A Study of the Long-Term Safety and Tolerability of Experimental ZYN002 in Children and Adolescents With Fragile X Syndrome (FXS)

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Age: 3 to 18 years old
Gender:
Any
Healthy Volunteers: No
Keywords: Fragile X, Fragile X syndrome, cannabidiol
Type: Drug study, Phase [/2/,/ /3/]
Target:
8 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 to the “endocannabinoid system.” This is a certain pathway 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
- behavioral, social and emotional difficulties.

Cannabidiol (CBD) is part of the Cannabis/Marijuana plant. However, it does not have the same 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 evaluate the long-term safety, tolerability and effectiveness of the experimental drug ZYN002. It will be used in the treatment of symptoms of Fragile X Syndrome (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:

  • Participated in the ZYN2-CL-016 study.
  • Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.
  • Patients and parents/caregivers must be adequately informed of the nature, risks of the study, and give written informed consent prior to enrollment in ZYN2-CL-017.
  • In the Investigator's opinion, the patients and parents/caregivers are reliable and are willing and able to comply with all protocol requirements and procedures.
  • Females of childbearing potential must have a negative pregnancy test at all designated visits

Exclusion criteria:

  • Patient is receiving any investigational drugs (not ZYN002) or using any experimental devices.
  • Patient has an ongoing serious adverse event (SAE) or has experienced a SAE in ZYN2-CL-016, which in the opinion of the Investigator, should exclude them from participation.
  • Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception for the duration of therapy and for three months after the last dose of trial drug.
  • Patients who have alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels >= 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels >= 3 times the ULN as determined from patient safety laboratories.
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 12 months after the screening period.
Recruitment period
From May 1, 2019
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Laura Pulido-Ramirez
Research Topic
Conditions:
  • Fragile X Syndrome