Clinical study Of caNNabidiol in childrEn and adolesCenTs with Fragile X

INTERVENTION: ZYN002 is a pharmaceutically manufactured Cannabidiol (CBD) that is developed as a clear gel that can be applied to the skin (called transdermal delivery). The gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms. All participants will undergo a screening process. Eligible participants will then participate in up to a 14 week treatment period, where all participants may receive placebo or active study drug Treatment group A (ZYN002): Parents/caregivers will apply the study gel twice daily for the treatment period. Participants who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours). • Participants who weigh more than 35 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours). Participants who are taking Anti‐epileptic drugs may have an additional one or two weeks of blinded treatment to taper off study treatment. After the final dose, patients will be followed weekly for 4 weeks by telephone, prior to discharge from the study. Patient compliance with the intervention will be monitored by the study coordinator through drug accountability at each study visit. CONDITION: Fragile X Syndrome PRIMARY OUTCOME: The change in the Aberrant Behavior Checklist‐Community Fragile X factor (ABC‐C) Pre‐specified Subscale 1 SECONDARY OUTCOME: Change in CGI‐I Change in the ABC‐C Fragile X factor Pre‐specified Subscale 2 ; Change in the ABC‐C Fragile X factor Pre‐specified Subscale 3 To evaluate the safety and tolerability of ZYN002 ; Safety assessments will include regular collection of any adverse events, physical and neurological exams (including Tanner staging), clinical laboratory safety assessments (hematology, chemistry and urinalysis), vital signs and 12‐lead ECGs. In addition, the Behavior Checklist and Penn Physician Withdrawal Checklist will be utilized to assess for any potential withdrawal characteristics of ZYN002. Finally, as the study drug is delivered transdermally, tolerability to study drug application will be assessed through skin check examinations performed at each visit after treatment with study gel has been initiated. INCLUSION CRITERIA: 1. Male or female children and adolescents aged 3 to less than 18 years, at the time of Screening. 2. Judged by the Investigator to be in generally good health at Screening based upon the results of a medical history, physical examination, 12‐lead ECG, and clinical laboratory test results. Laboratory results outside of the reference range must be documented as not clinically significant by both the Investigator and Sponsor. 3. Patients must have a diagnosis of FXS through molecular documentation of FMR1 full mutation. 4. Patients must be assessed by the Investigator as being moderately to severely impacted due to FXS. 5. Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of one or two AEDs, or must be seizure‐free for one year if not currently receiving AEDs. 6. Patients who are taking psychotropic medication(s) should be on a stable regimen of no more than two such medications for at least fo