EUCTR2011-005042-35-GB进行中（未招募）1 期
A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 9
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
入选标准
- •1. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with BMN 053 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 300 metres in the 6 minute walking distance (6MWD) test at the first screening visit and also at the baseline visit. In addition, results of 2 of any of the 3 pretreatment 6MWD tests (assessed screen 1, screen 2, baseline) must be within ±30 metres of each other prior to first BMN 053 administration. Subjects must also be able to rise from the
- •floor in = 7 seconds at the first screening visit and also at the baseline visit.
- •3. Adequate quality for biopsy (confirmed with MRI) of the lateral head of the gastrocnemius muscle. Only under exceptional circumstances will an alternative muscle (preferably brachii) be considered for biopsy and only following discussion between the Principal Investigator and the BioMarin Medical Monitor.
- •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 053 administration. Subjects must have been receiving glucocorticosteroids for at least 6 months prior to the first BMN 053 administration.
- •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.
- •9. Anticipated adequate vein access for intravenous (IV) infusion.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 45
- •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. Current or history of liver disease or impairment.
- •2. Current or history of renal disease or impairment.
- •3. Screening aPTT above upper limit of normal (ULN) within the last month prior to first dose of BMN 053.
- •4. Screening platelet count below the lower limit of normal (LLN).
- •5. Acute illness within 4 weeks prior to first dose of BMN 053 which may interfere with the study assessments.
- •6. Severe mental retardation and/or behavioural problems which, in the opinion of the Investigator, prohibit participation in this study.
- •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. Expected need for daytime mechanical ventilation within the next year.
- •9. Use of anticoagulants, antithrombotics or antiplatelet agents.
- •10. Use of idebenone or other forms of coenzyme Q10 within 1 month prior to the start of the screening for the study.
- •11. Use of nutritional or herbal supplements which, in the opinion of the Investigator, may influence muscle performance within 1 month prior to first dose of BMN 053.
- •12. 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.
研究者
相似试验
进行中（未招募）
1 期
A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyDuchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skippingMedDRA version: 18.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-005042-35-BEBioMarin Nederland B.V.42
进行中（未招募）
1 期
A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyDuchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skippingMedDRA version: 16.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-005042-35-NLProsensa Therapeutics BV42
进行中（未招募）
1 期
A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyDuchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skippingMedDRA version: 18.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-005042-35-FRProsensa Therapeutics BV42
进行中（未招募）
1 期
A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyDuchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skippingMedDRA version: 15.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-005042-35-ITProsensa Therapeutics BV42
已完成
2 期
A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously knoen as PRO053) in subjects with Duchenne muscular dystrophyDuchenne muscular dystrophyNL-OMON43770BioMarin Pharmaceutical Inc5