Rasch-analysis of Clinical Severity in FSHD (ROC-FSHD)

University of Kansas

Status: Completed

Conditions: Facioscapulohumeral Muscular Dystrophy

Study type: Observational

Funder types: Other; NIH

Identifiers

NCT02766985

UL1TR000001 (U.S. NIH Grant/Contract)

STUDY00003640

Details and patient eligibility

About

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).

Enrollment: 44 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

• Do not understand English
• Unable to provide informed consent

Trial design

44 participants in 1 patient group

FSHD

Description:

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.