EUCTR2014-005296-81-BE进行中（未招募）1 期
A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Muscular Dystrophy - Drisapersen confirmatory study
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 104
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. Ambulant subjects with DMD resulting from a mutation/deletion within the dystrophin gene, confirmed by a Deoxyribonucleic acid (DNA) diagnostic technique covering all dystrophin gene exons (e.g. MLPA, CGH, SCAIP, H-RMCA) and correctable by drisapersen sodium-induced exon 51 skipping.
- •2. Male subjects aged =5 years at Screening.
- •3. Able to Rise from Floor in = 7 seconds on at least 2 of the 3 pre-treatment visits (screening 1, screening 2, and baseline).
- •4. Able to walk at least 300m on the 6MWD on at least 2 of the 3 pre-treatment visits (screening 1, screening 2, and baseline).
- •5. Glucocorticosteroid use which is stable for at least 3 months prior to the first screening visit. Subjects must have been receiving glucocorticosteroids for at least 6 months prior to the first screening visit.
- •6. Willing and able to adhere to the study visit schedule and other protocol requirements.
- •7. Written informed consent signed (by parent(s)/legal guardian and/or the subject, according to the local regulations).
- •8. 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: 104
- •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
排除标准
- •1. More than a 30º (degree) flexion of either ankle, measure by goniometry.
- •2. Any additional mutations for DMD that cannot be treated by drisapersen sodium.
- •3. Current or history of liver or renal disease or impairment.
- •4. Screening platelet count below the lower limit of normal (LLN).
- •5. Acute illness within 4 weeks prior to first study drug administration which may interfere with the study assessments.
- •6. History of significant medical disorder which may confound the interpretation of efficacy or safety data (e.g. inflammatory disease, severe mental retardation and/or behavioral problems).
- •7. Severe cardiomyopathy which, in the opinion of the Investigator prohibits participation in this study. If a subject has a left ventricular ejection fraction <45% at screening, the Investigator should discuss inclusion of the subject with the Medical Monitor.
- •8. Chronic use of anti-coagulants, anti-thrombotics or anti-platelet agents within 1 month of the first administration of study drug.
- •9. Use of idebenone or other forms of coenzyme Q10 within 1 month prior to the start of the screening for the study.
- •10. Use of any investigational product or participation in another trial with an investigational product, within the half-life of that investigational product or a minimum of 6 months prior to the start of screening for the study.
- •11. Previous use of drisapersen sodium or eteplirsen.
- •12. NOTE: Subjects who fail on an entry criterion (apart from those subjects deemed to be ineligible for safety reasons) may be allowed to be re-screened at a later date, following discussion with the Medical Monitor.
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