EUCTR2020-002100-39-FRActive, not recruitingPhase 1
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) - MIT-E
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- PTC THERAPEUTICS, INC.
- Enrollment
- 80
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •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:
- •?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 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: 80
- •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
Exclusion Criteria
- •Allergy to vatiquinone or sesame oil.
- •Aspartate transaminase (AST) or alanine transaminase (ALT) =2 × upper level of normal (ULN) at time of screening.
- •International normalized ratio (INR) =1.5 × 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.
- •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).
Investigators
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