Current Studies at Mid-Atlantic Epilepsy and Sleep Center
Click here for recent research publications from Dr. Klein and the Mid-Atlantic Epilepsy and Sleep Center
New Epilepsy Drugs in Development: Current Studies
This Is Only A Partial List. Please Contact Our Office If You Would Be Interested In Participating In One Of Our Studies.
- Clobazam adjunctive treatment in adults with refractory focal epilepsy – Clobazam is an antiseizure medication that was approved by the FDA in 2012 for use in patients with a particular epilepsy condition called Lennox Gastaut syndrome – a condition with specific type of seizures called “drop attacks.” However, it has been used in Europe and Canada since 1980s for other types of seizures, including focal seizures. In a couple of studies of patients with focal epilepsy in Europe and Canada, clobazam was found to be effective and safe. Nevertheless, there have been no studies in the US of using clobazam in focal epilepsy. The purpose of this study is to evaluate effectiveness and safety of clobazam in patients with focal epilepsy that has continued in spite of treatment with other anti-seizure medications.
- Perampanel rational polytherapy in in adults with refractory focal epilepsy – Perampanel is an antiseizure medication that was approved by the FDA in 2012 for use in patients with “refractory” focal epilepsy. Perampanel has a new way of suppressing seizures that is different from all other existing anti-seizure drugs. It is possible that it may have a dramatic effect (for instance 75% or greater seizure reduction or complete freedom from seizures) when it is combined with particular medications with different mechanism of action. The goal of this study is to evaluate systematically the combination of perampanel with other antiseizure medications.
- Lacosamide adjunctive treatment in patients with refractory primary generalized epilepsy – A double blind, randomized, placebo controlled parallel group, multicenter study to evaluate the efficacy and safety of lacosamide as adjunctive therapy for uncontrolled primary generalized tonic-clonic seizures in subjects with idiopathic generalized epilepsy.
- Intranasal midazolam treatment of acute recurrent seizures. – A randomized, double blind, placebo controlled study of the safety and Efficacy of intranasal Midozolam in the outpatient treatment of subjects with seizure clusters.
- A Phase 3 Open Label (no placebo) Multicenter, Safety and Pharmacokinetic Study of YKP 3089 (Cenobamate) as Adjunctive Therapy in Subjects with Partial Onset Seizures. Principal Investigator: Pavel Klein, M.D
This study evaluates the effectiveness of a new antiseizure medication, YKP3089. This is a “Phase 2” study, i.e. a study at an earlier stage of evaluation. The goal of the study is to evaluate the safety and effectiveness of the medication in patients with partial seizures unresponsive to other medications. The study plan is the same as that described for study # 1 above. Patients continue to take their regular anti-seizure medications. They are observed for 8 weeks “baseline” for 8 weeks, after which YKP3089 (or placebo) is added for 3 months. Seizures are counted and their frequency is compared between the “baseline” period and the period of treatment with YKP3089 to see whether they have improved on the medication compared to baseline. We are looking for patients aged 18-65 years for this study.
- The Human Epilepsy Project: a prospective, observational study; this means that the patient will not be given any additional treatment as part of the study. The purpose of this study is to identify factors that can predict how patients with focal epilepsy will do on a given seizure medication, whether they will have side effects, and whether they will have more seizures. The study team will gather different information from the patient over a period of 3 years or longer.
New Epilepsy Drugs in Development: Recently Completed Studies
(A partial list. Study titles are shortened version of the formal titles)
- A Phase 2, placebo-controlled study of VX-765 in patients with treatment-resistant partial seizures. Principal Investigator: Pavel Klein, M.D.
This study was the first study in patients with epilepsy of a medication which reduces inflammation. In animals, some molecules produced during inflammation (for instance during infection) promote seizures. One of those molecules is called Interleukin 1 ß. The medication VX-765 blocks the production of interleukin 1 ß. This study was done to evaluate safety of this medication in patients with epilepsy. Mid-Atlantic Epilepsy and Sleep Center was one of 10 sites nationwide to evaluate the medication. The study is now completed, and will be followed by further studies to evaluate the effectiveness of the medication.
