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Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT01506518
Recruitment Status : Recruiting
First Posted : January 10, 2012
Last Update Posted : January 18, 2018
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
University of North Carolina, Chapel Hill

Study Description
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Brief Summary:
This is a pilot clinical trial to assess the ability of a new ultrasound-based imaging method, Double-Push Acoustic Radiation Force (DP ARF) ultrasound, to monitor the progression of Duchenne muscular dystrophy. The hypothesis being tested is that DP ARF ultrasound delineates changes in muscle composition and function in individual dystrophic muscles, from early through late stages of disease development, that correlate to time to loss of ambulation in patient volunteers.

Condition or disease
Muscular Dystrophy, Duchenne

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Study Design
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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Muscle Degeneration in Duchenne Muscular Dystrophy
Study Start Date : January 2012
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : September 2019

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Groups and Cohorts
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Group/Cohort
Duchenne muscular dystrophy and age 5-6 years
Boys diagnosed with Duchenne muscular dystrophy and age 5-6 years at enrollment.
Duchenne muscular dystrophy and age 7-8 years
Boys diagnosed with Duchenne muscular dystrophy and age 7-8 years at enrollment.
Duchenne muscular dystrophy and age 9-10 years
Boys diagnosed with Duchenne muscular dystrophy and age 9-10 years at enrollment.
No known neuromuscular disorders and age 5-14 years
Volunteer boys ages 5-14 years with no known neuromuscular disorders to serve as controls.


Outcome Measures
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Primary Outcome Measures :
  1. Change in DP ARF marginal peak displacement [ Time Frame: once every 4 months for 4 years for 12 total measures ]
    Marginal peak displacement (MPD) is a metric developed to qualitatively describe the degree of nonlinearity in the viscoelastic properties of tissue: MPD = (P2-D)/P1, where P1 and P2 are the first and second peak displacement achieved in tissue by the first and second ARF excitations, respectively, and d is the ARF-induced displacement remaining at the time of the second push.


Secondary Outcome Measures :
  1. Rate of change in DP ARF marginal peak displacement [ Time Frame: 4 months to 4 years ]
    rates of change in marginal peak displacement will be measured from time-point to time-point (every 4 months) and across multiple time points (spanning 8 months to 4 years).

  2. Change in quantitative muscle testing score of maximum voluntary isometric contraction (MVIC) [ Time Frame: every 4 months for 4 years ]
    standard quantitative muscle testing of maximum voluntary isometric contraction (MVIC) in the rectus femoris, cranial sartorius, gastrocnemius, and lateral deltoid muscles of the right limbs.

  3. Change in time to rise from supine position to standing [ Time Frame: every 4 months for 4 years ]
    standard time to standing timed function test

  4. Change in distance walked in six minutes [ Time Frame: every 4 months for 4 years ]
    standard six-minute walk timed function test

  5. Change in time to walk 30 feet [ Time Frame: every 4 months for 4 years ]
    standard 30-feet walk timed function test

  6. Rate of change in maximum voluntary isometric contraction (MVIC) [ Time Frame: 4 months to 4 years ]
    rate of change in maximum voluntary isometric contraction (MVIC) will be assessed from time-point to time-point (4 month separation between measures) and across time-points (8 months to 4 years time separation between measures).

  7. Rate of change in time to rise from supine to standing position [ Time Frame: 4 months to 4 years ]
    rate of change in time to standing timed function test score will be assessed from time-point to time-point (4 month separation between measures) and across time-points (8 months to 4 years time separation between measures).

  8. Rate of change in distance walked in six minutes [ Time Frame: 4 months to 4 years ]
    rate of change in six-minute walk timed function test score will be assessed from time-point to time-point (4 month separation between measures) and across time-points (8 months to 4 years time separation between measures).

  9. Rate of change in time to walk 30 feet [ Time Frame: 4 months to 4 years ]
    rate of change in 30-feet walk timed function test score will be assessed from time-point to time-point (4 month separation between measures) and across time-points (8 months to 4 years time separation between measures).

