease. |
| Primary outcome measure(s) | Rate of strength decline over time |
| Secondary outcome measure(s) | No secondary outcome measures |
| Sources of funding | Muscular Dystrophy Campaign (UK) |
| Trial website | |
| Publications | |
| Contact name | Dr Matthew Parton |
| Address |
Institute of Neurology
Queen Square |
| City/town | London |
| Zip/Postcode | WC1N 3BG |
| Country | United Kingdom |
| matt.parton@uclh.nhs.uk | |
| Sponsor | University College London Hospitals NHS Foundation Trust (UK) |
| Address |
National Hospital for Neurology and Neurosurgery
Gower Street |
| City/town | London |
| Zip/Postcode | WC1E 6BT |
| Country | United Kingdom |
| Sponsor website: | http://www.uclh.org/ |
| Date applied | 23/08/2012 |
| Last edited | 12/03/2013 |
| Date ISRCTN assigned | 21/02/2013 |
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