Keywords: Huntington's Disease (HD) , Observational , Prospective Registry , Multi-national , Mutli-center
IRB Number: 00056631
Specialty: Neurology, Neurology, Neurology
Sub Specialties: Movement Disorders, Chorea, Huntington Disease
Enroll-HD is an observational, open-ended, prospective, multi-national, multi-centre study without experimental treatment.
Objective 1: To improve the understanding of the dynamic phenotypic spectrum and the disease mechanisms of HD by:
a. collecting natural history data covering the cognitive, behavioral and motor domains permitting estimates of rates of progression in HD and allowing insights into the neurobiology of HD,
b. collecting data and biologic samples to identify genetic and environmental factors influencing and/or modifying the HD phenotype and disease progression, and
c. promoting interrogatory studies that may provide clues to the pathogenesis of HD.
Objective 2: To promote the development of evidence-based guidelines to inform clinical decision making and improve health outcomes for the participant/family unit by:
a. assisting in the identification of beneficial interventions (clinical, pharmaco-therapeutic, non-pharmacologic),
b. facilitating the dissemination and implementation of currently proposed best clinical practices,
c. providing a platform for conducting outcome research, and
d. promoting exploratory data analysis projects that may identify processes to further improve the healthcare of affected individuals and their families.
Objective 3: To provide a platform to support the design and conduct of clinical trials by:
a. providing a resource to identify, develop and qualify novel assessment tools, clinical endpoints and biomarkers,
b. collecting longitudinal data to inform disease modeling studies, and
c. facilitating the identification of potential trial participants informing the selection of potential trial participants using data to estimate and quantify slopes/rates of disease progression (providing “run-in” data).
To achieve these objectives suitably de-identified and coded clinical information and biological samples collected from study participants will be made available to investigators for research purposes in accordance with procedures adopted by the steering committee.