Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Fragile X Syndrome

In brief

This trial will evaluate the efficacy and safety of ZYN002, a clear cannabidiol gel that can be applied to the skin (called transdermal application) twice a day for the treatment of behavioral symptoms of Fragile X Syndrome (FXS). Eligible participants will then participate in up to a 14 week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to \< 18 years, will be eligible to participate.

Key facts

Study ID

NCT03614663

Run by

Zynerba Pharmaceuticals, Inc.

People needed

212

Starts

2018-06-12

Expected to finish

2020-06-14

Last updated by the study team

2022-07-06

Who can join

Age: 3 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female children and adolescents aged 3 to less than 18 years, at the time of Screening.
• Diagnosis of FXS through molecular documentation of FMR1 full mutation.
• Judged to be in good health based on physical exam, 12-lead ECG and clinical laboratory test results.
• Patients must be assessed by the Investigator as being moderately to severely impacted due to FXS.
• Patients taking psychotropic medication(s) should be on a stable regimen of not more than two 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 and parents/caregivers must be adequately informed of the nature and risks of the study and given written informed consent prior to Screening.
• In the Investigator's opinion, patients and parents/caregivers are reliable and willing and able to comply with all protocol requirements and procedures.

You may not qualify if…

• Females who are pregnant, nursing or planning a pregnancy.
• Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin levels greater than or equal to 2 times the upper limit of normal or alkaline phosphatase levels greater than or equal to 3 times the upper limit of normal.
• Use of a strong inhibitor/inducer of CYP3A4 or sensitive substrate of CYP3A4.
• Use of minocycline for 30 days prior to screening or throughout the study.
• Use of any benzodiazepine at screening or throughout the study.
• Use of THC or CBD-containing product within three months of Screening Visit or during the study.
• Change in pharmacologic or non-pharmacologic intervention during the course of the study.
• 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.
• Patient is using the following ASMs: clobazam, phenobarbital, ethosuximide, felbamate or vigabatrin.
• Patients 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 suspected or confirmed 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, risk factors for Torsades de pointes (e.g. heart failure, hypokalemia, family history of Long QT Syndrome) or other serious cardiac problems.
• History of treatment for, or evidence of drug abuse within the past year.
• Patient responds "yes" to Question 4 or 5 on the C-SSRS (Children) during Screening or at any time on study.

Where it is running

• Southwest Autism Research and Resource Center — Phoenix, Arizona, United States
• Phoenix Children's Hospital — Phoenix, Arizona, United States
• UC Davis Health System, MIND Institute — Sacramento, California, United States
• Children's Hospital of Colorado — Denver, Colorado, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Fragile X Center of Atlantic Health System — Morristown, New Jersey, United States
• The Fragile X Spectrum Disorder Clinic at Icahn School of Medicine at Mount Sinai, Division of Medical Genetics — New York, New York, United States
• University of North Carolina — Chapel Hill, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
• Central States Research — Tulsa, Oklahoma, United States
• Suburban Research Associates — Media, Pennsylvania, United States
• Greenwood Genetic Center — Greenville, South Carolina, United States
• University of Washington Center for Human Development and Disability — Seattle, Washington, United States
• Westmead Children's Hospital — Sydney, New South Wales, Australia
• Lady Cilento Children's Hospital - South Brisbane — Brisbane, Queensland, Australia
• Genetics Clinics Australia — Melbourne, Victoria, Australia
• Wellington Hospital — Wellington, New Zealand

Full record on ClinicalTrials.gov

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