MIT-E Study
Research type
Research Study
Full title
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E)
IRAS ID
1003429
Contact name
Shamima Rahman
Contact email
Eudract number
2020-002100-39
Clinicaltrials.gov Identifier
Research summary
This study will test whether vatiquinone (the study drug) will reduce the number of seizures in participants with mitochondrial
disease. The study will also test the safety of the study drug. We hope to learn whether the study drug is useful and safe for
treating seizures as a symptom of mitochondrial disease.
Patients will be invited to participate in this study because they have been diagnosed with mitochondrial disease and they
experience treatment resistant seizures. Only individuals under the age of 18 years with a caregiver who can ensure study
activities can participate in the study.
The study is divided into 4 parts and participation will last 83 weeks (around 20 months):
1. Screening/run-in phase (around 6 weeks)
2. Placebo controlled phase (24 weeks) Participants will be randomly assigned to receive either study drug or placebo. A placebo
looks like study drug but has no active ingredients. There is a 1 to 1 chance to receive placebo or active drug. Regardless of
what participants are assigned to, they will receive placebo or study drug three times a day. If the participant is receiving study
drug it will be at a dose of 15 mg/kg body weight (up to 200 mg maximum per dose), three times daily.
3. Long-term open label extension phase (48 weeks) Participants who choose to continue will receive study drug for the open
label phase of the study. All participants previously on placebo will receive study drug. In the long-term extension part of the
study, participants will receive study drug at a dose of 15 mg/kg up to 200 mg three times daily.
4. Follow up period (30 days) The study doctor will decide if additional observation or testing is needed to ensure the participant
is doing well.
In total, there will be 60 participants in this study at approximately 30 study sites around the world. The study is paid for by PTC
Therapeutics, Inc.REC name
London - Hampstead Research Ethics Committee
REC reference
20/LO/1289
Date of REC Opinion
1 Feb 2021
REC opinion
Further Information Favourable Opinion