Rasch-analysis of Clinical Severity in FSHD

Facioscapulohumeral Muscular Dystrophy Clinical Trial

— ROC-FSHD  

Official title:

Rasch-analysis of Clinical Severity in FSHD

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02766985
• Other study ID #: STUDY00003640
• Secondary ID: UL1TR000001
• Status: Completed
• First received: March 22, 2016
• Last updated: April 11, 2018
• Start date: March 2016
• Est. completion date: April 18, 2017

Study information

• Verified date: April 2018
• Source: University of Kansas Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 44
• Est. completion date: April 18, 2017
• Est. primary completion date: April 18, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

• Do not understand English

• Unable to provide informed consent

Related Conditions & MeSH terms

• Facioscapulohumeral Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Facioscapulohumeral

Locations

Country	Name	City	State
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Utah	Salt Lake City	Utah

Sponsors (2)

Lead Sponsor	Collaborator
University of Kansas Medical Center	National Institutes of Health (NIH)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	FSHD Clinical Severity Scale	Measure severity of FSHD.	1 day
Secondary	Brooks scale	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.	1 day
Secondary	Vignos scale	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.	1 day
Secondary	Iowa Oral Performance Instrument (IOPI)	IOPI measures lip and tongue strength by measuring the amount of pressure a person can exert on the tool.	1 day
Secondary	Manual muscle testing	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.	1 day
Secondary	Quantitative strength testing		1 day
Secondary	Functional testing		1 day
Secondary	Reparatory function testing		1 day
Secondary	Patient-reported outcome		1 day