Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)

Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS

The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid tool for measuring muscle strength in children with DMD. The data obtained from this study will be used to make recommendations for strength measurement endpoints in prospective muscular dystrophy trials and provide more reliable and accurate recommendations in the clinic for strength assessment. This study will be performed at six participating sites in the Cooperative International Neuromuscular Research Group (CINRG).

Study Overview

• Status: Completed
• Conditions: Duchenne Muscular Dystrophy

Detailed Description

We propose to compare the reliability of CQMS vs. HHM in the DMD population. The importance of this study is to be able to compare the results of clinical trials done by different networks using distinct strength endpoints. If results indicate a distinct difference in reliability of one tool over the other, a standardized tool could be established for research groups throughout the world to interpret strength in the context of clinical trials. If the results indicate minor differences then it would be possible to interpret and compare/contrast strength measurements used in different studies.

Understanding the relationship between the HHM vs CQMS will help us examine other surrogate measures capable of predicting functionality that are based on strength measurements.

• Study Type: Observational
• Enrollment (Anticipated): 30

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • Royal Children's Hospital
• Italy
  • Milano, Italy, 20162
    • Centro Clinico NeMO
• United States
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Children's National Medical Center
  • Missouri
    • St. Louis, Missouri, United States, 63110
      • Washington University - St. Louis
  • North Carolina
    • Charlotte, North Carolina, United States, 28207
      • Carolinas Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Probability Sample

Study Population

Participants will be selected from participating CINRG institution's neuromuscular clinics.

Description

Participants should meet the following criteria:

1. Confirmed clinical and molecular diagnosis of DMD
2. 6- 18 years of age
3. Ability to follow 2 step instructions
4. Ability to transfer to and from the wheelchair-mat with moderate assistance defined as no greater than 75% assistance.
5. Signed informed consent of parental or legal guardian(s) is required for participants. Assent from children 7-18 years old may also required.

Exclusion Criteria:

Participants must confirm:

1. No Surgical procedures were performed ≤ 8 weeks before study procedures.
2. No musculoskeletal injuries were experienced ≤ 8 weeks before study procedures.
3. Investigator assessment that patient or parent/legal guardian are not willing or able to comply with study procedures.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Crossover
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Compare the inter and intra rater reliability of HHM and CQMS by measuring Elbow and Knee Flexor/Extensor Strength in children ages 6-18 diagnosed with DMD tested by experienced clinical evaluators in both HHM and CQMS.	Muscle groups will be tested in a standardized order 1. Knee extension 2. Knee flexion 3. Elbow Flexion 4. Elbow extension with all tests sequencing following a right to left pattern. This will reduce assessment bias and the impact of muscle fatigue per muscle group. Study participants are randomized to two different sequences of four assessments, one sequence performed on one testing day (Visit 1) and another on a different testing day (Visit 2).	two-day visit

Collaborators and Investigators

• Sponsor: Cooperative International Neuromuscular Research Group
• Collaborators: Washington University School of Medicine; Children's National Research Institute; Sydney Children's Hospitals Network; Royal Children's Hospital; Carolinas Medical Center; Muscular Dystrophy Association; Centro Clinico Nemo
• Investigators: Principal Investigator: Tina T Duong, MPT, Children's National Research Institute

Publications and helpful links

Helpful Links

• CINRG website where all current and completed protocols are listed.

Study record dates

Study Major Dates

• Study Start: January 1, 2010
• Primary Completion (Actual): June 1, 2010
• Study Completion (Actual): August 1, 2010

Study Registration Dates

• First Submitted: May 17, 2010
• First Submitted That Met QC Criteria: May 17, 2010
• First Posted (Estimate): May 18, 2010

Study Record Updates

• Last Update Posted (Estimate): January 11, 2013
• Last Update Submitted That Met QC Criteria: January 10, 2013
• Last Verified: January 1, 2013

More Information

Terms related to this study

• Keywords: DMD; Duchenne; Muscular Dystrophy; Neuromuscular disease; Strength assessment; CINRG Quantitative Measurement System; CQMS; Hand Held Myometry; HHM
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: CNMC0609