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Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

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ClinicalTrials.gov Identifier: NCT03802799
Recruitment Status : Recruiting
First Posted : January 14, 2019
Last Update Posted : May 22, 2019
Sponsor:
Information provided by (Responsible Party):
Zynerba Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE December 26, 2018
First Posted Date  ICMJE January 14, 2019
Last Update Posted Date May 22, 2019
Actual Study Start Date  ICMJE November 9, 2018
Estimated Primary Completion Date September 30, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 9, 2019)
Incidence of treatment-emergent adverse events (safety and tolerability) [ Time Frame: Up to 1 year ]
Safety assessment will include collection of any treatment emergent adverse events
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03802799 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 9, 2019)
  • Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 [ Time Frame: Change from baseline to end of treatment, an average of 1 year ]
    The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
  • Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 [ Time Frame: Change from baseline to end of treatment, an average of 1 year ]
    The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
  • Clinical Global Impression- Improvement (CGI-I) [ Time Frame: Change from baseline to end of treatment, an average of 1 year ]
    The CGI-I global improvement item is a 7-point Likert scale designed to measure behavioral symptomatic change at a specific time compared to baseline. CGI-I is a standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
Official Title  ICMJE 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)
Brief 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.

Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Intervention Model Description:
Open-Label
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fragile X Syndrome
Intervention  ICMJE Drug: ZYN002 - CBD Transdermal Gel
Pharmaceutically manufactured. Cannabidiol (CBD) formulated as a clear gel (transdermal delivery)
Other Name: Cannabidiol Transdermal Gel
Study Arms  ICMJE Experimental: ZYN002
ZYN002 - CBD Transdermal Gel
Intervention: Drug: ZYN002 - CBD Transdermal Gel
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 9, 2019)
300
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 30, 2020
Estimated Primary Completion Date September 30, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Nancy Tich, PhD 973-727-4117 tichn@zynerba.com
Contact: Donna Gutterman, PharmD 919-522-8828 guttermand@zynerba.com
Listed Location Countries  ICMJE Australia,   New Zealand,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03802799
Other Study ID Numbers  ICMJE ZYN2-CL-017
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Zynerba Pharmaceuticals, Inc.
Study Sponsor  ICMJE Zynerba Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Zynerba Pharmaceuticals, Inc.
Verification Date May 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP