Clinical Evaluator Outcomes Reliability Study

Cooperative International Neuromuscular Research Group (CINRG) Clinical Evaluator Outcomes Reliability Study

This study outlines structured Clinical Evaluator (CE) testing techniques that are proposed to be implemented across all sites participating in the Cooperative International Neuromuscular Research Group (CINRG) research studies. The study will determine if the selected techniques are reliable and reproducible across the CINRG network by evaluating the reliability and reproducibility of the measures between CEs

Study Overview

• Status: Unknown
• Conditions: Dystrophinopathies

• Study Type: Observational
• Enrollment (Anticipated): 55

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Dystrophinopathies (Duchenne Muscular Dystrophy and Becker Muscular Dystrophy)

Description

Dystrophinopathy Participant Inclusion Criteria:

• Site confirmed genetic diagnosis of a dystrophinopathy
• Aged 6 years and older
• Able to transfer to testing table
• Able to walk 10 meters without an assistive device

Dystrophinopathy Participant Exclusion Criteria:

• Investigator assessment of inability to comply with protocol assessments
• Participants who have attention deficient disorder or other cognitive conditions that may limit attention span to perform protocol assessments

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort
Dystrophinopathies
Gold Standard Clinical Evaluators (GS-CEs)
Sites Clinical Evaluators (CEs)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Evaluator Assessments	The following assessments are conducted by certified physical therapists: Anthropometrics, goniometry, Brooke upper extremity and Vignos lower extremity scales, Performance Upper Limb Scale (PULS), North Star Ambulatory Assessment (NSAA), Six Minute Walk Test (6MWT), time to rise from floor, time to stand from a chair, time to climb four stairs, time to walk/run 10 meters, 9 Hole Peg Test (9HPT), Quantitative Muscle Tests (QMT), Manual Muscle testing (MMT), Force Vital Capacity (FVC), Force Expiratory Volume (FEV1), Maximum Inspiratory Pressure (MIP), Maximum Expiratory Pressure (MEP), Peak Expiratory Flow Rate (PEFR) and Peak Cough Flow (PCF). The reliability of these commonly used measurements is fundamental to clinical research, our ability to have confidence in the data we collect and our ability to draw rational conclusions from the data.	One time visit

Collaborators and Investigators

• Sponsor: Cooperative International Neuromuscular Research Group
• Investigators: Principal Investigator: Lauren Morgenroth, MS, CGC, Children's National Research Institute; Principal Investigator: Tina Duong, MPT, Stanford University

Study record dates

Study Major Dates

• Study Start: May 1, 2014
• Primary Completion (Anticipated): December 1, 2016
• Study Completion (Anticipated): December 1, 2016

Study Registration Dates

• First Submitted: May 14, 2014
• First Submitted That Met QC Criteria: May 21, 2014
• First Posted (Estimate): May 26, 2014

Study Record Updates

• Last Update Posted (Estimate): February 2, 2016
• Last Update Submitted That Met QC Criteria: February 1, 2016
• Last Verified: February 1, 2016

More Information

Terms related to this study

• Other Study ID Numbers: CNMC0713