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

Purpose

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
Muscular Dystrophy, Duchenne

Study Type:	Observational
Study Design:	Observational Model: Cohort Time Perspective: Prospective
Official Title:	Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Muscle Degeneration in Duchenne Muscular Dystrophy

Resource links provided by NLM:

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy

MedlinePlus related topics: Muscular Dystrophy

U.S. FDA Resources

Further study details as provided by University of North Carolina, Chapel Hill:

Primary Outcome Measures:

Change in DP ARF marginal peak displacement [ Time Frame: once every 4 months for 4 years for 12 total measures ] [ Designated as safety issue: No ]
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:

Rate of change in DP ARF marginal peak displacement [ Time Frame: 4 months to 4 years ] [ Designated as safety issue: No ]
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).
Change in quantitative muscle testing score of maximum voluntary isometric contraction (MVIC) [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
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.
Change in time to rise from supine position to standing [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
standard time to standing timed function test
Change in distance walked in six minutes [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
standard six-minute walk timed function test
Change in time to walk 30 feet [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
standard 30-feet walk timed function test
Rate of change in maximum voluntary isometric contraction (MVIC) [ Time Frame: 4 months to 4 years ] [ Designated as safety issue: No ]
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).
Rate of change in time to rise from supine to standing position [ Time Frame: 4 months to 4 years ] [ Designated as safety issue: No ]
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).
Rate of change in distance walked in six minutes [ Time Frame: 4 months to 4 years ] [ Designated as safety issue: No ]
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).
Rate of change in time to walk 30 feet [ Time Frame: 4 months to 4 years ] [ Designated as safety issue: No ]
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).
Age at loss of ambulation [ Time Frame: 4 years ] [ Designated as safety issue: No ]
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.
Change in percent degenerative muscle composition [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
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.
Change in percent necrotic tissue area [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
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.
Change in percent fat tissue area [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
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.
Change in percent fibrotic tissue area [ Time Frame: every 4 months for 4 years ] [ Designated as safety issue: No ]
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.

Estimated Enrollment:	30
Study Start Date:	January 2012
Estimated Study Completion Date:	September 2016
Estimated Primary Completion Date:	September 2016 (Final data collection date for primary outcome measure)

Groups/Cohorts
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.

Show Detailed Description

Eligibility

Ages Eligible for Study:	5 Years to 13 Years
Genders 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

Please refer to this study by its ClinicalTrials.gov identifier: NCT01506518

Contacts

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

No publications provided

Responsible Party:	University of North Carolina, Chapel Hill
ClinicalTrials.gov Identifier:	NCT01506518 History of Changes
Other Study ID Numbers:	11-1509, 1R01NS074057
Study First Received:	December 22, 2011
Last Updated:	April 2, 2013
Health Authority:	United States: Institutional Review Board

Keywords provided by University of North Carolina, Chapel Hill:

Muscular Dystrophy, Duchenne
Ultrasound
Imaging
Acoustic Radiation Force
Double Push

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 May 21, 2013