SHIELD HD is an international, multisite, prospective, longitudinal cohort natural history study to assess the natural history of HD and its biomarkers that are associated with modulation of the number of cytosine-adenine-guanine (CAG) repeats in the mutant Huntingtin (HTT) gene. Approximately 60 patients will be enrolled into the study and followed for up to 24 months at clinical sites in North America and Europe. The results of this study will inform assessments for a future interventional treatment trial.
The rationale for this study is to obtain longitudinal information related to Somatic Instability and DNA damage response genes in HDGECs at various stages of the disease. Established assessments of disease progression will also be recorded.
Study Type
OBSERVATIONAL
Enrollment
70
University of California, San Diego (UCSD)
San Diego, California, United States
Rocky Mountain Movement Disorders Center
Englewood, Colorado, United States
Beth Israel Deaconess
Boston, Massachusetts, United States
DDR gene expression
To assess deoxyribonucleic acid (DNA) damage repair (DDR) gene expression in accessible biofluids and disease trajectories for established and novel biomarkers and clinical outcomes.
Time frame: 2 years
Compare rates of change in biomarkers for disease progression
To compare the rates of change for different outcomes and cytosine adenine guanine (CAG) age product (CAP) Scores.
Time frame: 2 years
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Columbia University
New York, New York, United States
Inland Northwest Research
Spokane, Washington, United States
Centre for Movement Disorders
Toronto, Ontario, Canada
North York General Hospital
Toronto, Ontario, Canada
ICM - Institut du Cerveau et de la Moelle épinière
Paris, France
George-Huntington-Institut (GHI)
Münster, Germany
University College London - Institute of Neurology & The National Hospital for Neurology and Neurosurgery
London, United Kingdom