Vicki Wheelock MD HDSA Center of Excellence at UC Davis ...

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What are double blind, randomized, controlled studies? Randomized: the study participant is assigned randomly to either active drug or placebo Controlled: The study drug is compared to an known treatment (active control) or to a placebo (inactive compound that appears identical to the active drug).
Who can participate in clinical trials? • The study protocol is a research plan or roadmap designed to best answer the question posed by the study. • The protocol includes criteria for who is eligible. This could include: – people with HD at early, mid- or late-stage – People at 50% risk for the HD CAG expansion – Family members who are not at risk for HD, or people who have undergone predictive testing and don’t have the HD CAG expansion – People with the HD gene expansion who don’t yet have symptoms
Page 1 and 2: Vicki Wheelock MD HDSA Center of Ex
Page 3 and 4: Presenter Disclosures Dr. Vicki Whe
Page 5 and 6: Clinical research studies, especial
Page 7 and 8: The pathway to finding new drugs Pr
Page 9: What are double blind, randomized,
Page 13 and 14: How much time is required? • Stud
Page 15 and 16: What kinds of activities take place
Page 17 and 18: What kinds of activities take place
Page 19 and 20: What about privacy? • Confidentia
Page 21 and 22: Current observational studies Study
Page 23 and 24: The HD Pipeline Source: Marsha Mill
Page 25 and 26: Results: Tetrabenazine H · S · G
Page 27 and 28: Getting involved in research: Five
Page 29 and 30: There are reasons for and against v
Page 31 and 32: COHORT COHORT • Cooperative Hunti
Page 33 and 34: • Sponsor: NIH PREDICT study •
Page 36 and 37: 2CARE Study • Sponsor: NIH, Phase
Page 38 and 39: HART trial • Sponsor: Neurosearch
Page 40 and 41: CREST-E study • Sponsor: NIH, Pha
Page 42: H · S · G Huntington · Study ·

Vicki Wheelock MD HDSA Center of Excellence at UC Davis ...

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