NL-OMON43779已完成2 期
A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy - PRO045-CLIN-01
BioMarin Parmaceutical Inc.0 个研究点目标入组 5 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 11（—）
入选标准
- •1. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with BMN 045 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)
- •2. 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 BMN 045 administration
- •3. Adequate quality for biopsy (confirmed with MRI) of the lateral head of the gastrocnemius muscle.
- •4. Life expectancy of at least 3 years after inclusion in the study.
- •5. Glucocorticosteroid use which is stable for at least 3 months prior to first BMN 045 administration. Subjects must have been receiving glucocorticosteroids for at least 6 months prior to the first BMN 045 administration.
排除标准
- •1. 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).
- •2. Current or history of liver disease or impairment
- •3. Current, or history of, renal disease or impairment.
- •4. at least two aPTT above ULN within the last month
- •5. Screening platelet count below the lower limit of normal (LLN).
- •6. Acute illness within 4 weeks prior to first dose of BMN 045 which may interfere with the study assessments.
- •7. Severe mental retardation or behavioural problems which, in the opinion of the investigator, prohibit participation in this study
- •8.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.
- •9. Expected need for daytime mechanical ventilation within the next year.
- •10. Use of anticoagulants, antithrombotics or antiplatelet agents.
- •11. Use of idebenone or other forms of coenzyme Q10 within 1 month prior to the start of the screening for the study.
- •12. Use of nutritional or herbal supplements which, in the opinion of the investigator, may influence muscle performance, within 1 month of the study
- •13. Use of any other investigational product or participation in another trial with
- •an investigational product, within 6 months prior to the start of the screening
- •for the study.
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