A Clinical Study for Children with Doose Syndrome, also know as Epilepsy with Myoclonic-Atonic Seizures (EMAS).

A study (also called a clinical trial) is a medical study that helps to answer important questions about an investigational medication, including how well the medication works for a certain condition and how safe and tolerable the medication is.

All medications must be tested in clinical research studies before they can be approved by regulatory authorities and prescribed to patients. Even if a medication is already approved for a certain condition, it must be tested again before it can be approved for a different condition.

A study is the process by which new and innovative medications, interventions, and treatments are approved and brought to the general public, so people can hopefully live happier and healthier lives. Even over-the-counter medications, such as aspirin that you may have used for a headache, have gone through the clinical research study process before they were available at your local pharmacy.

By participating in a clinical study, you can help advance medical science and help your loved ones and others in the community by making new treatments available.

The purpose of this study is to assess the effect of a study medicine, compared with a placebo, on the frequency of seizures in children and adolescents between 1 and 18 years of age who have been diagnosed with EMAS or are suspected of having EMAS. The study will also assess how well the body tolerates the study medicine and how well it works over a longer period of time.

The investigational medicine in this study is an oral cannabidiol, or CBD, solution that has been approved in the United States, European Union, and the United Kingdom for the treatment of certain types of seizures. It is not approved to treat people with EMAS and is therefore called “investigational.”

The CinEMAS study is looking at how well the body tolerates the study medicine and whether it works in children and adolescents with EMAS. The study treatment will be taken in addition to the participant’s other current treatments. Like all medication, the trial medication may cause side effects. If your child experiences any side effects, you should let your child’s trial physician know immediately.

CinEMAS is a two-part study: the Main Study (Part A) will assess the how well the study medicine works and how safe it is compared to a placebo and the Extension Study (Part B) will assess how well the body tolerates the study medicine and how well it works over a longer period of time.

The duration of participation in the Main Study will be approximately 26 weeks, which includes a 1- to 3-week Screening Period, 4-week Seizure Observation Period, 14-week Dose Optimization Treatment Period, 10-day Taper Period, and a Safety Follow-up Period (4 weeks after end of taper visit).

If qualified for the study, participants will be randomly assigned, like drawing straws, to one of two treatment groups to take either the study medicine (oral solution) or a placebo. A placebo looks like the study medicine but does not contain the active ingredient. The Main Study is double-blind, which means that neither participants nor the study doctor will know which treatment they are taking during the study.

During the 14-week Treatment Period of the study, participants will take their study treatment twice a day at home as instructed by the study doctor (the first dose will be taken by the patient at the study site). The study treatment will be taken in addition to the participant’s other current treatments.

An electronic diary using a smartphone app (phone to be provided) will be completed throughout the study by the patient’s main caregiver to document various aspects of treatment. Visits will be required throughout the study, comprised of study site visits and phone calls. During these visits, a variety of tests, procedures, and questionnaires will be completed. It may be possible to reduce the number of study site visits by having some assessments performed at your home.

Upon completion of the Main Study, participants will have an option to continue in the Extension Study. The Extension Study will assess how well the body tolerates the study medicine and how well it works over a longer period of time. Patients who received the placebo in the Main Study will be switched to the study medicine. The duration of study participation in the Extension Study will be approximately 54 weeks and the electronic diary will be completed at least weekly.

Caregivers of children who qualify to take part in the study may receive reimbursement for time and travel. Please discuss this with the study team.

There is no cost to participate in the CinEMAS study. If your child is eligible and you decide to take part:

• Your child will receive study-related care from a team of experienced doctors and nurses throughout the study
• All study-related visits, tests, assessments, and investigational medication will be provided at no cost to you

The research team will be able to explain more about what the CinEMAS study will involve, and it is up to you to decide if you want your child to take part. Participation in this study is voluntary. Your decision to participate or not participate will have no effect on the medical care your child receives now or in the future. If your child is eligible and you choose to have them participate in the CinEMAS study, your child may leave the study at any time without providing any reason.

The global CinEMAS study is being conducted across several study sites in the United States, Australia, Italy, and the United Kingdom. Find a study site location near you.

Your child can still be considered for the CinEMAS study by one of the study doctors.  Please contact one of the study sites nearest to you and they can help to determine if your child can participate in the study or not.