A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With Fragile X Syndrome - RECONNECT

About this Study

The purpose of this study is to investigate of effective and safe ZYN002 transdermal gel is in patients with Fragile X Syndrom (FXS).  The Drug Product ZYN002 is a pharmaceutically manufactured Cannabidiol (CBD). It is being developed as a clear gel that can be applied to the skin (called transdermal delivery), to provide consistent, controlled levels of CBD in the blood when it is given twice a day

Sponsor Protocol ID:ZYN2-CL-033
IRB Number:2021-0614
Actively Enrolling
Interventional
Phase 3
January 24, 2022
Eligibility Criteria
3 years old
17 years old
Both Male and Female
No
Yes
Yes

Inclusion Criteria
  • Male or female children and adolescents aged 3 to 17
  • Generally in good health at Screening
  • Participants must have a diagnosis of FXS through molecular documentation of full mutation of the FMR1 gene documented through genetic testing at Screening.
  • Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of no more than two anti-seizure medications (ASMs) for the four weeks preceding study Screening; or must be seizure-free for one year if not currently receiving ASMs.
  • Patients taking psychoactive medication(s) should be on a stable regimen of not more than three such medications for at least fours weeks preceding Screening and must maintain that regimen throughout the study. 
  • If patients are receiving non-pharmacological, behavioral and/or dietary interventions, they must be stable and have been doing so for three months prior to screening.
  • Patients have a body mass index between 12-30 kg/m2 (inclusive).
  • Females of childbearing potential must have a negative serum pregnancy test at the Screening Visit
  • Patients and parents/caregivers must be adequately informed of the nature and risks of the study and given written informed consent prior to Screening.
  • Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.

Exclusion Criteria
  • Females who are pregnant, nursing or planning a pregnancy
  • History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients.
  • Exposure to any investigational drug or device less than or equal to 30 days prior to Screening or at any time during the study.
  • Elevated Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin levels   
  • Patient has a positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines, and opiates.
  • Patient is using the following AEDs (medications for the treatment of seizures and/ or epilepsy): clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin, or vigabatrin.
  • Patient is using a strong inhibitor/inducer of CYP3A4 or sensitive substrate of CYP3A4 including but not limited to the following medications:
  • Patients may not be taking any benzodiazepines (except single doses administered for the purposes of obtaining blood samples and ECGs) at screening or throughout the study.
  • Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.
  • Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations.
  • Patient has acute or progressive neurological disease, psychosis, schizophrenia or any other psychiatric disorder or severe mental abnormalities (other than FXS) that are likely to require changes in drug therapy or interfere with the study objectives or ability to adhere to protocol requirements.
  • Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies.
  • Patient has known history of cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope,
  • Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study medication.
  • Any skin disease or condition including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration that may affect treatment application, application site assessments or absorption of the trial drug.
  • History of treatment for, or evidence of, drug abuse within the past year.
  • Previous participation in a ZYN002 study