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Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease
Scottsdale/Phoenix, AZ
This study is a 26 week, randomized, parallel group, double blind comparison of PF-02545920 5 mg, PF-02545920 20 mg, and placebo dosed BID in the treatment of motor impairment of subjects with Huntington's Disease. A total of approximately 260 subjects are planned to be randomized in the study. Primary endpoint is the change from baseline in the Total Motor Score (TMS) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 26 weeks of treatment. secondary endpoints will include change from baseline in the Total Maximum Chorea (TMC) score of the UHDRS after 13 and 26 weeks of treatment and ...
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A Study to Investigate the Long Term Safety, Tolerability And Effectiveness of Pf-02545920 in Subjects with Huntington's Disease Who Completed Study A8241021
Scottsdale/Phoenix, AZ
The purpose of this study is an extension following study A8241021 to assess the long-term safety, tolerability and effectiveness of PF-02545920 in patients with Huntington's disease.
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Gene Editing Research With a Patient Centered Approach
Jacksonville, FL; Rochester, MN
This is an interview study to understand the views of people with the lived experience of 10 different genetic conditions on gene modification therapies, and specifically on prenatal gene editing. Prenatal gene editing is not happening now, but it is possible that prenatal gene editing will be available in the next few years, at least in a research setting, and we want to know your thoughts about the direction this technology is going. We to hope speak with many different stakeholders (patients and their families, clinicians, scientists) with diverse perspectives to understand values and priorities for prenatal gene ...