Summary

for people ages 3-18 (full criteria)
at Sacramento, California and other locations
study started
estimated completion

Description

Summary

ZYN002 is a pharmaceutically manufactured Cannabidiol (CBD) that is developed as a clear gel that can be applied to the skin (called transdermal delivery). The gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms. Only participants from the ZYN2-CL-016 study who meet the inclusion criteria and none of the exclusion criteria for study ZYN2-CL-017 are eligible. Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.

Official Title

An Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With Fragile X Syndrome - CONNECT-FX Open Label Extension (OLE)

Details

This is an open-label extension, multiple-center study, to assess the long-term safety and tolerability of CBD administered as ZYN002, a transdermal gel, for the treatment of child and adolescent patients with Fragile X Syndrome (FXS). Male and female patients with FXS will be treated for up to 12 months. Up to 300 male and female patients, ages 3 to 18 years will be enrolled.

Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.

Participants who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours).

Participants who weigh more than 35 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours).

At the Investigator's discretion, the dose may be increased to a total of 4 sachets a day or decreased to a total of 2 sachets a day any time after the first month of treatment.

Participants whose weight changes during the course of the study may have their doses changed at the investigator's discretion on or after the Month 1 visit, or reduced due to tolerability issues at investigator's discretion.

Participants who are taking anti-epileptic drugs may have an additional one or two weeks of treatment after the 52 week treatment period to taper off study treatment.

Blood samples will be collected for safety analysis of ZYN002. Additionally, the parents/caregivers will be asked to complete some questionnaires. There will be other questionnaires and scales that will be completed at the site by the study doctor and/or with the participant and their parents/caregivers.

Keywords

Fragile X Syndrome Syndrome Epidiolex ZYN002 - CBD Transdermal Gel ZYN002

Eligibility

You can join if…

Open to people ages 3-18

  • 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

You CAN'T join if...

  • 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.

Locations

  • UC Davis Health System, MIND Institute accepting new patients
    Sacramento California 95817 United States
  • University of Washington Center for Human Development and Disability accepting new patients
    Seattle Washington 98198 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Zynerba Pharmaceuticals, Inc.
Links
Sign up for this study
ID
NCT03802799
Phase
Phase 2/3
Study Type
Interventional
Last Updated