HIBM Natural history study
Research type
Research Study
Full title
Hereditary Inclusion Body Myopathy-Patient Monitoring Program (HIBM-PMP): A Registry and Prospective Observational Natural History Study to Assess HIBM Disease
IRAS ID
120840
Contact name
Hanns Lochmüller
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Hereditary inclusion body myopathy (HIBM) is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and ultimately a wheelchair-bound state. The HIBM Natural study is a prospective observational study conducted at specific centers worldwide. The only UK site will be located in Royal Victoria Infirmary (Newcastle upon Tyne). The study will monitor the condition of HIBM patients with broad spectrum of disease severity to determine the rate of decline in functional outcomes over time. Characterization of the decline in functional outcome and the relationship with disease severity will greatly aid the planning of future interventional studies of HIBM. Patients who meet the inclusion criteria and agree to participate will be included in the study. After enrollment in the study, patients will participate in one day study evaluation visits at baseline, 6 months, 12 months and then yearly after to repeat the study evaluations. The study will continue for 3 years and potentially may be extended for up to 15 years. At the visits, a medical history (baseline only) or interval medical history will be obtained. Patients will be evaluated by tests of muscle strength and function, patient-reported outcomes measures, and a blood sample taken for evaluation of biomarkers. No investigational agents or experimental interventions will be administered to subjects as part of the HIBM Natural History Study.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
13/NE/0124
Date of REC Opinion
22 Jun 2013
REC opinion
Further Information Favourable Opinion