A clinical study to investigate the pharmacokinetics safety, and tolerability of a single dose of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy (GSK Study Number DMD114118)
- Conditions
- Duchenne Muscular DystrophyMedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2010-024566-22-Outside-EU/EEA
- Lead Sponsor
- GlaxoSmithKline Research and Development LTD
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active
- Sex
- Male
- Target Recruitment
- 32
Subjects eligible for enrolment in the study must meet all of the following criteria:
1. Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe
Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single
Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting
Curve Analysis), and correctable by treatment with GSK2402968.
2. Age 9 years old or greater at Screening;
3. Male;
4. Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
5. Life expectancy at least three years;
6. Willingness and ability to comply with all protocol requirements and procedures;
7. QTc <450msec (based on single or average QTc value of triplicate ECGs obtained
over a brief recording period). Note: QTc may be either QTcB or QTcF, machine
read or manual overread;
8. Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
9. In France, a subject will be eligible for inclusion in this study only if either affiliated
to or a beneficiary of a social security category.
Are the trial subjects under 18? yes
Number of subjects for this age range: 32
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Subjects meeting any of the following criteria must not be enrolled in the study:
1. Any additional mutation (such as an additional missing exon for DMD) that cannot
be treated with GSK2402968;
2. Current or history of liver or renal disease;
3. Acute illness within 4 weeks of anticipated administration of study medication,
which may interfere with study assessments;
4. Daytime ventilator-dependency (except for daytime naps);
5. Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, within 6 months of the first administration of study
medication; and idebenone or other forms of Coenzyme Q10 within 1 month of study
medication.
6. Start of glucocorticosteroids within 6 months or non-stable use of
glucocorticosteroids within 3 months of the anticipated first administration of study
medication;
7. Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or
human immunodeficiency virus (HIV) test at Screening;
8. Symptomatic cardiomyopathy;
9. Use of alcohol from Screening through to the 1 month Follow-up visit (Day 29-35,
inclusive);
10. Any Child in Care. (The definition of a Child in Care is a child who has been placed
under the control or protection of an agency, organization, institution or entity by the
courts, the government or a government body, acting in accordance with powers
conferred on them by law or regulation. The definition of a child in care can include
a child cared for by foster parents or living in a care home or institution, provided
that the arrangement falls within the definition above. The definition of a child in
care does not include a child who is adopted or has an appointed legal guardian).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method