NCT01451281

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

Completed Last updated 22 May 2019

What this trial tests

trial in Muscular Dystrophy in 35 participants. Completed in 20 May 2019.

Timeline

15 September 2011

20 May 2019

Quick facts

Lead sponsor	National Institute of Neurological Disorders and Stroke (NINDS)
Status	Completed
Study type	OBSERVATIONAL
Enrollment	35
Start date	15 September 2011
Estimated completion	20 May 2019
Sites	1 location across United States

Conditions studied

• Muscular Dystrophy — all drugs for Muscular Dystrophy →
• Muscular Disease — all drugs for Muscular Disease →

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Who can join

Adults 5 to 17, male only, with Muscular Dystrophy or Muscular Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Quantifying disease activity in fatty-infiltrated skeletal muscle by IDEAL-CPMG in Duchenne muscular dystrophy.
   Mankodi A, Bishop CA, Auh S, Newbould RD, et al · · 2016 · cited 44× · PMID 27593185 · DOI 10.1016/j.nmd.2016.07.013
2. Skeletal muscle water T<sub>2</sub> as a biomarker of disease status and exercise effects in patients with Duchenne muscular dystrophy.
   Mankodi A, Azzabou N, Bulea T, Reyngoudt H, et al · · 2017 · cited 27× · PMID 28601553 · DOI 10.1016/j.nmd.2017.04.008
3. Respiratory magnetic resonance imaging biomarkers in Duchenne muscular dystrophy.
   Mankodi A, Kovacs W, Norato G, Hsieh N, et al · · 2017 · cited 21× · PMID 28904987 · DOI 10.1002/acn3.440
4. Plasma lipidomic analysis shows a disease progression signature in mdx mice.
   Tsonaka R, Seyer A, Aartsma-Rus A, Spitali P. · · 2021 · cited 15× · PMID 34155298 · DOI 10.1038/s41598-021-92406-6
5. Upper arm and cardiac magnetic resonance imaging in Duchenne muscular dystrophy.
   Gaur L, Hanna A, Bandettini WP, Fischbeck KH, et al · · 2016 · cited 14× · PMID 28097207 · DOI 10.1002/acn3.367
6. Precurved, Fiber-Reinforced Actuators Enable Pneumatically Efficient Replication of Complex Biological Motions.
   Hu L, Gau D, Nixon J, Klein M, et al · · 2022 · cited 1× · PMID 34000210 · DOI 10.1089/soro.2020.0087