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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT03802799
Other study ID # ZYN2-CL-017
Secondary ID
Status Enrolling by invitation
Phase Phase 2/Phase 3
First received
Last updated
Start date November 9, 2018
Est. completion date August 31, 2027

Study information

Verified date January 2024
Source Zynerba Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

This is an open-label extension, multiple-center study, to assess the long-term safety and tolerability of cannabidiol administered as ZYN002, a transdermal gel, for the treatment of child and adolescent patients with Fragile X Syndrome (FXS). Male and female patients with FXS will be treated for up to 72 months. Up to 450 male and female patients, ages 3 to 18 years will be enrolled. 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.


Recruitment information / eligibility

Status Enrolling by invitation
Enrollment 450
Est. completion date August 31, 2027
Est. primary completion date August 31, 2027
Accepts healthy volunteers No
Gender All
Age group 3 Years to 18 Years
Eligibility 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 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


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
Australia Lady Cilento Children's Hospital - South Brisbane Brisbane Queensland
Australia Genetics Clinics Australia Melbourne Victoria
Australia Westmead Children's Hospital Sydney New South Wales
New Zealand Wellington Hospital Wellington
United Kingdom Children's Clinical Research Facility, Royal Hospital for Children and Young People Edinburgh Lothian
United Kingdom Research Space Leicester Leicestershire
United Kingdom Manchester University NHS Foundation Trust Manchester
United States Emory University School of Medicine Atlanta Georgia
United States Han Phan Atlanta Georgia
United States Kennedy Krieger Institute Baltimore Maryland
United States Boston Children's Hospital Boston Massachusetts
United States Rush University Medical Center Chicago Illinois
United States Cincinnati Children's Hospital Medical Center Cincinnati Ohio
United States Science 37 Culver City California
United States Children's Hospital of Colorado Denver Colorado
United States Greenwood Genetic Center Greenville South Carolina
United States Penn State Milton S. Hershey Medical Center Hershey Pennsylvania
United States University of Mississipi Medical Center Jackson Mississippi
United States Suburban Research Associates Media Pennsylvania
United States University of Miami Miami Florida
United States Masonic Institute of Developing Brain Minneapolis Minnesota
United States Fragile X Center of Atlantic Health System Morristown New Jersey
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 Southwest Autism Research and Resource Center Phoenix Arizona
United States UC Davis Health System, MIND Institute Sacramento California
United States Primary Children's Hospital Salt Lake City Utah
United States University of Washington Center for Human Development and Disability Seattle Washington
United States Central States Research Tulsa Oklahoma

Sponsors (1)

Lead Sponsor Collaborator
Zynerba Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Australia,  New Zealand,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 72 month.
Secondary To evaluate the long-term efficacy of ZYN002 in the treatment of symptoms of FXS. Change from Baseline (last assessment prior to the first dose of ZYN002) in the ABC-CFXS subscale scores. Change from baseline to end of treatment
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