DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls

NCT01523964 | Completed Results

• 1 other identifier: CMD-001
• Study Type: interventional
• Target: 92
• Locations: 1 country
• Sites: 6

Brief Summary

Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance Myography (EIM) has the potential to serve as a non-invasive, quantifiable, diagnostic tool for neuromuscular disease. If successful, these devices will allow for improved ability to diagnose neuromuscular disease and to assess disease progression or remission, allowing for better care of individual patients as well as for use in clinical trials, where improved outcome measures for neuromuscular diseases is being sought.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; EIM; Duchenne Muscular Dystrophy; electrical impedance myography

Outcome Measures

Primary Outcomes (1)

• Number of Subjects With an Adverse Event.

  Adverse events will be assessed during the time the subject is enrolled in the trial.

  1 day

Study Arms (4)

DMD subject ages 3-7 inclusive

OTHER

Young DMD Testing with EIM

Other: Testing with EIM

DMD subject ages 8-12 inclusive

OTHER

Older DMD Testing with EIM

Other: Testing with EIM

Healthy Control ages 3-7 inclusive

OTHER

Young Healthy Testing with EIM

Other: Testing with EIM

Healthy Control ages 8-12 inclusive

OTHER

Older Healthy Testing with EIM

Other: Testing with EIM

Interventions

Testing with EIM OTHER

DMD subject ages 3-7 inclusive; DMD subject ages 8-12 inclusive; Healthy Control ages 3-7 inclusive; Healthy Control ages 8-12 inclusive

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• DMD Subject Cohorts
• Male subjects
• Subjects with a chronologic age of 3 to 7 years inclusive for Cohort A, and 8 to 12 years inclusive for Cohort B
• Subjects with DMD diagnosed with mutational testing and/or absence of dystrophin on muscle biopsy
• Subjects with proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
• Subjects who can walk 10 meters unassisted (ie, without braces, canes, or other aids)
• Subjects who are taking systemic corticosteroids and/or any other medication which, in the judgment of the investigator, could impact muscle strength or physical activity levels, must be on a stable dose for at least 4 weeks prior to initiation of study measurements
• Subjects who provide assent, as stipulated by IRB requirements, and whose parent/guardian signs an informed consent form
• Subjects who are willing and able to cooperate and comply with all protocol requirements and procedures
• Healthy Control Cohort
• Healthy males with normal neuromuscular examination
• Subjects with a chronologic age of 3 to 7 years inclusive for Cohort C, and 8 to 12 years inclusive for Cohort D
• Subjects who provide assent, as stipulated by IRB requirements, and whose parent/guardian signs an informed consent form
• Subjects who are willing and able to cooperate and comply with all protocol requirements and procedures

You may not qualify if:

• Subjects with daytime ventilatory dependence (non-invasive or tracheostomy)
• Subjects enrolled in a DMD therapeutic clinical trial concomitantly or within the past 4 weeks
• Subjects with any physical or mental condition which may, in the investigator's opinion, render the subject unable to complete the tasks of the study appropriately

Sponsors & Collaborators

• Dart Therapeutics. LLC: lead

Study Sites (6)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Massauchusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

The University of Texas Southwestern Medical Center-Dallas

Dallas, Texas, 75220, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, 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

Results Point of Contact

• Title: Chief Operating Officer
• Organization: DART Therapeutics

egoldstein@dartrx.com

415-341-5783

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 30, 2012
• First Posted: February 1, 2012
• Study Start: February 1, 2012
• Primary Completion: August 1, 2012
• Study Completion: August 1, 2012
• Last Updated: December 9, 2013
• Results First Posted: December 9, 2013

Record last verified: 2013-10

Locations

US (6)