Queen Square Centre for Neuromuscular Diseases



CBYM338B2302: A Prospective Natural History Study in Sporadic Inclusion Body Myositis (sIBM)

Sponsor / Funder Novartis
CI Dr Pedro Machado/ Professor Michael Hanna
Sites London Queen Square
Contact details p.machado@ucl.ac.uk

This is a longitudinal, multinational, multi-centre natural history study of patients diagnosed with sIBM.

Primary objective

To characterize the clinical progression and functional impact of sIBM on patients and their caregivers over time. This will be assessed by:

  • functional performance measures
  • sporadic Inclusion Body Myositis Physical Functioning Assessment.

The link between functional impairment and long-term outcomes, such as loss of mobility and falls, will be examined. Also, this study will identify both the economic and humanistic burden of this disease.

Epidemiological data collected will document basic demographic characteristics of the sIBM patient population.

Composition MRI imaging in a group of 30 sIBM patients* will assess the changes in muscle quality.

*only patients in the MRC Centre for Neuromuscular Diseases