Muscular Dystrophy Clinical Trial
Official title:
Evaluation of Skeletal Muscle, Cardiac, and Diaphragm Imaging Biomarkers for GSK2402968 Effects in Ambulatory Boys With Duchenne Muscular Dystrophy
Verified date | May 20, 2019 |
Source | National Institutes of Health Clinical Center (CC) |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Completed |
Enrollment | 35 |
Est. completion date | May 20, 2019 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 5 Years to 17 Years |
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 |
Country | Name | City | State |
---|---|---|---|
United States | National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Institute of Neurological Disorders and Stroke (NINDS) |
United States,
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. Review. — View Citation
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. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. Erratum in: N Engl J Med. 2011 Oct 6;365(14):1361. — View Citation
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. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | 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... | |||
Secondary | 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. |
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