Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

Running, not enrolling · Phase 2/Phase 3

Conditions studied: Fragile X Syndrome

In brief

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.

Key facts

Study ID

NCT03802799

Run by

Zynerba Pharmaceuticals, Inc.

People needed

476

Starts

2018-11-09

Expected to finish

2026-06-30

Last updated by the study team

2026-05-27

Who can join

Age: 3 and older, up to 29. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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