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
Study Status: Study Status Actively Enrolling: The study is currently recruiting participants. Closed for Enrollment: The study is not currently recruiting participants.	Actively Enrolling
Study Type: Study Type Interventional: A type of clinical study in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. The assignments are determined by the study's protocol. Participants may receive diagnostic, therapeutic, or other types of interventions. Observational: A type of clinical study in which participants are identified as belonging to study groups and are assessed for biomedical or health outcomes. Participants may receive diagnostic, therapeutic, or other types of interventions, but the investigator does not assign participants to a specific interventions/treatment. A patient registry is a type of observational study.	Interventional
Study Phase: Study Phase Early Phase 1 Trials (formerly listed as Phase 0): Exploratory trials conducted before traditional phase 1 trials to investigate how or whether a drug affects the body. They involve very limited human exposure to the drug and have no therapeutic or diagnostic goals (for example, screening studies, microdose studies). Phase 1 Trials: Trials that focus on the safety of a drug. They are usually conducted with healthy volunteers, and the goal is to determine the drug's most frequent and serious adverse events and, often, how the drug is broken down and excreted by the body. These trials usually involve a small number of participants. Phase 2 Trials: Trials that gather preliminary data on whether a drug works in people who have a certain condition/disease (that is, the drug's effectiveness). For example, participants receiving the drug may be compared to similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied. Phase 3 Trials: Trials that gather more information about a drug's safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. These studies typically involve more participants. Phase 4 Trials: Trials occurring after FDA has approved a drug for marketing. They include postmarket requirement and commitment studies that are required of or agreed to by the study sponsor. These trials gather additional information about a drug's safety, efficacy, or optimal use.	Phase 3
Online Start Date: Online Start Date The online start date is the day that this trial first appeared on the UMMC website, which is not necessarily the day that the trial first began at UMMC.	January 24, 2022

Eligibility Criteria

Minimum Age Age Requirement Studies may have specific age requirements. Some may only accept participants over a specified age and/or under a specified age. Please read the study description and additional eligibility criteria for specific information.	3 years old
Maximum Age Age Requirement Studies may have specific age requirements. Some may only accept participants over a specified age and/or under a specified age. Please read the study description and additional eligibility criteria for specific information.	23 years old
Biological Sex Biological Sex Requirement Indicates the sex of people who may participate in a clinical study (all, female, male). Sex is a person's classification as female or male based on biological distinctions. Sex is distinct from gender-based eligibility.	Both Male and Female
Cancer Related? Cancer Related Some studies may be targeted to cancer-related treatments or research. Please read the study description and additional eligibility criteria for specific information.	No
Pediatric Related? Pediatric Related Some studies may be targeted to child-related treatments or research. Please read the study description and additional eligibility criteria for specific information.	Yes
Accepts Healthy Volunteers? Accepts Healthy Volunteers Indicates whether people who do not have the condition/disease being studied can participate in the clinical study. Healthy volunteers play an important role in research because they help define "normal" ranges. While they may receive no direct benefit, healthy volunteers choose to participate in clinical research because they want to help others and contribute to advancing science.	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