Insys Therapeutics’ Phase 2 trial to evaluate the safety and effectiveness of its cannabidiol (CBD) oral solution in pediatric patients with Prader-Willi syndrome (PWS) will soon begin recruiting participants.
More information about the study and dates of enrollment will be announced on the clinical trial’s official webpage, found here (NCT02844933).
The Insys announcement also recognizes May as PWS Awareness Month.
The trial expects to enroll 66 children and teens, ages 8 to 17, at 10 clinical sites in the United States. Patients will be randomized to receive either the CBD oral solution (delivered as 40 mg/kg per day, divided into two daily doses) or a matching placebo control.
The trial’s primary objective (endpoint) is to determine the therapy’s impact on patients hyperphagia — an insatiable appetite that characterizes Prader-Willi syndrome and is linked to obesity, type 2 diabetes, and premature death.
Researchers will measure patient outcomes as changes from the start of the study through 13 weeks of treatment through the Hyperphagia Questionnaire for Clinical Trial.
Additional (secondary) endpoints will evaluate changes in total body weight, patient global impression of change and severity, quality of life, physical activity and fatigue, and other parameters.
Results from the cannabidiol trial are expected by the end of 2019.
In December 2017, Insys’ oral cannabidiol solution for the treatment of Prader-Willi syndrome was granted fast track designation by the U.S. Food and Drug Administration.
“Individuals with Prader-Willi syndrome face relentless hunger, which requires strict environmental controls, such as locking pantries and refrigerators to prevent life-threatening obesity,” Theresa Strong, director of research programs at the Foundation for Prader-Willi Research, said in a press release.
“Currently there are no treatments for the most challenging aspects of the disorder, which takes a toll on patients’ families as well,” she added.
“While Prader-Willi syndrome presents a complex constellation of symptoms to address, our study will focus on one of the most challenging, hyperphagia,” said Steve Sherman, senior vice president of regulatory affairs for Insys. “Our formulation of CBD has the potential to be an important addition to the limited range of treatment options for this condition.”
Insys is also evaluating its cannabidiol oral formulation in a Phase 3 clinical trial in infantile spasms and a Phase 2 trial in childhood absence epilepsy.