Phase 2 Study Results Published: Diazoxide Choline Controlled-Release

In 2015 FPWR co-funded a phase 2 study to investigate diazoxide choline controlled-release in patients with PWS. Results from the study were recently published in PLoS ONE and are summarized below.

The phase 2 study included a 10-week Open-Label Treatment Period during which time subjects were dose escalated, followed by a 4-week Double-Blind, Placebo Controlled Treatment Period.

13 overweight or obese, adolescent and adult subjects with genetically-confirmed PWS with an average age of 15.5±2.9 years were enrolled in the study. There was a statistically significant reduction in hyperphagia at the end of the Open-Label Treatment Period (-4.32, n = 11, p = 0.006). The onset of effect on hyperphagia was rapid and greater reductions in hyperphagia were seen in subjects with moderate to severe Baseline hyperphagia (-5.50, n = 6, p = 0.03), in subjects treated with the highest dose (-6.25, n = 4, p = 0.08), and in subjects with moderate to severe Baseline hyperphagia treated with the highest dose (-7.83, n = 3, p = 0.09).

DCCR treatment resulted in:

  • a reduction in the number of subjects displaying aggressive behaviors (-57.1%, n = 10, p = 0.01)
  • clinically relevant reductions in fat mass (-1.58 kg, n = 11, p = 0.02), and
  • increases in lean body mass (2.26 kg, n = 11, p = 0.003).

There was a corresponding decrease in waist circumference, and trends for improvements in lipids and insulin resistance. The most common adverse events were peripheral edema and transient increases in glucose. Many of the adverse events were common medical complications of PWS and diazoxide.

A phase 3 study of DCCR is now underway and enrolling people with PWS ages 4 and up.

Learn more about the phase 3 study on our PWS clinical trials directory.


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Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.