Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
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ClinicalTrials.gov Identifier: NCT03458832 |
Recruitment Status
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Recruiting
First Posted
: March 8, 2018
Last Update Posted
: April 11, 2018
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Tracking Information | |||||||||
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First Submitted Date | January 11, 2018 | ||||||||
First Posted Date | March 8, 2018 | ||||||||
Last Update Posted Date | April 11, 2018 | ||||||||
Actual Study Start Date | March 5, 2018 | ||||||||
Estimated Primary Completion Date | March 2022 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures |
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Original Primary Outcome Measures | Same as current | ||||||||
Change History | Complete list of historical versions of study NCT03458832 on ClinicalTrials.gov Archive Site | ||||||||
Current Secondary Outcome Measures |
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Original Secondary Outcome Measures | Same as current | ||||||||
Current Other Outcome Measures | Not Provided | ||||||||
Original Other Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title | Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD | ||||||||
Official Title | Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD | ||||||||
Brief Summary | The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures and optimize eligibility criteria by testing 160 patients in 7 sites over a period of 18 months. | ||||||||
Detailed Description | The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a normally silenced gene, DUX4, resulting in a toxic gain-of-function. This disease mechanism is particularly amenable to knock-down of DUX4 using epigenetic strategies or RNA therapies, as well as to other interventions targeting the downstream effects of DUX4 expression. There are many drug companies actively working towards disease-targeted therapies, and two clinical trials either under way now, or planned to start in early Fall 2016. However, meetings with industry, advocacy groups, and FSHD researchers have identified several gaps in the clinical trial arsenal, and clinical trial planning as a major goal for the community. Consequently, there is an urgent need to establish the tools necessary for the conduct of currently planned and expected therapeutic trials in FSHD. To this end, the researchers propose to develop two novel clinical outcome assessments (COA), a composite functional outcome measure (FSH-COM) and skeletal muscle biomarker, electrical impedance myography (EIM). In addition there is broad consensus a better understanding of the relationship of genetic and demographic features to disease progression will be necessary for enumerating eligibility criteria. The specific aims are to: 1. Determine the multi-site validity of the COAs, 2. Compare the responsiveness of new COAs to other FSHD outcomes and determine the minimal clinically meaningful changes, and 3. establish FSHD cohort characteristics useful for determining clinical trial eligibility criteria. To achieve these aims, the researchers are conducting a multicenter, prospective, 18 months study of 160 subjects. |
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Study Type | Observational | ||||||||
Study Design | Observational Model: Cohort Time Perspective: Prospective |
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Target Follow-Up Duration | Not Provided | ||||||||
Biospecimen | Retention: Samples With DNA Description: Each subject will have approximately 15 mL of blood collected at baseline and month 3 for genetic testing. The month 3 sample will be stored for use as a back-up for any lost samples, or for use in future studies. |
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Sampling Method | Non-Probability Sample | ||||||||
Study Population | Participants with FSHD that are seen in the researchers clinic. | ||||||||
Condition | Facioscapulohumeral Muscular Dystrophy | ||||||||
Intervention |
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Study Groups/Cohorts | FSHD-COM
All participants will be asked to undergo FSHD-specific functional rating scale tests and procedures and Electrical Impedance Myography.
Interventions:
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Publications * | Not Provided | ||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status | Recruiting | ||||||||
Estimated Enrollment |
150 | ||||||||
Original Estimated Enrollment | Same as current | ||||||||
Estimated Study Completion Date | March 2022 | ||||||||
Estimated Primary Completion Date | March 2022 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender |
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Ages | 18 Years to 75 Years (Adult, Senior) | ||||||||
Accepts Healthy Volunteers | No | ||||||||
Contacts |
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Listed Location Countries | United States | ||||||||
Removed Location Countries | |||||||||
Administrative Information | |||||||||
NCT Number | NCT03458832 | ||||||||
Other Study ID Numbers | STUDY00140842 U01NS101944 ( U.S. NIH Grant/Contract ) |
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Has Data Monitoring Committee | No | ||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement |
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Responsible Party | University of Kansas Medical Center | ||||||||
Study Sponsor | University of Kansas Medical Center | ||||||||
Collaborators | National Institute of Neurological Disorders and Stroke (NINDS) | ||||||||
Investigators |
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PRS Account | University of Kansas Medical Center | ||||||||
Verification Date | April 2018 |