EUCTR2020-002100-39-IT进行中（未招募）1 期
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 80
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
- •- Signed informed consent form.
- •- Genetic confirmation of inherited mitochondrial disease (secondary to either nuclear or mitochondrial DNA mutation) with associated epilepsy phenotype
- •- Despite ongoing treatment with at least 2 antiepileptic drugs:
- •a) have >or= 6 observed motor seizures occurring during the 28 days prior to the baseline visit (Day 0).
- •b) have >or= 2 observed motor seizures in the first 14 days and >or= 2 in the second 14 days of the Run-in period (Day -14).
- •c) do not have a consecutive 20-day seizure free period.
- •d) have at least 80% of seizure diary data.
- •No changes to the AED regimen will be allowed during the first 24-week period.
- •- Documented medical history of epilepsy symptoms associated with mitochondrial disease for at least 6 months prior to screening.
- •- Consent to abstain from non-approved therapies for 30 days prior to the baseline visit (Day 0) and for the duration of the study.
- •- Stable dose regimen of antiepileptic therapies 30 days prior to the baseline visit (Day 0).
- •- Stable regimen of dietary supplements 30 days prior and, if on a ketogenic diet, stable ketogenic diet 90 days prior to the baseline visit (Day 0) and for duration of the study.
- •- Electroencephalogram (EEG) at screening or historical EEG (taken within the last 30 days) for diagnostic confirmation of epilepsy
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •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
- •- Allergy to vatiquinone or sesame oil.
- •- Aspartate transaminase (AST) or alanine transaminase (ALT) >or= 2 × upper level of normal (ULN) at time of screening.
- •- International normalized ratio (INR) >or= 1.5 × ULN at time of screening.
- •- Serum creatinine >or= 1.5 × ULN at time of screening.
- •- Participation in another interventional clinical trial 60 days prior to randomization or for the duration of this clinical trial
- •- Previously received vatiquinone.
- •- Concomitant treatment with drug(s) that have not received regulatory agency approval for the treatment of mitochondrial diseases.
- •- Ongoing treatment with cytochrome P450 (CYP) inhibitors such as itraconazole or CYP inducers such as rifampin. Treatment with these agents must be completed at least 4 weeks prior to enrollment.
- •- Pregnant or lactating participants or those sexually active participants who are unwilling to comply with proper birth control methods. Females of childbearing potential must have a negative pregnancy test at screening and during the baseline visit (Day 0).
研究者
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