Condition category
Nervous System Diseases
Date applied
23/08/2012
Date assigned
21/02/2013
Last edited
09/08/2013
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

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 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 to 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 until 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

Trial website

Contact information

Type

Scientific

Primary contact

Dr Matthew Parton

ORCID ID

Contact details

Institute of Neurology
Queen Square
London
WC1N 3BG
United Kingdom
matt.parton@uclh.nhs.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.

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

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

Not specified

Trial type

Screening

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

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.

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

01/11/2014

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 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.

Participant type

Patient

Age group

Not Specified

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

01/11/2014

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Institute of Neurology
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

http://www.uclh.org/

Funders

Funder type

Charity

Funder name

Muscular Dystrophy Campaign (UK)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes