Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
An Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of ZYN002 Administered as a Transdermal Gel to Children, Adolescents, and Young Adults With Fragile X Syndrome
1 other identifier
interventional
550
4 countries
26
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 Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Nov 2018
Longer than P75 for phase_2
26 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 9, 2018
CompletedFirst Submitted
Initial submission to the registry
December 26, 2018
CompletedFirst Posted
Study publicly available on registry
January 14, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 31, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 31, 2027
November 5, 2025
February 1, 2025
8.8 years
December 26, 2018
November 4, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
To evaluate the long-term safety and tolerability of ZYN002 administered as a transdermal gel formulation.
Safety assessment will include collection of any treatment emergent adverse events
Up to 96 month.
Secondary Outcomes (1)
To evaluate the long-term efficacy of ZYN002 in the treatment of symptoms of FXS.
Change from baseline to end of treatment
Study Arms (1)
ZYN002
EXPERIMENTALZYN002 - cannabidiol Transdermal Gel
Interventions
Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (26)
Southwest Autism Research and Resource Center
Phoenix, Arizona, 85006, United States
Science 37
Culver City, California, 90230, United States
Amnova Clinical Research, LLC
Irvine, California, 92604, United States
UC Davis Health System, MIND Institute
Sacramento, California, 95817, United States
Children's Hospital of Colorado
Denver, Colorado, 80045, United States
Children's Research Institute
Washington D.C., District of Columbia, 20010, United States
University of Miami
Miami, Florida, 33136, United States
Han Phan
Atlanta, Georgia, 30329, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Masonic Institute of Developing Brain
Minneapolis, Minnesota, 55414, United States
University of Mississipi Medical Center
Jackson, Mississippi, 39216, United States
The Fragile X Spectrum Disorder Clinic at Icahn School of Medicine at Mount Sinai, Division of Medical Genetics
New York, New York, 10029, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Central States Research
Tulsa, Oklahoma, 74136, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Greenwood Genetic Center
Greenville, South Carolina, 29605, United States
Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
Westmead Children's Hospital
Sydney, New South Wales, 2145, Australia
Lady Cilento Children's Hospital - South Brisbane
Brisbane, Queensland, 4101, Australia
Genetics Clinics Australia
Melbourne, Victoria, 3161, Australia
Wellington Hospital
Wellington, 6021, New Zealand
Research Space
Leicester, Leicestershire, LE15WW, United Kingdom
Children's Clinical Research Facility, Royal Hospital for Children and Young People
Edinburgh, Lothian, EH105HF, United Kingdom
Manchester University NHS Foundation Trust
Manchester, M13 9WL, United Kingdom
Related Publications (2)
Berry-Kravis E, Hagerman R, Cohen J, Budimirovic D, Buchanan CB, Silove N, Tich N, Thibodeau A, Dobbins T, Sebree T, O'Quinn S, Albers DS, Bzdek KG, Nomikos G, Budur K. Long-term safety and tolerability of transdermal cannabidiol gel in children and adolescents with Fragile X syndrome (ZYN2-CL-017): an interim analysis of an ongoing open-label extension study. J Neurodev Disord. 2025 Nov 18;17(1):69. doi: 10.1186/s11689-025-09657-x.
PMID: 41254489DERIVEDHeussler 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.
PMID: 33395098DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 26, 2018
First Posted
January 14, 2019
Study Start
November 9, 2018
Primary Completion (Estimated)
August 31, 2027
Study Completion (Estimated)
August 31, 2027
Last Updated
November 5, 2025
Record last verified: 2025-02
Data Sharing
- IPD Sharing
- Will not share