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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03802799
Ethics application status
Date submitted
26/12/2018
Date registered
14/01/2019
Date last updated
25/07/2024

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
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
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

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

Experimental: ZYN002 - ZYN002 - cannabidiol Transdermal Gel


Treatment: Drugs: ZYN002 - Cannabidiol Transdermal Gel
Pharmaceutically manufactured. Cannabidiol 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
To evaluate the long-term safety and tolerability of ZYN002 administered as a transdermal gel formulation.
Timepoint [1] 0 0
Up to 72 month.
Secondary outcome [1] 0 0
To evaluate the long-term efficacy of ZYN002 in the treatment of symptoms of FXS.
Timepoint [1] 0 0
Change from baseline to end of treatment

Eligibility
Key inclusion criteria
* Participated in study ZYN2-CL-016 or Study ZYN2-CL-033.
* 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
Sex
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 or ZYN2-CL-033, 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
Not applicable
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
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
9/11/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/08/2027
Actual
Sample size
Target
450
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
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United States of America
State/province [1] 0 0
Arizona
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United States of America
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California
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Colorado
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Florida
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Georgia
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United States of America
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Illinois
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Maryland
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United States of America
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Massachusetts
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United States of America
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Minnesota
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Mississippi
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New Jersey
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New York
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Ohio
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Oklahoma
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Pennsylvania
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South Carolina
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Utah
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Washington
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New Zealand
State/province [19] 0 0
Wellington
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Leicestershire
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Lothian
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Manchester

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 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.

Participants from the ZYN2-CL-016 and ZYN2-CL-033 studies 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/study/NCT03802799
Trial related presentations / publications
Heussler HS. Emerging Therapies and challenges for individuals with Angelman syndrome. Curr Opin Psychiatry. 2021 Mar 1;34(2):123-128. doi: 10.1097/YCO.0000000000000674. Erratum In: Curr Opin Psychiatry. 2021 Sep 1;34(5):514. doi: 10.1097/YCO.0000000000000738.
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

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