2017-2018
2017-2018
| Project title: | Trial readiness to solve barriers to drug development in FSHD |
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| Investigator: | Jeff Statland, MD, Kansas University Medical Center |
| Approval: | 2/2018 |
| Study Type: | Recruitment |
| Description: | As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development. This study will develop two new outcome measures and optimize eligibility criteria by testing 160 patients in 8 sites over a period of 18 months. |
| Project title: | Study of testosterone and rHGH in FSHD (STARFISH) |
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| Investigator: | Chad Heatwole, MD, University of Rochester Medical Center |
| Approval: | 1/2018 |
| Study Type: | Recruitment |
| Description: | The purpose of this study is to evaluate whether or not a combination of two drugs (recombinant human growth hormone (rHGH) and testosterone) can be safely given to patients with FSHD and possibly improve walking, strength, muscle mass, quality-of-life, and functional ability. |
| Project title: | Clinical Establishing Biomarkers and Clinical Endpoints in Mytonic Dystrophy Type 1 (END-DM1) |
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| Investigator: | Charles Thornton, MD, University of Rochester Medical Center/ Nicholas Johnson, MD, Virginia Commonwealth University |
| Approval: | 1/2018 |
| Study Type: | Recruitment |
| Description: | Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), this study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of DM1. 650 people with DM1 will be enrolled across 16 centers in the US and Europe. |
| Project title: | Bioresources Core: Resources for FSHD Research and Clinical Trials |
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| Investigator: | Rabi Tawil, MD, University of Rochester Medical Center |
| Approval: | 6/2017 |
| Study Type: | Recruitment |
| Description: | This study will help test the best ways to measure FSHD symptoms and understand how they change over time. This information is essential for planning future studies to test experimental treatments for FSHD. The study will perform genetic testing for FSHD, measure “biomarkers” of disease manifestations, and conduct questionnaires, muscle strength and function testing, physical exam, blood testing and skin and muscle biopsies. |
| Project title: | A study of depression and its treatment in members of the National Registry of DM and FSHD |
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| Investigator: | Amy Parkhill, PhD, St. John Fisher College |
| Approval: | 5/2017 |
| Study Type: | Recruitment |
| Description: | The purpose of the study is to evaluate the prevalence of depression and if and how it’s being managed in Registry participants. This information may help guide clinical care and help researchers, clinicians, and pharmacists better understand how the manifestations of DM and FSHD may impact depression. Patients participate by completing a questionnaire. |
| Project title: | Natural History Data Analysis in FSHD |
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| Investigator: | Sanjay Shukla, MD, Atyr Pharmaceuticals |
| Approval: | 1/2017 |
| Study Type: | Data analysis |
| Description: | This study will help industry to better understand the natural history or progression of FSHD over time and help define the patient population. This information will help Atyr plan for clinical trials in FSHD. |