As a result, Zynerba informed clinical sites that screening has closed. The company expects to include at least 204 participants, ages 3 to 17, with fragile X syndrome. Topline results are expected in the second quarter of 2020.
Before being enrolled in the CONNECT-FX trial (NCT03614663), patients were screened to assess the severity of their symptoms and whether they were taking any medication. Their medical history was reviewed, including genetic confirmation of their diagnosis.
Participants will be randomly assigned to treatment with Zygel or a placebo. Daily doses will depend on body weight, with two sachets given to those weighing 35 kg (about 77 pounds) or less, and four sachets administered to heavier participants.
The study’s primary goal is to determine changes in social avoidance after 14 weeks of treatment. Pending positive results, Zynerba is expected to meet with the U.S. Food and Drug Administration with the ultimate goal of getting regulatory approval for Zygel by mid-year in 2021.
Those who successfully complete the study may continue on Zygel in an open-label extension trial (NCT03802799) regardless of perceived response or treatment assignment in the initial trial.
“This is an important milestone for patients with Fragile X syndrome and their caregivers as we move toward completion of enrollment in this pivotal trial,” Armando Anido, Zynerba’s CEO, said in a press release.
“I would like to thank all of our clinical investigators and their staff for their assistance thus far. Through strict entry criteria and overall trial design, we expect to enhance the study’s ability to demonstrate a strong signal of activity and minimize response variability,” he added.
The endocannabinoid system has a crucial role in nerve cell communication and protection against damage. Cannabidiol (CBD) — a naturally occurring chemical derived from cannabis that does not produce psychoactive effects or a “high” — is being tested as a potential treatment for modulating the endocannabinoid system. Zygel is intended to provide controlled amounts of CBD into the bloodstream via the skin.
In FAB-C — which stands for Treatment of Fragile X Syndrome Anxiety and Behavioral Challenges with CBD — a total of 20 participants, 6–17 years old, received Zygel twice daily for 12 weeks as an add-on therapy. During the first six weeks, the researchers tested increasing doses of the gel, ranging from 50 mg up to 250 mg daily. From week 7, participants received Zygel at a stable dose — the highest tolerated dose given during the first six weeks.
Results showed that Zygel was well-tolerated and led to clinically meaningful reductions in anxiety and other behavioral symptoms in children and teenagers with fragile X.