This two-year natural history study of HDGECs, sponsored by Triplet Therapeutics, is collecting longitudinal information related to somatic instability and DNA damage response genes, along with established assessments of disease progression. The study has enrolled 70 participants to date, and follow-up assessments are ongoing at sites in North America and Europe. The results will inform assessments ahead of a future treatment trial.
SHIELD HD is an International study that will follow a group of people over time who have or are at risk of developing Huntington´s Disease. SHIELD HD will collect health information that includes medical history, blood and cerebrospinal fluid samples. Participants will also undergo MRIs, cognitive and motor function tests. The information will be used to evaluate the natural history of HD and its biomarkers that are associated with the modulation of the number of CAG (cytosine-adenine-guanine) repeats in the mutant Huntingtin (mHTT) gene. The results of this study will inform assessments for a future interventional treatment trial.
This international study will follow a group of people over time who have or are at risk of developing HD. Health information will be collected including medical history, blood and CSF samples. Participants will also undergo MRIs, cognitive and motor function tests. The information will be used to look at the natural history of HD and its biomarkersthat are linked with changes in the CAG repeat.
Any conditions, including severe chorea and dementia, that would prevent either writing or performing pen and paper, tablet, or computer based tasks as determined by the Investigator
Treatment with an investigational drug within 30 days prior to screening or within 5 half lives of the investigational drug, whichever is longer
History of gene therapy or cell transplantation or any other experimental brain surgery
LOCATIONS
COUNTRIES
FRANCE
TRIAL SITE: ICM – Institut du Cerveau et de la Moelle épinière
Address: Paris, France, 75013
Active, Not Recruiting
GERMANY
TRIAL SITE: George-Huntington-Institut
Address: Münster, Germany, 48149.
Active, Not Recruiting
UNITED KINGDOM
TRIAL SITE: University College London – Institute of Neurology & The National Hospital for Neurology and Neurosurgery
A clear fluid produced by the brain, which surrounds and supports the brain and spinal cord.
(from HDbuzz.org)
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Biomarker
A biomarker, or biological marker is a measurable indicator of some biological state or condition. It can be a level of something in your blood or hormon levels for instance. Biomarkers are often measured and evaluated to examine normal biological processes – like measuring the level of hemoglobin in your blood or your blood pressure. In reference to research on Huntingtons disease, a biomarker is an indicator of disease progression. Examples are changes within the cells related to so-called metabolism (energy processes) or levels of mutant Huntingtin in nervecells. It can also be changes in organs – like loss of muscle or brain volume. We use biomarkers to measure how the disease progress and potentially how a treatment influence the disease progression.
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Inclusion Criteria
A type of eligibility criteria. These are the reasons that a person is allowed to participate in a clinical study.
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Exclusion Criteria
A type of eligibility criteria. These are reasons that a person is not allowed to participate in a clinical study.
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CAG repeat
The stretch of DNA at the beginning of the HD gene, which contains the sequence CAG repeated many times, and is abnormally long in people who will develop HD. (from HDbuzz.org)
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At risk
You do not know if you carry the genetic mutation for HD gene
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mHTT
Mutant huntingtin protein. The protein produced by the faulty HD gene.