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

Sponsor
Zynerba Pharmaceuticals, Inc. (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT03802799
Collaborator
(none)
450
Enrollment
22
Locations
1
Arm
102.7
Anticipated Duration (Months)
20.5
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

ZYN002 is a pharmaceutically manufactured Cannabidiol 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.

Participants from the ZYN2-CL-016 and ZYN2-CL-033 studies 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.

Condition or Disease Intervention/Treatment Phase
  • Drug: ZYN002 - Cannabidiol Transdermal Gel
 Phase 2/Phase 3

Detailed Description

This is an open-label extension, multiple-center study, to assess the long-term safety and tolerability of cannabidiol 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 450 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 from study ZYN-CL-016 who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours).

Participants from study ZYN-CL-016 who weigh more than 35 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours).

Participants from study ZYN-CL-033 who weigh less than or equal to 30 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours).

Participants from study ZYN-CL-033 who weigh more than 30 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours).

Participants from study ZYN-CL-033 who weigh more than 50 kg will receive 3 sachets of ZYN002, applied every 12 hours (± 2 hours).

At the Investigator's discretion and medical monitor approval, the dose may be increased to a total of 6 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 Design

Study Type:
Interventional
Anticipated Enrollment :
450 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
Open-LabelOpen-Label
Masking:
None (Open Label)
Primary Purpose:
Treatment
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)
Actual Study Start Date :
Nov 9, 2018
Anticipated Primary Completion Date :
May 31, 2027
Anticipated Study Completion Date :
May 31, 2027

Arms and Interventions

Arm Intervention/Treatment
Experimental: ZYN002

ZYN002 - cannabidiol Transdermal Gel

Drug: ZYN002 - Cannabidiol Transdermal Gel
Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
Other Names:
  • Cannabidiol Transdermal Gel

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Incidence of treatment-emergent adverse events (safety and tolerability) [Up to 1 year]

      Safety assessment will include collection of any treatment emergent adverse events

    Secondary Outcome Measures

    1. Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 [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.

    2. Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 [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.

    3. Clinical Global Impression- Improvement (CGI-I) [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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    3 Years to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Participated in study ZYN2-CL-016 or Study Zyn2-CL-033.

    • 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 or ZYN2-CL-033, 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Southwest Autism Research and Resource Center Phoenix Arizona United States 85006
    2 Phoenix Children's Hospital Phoenix Arizona United States 85016
    3 UC Davis Health System, MIND Institute Sacramento California United States 95817
    4 Children's Hospital of Colorado Denver Colorado United States 80045
    5 Emory University School of Medicine Atlanta Georgia United States 30322
    6 Han Phan Atlanta Georgia United States 30329
    7 Rush University Medical Center Chicago Illinois United States 60612
    8 Kennedy Krieger Institute Baltimore Maryland United States 21205
    9 Boston Children's Hospital Boston Massachusetts United States 02115
    10 Fragile X Center of Atlantic Health System Morristown New Jersey United States 07960
    11 The Fragile X Spectrum Disorder Clinic at Icahn School of Medicine at Mount Sinai, Division of Medical Genetics New York New York United States 10029
    12 University of North Carolina Chapel Hill North Carolina United States 27510
    13 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    14 University Hospitals Cleveland Medical Center Cleveland Ohio United States 44106
    15 Central States Research Tulsa Oklahoma United States 74136
    16 Suburban Research Associates Media Pennsylvania United States 19063
    17 Greenwood Genetic Center Greenville South Carolina United States 29605
    18 University of Washington Center for Human Development and Disability Seattle Washington United States 98198
    19 Westmead Children's Hospital Sydney New South Wales Australia 2145
    20 Lady Cilento Children's Hospital - South Brisbane Brisbane Queensland Australia 4101
    21 Genetics Clinics Australia Melbourne Victoria Australia 3161
    22 Wellington Hospital Wellington New Zealand 6021

    Sponsors and Collaborators

    • Zynerba Pharmaceuticals, Inc.

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Zynerba Pharmaceuticals, Inc.
    ClinicalTrials.gov Identifier:
    NCT03802799
    Other Study ID Numbers:
    • ZYN2-CL-017
    First Posted:
    Jan 14, 2019
    Last Update Posted:
    Aug 22, 2022
    Last Verified:
    Apr 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Fragile X Syndrome
    Syndrome
    Cannabidiol

    Study Results

    No Results Posted as of Aug 22, 2022