MysIBM: A Non-interventional Study in Sporadic Inclusion Body Myositis
Research type
Research Study
Full title
A Prospective Natural History Study in Sporadic Inclusion Body Myositis (sIBM).
IRAS ID
166966
Contact name
Parul Houston
Contact email
Sponsor organisation
Novartis Pharma Services AG (NPSAG)
Duration of Study in the UK
3 years, 1 months, 1 days
Research summary
Sporadic inclusion body myositis (sIBM) is a progressive muscle disorder characterised by slow progressive muscle weakness that generally develops after age 50. Muscle weakness typically leads to loss of manual control, impaired mobility, and increased susceptibility to falls.
This non-interventional study aims to characterise the clinical progression and functional impact of sIBM on patients and their caregivers over time assessed by functional performance measures and patient questionnaires. The association between functional impairment and long-term outcomes, such as loss of mobility and falls, will be examined. In addition, this study will characterise both the economic and humanistic burden of this disease. Furthermore, epidemiological data collected in this study will document basic demographic characteristics of the sIBM patient population.Participating sites will complete a brief questionnaire or Patient-reported outcome (PRO) with input from the patient to capture demographic and medical history information at the initial patient visit to the clinic and annually for 2 years with an optional end-of-study assessment. To assess stage and severity of disease, sites will perform clinical functional assessments on the same schedule. A brief patient questionnaire will be administered monthly, and a more detailed patient questionnaire will be administered every 6 months for up to 2.5 years with an optional end-of-study questionnaire. Additionally, MRI imaging will be performed for 30 patients at one UK centre.
The study will be conducted in 4 countries—Canada, Sweden, UK, and US—and will target at least two specialty sites (neurology, neuromuscular, or rheumatology treatment centres) per country. The goal is to have a diverse group of patients covering all areas of functional ability. It is anticipated that a maximum of 300 patients with a targeted minimum of 70 patients without the ability to walk > 1 meter unaided by another person will be enrolled. The study enrollment period will be up to 6 months.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
14/SC/1419
Date of REC Opinion
4 Dec 2014
REC opinion
Further Information Favourable Opinion