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NYSDOH Epidiolex® Expanded Access Studies For Treatment-Resistant Epilepsy In Children and Young Adults
Thursday, January 21, 2016
STATE HEALTH DEPARTMENT ANNOUNCES COMPASSIONATE USE STUDIES OF CANNABIDIOL FOR CHILDREN AND YOUNG ADULTS WITH TREATMENT-RESISTANT EPILEPSY
Studies to be regulated under FDA’s Expanded Access Program
100 Children and Young Adults in New York Will be Enrolled at Five Sites Statewide
The New York State Department of Health (DOH) today announced that compassionate use studies using Epidiolex®, an investigational pharmaceutical-grade form of cannabidiol, will soon begin at each of the following five sites across New York State:
- Montefiore Medical Center, Bronx
- University of Buffalo - Women & Children’s Hospital of Buffalo
- NYU Langone Medical Center
- Mount Sinai Beth Israel
- University of Rochester Medical Center
Patients between the ages of one and twenty-one who meet a clinical definition of severe, treatment-resistant epilepsy may be eligible. This definition includes patients who have tried at least four anti-epileptic drugs (AEDs), including a combination of two concomitant drugs, without successful control of their seizures. Patients with a confirmed diagnosis of a treatmentresistant epilepsy syndrome may also be eligible. Initial enrollment of selected patients is expected to begin over the next few months.
“Given the devastating effect seizures have on development and the quality of life for these children and young adults, we are thrilled to be able to provide this opportunity to patients who may qualify,” said Health Commissioner Dr. Howard Zucker. “These studies will give patients in New York State who are not responding to conventional anti-epileptic therapy access to a drug that has shown promise in previous expanded access studies.”
Children and young adults will be referred by their treating neurologist for consideration as participants at one of the five sites. The studies, which are FDA-approved, will last one year. DOH is currently notifying New York State neurologists of the studies and providing further guidance on the referral process.
Epidiolex® is not FDA approved, and therefore, other than in formal clinical trials and one existing physician-sponsored compassionate access study, it is unavailable to patients being treated in New York State at this time. GW Pharmaceuticals (GW) is conducting randomized controlled trials now throughout the US for two epilepsy syndromes (Dravet and Lennox-Gastaut) to gather the safety and efficacy data to support an application for FDA approval of this drug.
GW has agreed to make Epidiolex® available free of charge for the DOH-sponsored expanded access studies. Only children and young adults who are not eligible for the current GW trials will be eligible for participation in the DOH sponsored studies.
Data gathered from these studies will contribute to knowledge of the safety and tolerability of this investigational drug in young people with intractable epilepsy and will expand the number of practitioners with experience in providing this drug to epilepsy patients. Information on interactions with other antiepileptic drugs will also be gathered.
Click on the links below for further information:
- Letter to NYS Neurologists describing the studies (appendices are listed separately below)
- Appendix A: Clinical Study Principal Investigators and Sites
- Appendix B: Study Summary
- Appendix C: Subject Selection (Inclusion and Exclusion Criteria)
- Appendix D: GW Pharmaceuticals RCT Inclusion and Exclusion Criteria
- Appendix E: Patient Visit Schedule
- Appendix F: Seizure Diary
- Study Referral Form
- De-Identified Selection Worksheet