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

This study has been completed.
Sponsor:
Collaborators:
Muscular Dystrophy Association
Children's Research Institute
Washington University School of Medicine
Royal Children's Hospital
Children's Hospital at Westmead
Centro Clinico Nemo
Carolinas Medical Center
Information provided by:
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT01125709
First received: May 17, 2010
Last updated: January 10, 2013
Last verified: January 2013
  Purpose

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).


Condition
Duchenne Muscular Dystrophy

Study Type: Observational
Study Design: Observational Model: Case-Crossover
Time Perspective: Prospective
Official Title: Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS

Resource links provided by NLM:


Further study details as provided by Cooperative International Neuromuscular Research Group:

Primary Outcome Measures:
  • 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. [ Time Frame: two-day visit ] [ Designated as safety issue: No ]
    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).


Estimated Enrollment: 30
Study Start Date: January 2010
Study Completion Date: August 2010
Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   6 Years to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

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

Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01125709

Locations
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Missouri
Washington University - St. Louis
St. Louis, Missouri, United States, 63110
United States, North Carolina
Carolinas Medical Center
Charlotte, North Carolina, United States, 28207
Australia, Victoria
Royal Children's Hospital
Parkville, Victoria, Australia, 3052
Italy
Centro Clinico Nemo
Milano, Italy, 20162
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
Muscular Dystrophy Association
Children's Research Institute
Washington University School of Medicine
Royal Children's Hospital
Children's Hospital at Westmead
Centro Clinico Nemo
Carolinas Medical Center
Investigators
Principal Investigator: Tina T Duong, MPT Children's Research Institute
  More Information

Additional Information:
No publications provided

Responsible Party: Tina Duong, MPT, Children's National Medical Center, Children's Research Institute
ClinicalTrials.gov Identifier: NCT01125709     History of Changes
Other Study ID Numbers: CNMC0609
Study First Received: May 17, 2010
Last Updated: January 10, 2013
Health Authority: United States: Institutional Review Board
Australia: Human Research Ethics Committee
Italy: Ethics Committee

Keywords provided by Cooperative International Neuromuscular Research Group:
Neuromuscular disease
Duchenne
Muscular Dystrophy
DMD
Strength assessment
CINRG Quantitative Measurement System
CQMS
Hand Held Myometry
HHM

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 20, 2014