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

NCT01506518 | Completed

• 2 other identifiers: 11-15091R01NS074057
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

Muscular Dystrophy, Duchenne

Keywords

Muscular Dystrophy, Duchenne; Ultrasound; Imaging; Acoustic Radiation Force; Double Push

Outcome Measures

Primary Outcomes (1)

• Change in DP ARF marginal peak displacement

  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.

  once every 4 months for 4 years for 12 total measures

Secondary Outcomes (14)

• Rate of change in DP ARF marginal peak displacement

  4 months to 4 years

• Change in quantitative muscle testing score of maximum voluntary isometric contraction (MVIC)

  every 4 months for 4 years

• Change in time to rise from supine position to standing

  every 4 months for 4 years

• Change in distance walked in six minutes

  every 4 months for 4 years

• Change in time to walk 30 feet

  every 4 months for 4 years

Study Arms (4)

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.

Eligibility Criteria

• Age: 5 Years - 13 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (1)

The University of North Carolina at Chapel Hill Hospitals

Chapel Hill, North Carolina, 27599, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Caterina M Gallippi, Ph.D.

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2011
• First Posted: January 10, 2012
• Study Start: January 1, 2012
• Primary Completion: May 31, 2020
• Study Completion: May 31, 2020
• Last Updated: August 2, 2021

Record last verified: 2021-05

Locations

US (1)