CONNECT-FX TRIAL RATIONALE
Fragile X Syndrome (FXS) is a genetic condition that causes intellectual disability, behavioral and learning challenges and is also the most common known single gene cause of autism spectrum disorder. There is currently no therapeutic option approved for the treatment of behavioral symptoms associated with FXS. CONNECT-FX is enrolling eligible patients to help researchers find a potential treatment for these patients and their families.
ZYN002 is an experimental treatment. This means that it is not approved by government regulatory bodies, including the United States Food and Drug Administration (FDA) and other agencies and must be tested to see if it is an effective and safe treatment.
Cannabinoids are a class of chemical compounds that occur naturally in the human body and in the cannabis plant, and interact with numerous signaling systems and receptors in the central nervous system. Cannabidiol, or CBD, is one of those naturally occurring compounds. Two primary cannabinoids contained in Cannabis are CBD and ∆9-tetrahydrocannabinol, or THC. Of the more than 100 cannabinoids identified, CBD and THC are the most known and characterized. An important distinction between these two cannabinoids is that, unlike THC, CBD is non-euphoric, meaning it does not create “the high” often associated with cannabis or marijuana. The drug under investigation in this trial, ZYN002, is pharmaceutically manufactured CBD, does not contain THC and, therefore, lacks euphoric effects.
CONNECT-FX is a clinical trial evaluating ZYN002, an experimental transdermally delivered CBD gel, as a treatment for some of the common and debilitating behaviors in patients with FXS. Transdermal delivery means that the drug is applied directly to the skin and it is then absorbed into the bloodstream.
CONNECT-FX Trial Objective
The purpose of CONNECT-FX is to evaluate the efficacy and safety of ZYN002 in improving certain behavioral symptoms that patients with FXS exhibit. The trial will include 204 patients (children and adolescents ages three through 17 years) at approximately 20 clinical sites in the United States, Australia and New Zealand who have been diagnosed with FXS.
CONNECT-FX Trial Design and Procedures
All patients enrolled in CONNECT-FX will receive either active study drug (ZYN002) or placebo (an identical version of the study treatment with no active drug included). CONNECT-FX is a double-blind trial, meaning that the study patient, parent, doctor, site staff and study sponsor will not know which study treatment, active study drug or placebo, has been given to the patient. These types of studies are important to help investigators establish the benefits and risks of treatment and to allow government regulatory bodies, such as the United States Food and Drug Administration (FDA) and other country regulatory agencies, determine if a drug should or should not be approved for marketing and made available to patients.
The CONNECT-FX trial will last 14 weeks. Prior to starting the trial, parents or caregivers of eligible patients will provide written informed consent. During the screening period, the CONNECT-FX clinical site coordinator and staff will review the eligibility criteria and any medications including over-the-counter (OTC)
medications the patient is taking, obtain patient’s medical history including details related to their FXS diagnosis, note any seizure history and demographics, check their vital signs, perform an electrocardiogram (ECG), assess stage of puberty (if applicable), perform a physical and neurological exam and a skin assessment, obtain blood and urine for analysis, and administer assigned scales.
Enrolled patients in the trial will be randomized (assigned by chance, not choice) and given either the active study drug or placebo.
Patients randomized in the trial (whether on placebo or active study drug) who have been at least 90% compliant with study protocol may have the option to enroll in a one-year open-label extension trial, during which all patients receive the active study drug.
CONNECT-FX Trial Eligibility
Patients may be eligible to participate in CONNECT-FX if they are children or adolescents (boys and girls) ages three through 17 years, diagnosed with FXS. For more details about this trial, including a list of requirements for entering the trial, visit www.clinicaltrials.gov and www.anzctr.org.au. This trial has the clinical trials identifier: 03614663 AND ACTRN12618001063202.
A total of 204 patients are being recruited to participate in this trial at approximately 20 sites in the United States, Australia and New Zealand. To locate your nearest trial center, please use the map below.
The CONNECT-FX team at the clinical site will meet with eligible patients, and their parents and/or caregivers, to discuss the trial, obtain medical history and perform relevant screening tests to determine if the child or adolescent meets all of criteria to participate. Feel free to discuss any questions with a medical professional or the CONNECT-FX team to help further inform your decision to enroll (if eligible) in CONNECT-FX.
Frequently Asked Questions
What are clinical trials?
Clinical trials are studies of drugs that are still in development or have not been approved to treat patients with the specific condition being studied. A drug being studied in a clinical trial is called an investigational drug. In a clinical trial, a drug is evaluated to determine whether or not it is safe and effective. The drug may be compared with a placebo (a substance not expected to have any real medical effect) or a different treatment. Clinical trials of drugs provide information about:
- Whether the drug has the effect it is supposed to have
- How much of the drug to give to a patient (also called the treatment dose) and how often (also called the treatment regimen)
- What side effects are associated with the drug, and how they can best be managed
- How a drug is broken down in the body, and how long it stays in the body
- Which foods, drinks, or other drugs can be used at the same time, or should be avoided
Clinical trial results allow the FDA, or other regulatory agencies, to make decisions about whether or not a drug should be approved for marketing.
Why should patients participate in a clinical trial?
People participate in clinical trials for a variety of reasons.
- Healthy volunteers participate to help others and contribute to moving science forward so doctors and researchers can learn more about a disease and improve health care for others in the future.
- Participants with an illness, disease or condition also participate to help others, but also to possibly receive new, experimental drugs or devices – especially if there are no other treatment options or if all of other treatment options have failed.
What is informed consent?
Informed consent provides key information and details about a clinical trial in a written document to patients before they decide whether to take part, and continues throughout the trial. This information typically includes the purpose of the clinical trial, how long it should last, tests or procedures done as part of the trial, potential risks and benefits, and contact information for the trial coordinator and others involved in leading the trial. It is important to note that taking part in a clinical research trial is voluntary, and patients can withdraw on their own at any time.
Are there costs associated with participating in a clinical trial?
How many times will CONNECT-FX patients have to visit the study site?
Will patients enrolled in the CONNECT-FX trial be allowed to continue taking medications he/she might currently be taking?
How does a patient qualify to participate in the CONNECT-FX trial?
Addressing Safety in the
Any research has some risks, which may include things that could make a child or adolescent feel bad or uncomfortable. In a research trial like CONNECT-FX, not every risk or side effect may be known or can be predicted. Each person’s reaction to a test, drug, or procedure may be different. A child or adolescent may have a side effect or be at risk for symptoms, illnesses and/or complications that could not be predicted by the trial doctor or sponsor. To date, the study drug has been given to over 570 subjects.
Safety assessments in CONNECT-FX will include the collection of adverse events. Throughout the study, the trial investigator will monitor each patient for evidence of drug intolerance and for the development of clinical and/or laboratory evidence of an adverse event. An adverse event assessment will be made by the investigator on a routine basis at each study visit.