MIT-E Study

What is this study about?

PTC Therapeutics is conducting a clinical trial (MIT-E) to evaluate the safety and efficacy of an investigational treatment called vatiquinone in patients with mitochondrial disease with refractory epilepsy.

Who can take part? 

The study is aiming to involve 60 participants, up to 20 years of age with genetically confirmed Mitochondrial Diseases Drug Resistant Epilepsy; Leigh Disease, Leigh Syndrome, MELAS, Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease or Alpers Syndrome.

The criteria to enter the trial are quite specific and they are aimed at identifying children with genetically confirmed mitochondrial disease and relatively stable but refractory epilepsy (epilepsy that is not fully controlled by medication).

In order to be eligible for this trial, participants must have tried at least 2 antiepileptic drugs and have documented medical history of epilepsy associated with mitochondrial disease for at least 6 months prior to screening. They must also have a relatively large number of seizures (at least 6 per month).

Participants must satisfy a number of additional criteria there are also certain exclusion criteria. Further details are available here.

What's involved in taking part?

This study is a parallel-arm, double-blind, placebo-controlled study. This means that after a 28-week run in phase, participants will be randomly assigned to receive either the study medication (vatiquinone) or placebo over 24-weeks. This phase is known as the 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 treatment with the study medication vatiquinone.

Are there any risks?

Like all clinical trials involving investigational treatments, there may be risks associated with taking part. Each patient should discuss the benefits and risks of participating in a clinical trial with their doctor who is best placed to advise them individually.

Who will benefit?

Clinical trials are a type of research that studies new tests and treatments and evaluates their effects on humans including the safety and effectivness. Clinical trials are carefully designed, reviewed and approved before they can start. People volunteer to take part in clinical trials. Patients should discuss with their treating doctor whether participation into a particular trial is appropriate for them.

Who is running the study?

PTC Therapeutics are running this study. Currently there are a number of centres in the Unites States recruiting patients. However, it is expected that more centres in other locations, including the UK, may soon begin to enrol participants. Study sites in the UK are in the locations: London (Great Ormond Street) and Newcastle, and are now active and recruiting participants.

How do I find out more?

For further details on this trial, please contact Dr Naomi Thomas (Newcastle), telephone: 0191 2083105, email: [email protected] or Victor Maduekwe (London), telephone: 020 78298261 (x8261), email: [email protected]