Clinical Trial: MYOPROSP - a Prospective Cohort Study in Myositis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

Official Title: MYOPROSP: A Prospective Cohort Study to Identify a Stratified Approach in the Diagnosis, Treatment and Delivery of Care in Adult Idiopathic Inflammatory Myopathy

Brief Summary:

Adult patients with suspected or confirmed idiopathic inflammatory myopathy (IIM) will be recruited. Patients will be approached, consented, have baseline demographics, diagnostics and disease activity measures recorded, and blood taken. The collection of data and biological material will mirror usual clinical practice as far as possible. Subjects will ideally attend further visits at 3, 6 and 12 months to have bloods taken, outcome measures recorded and questionnaires completed.

In addition, blood, muscle biopsies and imaging undertaken as part of usual care will also be collected for research purposes to measure a number of biomarkers for the assessment of diagnostic accuracy and clinical utility evaluation. As per usual practice, a muscle biopsy will be performed at baseline, and a further biopsy offered at 6 months to assess treatment response. A magnetic resonance (MR) muscle protocol will also be performed as per usual clinical practice, and a gadolinium-enhanced MR heart scan offered. Both these scans will be repeated at 6 months. An existing electronic database entry system will be used for data entry and capture on an anonymised basis.

The study will thus be based around diagnostic evaluations and outcome measures to improve quality of care in IIM.


Detailed Summary:

The bioresource from this protocol, ethics application and future funding will comprise the following:

i) UKMYONET cross-sectional study ii) MYOPROSP inception cohort study iii) MYOACT novel therapies registry iv) UK neuromuscular biobank

Primary endpoint The primary study endpoint will be sensitivity to change of the biomarkers of interest.

Secondary endpoints

This will include but not limited to:

  • Response to treatment
  • Drug reductions and cessations due to treatment failure, adverse events and clinical remission
  • Differentiation of myositis subgroups using newer imaging modalities
  • Sensitivity of biomarkers to diagnose extramuscular manifestations (including myocardial and pulmonary lung disease) and predict their severity and clinical course
  • Identification of novel genetic variants associated with IIM and subtypes
  • Cost economic analysis
  • Validations of definition of improvement criteria

Cases will be adults >18 years, with a number of different IIM clinical phenotypes encountered in clinically practice. It is vitally important that patients with suspected, but not confirmed, IIM are also included so that the natural history and subsequent final diagnoses can be tracked. Subsequent investigations will often confirm a diagnosis that was not confirmed at initial presentation. Patients who do have confirmed PM/DM will have IIM according to the Bohan and Peter criteria, which remains t
Sponsor: University of Manchester

Current Primary Outcome: Number of participants with a significant change levels of diagnostic biomarkers. [ Time Frame: 5 years ]

This will depend on the specific biomarker/cytokine being measured


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of participants with a 20% improvement in myositis specific disease activity measures from baseline [ Time Frame: 5 years ]
    As per the published International Myositis Assessment & Clinical Studies Group (IMACS) outcome measures of definition of improvement (http://www.niehs.nih.gov/research/resources/imacs/definitions/index.cfm)
  • Differences in frequency of genetic variants associated with IIM and subtypes compared to population matched controls [ Time Frame: 5 years ]
    Specific identified genetic variants will be identified as part of large scale genetic studies


Original Secondary Outcome: Same as current

Information By: University of Manchester

Dates:
Date Received: December 5, 2014
Date Started: October 4, 2016
Date Completion: February 2021
Last Updated: January 31, 2017
Last Verified: January 2017