- A Placebo-controlled study to evaluate the efficacy and safety of Perampanel adjunctive therapy in patients with refractory partial seizures. Principal Investigator: Pavel Klein, M.D.
This was a Phase 3 study of a new antiseizure medication with a new mechanism of action. Perampanel blocks the excitatory effect on neurons (nerve cells) of the neurotransmitter glutamate by blocking one of the glutamate receptors (“AMPA” receptor). No other available antiseizure medication acts this way. The study evaluated the efficacy and safety of perampanel in treating seizures unresponsive to other antiseizure medications. The study is completed.
- A Placebo-controlled study to evaluate the efficacy and safety of Retigabine therapy in patients with refractory partial seizures. Principal Investigator: Pavel Klein, M.D.
This was also a Phase 3 study of a new antiseizure medication with a new mechanism of action. Retigabine changes one of the ion channels in neurons, the potassium channel, to make neurons less excitable and less prone to seizures. No other available antiseizure medication acts this way. The study also evaluated efficacy and safety of the medication in treating seizures unresponsive to other antiseizure medications. The study is completed.The medication was approved by the FDA in the summer of 2011 and it is expected that it will be available in the market later this year.
- A Placebo-controlled study to evaluate the efficacy and safety of Ganaxalone in subjects with refractory partial seizures. Principal Investigator: Pavel Klein, M.D.
This was a Phase 2 study of a new possible antiseizure medication, also with a new mechanism of action. Ganaxolone is a modified version of a molecule produced by the brain called allopregnanolone. Allopregnanolone is a natural metabolite of the hormone progesterone. It is produced in the brain and acts on the brain like valium. Like valium, it increases the effect of the neurotransmitter GABA (g-amino butyric acid) which inhibits neurons and makes them less excitable and less prone to seizures. No other available antiseizure medication acts this way. The main study outcome was efficacy and safety of the medication in treating seizures unresponsive to other antiseizure medications. The study is completed.
Diet Treatment Studies
- Adjunctive treatment of glioblastoma multiforme with ketogenic diet. – We will be evaluating the effect of ketogenic diet on GBM in patients in whom other treatments have not been effective. GBM cancer cells need glucose for their energy source and survival. Normal brain cells can use fat (“ketone bodies”) as a source of energy when glucose levels are reduced. Brain tumor cells cannot do so. A diet treatment that reduces glucose level and provides most energy in the form of fat may “starve” (kill) the tumor cells without affecting normal brain cells. Ketogenic diet (KD) reduces blood glucose levels while elevating levels of ketone bodies. It may therefore help in the treatment of GBM. There have been two reports of KD use in patients with GBM. They suggest that KD could be safely tested as a treatment in patients with malignant brain tumors.
- Adjunctive treatment of advanced GBM with ketogenic diet. – We will be evaluating the effect of ketogenic diet on GBM. Ketogenic diet will be used in addition to the standard treatment of radiation and chemotherapy. Standard therapy for glioblastoma multiforme (GBM) includes surgery followed by radiation and chemotherapy. Despite optimal treatment GBM often recurs or progresses. The current standard of treatment for glioblastoma is radiation and chemotherapy with temozolamide. Radiation and chemotherapy slow down the disease but GBM progresses in most patients. There is an urgent need for better therapies. Ketogenic diet (KD) is a high fat, low carbohydrate diet. In animals, brain tumor growth is dependent on blood levels of glucose. High blood glucose increase tumor growth. The same occurs in brain cancer patients in whom high blood glucose makes the tumor worse. In animals, reduction in circulating glucose levels, e.g., through ketogenic diet (KD), reduces tumor growth. In addition, ketone bodies (fat) themselves make tumor cells die and slow down tumor growth without affecting normal brain cells. Ketogenic diet (KD) reduces blood glucose levels while elevating levels of ketone bodies.