EUCTR2018-004089-33-DK进行中（未招募）1 期
A Phase 1/2 trial on the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of DYN101 in patients = 16 years of age with centronuclear myopathies caused by mutations in DNM2 or MTM1 - Research Using an Investigational Treatment for CNM (Unite-CNM)
Dynacure0 个研究点目标入组 18 人开始时间: 2019年6月11日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 18
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
- All
入选标准
- •1. Male or female aged = 16 years of age* on the date of signing the
- •main ICF. The first subject (i.e. the
- •sentinel subject) in each cohort must be = 18 years of age.
- •*for Germany, the subject must be = 18 years of age in accordance with
- •local and national regulations.
- •2. Have a documented mutation in DNM2 or MTM1.
- •3. Platelet count > 150,000/µL,
- •4. Have a symptomatic CNM in the opinion of the investigator, at least mild to moderately affected, i.e. showing clinical symptoms in at least 2 of the 4 relevant domains that will be investigated in this trial (respiratory function, muscle strength, muscle function, dysphagia), and be ambulatory, i.e. being able to walk 10 steps, if needed with support/assisted. If a subject is non-ambulatory but highly functioning in the view of the investigator, he/she may be included following discussion with the sponsor.
- •5. Have an understanding, ability and willingness to fully comply with visit frequency, trial procedures and restrictions, including contraceptive requirements.
- •6. Able to provide written, signed and dated informed consent/assent to participate in the trial. Parental consent (one or both parents) and an assent for subjects < 18 years may be required per local legislation.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 6
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 10
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Clinically significant liver disease.
- •2. Clinically significant renal disease.
- •3. Presence of significant co-morbidities or conditions other than CNM or clinically significant findings during screening of medical history, physical examination, laboratory testing, vital signs or ECG recording for which, in the opinion of the investigator and the medical monitor, participation would not be in the best interest of the subject (e.g. compromise the safety or well-being) or that could prevent, limit, or confound the protocol-specified assessments (e.g. taking a muscle biopsy).
- •4. For female subjects of child-bearing potential: pregnant or breastfeeding, or planning to become pregnant during the trial.
- •5. Current or past abuse of alcohol or recreational/narcotic drugs (with the exception of caffeine and nicotine), which in the investigator’s opinion would compromise the subject’s safety and/or compliance with the trial procedures.
- •6. Currently enrolled in any interventional trial or scheduled to participate in another trial whilst participating in this trial. Subjects are
- •allowed to participate in registry studies.
- •7. Current or relevant history of physical or psychiatric illness, any medical disorder that may require treatment or make the subject unlikely to fully complete the trial, or any condition that presents undue risk from the IMP or procedures.
- •8. Intake of any disallowed therapies as noted in the protocol within 12 weeks before the planned first IMP administration.
- •9. Known or suspected intolerance or hypersensitivity to IMP ingredients or closely-related compounds, or history of a significant allergic reaction to IMP ingredients as determined by the investigator, such as anaphylaxis requiring hospitalization.
- •10. Legally incapacitated or have limited legal capacity. Lack of mental capacity to fully understand the protocol requirements and complete all study required procedures.
研究者
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An early phase human drug trial (called phase 1/2) to investigate DYN101 in patients = 16 years of age with a rare muscle disease group called the centronuclear myopathies caused by genetic changes in DNM2 or MTM1 for the drug’s safety, tolerability, how the body processes the drug as well as interactions of the drug with the body and preliminary effects of the drug.Centronuclear myopathies (CNMs) in patients = 16 years of age with mutations in DNM2 or MTM1MedDRA version: 20.0Level: HLTClassification code 10028640Term: MyopathiesSystem Organ Class: 100000004859EUCTR2018-004089-33-GBDynacure18
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An early phase human drug trial (called phase 1/2) to investigate DYN101 in patients = 16 years of age with a rare muscle disease group called the centronuclear myopathies caused by genetic changes in DNM2 or MTM1 for the drug’s safety, tolerability, how the body processes the drug as well as interactions of the drug with the body and preliminary effects of the drug.Centronuclear myopathies (CNMs) in patients = 16 years of age with mutations in DNM2 or MTM1EUCTR2018-004089-33-FRDynacure SAS18