ZYN2-CL-017 - OLE
Research type
Research Study
Full title
An Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults with Fragile X Syndrome
IRAS ID
308782
Contact name
Andrew Stanfield, Dr
Contact email
Sponsor organisation
Zynerba Pharmaceuticals, Inc.
Eudract number
2021-006459-33
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
IND number, 130876
Duration of Study in the UK
4 years, 3 months, 0 days
Research summary
Fragile X Syndrome (FXS) is a genetic condition that causes intellectual disability, behavioural and learning challenges, and various physical characteristics. Though FXS occurs in both genders, males are more frequently affected than females, and generally with greater severity. People with FXS commonly display attention deficit and hyperactivity, anxiety with mood lability and autistic behaviours.
There is a high unmet need for effective therapies in the treatment of FXS, with current medications aimed at treating co-occurring symptoms of rather than focused at the underlying mechanism.
This is an open-label extension, multi-centre study, to investigate how tolerable and safe the study medication (ZYN002, a transdermal gel) is over a long term (for up to 48 months), in male and female children and adolescent patients with FXS.
Patients will be enrolled from the following patient populations:
-Roll-over Patients Who Completed Study ZYN2-CL-033 (RECONNECT);
-Patients Who Were Not Eligible for Randomisation at Visit 3 of Study ZYN2-CL-
033 (RECONNECT)
-Patients Who Were Screen Failures at Screening or Visit 2 of Study ZYN2-CL-033 (RECONNECT)Approximately 450 participants will be enrolled at approximately 25 clinical sites in Australia, Ireland, New Zealand, United Kingdom, and the United States.
There is no guarantee that participants will directly benefit from taking part in this study.
The study is being sponsored and funded by Zynerba Pharmaceuticals, Inc., United States, and Zynerba Pharmaceuticals Pty., Ltd., Australia.
REC name
Scotland A: Adults with Incapacity only
REC reference
22/SS/0029
Date of REC Opinion
6 May 2022
REC opinion
Favourable Opinion