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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03802799




Registration number
NCT03802799
Ethics application status
Date submitted
26/12/2018
Date registered
14/01/2019
Date last updated
22/05/2019

Titles & IDs
Public title
Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
Scientific 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)
Secondary ID [1] 0 0
ZYN2-CL-017
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fragile X Syndrome 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ZYN002 - CBD Transdermal Gel

Experimental: ZYN002 - ZYN002 - CBD Transdermal Gel


Treatment: Drugs: ZYN002 - CBD Transdermal Gel
Pharmaceutically manufactured. Cannabidiol (CBD) formulated as a clear gel (transdermal delivery)

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events (safety and tolerability) - Safety assessment will include collection of any treatment emergent adverse events
Timepoint [1] 0 0
Up to 1 year
Secondary outcome [1] 0 0
Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 - The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Timepoint [1] 0 0
Change from baseline to end of treatment, an average of 1 year
Secondary outcome [2] 0 0
Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 - The ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Timepoint [2] 0 0
Change from baseline to end of treatment, an average of 1 year
Secondary outcome [3] 0 0
Clinical Global Impression- Improvement (CGI-I) - 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.
Timepoint [3] 0 0
Change from baseline to end of treatment, an average of 1 year

Eligibility
Key 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
Minimum age
3 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2/Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Westmead Children's Hospital - Sydney
Recruitment hospital [2] 0 0
Lady Cilento Children's Hospital - South Brisbane - Brisbane
Recruitment hospital [3] 0 0
Genetics Clinics Australia - Melbourne
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
4101 - Brisbane
Recruitment postcode(s) [3] 0 0
3161 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
New Jersey
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oklahoma
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
South Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Washington
Country [16] 0 0
New Zealand
State/province [16] 0 0
Wellington

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Zynerba Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03802799
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Nancy Tich, PhD
Address 0 0
Country 0 0
Phone 0 0
973-727-4117
Fax 0 0
Email 0 0
tichn@zynerba.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03802799