A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Part… (NCT04378075) | Clinical Trial Compass
TerminatedPhase 2/3
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Stopped: Sponsor decision.
United States68 participantsStarted 2020-09-28
Plain-language summary
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.
Who can participate
Age range20 Years
SexALL
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Inclusion Criteria:
* Signed informed consent form.
* Participant or parent/legal guardian is able and willing to complete seizure diaries for the duration of the study.
* Genetic confirmation of inherited mitochondrial disease with associated epilepsy phenotype (Alpers/polymerase subunit gamma \[POLG\], Leigh syndrome, mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes \[MELAS\]), or other genetically confirmed mitochondrial disease 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).
* 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.
* Documented medical history of epilepsy associated with mitochondrial disease for at least 6 months prior to screening except for participants who are \<2 years of age at the time of screening (participants \<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 participants).
* Consent to abstain from non-approved…
What they're measuring
1
Percent Change From Baseline to Week 24 in the Number of Observable Motor Seizures Per 28 Days During the Double-blind Period