JPRN-jRCT2051190069已完成2 期
A Phase 2 Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Efficacy of MYD-0124 to Adult Patients with Myotonic Dystrophy Type 1 - MYD-0124 Study
akamori Masayuki0 个研究点目标入组 30 人开始时间: 2019年10月29日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
研究概览
简要总结
Patients with myotonic dystrophy experienced no issues with tolerating MYD-0124 treatment. While no statistically significant differences in efficacy endpoints were evident, the improvement in splicing biomarkers and the reduction in CK levels suggested that MYD-0124 might be effective for patients with myotonic dystrophy.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 20age old 至 <= 55age old（—）
- 性别
- All
入选标准
- •1. Males or females aged 20 to 55 years old at the time of informed consent
- •2. Genetic confirmation of DM1
- •3. Ambulatory (orthoses allowed, canes and walkers not allowed) and able to walk at least 25 meters at screening
排除标准
- •1. Treatment with macrolide antibiotics medication within 60 days prior to screening.
- •2. Clinically significant abnormal ECG or significant symptoms of cardiac dysfunction at Screening
- •3. Intellectual disability, or significant behavioral neuropsychiatric manifestations
- •4. Clinically significant abnormalities in screening laboratory values that would render the subject unsuitable for inclusion
- •5. Treatment with anti-myotonia medication within 30 days prior to screening. May include, but not be limited to: Phenytoin, Carbamazepine, Procainamide, Mexiletine
- •6. Recent history of or current drug or alcohol abuse
- •7. Have any condition, which, in the opinion of the investigator would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the study
研究者
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