Rasch-analysis of Clinical Severity in FSHD

NCT02766985 — Completed, 44 participants, University of Kansas Medical Center

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Registry	NCT02766985, acronym ROC-FSHD
Status	Completed
Study type	Observational
Enrollment	44 (Actual)
Sponsor	University of Kansas Medical Center (Other); collaborators: National Institutes of Health (NIH)
Conditions	Facioscapulohumeral Muscular Dystrophy
Dates	start 2016-03-01, primary completion 2017-04-18, completion 2017-04-18, first posted 2016-05-10, last updated 2018-04-12
Locations	2 sites in United States

-Summary

The purpose of this study is to make a standardized and scalable Rasch-built clinical severity scale to help in finding genetic and environmental modifiers of disease in Facioscapulohumeral muscular dystrophy (FSHD).

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+Arms and interventions

Arm	Type	Interventions	Description
FSHD			Participants with FSHD-1 or FSHD-2. No intervention is given to participants. Participants will undergo series of tests and procedures in order to make a standardized and scalable Rasch-built clinical severity scale.

+Outcome measures

Primary outcomes

FSHD Clinical Severity Scale [1 day]: Measure severity of FSHD.

Secondary outcomes

Brooks scale [1 day]: Measures upper extremity function.Scores on the scale range from 1-6. A score of 1 means the person can elevate their arms above their head while arms remain straight. 6 means the person cannot elevate their arms to their mouth and do not have useful function of their hands.
Vignos scale [1 day]: Measures lower extremity function. Scores on the scale range from 1-10. A score of 1 means the person can walk and climb stairs without assistance. A score of 10 means the person is bedridden.
Iowa Oral Performance Instrument (IOPI) [1 day]: IOPI measures lip and tongue strength by measuring the amount of pressure a person can exert on the tool.
Manual muscle testing [1 day]: Measure person's range of motion. Scored on a scale with range of 0-5. Score of 0 means no movement. Score of 5 means the person has a full range of movement.
Quantitative strength testing [1 day]
Functional testing [1 day]
Reparatory function testing [1 day]
Patient-reported outcome [1 day]

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

* Genetic confirmation of FSHD (either FSHD-1 or FSHD-2)

Exclusion Criteria:

* Do not understand English
* Unable to provide informed consent

+Locations (2)

Facility	City	Country	Status
University of Kansas Medical Center	Kansas City, Kansas	United States
University of Utah	Salt Lake City, Utah	United States

+Investigators

Jeffrey Statland, MD, Principal investigator, University of Kansas Medical Center

+Funding

NIH grants: UL1TR000001