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Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) (CONNECT-FX)

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ClinicalTrials.gov Identifier: NCT03614663
Recruitment Status : Recruiting
First Posted : August 3, 2018
Last Update Posted : May 22, 2019
Sponsor:
Information provided by (Responsible Party):
Zynerba Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE July 10, 2018
First Posted Date  ICMJE August 3, 2018
Last Update Posted Date May 22, 2019
Actual Study Start Date  ICMJE June 12, 2018
Estimated Primary Completion Date July 23, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 2, 2018)
Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 [ Time Frame: Change from Baseline to end of treatment (Week 14) ]
The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03614663 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 2, 2018)
  • Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 [ Time Frame: Change from Baseline to end of treatment (Week 14) ]
    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 3 [ Time Frame: Change from baseline to end of treatment (Week 14) ]
    The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
  • Clinical Global Impressions- Improvement (CGI-I) [ Time Frame: Change from baseline to end of treatment (Week 14) ]
    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 Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With Fragile X Syndrome
Brief Summary This study will evaluate the efficacy and safety of ZYN002, a clear cannabidiol (CBD) gel that can be applied to the skin (called transdermal application) twice a day for the treatment of behavioral symptoms of Fragile X Syndrome (FXS). Eligible participants will then participate in up to a 14 week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to < 18 years, will be eligible to participate.
Detailed Description

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of ZYN002, a pharmaceutically manufactured CBD, formulated as a transdermal gel, for the treatment of children and adolescent with FXS. Approximately 204 male and female patients, ages 3 to < 18 years, will undergo a screening process. Eligible participants will be randomized 1:1 to either trial drug or placebo and will undergo a 14-week treatment period. Randomization will be stratified by gender, weight category and geographic region. All participants may receive placebo during the trial. Participants who are taking anti-epileptic drugs may undergo an additional 1-2 weeks of blinded treatment to taper off study drug treatment. The assignment will be done by a computer generated system and neither the study doctor or the participant or their caregivers will know which treatment is being given to them. The dose of the treatment will depend on the weight of the participants. If the participants weigh less than or equal to 35 kg, they will receive 2 sachets of the gel twice a day (1 sachet approximately every 12 hours) and if they weigh more than 35 kg, they will receive 4 sachets of gel per day (2 sachets approximately every 12 hours). Parents/ caregivers will be instructed on proper application of the gel. The gel will be applied to clean, dry, intact skin of the upper arms/ shoulders.

Blood samples will be collected for safety analysis of ZYN002. An independent analytical laboratory will also perform CGG repeat and methylation status analyses. 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.

After the final dose, patients will be followed weekly for 4 weeks by telephone, prior to discharge from the study.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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: Placebo Transdermal Gel
    Placebo formulated as a clear gel (transdermal delivery)
    Other Names:
    • Placebo Comparator
    • Matching Placebo
Study Arms  ICMJE
  • Experimental: ZYN002 - CBD transdermal gel

    ZYN002 supplied as a transdermal gel. Patients weighing less than or equal to 35 kg will be randomized to receive either 125 mg CBD Q12H or placebo.

    Patients weighing greater than 35 kg will be randomized to receive 250 mg CBD Q12H or placebo.

    Intervention: Drug: ZYN002 - CBD Transdermal Gel
  • Placebo Comparator: Placebo transdermal gel
    Matching ZYN002 placebo supplied as a transdermal gel.
    Intervention: Other: Placebo 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: August 2, 2018)
204
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 29, 2019
Estimated Primary Completion Date July 23, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male or female children and adolescents aged 3 to less than 18 years, at the time of Screening.
  • Diagnosis of FXS through molecular documentation of FMR1 full mutation.
  • Judged to be in good health based on physical exam, 12-lead ECG and clinical laboratory test results.
  • Patients must be assessed by the Investigator as being moderately to severely impacted due to FXS.
  • Patients taking psychotropic medication(s) should be on a stable regimen of not more than two such medications for at least fours weeks preceding Screening and must maintain that regimen throughout the study.
  • If patients are receiving non-pharmacological, behavioral and/or dietary interventions, they must be stable and have been doing so for three months prior to screening.
  • Patients and parents/caregivers must be adequately informed of the nature and risks of the study and given written informed consent prior to Screening.
  • In the Investigator's opinion, patients and parents/caregivers are reliable and willing and able to comply with all protocol requirements and procedures.

Exclusion Criteria:

  • Females who are pregnant, nursing or planning a pregnancy.
  • Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin levels greater than or equal to 2 times the upper limit of normal or alkaline phosphatase levels greater than or equal to 3 times the upper limit of normal.
  • Use of a strong inhibitor/inducer of CYP3A4 or sensitive substrate of CYP3A4.
  • Use of minocycline for 30 days prior to screening or throughout the study.
  • Use of any benzodiazepine at screening or throughout the study.
  • Use of THC or CBD-containing product within three months of Screening Visit or during the study.
  • Change in pharmacologic or non-pharmacologic intervention during the course of the study.
  • Any skin disease or condition including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration that may affect treatment application, application site assessments or absorption of the trial drug.
  • Patient is using the following AEDs: clobazam, phenobarbital, ethosuximide, felbamate or vigabatrin.
  • Patients has an advanced, severe or unstable disease that may interfere with the study outcome evaluations.
  • Patient has acute or progressive neurological disease, psychosis, schizophrenia or any other psychiatric disorder or severe mental abnormalities (other than FXS) that are likely to require changes in drug therapy or interfere with the study objectives or ability to adhere to protocol requirements.
  • Patient has suspected or confirmed cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, risk factors for Torsades de pointes (e.g. heart failure, hypokalemia, family history of Long QT Syndrome) or other serious cardiac problems.
  • History of treatment for, or evidence of drug abuse within the past year.
  • Patient responds "yes" to Question 4 or 5 on the C-SSRS (Children) during Screening or at any time on study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Nancy R 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 NCT03614663
Other Study ID Numbers  ICMJE ZYN2-CL-016
Has Data Monitoring Committee Not Provided
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