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Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )
ClinicalTrials.gov Identifier:
NCT01451281
First received: October 8, 2011
Last updated: November 11, 2014
Last verified: May 2014
  Purpose

Background:

- Duchenne muscular dystrophy (DMD) is a disease in which the muscles are unable to make the protein dystrophin. Without this protein, the muscles become gradually weaker. A new medicine called GSK2402968 is being tested to see if it can help prevent or slow down this loss of muscle strength. In this study, boys with DMD and healthy volunteers will have different types of imaging studies to see which ones provide the best images of the muscles. This information will help researchers use these imaging techniques to test the safety and effectiveness of GSK2402968 and other agents.

Objectives:

- To test magnetic resonance imaging and ultrasound techniques that can detect changes in muscles of boys with DMD.

Eligibility:

  • Boys who have DMD and are in the GSK2402968 drug test study.
  • Healthy boys of the same age as the above study participants.

Design:

  • Participants will be screened with a medical history and physical exam.
  • Healthy volunteers will have one 2-hour visit with three tests. Magnetic resonance imaging (MRI) scans of the skeletal muscles and heart and diaphragm muscles will be carried out. Muscle ultrasound imaging of leg and arm muscles will also be done. Participants should not perform heavy physical activity like school sports or long walks during the week before the visit.
  • Participants in the GSK2402968 study will have the same series of tests as the healthy volunteers. The tests will be given during the study screening phase. They will be repeated after 3 months and 6 months of receiving the study agent (GSK2402968 or placebo) and at 6 months after stopping the GSK study.

Condition
Muscular Dystrophy
Muscular Disease

Study Type: Observational
Official Title: Evaluation of Skeletal Muscle, Cardiac, and Diaphragm Imaging Biomarkers for GSK2402968 Effects in Ambulatory Boys With Duchenne Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary Outcome Measures:
  • Changes in muscle fat content quantified by T1w GRE Dixon imaging method in skeletal muscles in the lower extremities at 24 weeks from baseline in the parent study in ambulatory boys with DMD receiving GSK2402968 or placebo

Secondary Outcome Measures:
  • Changes in muscle edema by T2w imaging and muscle fat/water content by IDEAL-CPMG method; myocardial fat/edema and cardiac function by MRI methods in DMD boys receiving GSK2402968 or placebo as well as DMD boys at baseline versus healthy volunte...
  • Changes in water diffusivity by MRI; muscle volume, fat, and fibrosis by ultrasound; and diaphragm function by dynamic breathing MRI methods in DMD boys receiving GSK2402968 or placebo as well as DMD boys at baseline versus healthy volunteers.

Enrollment: 35
Study Start Date: September 2011
  Show Detailed Description

  Eligibility

Ages Eligible for Study:   5 Years to 17 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   Yes
Criteria
  • INCLUSION CRITERIA:

DMD Subjects

  • Eligible for the parent study
  • Willing and able to comply with all protocol requirements and procedures, including MRI without sedation
  • Able to give informed assent and parent(s)/legal guardian to give informed consent in writing signed by the subject and/or parent(s)/legal guardian

Healthy Volunteers

  • Must be unaffected by a neuromuscular condition
  • Willing and able to comply with all protocol requirements and procedures, including MRI without sedation.
  • Able to give informed assent and parent(s)/legal guardian to give informed consent in writing signed by the subject and/or parent(s)/legal guardian.

EXCLUSION CRITERIA:

DMD Subjects and Healthy Volunteers

  • Having metal objects in his body that are not MRI-safe. These include the following objects: 1) pacemakers or other implanted electrical devices; 2) brain stimulators; 3) some types of dental implants; 4) aneurysm clips (metal clips on the wall of a large artery); 5) metallic prostheses (including metal pins and rods, heart valves, and cochlear implants; 6) implanted delivery pump; 7) permanent eye liner; or 8) shrapnel fragments.
  • Having a fear of closed spaces
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01451281

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
Investigators
Principal Investigator: Kenneth H Fischbeck, M.D. National Institute of Neurological Disorders and Stroke (NINDS)
  More Information

Additional Information:
Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )
ClinicalTrials.gov Identifier: NCT01451281     History of Changes
Other Study ID Numbers: 110261, 11-N-0261
Study First Received: October 8, 2011
Last Updated: November 11, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Ultrasonography
Muscular Dystrophy
Neuromuscular Disease
Oligonucleotide
Magnetic Resonance Imaging (MRI)
Duchenne Muscular Dystrophy
DND

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

ClinicalTrials.gov processed this record on November 19, 2014