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

Fragile X Syndrome Clinical Trial

Official 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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03802799
• Other study ID #: ZYN2-CL-017
• Status: Enrolling by invitation
• Phase: Phase 2/Phase 3
• 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
• 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.

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

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