A Study Of Experimental Cannabidiol in Children and Adolenscents With Fragile X Syndrome (CONNECT-FX Study)

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Age: 3 to 17 years old
Gender:
Any
Healthy Volunteers: No
Keywords: Fragile X Syndrome
Type: Drug study, Phase [2, 3]
Target:
15 Participants
Investigator:
Description
Fragile X Syndrome (FXS) is a genetic condition caused by a mutation in the Fragile X mental retardation 1 (FMR1) gene. This mutation is thought to cause changes the “endocannabinoid system” in the central nervous system. Disruption in this pathway can lead to FXS symptoms such as:
- mild to severe intellectual or developmental disability,
- speech and language difficulties, and
- behavioral, social and emotional difficulties.

Cannabidiol (CBD) is part of the Cannabis/Marijuana plant. It does not have the commonly known effects on the brain that recreational marijuana has. It may affect the endocannabinoid pathway. The Drug Product ZYN002 is a pharmaceutically manufactured CBD. It is being developed as a clear gel that can be applied to the skin (called transdermal delivery). This provides consistent, controlled levels of CBD in the blood when it is given once or twice a day.

The purpose of this study is to test the safety and effectiveness of ZYN002 transdermal gel in patients with FXS.

There are currently no FDA approved medications shown to treat FXS.

ZYN002 is an experimental treatment. This means that it is not approved by the FDA and must be tested to see if it is an effective treatment.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

Inclusion criteria:

  • 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.

Exclusion criteria:

  • 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 AEDs: 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.
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid to take part in this research study.
Resources
Schedule
Study duration and period
We expect that your child will be in this research study for up to 18 weeks after the screening period.
Recruitment period
From Nov. 8, 2018
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Research Topic
Conditions:
  • Fragile X Syndrome