RecruitingPhase 2ACTRN12618001868279

Clinical study Of caNNabidiol in children and adolesCenTs with Fragile X Open-Label Extension (CONNECT-FX OLE)

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 – CONNECT-FX Open Label Extension (OLE)

Sponsor

Zynerba Pharmaceuticals Pty. Ltd

Enrollment

300 participants

Start Date

Nov 8, 2018

Study Type

Interventional

Conditions

Fragile X Syndrome

Summary

This study is evaluating the long term and safety and tolerability of ZYN002, a clear gel that can be applied to the skin (called transdermal application) twice a day for treatment of symptoms of Fragile X Syndrome (FXS) Who is it for? Patients who have been diagnosed with Fragile X Syndrome and are aged between 3 and 18 years old who have participated in the ZYN2-CL-016 double-blind study and meet certain eligibility criteria. Study details Eligible participants will undergo up to a 52-week treatment period. Participants who are taking anti-epileptic drugs may undergo an additional 1-2 weeks of treatment to taper off study drug treatment. All participants will be assigned to ZYN002. Parents/ caregivers will be instructed on proper application of the gel. The gel will be applied to clean, dry, intact skin of the upper arms/ shoulders. Participants whose weight changes during the course of the study may have their doses changed at the investigator’s discretion on or after the Month 1 visit, or reduced due to tolerability issues at investigator’s discretion. 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. Participation in this study may help the child’s/ adolescent’s FXS symptoms; however, we cannot guarantee that he/ she will get any benefits from this study. The results of this study may benefit future patients.

Eligibility

Sex: Both males and femalesMin Age: 3 YearssMax Age: 18 Yearss

Inclusion Criteria5

Participated in a certain number of visits in the ZYN2-CL-016 study
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 Criteria4

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, 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 as outlined below 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.

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Interventions

ZYN002 is a pharmaceutically manufactured Cannabidiol (CBD) 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, in

ZYN002 is a pharmaceutically manufactured Cannabidiol (CBD) 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. Only participants from the ZYN2-CL-016 study 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. Participants who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours). Participants who weigh more than 35 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours). At the Investigator’s discretion, the dose may be increased to a total of 4 sachets a day or decreased to a total of 2 sachets a day any time after the first month of treatment. 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. Participant compliance with the intervention will be monitored by the study coordinator through drug accountability at each study visit.