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This study evaluates the drug GSK2402968 (Drisapersen) in people with Duchenne muscular dystrophy caused by a mutation/deletion in the DMD gene; the drug works via exon 51 skipping. It included ambulant boys over 5 whose mutation is correctable by exon 51 skipping. It was a Phase 3 study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
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Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation/deletion within the DMD gene, confirmed by a state-of-the-art 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 GSK2402968-induced DMD exon 51 skipping. * Males, aged at least 5 years, and with life expectancy of at least 1 year * Able to complete 6MWD test with minimal distance of at least 75m at each predrug visit. In addition, results of 6MWD must be within 20% of each other at each pre-drug visit * Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study * QTc \<450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period), or \<480 msec for subjects with Bundle Branch Block. Note: QTc may be either QTcB or QTcF, and machine read or manual overread. * Subjects, where appropriate, must be willing to use adequate contraception (condoms or abstinence) for the duration of the study and for at least 5 months after the last dose of study drug. * Willing and able to comply with all protocol requirements and procedures, * Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations). * French subjects: 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. Exclusion Criteria: * Any additional missing exon for DMD that cannot be treated with GSK2402968 * Current or history of liver or renal disease or impairment * Acute illness within 4 weeks of the first anticipated administration of study medication which may interfere with study assessments * 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 the first administration of study medication. * Current or anticipated participation in any investigational clinical studies * Positive hepatitis B surface antigen, hepatitis C antibody test (if verified via RIBA or PCA testing), or human immunodeficiency virus (HIV) test at screening, * Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction \<45% at Screening, the investigator should discuss inclusion of subject in the study with the medical monitor, * Children in Care. The definition of a Child in Care is a child who has been placed under the control or protection of an agency, organisation, 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.