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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01125709
• Other study ID #: CNMC0609
• Status: Completed
• Phase: N/A
• First received: May 17, 2010
• Last updated: January 10, 2013
• Start date: January 2010
• Est. completion date: August 2010

Study information

• Verified date: January 2013
• Source: Cooperative International Neuromuscular Research Group
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review BoardAustralia: Human Research Ethics CommitteeItaly: Ethics Committee
• Study type: Observational

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: August 2010
• Est. primary completion date: June 2010
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 18 Years

Eligibility

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 Design

Observational Model: Case-Crossover, Time Perspective: Prospective

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
Australia	Royal Children's Hospital	Parkville	Victoria
Italy	Centro Clinico Nemo	Milano
United States	Carolinas Medical Center	Charlotte	North Carolina
United States	Washington University - St. Louis	St. Louis	Missouri
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (8)

Lead Sponsor	Collaborator
Cooperative International Neuromuscular Research Group	Carolinas Medical Center, Centro Clinico Nemo, Children's Hospital at Westmead, Children's Research Institute, Muscular Dystrophy Association, Royal Children's Hospital, Washington University School of Medicine

Countries where clinical trial is conducted

United States,  Australia,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No

External Links

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