Recruitment took place at 21 clinical sites in the United States, Australia, and New Zealand.
Topline results from the study are expected by August 2020. If the results are positive, Zynerba hopes to meet with the U.S. Food and Drug Administration (FDA) to file a New Drug Application for Zygel as a treatment for fragile X. The approval could potentially be granted by mid-2021.
“We are excited about the potential for Zygel, if approved by the FDA, to be the first drug ever indicated to treat the behavioral symptoms of Fragile X, and completion of enrollment in this trial is an important step toward that goal,” Armando Anido, chairman and CEO of Zynerba, said in a press release.
Zygel is a gel that is placed on the skin of the shoulder or upper arm to provide controlled amounts of cannabidiol (CBD) into the bloodstream.
CBD — a naturally occurring chemical derived from cannabis that does not make users “high” — is being tested as a potential treatment for modulating the endocannabinoid system, which plays a critical role in nerve cell communication and protection against damage. Impairments in this system are linked to the behavioral abnormalities in people with fragile X and other neurological conditions.
CONNECT-FX trial (NCT03614663) is a double-blind study assessing the efficacy and safety of Zygel in 210 fragile X syndrome patients, ages 3–17. Participants will be randomly assigned to receive either Zygel or a placebo.
Daily doses depend on the participants’ weight, with two sachets given to those weighing 35 kg about (77 pounds) or less, and four sachets administered to participants over 35 kg.
The primary goal of the study is to assess changes in social avoidance after 14 weeks of treatment compared with the start of the trial. Secondary goals include assessing changes in irritability, unresponsive social behavior, and other behavioral symptoms measured using the Clinical Global Impression (CGI-I) score.
Updated results of the Phase 1/2 FAB-C trial (ACTRN12617000150347) showed that treatment with Zygel was well-tolerated and resulted in clinically meaningful reductions in anxiety and other behavioral symptoms in children and teenagers with fragile X.
As intended, participants in CONNECT-FX trial have more severe symptoms — specifically worse baseline scores in the six subscales of the Aberrant Behavior Checklist-Community FXS Specific — compared to those enrolled in the FAB-C trial. This included measurements of social avoidance, irritability, hyperactivity, social unresponsiveness, stereotypy (purposeless repetitive movements), and inappropriate speech.
Caregivers were informed that patients who successfully completed the study may be given the opportunity to receive Zygel in an open-label extension trial (NCT03802799), regardless of their perceived response or actual treatment assignment in CONNECT-FX.
So far, 97% of the 158 participants who completed the initial trial have enrolled in this extension study.
“I want to thank everyone who participated in this achievement, particularly the patients in the study, their families, the clinicians involved in CONNECT-FX and their staff. We look forward to announcing the topline results of this trial late in the second quarter of this year,” said Anido.
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