Clinical trial NCT01125709: Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)
Official title: Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Duchenne Muscular Dystrophy
Keywords: Neuromuscular disease
Duchenne
Muscular Dystrophy
DMD
Strength assessment
CINRG Quantitative Measurement System
CQMS
Hand Held Myometry
HHM
Lead sponsor: Cooperative International Neuromuscular Research Group
Sponsor class: NETWORK
Collaborators: name: Muscular Dystrophy Association
name: Children's National Research Institute
name: Washington University School of Medicine
name: Royal Children's Hospital
name: Sydney Children's Hospitals Network
name: Fondazione Serena Onlus - Centro Clinico NeMO Milano
name: Carolinas Medical Center
First posted: 2010-05-18
Enrollment: 30
Sex: MALE
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_CROSSOVER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants will be selected from participating CINRG institution's neuromuscular clinics.

Brief 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).

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 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.

Primary outcomes:
measure: 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.
timeFrame: two-day visit
Secondary outcomes:
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: St. Louis
country: United States
facility: Washington University - St. Louis
state: Missouri
city: Charlotte
country: United States
facility: Carolinas Medical Center
state: North Carolina
city: Parkville
country: Australia
facility: Royal Children's Hospital
state: Victoria
city: Milano
country: Italy
facility: Centro Clinico Nemo
Study officials:
affiliation: Children's National Research Institute
name: Tina T Duong, MPT
role: PRINCIPAL_INVESTIGATOR
References: