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

Phase 2

Recruiting

• Conditions: Fragile X Syndrome; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders; Neurological - Other neurological disorders

• Registration Number: ACTRN12618001868279
• Lead Sponsor: Zynerba Pharmaceuticals Pty. Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-11-16
• Last Update Posted: 26 November 2018

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

1.Participated in a certain number of visits in the ZYN2-CL-016 study
2.Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.
3.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.
4.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.
5.Females of childbearing potential must have a negative pregnancy test at all designated visits

Exclusion Criteria

1.Patient is receiving any investigational drugs (not ZYN002) or using any experimental devices.
2.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.
3.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.
4.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

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To evaluate the long-term safety and tolerability of ZYN002. <br>Safety assessments will include collection of any adverse events, physical and neurological exams (including Tanner staging), clinical laboratory safety assessments (hematology, chemistry and urinalysis), vital signs and 12-lead ECGs. Finally, as the study drug is delivered transdermally, tolerability to study drug application will be assessed through skin check examinations performed at each visit after treatment with study gel has been initiated.<br>[Safety and tolerability will be assessed at every study visit from Day 1 to end of treatment, including Day 1, Months 1, 3, 6, 9 and 12, any Unscheduled Visits and the taper period.]

Secondary Outcome Measures

Name	Time	Method
The change in the Aberrant Behavior Checklist – Community Fragile X factor (ABC-C) Pre-specified Subscale 1[Change from Baseline to End of Treatment. Subscale 1 to be assessed at all study visits: Months 1, 3, 6, 9 and 12/Early Termination (ET)];Change in the ABC-C Fragile X factor Pre-specifed Subscale 2[Change from Baseline to End of Treatment. Subscale 2 to be assessed at all study visits: Months 1, 3, 6, 9 and 12/Early Termination (ET)];Change in the ABC-C Fragile X factor Pre-specifed Subscale 3[Change from Baseline to End of Treatment. Subscale 3 to be assessed at all study visits: Months 1, 3, 6, 9 and 12/Early Termination (ET)];Change in Caregiver Global Impression of Change (CGI-)I[Change from Baseline to End of Treatment. Assessed at Months 6 and 12/ET]