Protocol Details

Evaluation of Skeletal Muscle, Cardiac, and Diaphragm Imaging Biomarkers for GSK2402968 Effects in Ambulatory Boys with Duchenne Muscular Dystrophy

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number	11-N-0261
Sponsoring Institute	National Institute of Neurological Disorders and Stroke (NINDS)
Recruitment Detail	Type: Participants currently recruited/enrolled Gender: Male Min Age: 5 Max Age: 17
Referral Letter Required	Yes
Population Exclusion(s)	Female
Special Instructions	Currently Not Provided
Keywords	Ultrasonography; Muscular Dystrophy; Neuromuscular Disease; Oligonucleotide; Magnetic Resonance Imaging (MRI)
Recruitment Keyword(s)	None; Duchenne Muscular Dystrophy; DND; Muscular Dystrophy
Condition(s)	Muscular Dystrophy; Muscular Disease
Investigational Drug(s)	None
Investigational Device(s)	None
Intervention(s)	None
Supporting Site	National Institute of Neurological Disorders and Stroke

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.

--Back to Top--

Eligibility

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

--Back to Top--

Citations:

Aartsma-Rus A, Van Deutekom JC, Fokkema IF, Van Ommen GJ, Den Dunnen JT. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve. 2006 Aug;34(2):135-44.

Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med. 2011 Apr 21;364(16):1513-22.

Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 Dec 24;51(6):919-28.

--Back to Top--

Contacts:

Principal Investigator	Referral Contact	For more information:
Kenneth H. Fischbeck, M.D. National Institute of Neurological Disorders and Stroke (NINDS) National Institutes of Health 35 CONVENT DR BG 35 RM 2A1000 MSC 3705 BETHESDA MD 20892-3705 (301) 435-9318 kf@ninds.nih.gov	Donovan P. Stock National Institute of Neurological Disorders and Stroke (NINDS) National Institutes of Health Building 10 Room 5S219 10 Center Drive Bethesda, Maryland 20892 (301) 594-3953 stockd@mail.nih.gov	Patient Recruitment and Public Liaison Office Building 61 10 Cloister Court Bethesda, Maryland 20892-4754 Toll Free: 1-800-411-1222 TTY: 301-594-9774 (local),1-866-411-1010 (toll free) Fax: 301-480-9793 prpl@mail.cc.nih.gov

Clinical Trials Number:

NCT01451281

--Back to Top--

QUESTIONS?

Contact the Patient Recruitment and Public Liaison Office for:

• Details on how to participate in a study
• Details on how to refer a patient to a study

NIH Clinical Studies Information Request

---

Contact the Office of Communications for:

• General information about the NIH Clinical Center

www.cc.nih.gov/contact.shtml

---

Contact the Department Clinical Research Informatics, (DCRI) for:

• Technical questions about Adobe Acrobat and the PDF format
• Technical questions about this web server

webmaster@cc.nih.gov