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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03614663
• Date of registration: 10/07/2018
• Prospective Registration: No
• Primary sponsor: Zynerba Pharmaceuticals, Inc.
• Public title: Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) CONNECT-FX
• Scientific title: 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
• Date of first enrolment: June 12, 2018
• Target sample size: 212
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03614663
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

Australia, New Zealand, United States

Key inclusion & exclusion criteria

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.



Age minimum: 3 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fragile X Syndrome

Intervention(s)

Other: Placebo Transdermal Gel

Drug: ZYN002 - CBD Transdermal Gel

Primary Outcome(s)

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)]

Secondary Outcome(s)

Clinical Global Impressions- Improvement (CGI-I) [Time Frame: Change from baseline to end of treatment (Week 14)]

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)]

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)]

Secondary ID(s)

ZYN2-CL-016

Source(s) of Monetary Support

Please refer to primary and secondary sponsors