EUCTR2020-002100-39-SE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
- 88
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.
- •Age <21 years at time of randomization.
- •Subjects with genetic confirmation of inherited mitochondrial disease with associated epilepsy phenotype (Alpers/POLG, Leigh syndrome, MELAS), or other genetically confirmed mitochondrial diseases secondary to mitochondrial mutations (Pontocerebellar Hypoplasia Type 6 [PCH6], nuclear DNA RARS2 mutation) or myoclonic epilepsy with ragged red fibers (MERRF, mitochondrial DNA [mtDNA] mitochondrially encoded tRNA lysine [MT-TK] mutation) are eligible.
- •Despite treatment with at least 2 antiepileptic drugs:
- •?have =6 observed motor seizures occurring during the 28 days prior to the baseline visit (Day 0).
- •?have =2 observed motor seizures in the first 14 days and =2 in the second 14 days of the Run-in period (Day -14).
- •?do not have a consecutive 20-day seizure free period.
- •?have at least 80% of seizure diary data.
- •No changes to the AED regimen will be allowed (except weight-based dose adjustments) during the first 24-week period.
- •Documented medical history of epilepsy symptoms associated with mitochondrial disease for at least 6 months prior to screening except for subjects who are <2 years of age at the time of screening (subjects <2 years of age can be considered for enrollment if all other screening criteria are met due to the potential for rapid progression in these subjects).
- •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 screening visit
- •Stable regimen of dietary supplements 30 days prior and, if on a ketogenic diet, stable ketogenic diet 90 days prior to the screening visit and for duration of the study.
- •Electroencephalogram (EEG) at screening or historical EEG up to 6 months prior to screening for diagnostic confirmation of epilepsy.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 88
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 8
- •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) =3×ULN at time of screening
- •INR > ULN at time of screening
- •Serum creatinine =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 and use of artisanal (non-Epidiolex cannabidiol) cannabidiol therapies.
- •Ongoing treatment with strong cytochrome P450 (CYP) inhibitors such as itraconazole or strong 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 male and female sexually active subjects 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).
- •Comorbidities that may confound study results (eg, fat malabsorption syndrome, other mitochondrial disorders) in the opinion of the investigator
研究者
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