Plain English Summary
Background and study aims
Inclusion body myositis (IBM) is the most common 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 from clinical experience, rather than on firm fact. The largest published series of data on the natural history of the illness followed only 11 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 are asked to volunteer to undergo a standardised assessment at least annually for a five-year period. Background information is collected 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 data, participants are also asked to donate a small blood sample for storage and extraction of DNA and serum. The serum and DNA samples are stored for used in future studies of the disease. All data is recorded on a secure central computer database. This is a multi-centre study and to allow as many people as possible to participate data entry is possible by other muscle disease specialists around the UK (over a secure internet link). This allows people to be seen near to home. Alternatively, the necessary data can be sent to the hospital for the researchers to enter into the database. The assessments are repeated over five years.
What are the possible benefits and risks of participating?
This study will improve knowledge of IBM and allow doctors to give a more accurate prediction of the likely progression of IBM. In time, analysis of the 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 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?
January 2012 to April 2022
Who is funding the study?
1. Muscular Dystrophy Campaign (UK)
2. National Institute for Health Research (NIHR) Rare Diseases Translational Research Collaboration (UK)
Who is the main contact?
1. Dr Pedro Machado (p.machado@ucl.ac.uk)
2. Iwona Skorupinska (Iwona.Skorupinska@uclh.nhs.uk)
Trial website
Contact information
Type
Scientific
Primary contact
Ms Iwona Skorupinska
ORCID ID
Contact details
Institute of Neurology
Queen Square
London
WC1N 3BG
United Kingdom
-
Iwona.Skorupinska@uclh.nhs.uk
Type
Scientific
Additional contact
Dr Pedro Machado
ORCID ID
http://orcid.org/0000-0002-8411-7972
Contact details
Institute of Neurology
Queen Square
London
WC1N 3BG
United Kingdom
-
p.machado@ucl.ac.uk
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
11688
Study information
Scientific title
The natural history of inclusion body myositis: an observational cohort study
Acronym
Study hypothesis
Inclusion body myositis (IBM) is the most common 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 11 patients for six months. Thus, information on the pattern and prognosis of IBM is based on anecdote from clinical experience, rather than firm fact.
This 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.
Ethics approval
East Central London Research Ethics Committee, 07/06/2010, ref: 10HO72128
Study design
Non-randomised observational cohort study
Primary study design
Observational
Secondary study design
Cohort study
Trial setting
Hospitals
Trial type
Other
Patient information sheet
Not available in web format, please contact Gisela Barreto (Gisela.barreto@uclh.nhs.uk) to request a patient information sheet
Condition
Inclusion body myositis
Intervention
Participants in the project will be asked to volunteer to undergo a standardised assessment at least annually for a five-year period. This will consist of 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. All data will be recorded on a secure central computer database. To allow as many people as possible to participate in the study data entry will be 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 the trialist's hospital to enter into the database. By repeating the assessments over five years, the trialists will be able to give a much more reliable and accurate prediction of the course of the disease. As well as this clinical data, participants will also be asked 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.
Intervention type
Other
Phase
Not Specified
Drug names
Primary outcome measures
Rate of strength decline over time
Secondary outcome measures
No secondary outcome measures
Overall trial start date
27/01/2012
Overall trial end date
01/04/2022
Reason abandoned
Eligibility
Participant 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 1518 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.
Participant type
Patient
Age group
Adult
Gender
Both
Target number of participants
UK sample size: 120
Participant exclusion criteria
Does not meet inclusion criteria
Recruitment start date
27/01/2012
Recruitment end date
01/04/2017
Locations
Countries of recruitment
United Kingdom
Trial participating centre
National Hospital for Neurology and Neurosurgery
London
WC1N 3BG
United Kingdom
Sponsor information
Organisation
University College London Hospitals NHS Foundation Trust (UK)
Sponsor details
National Hospital for Neurology and Neurosurgery
Gower Street
London
WC1E 6BT
United Kingdom
Sponsor type
Not defined
Website
Funders
Funder type
Charity
Funder name
Muscular Dystrophy Campaign (UK)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Funder name
National Institute for Health Research (NIHR) Rare Diseases Translational Research Collaboration
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Planned publication in a high-impact peer reviewed journal and presentation at medical conferences.
IPD sharing plan
The current data sharing plans for the current study are unknown and will be made available at a later date.
Intention to publish date
Participant level data
To be made available at a later date
Results - basic reporting
Publication summary
2013 results in: https://www.ncbi.nlm.nih.gov/pubmed/23489664