Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy

NCT01491555 | Completed

• 1 other identifier: IRB-P00001218
• Study Type: observational
• Target: 73
• Locations: 1 country
• Sites: 1

Brief Summary

Researchers at Children's Hospital Boston Neurology Department invite children to participate in a new research study. Researchers are looking for boys ages 2 - 30 with Duchenne Muscular Dystrophy (DMD) and healthy boys ages 2 - 30 (without any nerve or muscle concerns) to serve as controls. The study is evaluating a new technique that will test nerve and muscle function. The testing is all pain free. Children participating in the study will come in for 10 visits over two years. Visits will take place every month at first, then less often for the remaining visits. The tests for the study itself take approximately 2hours. If participants are interested or would like to learn more about the study, please call Lavanya Madabusi at 617-919-3554 or Lavanya.Madabusi@childrens.harvard.edu. All inquiries will be kept strictly confidential.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; DMD; controls; EIM; QUS

Outcome Measures

Primary Outcomes (1)

• The rate of decline of DMD patients versus normal subjects as assessed by EIM and quantitative ultrasound

  With the successful completion of this aim, the investigators will establish that alterations in both EIM and QUS provide meaningful surrogate measures of disease progression in DMD.

  up to 45 months

Secondary Outcomes (1)

• The rate of decline of DMD patients versus normal subjects as assessed by handheld dynamometry, 6-minute walk, and other functional tests.

  up to 45 months

Study Arms (2)

DMD patients

35 boys ages 2 through 30 with DMD

Control Group

35 healthy boys ages 2 through 30

Eligibility Criteria

• Age: 2 Years - 30 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

This study will involve boys with DMD and healthy male controls.

You may qualify if:

• Genetically or histologically established diagnosis of DMD
• Male, age 2 - 30
• \. Male, age 2 - 30

You may not qualify if:

• Presence of implanted pacemaker or other electrical device
• Presence of a superimposed neuromuscular or other medical condition that substantially impacts the individual's health
• Presence or past history of a neuromuscular disorder or other disease that substantially impacts health
• Presence of implanted pacemaker or other electrical device.

Sponsors & Collaborators

• Boston Children's Hospital: lead
• Beth Israel Deaconess Medical Center: collaborator

Study Sites (1)

Children's Hospital Boston

Boston, Massachusetts, 02115, 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

• Basil Darras, MD

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

• Seward Rutkove, MD

  Beth Israel Deaconess Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Basil Darras, M.D.

Study Record Dates

• First Submitted: December 5, 2011
• First Posted: December 14, 2011
• Study Start: April 1, 2012
• Primary Completion: September 1, 2015
• Study Completion: September 1, 2015
• Last Updated: March 24, 2016

Record last verified: 2016-03

Locations

US (1)