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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 : April 9, 2019
Sponsor:
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 : September 30, 2019
Estimated Study Completion Date : September 30, 2020

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

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

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 ClinicalTrials.gov identifier (NCT number): NCT03802799


Contacts
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Contact: Nancy Tich, PhD 973-727-4117 tichn@zynerba.com
Contact: Donna Gutterman, PharmD 919-522-8828 guttermand@zynerba.com

  Show 21 Study Locations
Sponsors and Collaborators
Zynerba Pharmaceuticals, Inc.

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Responsible Party: Zynerba Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03802799     History of Changes
Other Study ID Numbers: ZYN2-CL-017
First Posted: January 14, 2019    Key Record Dates
Last Update Posted: April 9, 2019
Last Verified: April 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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Epidiolex
Syndrome
Fragile X Syndrome
Disease
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
Anticonvulsants