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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03802799
Recruitment Status : Recruiting
First Posted : January 14, 2019
Last Update Posted : January 18, 2020
Information provided by (Responsible Party):
Zynerba Pharmaceuticals, Inc.

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.

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: ZYN002 - CBD 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 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 300 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Open-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 : November 9, 2018
Estimated Primary Completion Date : February 28, 2022
Estimated Study Completion Date : February 28, 2022

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: ZYN002
ZYN002 - CBD Transdermal Gel
Drug: ZYN002 - CBD Transdermal Gel
Pharmaceutically manufactured. Cannabidiol (CBD) formulated as a clear gel (transdermal delivery)
Other Name: Cannabidiol Transdermal Gel

Primary Outcome Measures :
  1. 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

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03802799

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Contact: Nancy Tich, PhD 973-727-4117
Contact: Donna Gutterman, PharmD 919-522-8828

Hide Hide 21 study locations
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United States, Arizona
Southwest Autism Research and Resource Center Recruiting
Phoenix, Arizona, United States, 85006
Contact: Raun Melmed, MD   
Phoenix Children's Hospital Recruiting
Phoenix, Arizona, United States, 85016
Contact: Richard Frye, MD   
United States, California
UC Davis Health System, MIND Institute Recruiting
Sacramento, California, United States, 95817
Contact: Randi Hagerman, MD   
United States, Colorado
Children's Hospital of Colorado Recruiting
Denver, Colorado, United States, 80045
Contact: Nicole Tartaglia, MD   
United States, Georgia
Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30322
Contact: Amy Talboy, MD   
United States, Illinois
Rush University Medical Center Recruiting
Chicago, Illinois, United States, 60612
Contact: Elizabeth Berry-Kravis, MD   
United States, Maryland
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
Contact: Dejan Budimirovic, MD   
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Lisa Prock, MD   
United States, New Jersey
Fragile X Center of Atlantic Health System Recruiting
Morristown, New Jersey, United States, 07960
Contact: Darius Adams, MD   
United States, New York
The Fragile X Spectrum Disorder Clinic at Icahn School of Medicine at Mount Sinai, Division of Medical Genetics Recruiting
New York, New York, United States, 10029
Contact: Reymundo Lozano, MD         
Contact: Katherine Graham-Oregan    212-659-8645   
United States, North Carolina
University of North Carolina Recruiting
Chapel Hill, North Carolina, United States, 27510
Contact: Laura Politte, MD   
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Craig Erickson, MD   
University Hospitals Cleveland Medical Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: Nora McNamara, MD   
United States, Oklahoma
Central States Research Recruiting
Tulsa, Oklahoma, United States, 74136
Contact: Whitney Deneen    918-645-5400      
Principal Investigator: Sarah Land, MD         
United States, Pennsylvania
Suburban Research Associates Recruiting
Media, Pennsylvania, United States, 19063
Contact: Shivkumar Hatti, MD   
United States, South Carolina
Greenwood Genetic Center Recruiting
Greenville, South Carolina, United States, 29605
Contact: Carrie Buchanan, MD   
United States, Washington
University of Washington Center for Human Development and Disability Recruiting
Seattle, Washington, United States, 98198
Contact: Raphael Bernier, PhD   
Australia, New South Wales
Westmead Children's Hospital Recruiting
Sydney, New South Wales, Australia, 2145
Contact: Natalie Silove, MD   
Australia, Queensland
Lady Cilento Children's Hospital - South Brisbane Recruiting
Brisbane, Queensland, Australia, 4101
Contact: Honey Heussler, MD   
Australia, Victoria
Genetics Clinics Australia Recruiting
Melbourne, Victoria, Australia, 3161
Contact: Jonathan Cohen, MD   
New Zealand
Wellington Hospital Recruiting
Wellington, New Zealand, 6021
Contact: Andrew Marshall, MD   
Sponsors and Collaborators
Zynerba Pharmaceuticals, Inc.

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Responsible Party: Zynerba Pharmaceuticals, Inc. Identifier: NCT03802799    
Other Study ID Numbers: ZYN2-CL-017
First Posted: January 14, 2019    Key Record Dates
Last Update Posted: January 18, 2020
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Fragile X Syndrome
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System