Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
NCT ID: NCT03802799
Last Updated: 2025-11-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
PHASE2/PHASE3
550 participants
INTERVENTIONAL
2018-11-09
2027-08-31
Brief Summary
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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.
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Detailed Description
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Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.
Participants from study ZYN-CL-016 who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours).
Participants from study ZYN-CL-016 who weigh more than 35 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours).
Participants from study ZYN-CL-033 who weigh less than or equal to 30 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours).
Participants from study ZYN-CL-033 who weigh more than 30 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours).
Participants from study ZYN-CL-033 who weigh more than 50 kg will receive 3 sachets of ZYN002, applied every 12 hours (± 2 hours).
At the Investigator's discretion, the dose may be increased or decreased at any time after the first month of treatment (for example due to weight changes during the course of the study).
Participants who are taking anti-epileptic drugs may have an additional one or two weeks of treatment after the 52 week treatment period to taper off study treatment.
Blood samples will be collected for safety analysis of ZYN002. 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 and/or with the participant and their parents/caregivers.
Patients that complete Month 12 will be allowed to continue for another 12 months of treatment if they qualify. Patients will be evaluated again annually at Months 24 and 36, and 48 in order to remain in the study.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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ZYN002
ZYN002 - cannabidiol Transdermal Gel
ZYN002 - Cannabidiol Transdermal Gel
Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
Interventions
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ZYN002 - Cannabidiol Transdermal Gel
Pharmaceutically manufactured. Cannabidiol formulated as a clear gel (transdermal delivery)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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.
3 Years
29 Years
ALL
No
Sponsors
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Zynerba Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Southwest Autism Research and Resource Center
Phoenix, Arizona, United States
Science 37
Culver City, California, United States
Amnova Clinical Research, LLC
Irvine, California, United States
UC Davis Health System, MIND Institute
Sacramento, California, United States
Children's Hospital of Colorado
Denver, Colorado, United States
Children's Research Institute
Washington D.C., District of Columbia, United States
University of Miami
Miami, Florida, United States
Han Phan
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Masonic Institute of Developing Brain
Minneapolis, Minnesota, United States
University of Mississipi Medical Center
Jackson, Mississippi, United States
The Fragile X Spectrum Disorder Clinic at Icahn School of Medicine at Mount Sinai, Division of Medical Genetics
New York, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Central States Research
Tulsa, Oklahoma, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Greenwood Genetic Center
Greenville, South Carolina, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Westmead Children's Hospital
Sydney, New South Wales, Australia
Lady Cilento Children's Hospital - South Brisbane
Brisbane, Queensland, Australia
Genetics Clinics Australia
Melbourne, Victoria, Australia
Wellington Hospital
Wellington, , New Zealand
Research Space
Leicester, Leicestershire, United Kingdom
Children's Clinical Research Facility, Royal Hospital for Children and Young People
Edinburgh, Lothian, United Kingdom
Manchester University NHS Foundation Trust
Manchester, , United Kingdom
Countries
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References
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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.
Other Identifiers
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ZYN2-CL-017
Identifier Type: -
Identifier Source: org_study_id
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