International HIBM Registry
Research type
Research Database
Full title
International HIBM Registry
IRAS ID
119635
Contact name
Volker Straub
Contact email
Research summary
International HIBM Registry
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
13/NE/0123
Date of REC Opinion
18 Jun 2013
REC opinion
Further Information Favourable Opinion
Data collection arrangements
The HIBM Registry is an online registry for HIBM patients. The data to be stored in the registry will include some personal and demographic data, relevant medical data and results of the disease specific genetic and biopsy tests.
Data collection form is divided in three sections. First part contains general demographic information, clinical diagnosis and relevant HIBM medical information. It was created to be compatible with the existing National HIBM Registry in Japan to facilitate general de-identified data analysis. Second part collects the data on disability (mainly motor function) and support in daily living activities. Final part includes validated questionnaires “Your Health and Well-Being” (SF12) and Body Myopathy Functional Activities Scale (FAS).
The data will be entered through an online portal and will be initiated by the data subject.Patients will be directed (either by themselves, their physicians, or patient advocacy groups) to the registry portal. Data will be entered in the registry at baseline then yearly thereafter for up to 15 years.
The HIBM Disease Registry will allow patients to review their own data collected over time. Physicians will be allowed to access the information for the subjects they personally manage and track their progress through customized physician reports. The registry will be linked to the natural history study of the disease progression. The two modules (registry and natural history study) of one Patient monitoring program will provide the insight in the clinical presentation, progression and variability of the disease.Research programme
The Neuromuscular Research community will be supported directly through it links to TREAT-NMD. The TREAT-NMD Alliance brings together researchers, clinicians, patients organizations and industry on a global level to help to improve treatments and standards of care for all neuromuscular disorders. This database will provide a first step in Patient Monitoring Program which will help to describe clinical presentation, natural progression and variation of the disease. An additional goal of this disease registry is to make the information provided by patients/physicians accessible to an appropriate research community, while protecting subject identity. Aggregated de-identified data gathered from the profiles will be made available to the community of registered subjects, families, and medical researchers in the hope that this information will provide insight into the disease, and help drive clinical trials and research that could lead to better ancillary treatment strategies, including physical therapy guidelines.
Research database title
International HIBM Registry
Establishment organisation
Newcastle University
Establishment organisation address
Framlington place
Newcastle upon Tyne
United Kingdom
NE2 4HH