  10. Age at loss of ambulation [ Time Frame: 4 years ]
    Loss of ambulation will be diagnosed by the patient volunteer's physician. The patient volunteer's age at the time loss of ambulation is first diagnosed will be recorded.

  11. Change in percent degenerative muscle composition [ Time Frame: every 4 months for 4 years ]
    Muscle boundaries will be hand-delineated using matched B-Mode image guidance in DP ARF marginal peak displacement parametric images. Within each 2D muscle image, percent degenerative area (Ad) will be calculated as T/N, where N is the total muscle area (number of pixels x area/pixel) and T is the muscle area in which marginal peak displacement values are within thresholds for necrosis, fat or fibrous tissue identification.

  12. Change in percent necrotic tissue area [ Time Frame: every 4 months for 4 years ]
    Muscle boundaries will be hand-delineated using matched B-Mode image guidance in DP ARF marginal peak displacement parametric images. Within each 2D muscle image, percent necrotic area (An) will be calculated as n/N, where N is the total muscle area (number of pixels x area/pixel) and n is the muscle area in which marginal peak displacement values are within thresholds for necrosis.

  13. Change in percent fat tissue area [ Time Frame: every 4 months for 4 years ]
    Muscle boundaries will be hand-delineated using matched B-Mode image guidance in DP ARF marginal peak displacement parametric images. Within each 2D muscle image, percent fatty deposition area (Af) will be calculated as f/N, where N is the total muscle area (number of pixels x area/pixel) and f is the muscle area in which marginal peak displacement values are within thresholds for fat tissue identification.

  14. Change in percent fibrotic tissue area [ Time Frame: every 4 months for 4 years ]
    Muscle boundaries will be hand-delineated using matched B-Mode image guidance in DP ARF marginal peak displacement parametric images. Within each 2D muscle image, percent degenerative area (Ac) will be calculated as c/N, where N is the total muscle area (number of pixels x area/pixel) and c is the muscle area in which marginal peak displacement values are within thresholds for fibrous tissue identification.


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 13 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
The study population will include patient volunteers with Duchenne muscular dystrophy (DMD). Thirty volunteers (boys with DMD, enrolling at ages 5 (n=10), 7 (n=10), or 9 (n=10)) having clinical diagnosis of DMD with clinical onset by age 5 will be considered for enrollment.
Criteria

Inclusion Criteria:

  • Clinical diagnosis of Duchenne muscular dystrophy with clinical onset by age 5
  • Ability to stand, alone or with assistance, at time of enrollment
  • Ability to communicate with pertinent staff
  • Ability to understand and comply with study requirements
  • Ability to give informed consent.

Exclusion Criteria:

  • Confirmed diagnosis of other muscle disease
  • Previous compartment syndrome
  • Previous injury to selected limbs
  • Previous vascular surgery to selected limbs
  • History of a compressive neuropathy (e.g., sciatic, femoral or tibial palsy in leg)
  • History of rhabdomyolysis
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01506518


Contacts
Contact: Manisha Chopra, BS 919-843-7857 chopram@neurology.unc.edu
Contact: Caterina Gallippi, Ph.D. 919-843-6647 cmgallip@email.unc.edu

Locations
United States, North Carolina
The University of North Carolina at Chapel Hill Hospitals Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: James Howard, MD    919-966-5522    howardj@neurology.unc.edu   
Contact: Manisha Chopra, BS    919-843-7857    chopram@neurology.unc.edu   
Sponsors and Collaborators
University of North Carolina, Chapel Hill
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
Principal Investigator: Caterina M Gallippi, Ph.D. University of North Carolina, Chapel Hill
More Information
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Responsible Party: University of North Carolina, Chapel Hill
ClinicalTrials.gov Identifier: NCT01506518     History of Changes
Other Study ID Numbers: 11-1509
1R01NS074057 ( U.S. NIH Grant/Contract )
First Posted: January 10, 2012    Key Record Dates
Last Update Posted: January 18, 2018
Last Verified: January 2018

Keywords provided by University of North Carolina, Chapel Hill:
Muscular Dystrophy, Duchenne
Ultrasound
Imaging
Acoustic Radiation Force
Double Push

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