Researchers are conducting this study for children ages 4 to less than 12 years old with epilepsy. This research will look to see if the study drug is safe and effective in children with a certain type of epilepsy (PGTC) and determine the best dose of the study drug in this age group. While epilepsy is a common neurological brain condition, one out of four patients with epilepsy may not achieve long-term freedom from seizures. New medications have become available to treat epilepsy, but because some drugs work differently in children than they do in adults, it is important to conduct clinical studies in pediatric patients with epilepsy to evaluate how the drug works in children.
In this study, your child will be receiving an investigational epilepsy drug. Although the study drug has been approved for adjunctive use for PGTC in adults and adolescents in the US and more than 50 countries, it is “investigational” in this study because it has not been approved for use in children in this age group.
CAN MY CHILD JOIN THIS STUDY?
The study doctor is looking for patients who:
• Have been diagnosed with epilepsy – PGTC
• Are at least 4 years old but less than 12 years old
• Weigh 35 pounds (about 16kg) or more
• Are currently being treated for epilepsy
Additional requirements will apply.
AS A PARENT WHAT WILL I NEED TO DO?
• Give correct and accurate information about your child’s medical history and medical condition.
• Tell your study doctor at every appointment about any physical problems or emotional changes in your child.
• Complete a daily seizure diary and bring it to every appointment.
• Give your child the study medication as instructed by the study doctor.
WHAT CAN WE EXPECT IF MY CHILD JOINS THE STUDY?
The study doctor will explain the study to you and your child. You will sign an informed consent document that explains potential risks and benefits of joining the study. This is not a binding contract; you can remove your child from the study at any time, for any reason, with no penalty to you or your child.
Your child will attend a screening visit and tests will be done to see if your child is a good match for the study. The study drug will not be given until these tests are performed.
Once it’s determined that your child is eligible to participate in the study, he or she may be in the study for a little over a year (56 weeks) and visit the clinic up to 13 times. Your child will be closely monitored by the study doctor throughout the study, and you can stay with your child during his or her appointments.
Your child will take the study drug (which is a liquid) by mouth every night before bedtime. He or she may continue to take routine epilepsy drugs in addition to the study drug.
Participation in a research study is entirely voluntary. You can stop at any time without giving a reason, and leaving will not affect your child’s regular care. If you exit the study, you will be asked to bring your child to the study clinic for a final medical exam for his or her safety. There is no charge for study-related tests and study drug. You may be reimbursed for your reasonable travel expenses.
CRCNJ is now enrolling patients in blüm, a Phase III clinical trial for CM-AT, a potential first-in-class treatment for autism. CM-AT is developed by Curemark, LLC, a drug research and development company focused on the treatment of neurological diseases.
CM-AT is based on Curemark’s recent research that showed enzyme deficiencies in some autistic children result in an inability to digest protein. The inability to digest protein affects the availability of amino acids, the building blocks of chemicals essential for brain function.
CRCNJ is one of over 20 sites across the country conducting the blüm Phase III clinical trial of CM-AT in autism.
STUDY COMPLETED, CLOSED FOR ENROLLMENT.
NeurAbilities (Voorhees, NJ) was awarded a two-year grant to investigate for genetic abnormalities in those with Autism Spectrum Disorders (ASD) associated with intellectual disabilities and/or epilepsy, and determine their clinical manifestations. Findings from this research project may discover new biological causes of ASDs and associated complications, as well as assist clinicians in determining treatments targeting mechanisms of disease rather than symptoms. The Center for Neurological and Neurodevelopmental Health (CNNH) will lead this project, with three collaborating institutions: Courtagen Life Sciences (Woburn, Massachusetts), Coriell Institute for Medical Research (Camden, NJ), and the Mitochondrial Disease Laboratory of St. Christopher’s Hospital for Children (Philadelphia, PA). These four specialized and innovative organizations will work collaboratively to investigate how certain genetic variations are associated with impairments in brain energy production and function, and can subsequently cause ASDs and/or lead to complications of ASDs, such as epilepsy, gastrointestinal problems, and serious behavioral disturbances.
Children and adults with Autism Spectrum Disorders and associated Intellectual Disabilities and/or Epilepsy between the ages of 6 and 30 years of age are eligible to participate in the study. Comprehensive clinical assessments include the following:
• Specialized Genetic Testing
• Dense Array Electroencephalographic (“brain wave”) Testing
• Neurological and Medical Evaluation
• Neuropsychological Testing
• Functional Behavioral Analysis
• Sleep Assessment
• Laboratory Testing
Help increase our knowledge of autism spectrum conditions and consider participation in this study.
This clinical research is funded in part by The New Jersey Governor’s Council for Medical Research and Treatment of Autism and the New Jersey Department of Health.
ONGOING STUDY, CLOSED FOR ENROLLMENT.
CRCNJ is conducting various clinical trials that are not yet listed on this page, please contact us to find out if there is a clinical trial right for you.