A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of PRO045 in subjects with Duchenne muscular dystrophy

Prosensa Therapeutics BV0 个研究点目标入组 45 人2015年6月16日

适应症Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping MedDRA version: 18.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

概览

阶段: 1 期
干预措施: 未指定
疾病 / 适应症: Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping
发起方: Prosensa Therapeutics BV
入组人数: 45
状态: 进行中（未招募）
最后更新: 8年前

概览研究者入排标准结局指标相似试验

概览

简要总结

暂无简介。

注册库

who.int

开始日期

2015年6月16日

结束日期

待定

最后更新

8年前

研究类型

Interventional clinical trial of medicinal product

性别

Male

研究者

发起方

Prosensa Therapeutics BV

入排标准

入选标准

•Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO045 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 HRMCA (High\-Resolution Melting Curve Analysis).
•Ambulant boys aged at least 5 years on the day of first dosing able to walk for at least 230 meters in the 6 minute walking distance (6MWD) at the first screening visit and also at the baseline visit. In addition, 2 of the 3 pre\-treatment 6MWD tests (screen 1, screen 2, baseline) must be within ± 30 meters of each other prior to first PRO045 administration.
•Adequate quality for biopsy (confirmed with MRI) of the lateral head of the gastrocnemius muscle. An alternative muscle may be considered for biopsy but only following discussion between the Principal Investigator and the Prosensa Medical Monitor.
•Life expectancy of at least 3 years after inclusion in the study.
•Glucocorticosteroid use which is stable for at least 3 months prior to first PRO045 administration. Subjects must have been receiving glucocorticosteroids for at least 6 months prior to the first PRO045 administration.
•Willing and able to adhere to the study visit schedule and other protocol requirements.
•Written informed consent signed (by parent(s)/legal guardian and/or the subject, according to the local regulations).
•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: 45

排除标准

•Known presence of dystrophin in \=5% of fibres in a pre\-study diagnostic muscle biopsy (i.e. historic muscle biopsy taken prior to written informed consent for this study).
•Current or history of liver disease or impairment.
•Current or history of renal disease or impairment.
•At least two aPTT above ULN within the last month.
•Screening platelet count below the lower limit of normal (LLN).
•Acute illness within 4 weeks prior to first dose of PRO045 which may interfere with the study assessments.
•Severe mental retardation or behavioural problems which, in the opinion of the investigator, prohibit participation in this study.
•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.
•Expected need for daytime mechanical ventilation within the next year.
•Use of anticoagulants, antithrombotics or antiplatelet agents.