Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD
- Conditions
- Huntington Disease
- Registration Number
- NCT04406636
- Lead Sponsor
- CHDI Foundation, Inc.
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 70
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method DDR gene expression 2 years To assess deoxyribonucleic acid (DNA) damage repair (DDR) gene expression in accessible biofluids and disease trajectories for established and novel biomarkers and clinical outcomes.
- Secondary Outcome Measures
Name Time Method Compare rates of change in biomarkers for disease progression 2 years To compare the rates of change for different outcomes and cytosine adenine guanine (CAG) age product (CAP) Scores.
Trial Locations
- Locations (10)
Columbia University
🇺🇸New York, New York, United States
Inland Northwest Research
🇺🇸Spokane, Washington, United States
ICM - Institut du Cerveau et de la Moelle épinière
🇫🇷Paris, France
University of California, San Diego (UCSD)
🇺🇸San Diego, California, United States
George-Huntington-Institut (GHI)
🇩🇪Muenster, Germany
University College London - Institute of Neurology & The National Hospital for Neurology and Neurosurgery
🇬🇧London, United Kingdom
Rocky Mountain Movement Disorders Center
🇺🇸Englewood, Colorado, United States
Beth Israel Deaconess
🇺🇸Boston, Massachusetts, United States
Centre for Movement Disorders
🇨🇦Toronto, Ontario, Canada
North York General Hospital
🇨🇦Toronto, Ontario, Canada