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Clinical Trials

Zynerba starts phase 2 trial of ZYN002 in Fragile X syndrome

PBR Staff Writer Published 04 January 2017

Zynerba Pharmaceuticals has started an exploratory phase 2 clinical trial of ZYN002 cannabidiol (CBD) topical gel in children with Fragile X syndrome, genetic disorder that causes intellectual disability, behavioral and learning challenges.

The 16-subject FAB-C (Treatment of Fragile X Syndrome Anxiety and Behavioral Challenges with CBD) trial will assess 50 mg of CBD in ZYN002 4.2% gel once daily. It may be titrated up to 125 mg twice daily during the six-week titration period.

The primary outcome measures include changes in anxiety, depression and mood as measured by the ADAMS scale, a validated patient reported outcomes questionnaire.

Other measurements include the Aberrant Behavior Checklist and visual analog scale (VAS) to assess for hyperactivity/impulsivity.

Zynerba chairman and CEO Armando Anido said: “Fragile X syndrome is devastating to patients and their caregivers and we are very hopeful that ZYN002 CBD gel can provide relief for this underserved patient group.

“We anticipate having top line results for the FAB-C trial late in the first half of 2017.  With an orphan drug designation granted by the Food and Drug Administration for Fragile X syndrome, we look forward to working with the FDA to bring a treatment to patients as fast as possible.”

ZYN002 is designed to offer consistent, controlled drug delivery transdermally with twice-daily dosing.

Zynerba said transdermal delivery of ZYN002 prevents the gastrointestinal tract and potential stomach acid degradation.

In clinical studies, ZYN002 showed no reports of somnolence or fatigue and a very low incidence of gastrointestinal events.


Image: Zynerba starts phase 2 trial of ZYN002 in Fragile X syndrome. Photo: courtesy of jk1991 at FreeDigitalPhotos.net.