Hereditary Inclusion Body Myopathy-Patient Monitoring Program (HIBM-PMP): A Registry and Prospective Natural History Study to Assess HIBM Disease

Background

HIBM is a severe progressive myopathy. It typically starts in early adulthood as muscle weakness in the legs and progresses, ultimately leading to a wheelchair-bound state. The rate of progression is gradual and variable over the course of 10-20 years or longer.

There is a need to:

• better understand the specific features of HIBM to improve awareness
• help early diagnosis
• identify patients
• expand knowledge of the clinical presentation, progression and variation of the disease
• identify and validate biomarkers and other outcome measures
• inform on the design and interpretation of clinical trials of new drugs
• and eventually to optimize patient management

Recruitment

Up to 10 centres in:

• North America
• European Union
• and Middle East

Study information

HIBM Disease Registry is a database where patients can self-report their condition. Registry patients will not necessarily be associated with a clinical site.

Those registry patients who are in the same country as a study site may choose to have their data confirmed by one of those centres or opt-in to the HIBM Natural History Study.

Primary objective

To better understand HIBM.

HIBM Disease Registry's specific objectives:

• Identify HIBM patients worldwide
• Promote awareness and help diagnosis of HIBM disease in the neuromuscular field
• Obtain an assessment of the medical history, clinical presentation and progression of disease in HIBM patients
• Provide a connection for patients to the broader HIBM community and associated programs
• Provide customized information to patients and their physicians that desire information on their disease status and progression.

HIBM Natural History Study's specific objectives:

• Characterize HIBM disease presentation and progression over time using relevant clinical assessments
• Obtain information using patient-reported outcomes (quality of life and timing of life changing events)