Clinical Trial on DCCR As PWS Treatment: Results + Next Phase [VIDEO]

Update: DCCR is currently enrolling for its Phase 3 study. Information on this study can be found here

This blog contains excerpts from a presentation on a PWS clinical trial of DCCR, the diazoxide choline controlled release tablet. The presentation was given at the FPWR 2017 conference by Dr. Neil Cowen, president and chief scientific officer of Essentialis Therapeutics, as part of a PWS clinical trials update. You can watch his complete presentation by clicking on the embedded video below.

In case you don't have time to watch the full video, we've captured some of the key points in the notes below.


DCCR – Extensive Pre-Clinical and Clinical Data


  • Historically approved product
  • Approved to treat rare diseases in adults and young children relating to hypoglycemia
  • Used as an oral suspension
  • Rare disease conditions occur about one per million
  • Health and safety data exists that helps drive decisions about the drug

Diazoxide Choline Controlled Release (DCCR) Tablet

  • Once a day tablet
  • 8 clinical trials so far, including phases 1 and 2
  • Studies done in obese, dyslipidemic patients and patients with PWS
  • Treated more than 200 patients 
  • Protected by multiple issued patents

Appetite and Hyperphagia

  • Appetite is regulated by a couple sets of neurons in the hypothalamus
  • One set of neurons, NPY/AgRP, acts as the accelerator of appetite
  • The second set, the POMC neurons, act as the brakes
  • Neurons are regulated by leptin and insulin, hormones produced elsewhere in the body
  • Insulin regulates so that after a meal, your hunger level drops
  • Leptin relates to the longer term: If you’ve eaten well for a long period of time, your appetite is suppressed
  • In PWS, because of the loss of at least one gene, the appetite accelerator is active, but the brakes aren’t working

Treatment with DCCR

  • Drug amplifies effects of leptin and insulin
  • Reduces hyperphagia by easing up off the accelerator and applying the brakes 

DCCR Study Involving Patients with PWS

  • Randomized
  • Single-center
  • Overweight or obese patients
  • Genetically confirmed PWS diagnosis
  • Ages 10-22
  • 10 weeks: patients knew they were on the drug
  • Drug doses were gradually increased
  • Phase 2 entered a double-blind period in which half the patients were switched to placebo and half remained on the drug
  • Some came back for a six-month treatment period

How Did They Measure Hyperphagia?

  • Questionnaire by parent / caregiver about food-related behaviors
  • As patient doses increased, researchers saw improvements in hyperphagia
  • There was a 30-35 percent reduction in hyperphagia
  • Higher doses led to increased improvement
  • When patients were moved to placebo, they shifted back toward baseline
  • Those who stayed on DCCR tended to have improvement maintained 

Body Composition Changes at 10 Weeks

  • In patients who finished the 10-week, open-label phase of the study, body fat was reduced by almost 4 percent
  • Lean body mass increased by about 5 percent
  • The ratio of lean body mass to body fat increased almost 10 percent
  • At the dose being targeted in the future study, patients experienced a six percent loss of body fat
  • They gained almost 9 percent of lean body mass
  • Ratio improved by almost 17 percent
  • Results were reproduced over 6 months

Aggressive and Destructive Behaviors

  • Unrelated to the hyperphagia, at baseline, researchers looked at a series of aggressive behaviors
  • At the beginning of the study, about 70 percent of patients had aggressive behaviors
  • By 10 weeks of treatment, only 30 percent were displaying the behaviors 
  • Patients had to completely stop the behavior before it was noted as an improvement
  • If it’s reproducible, it’s a major benefit of the drug, in addition to the impact on hyperphagia

Improvements in Cholesterol Levels

  • Patients had expected reductions in bad cholesterol and increases in good cholesterol 
  • Patients who remained for the double-blind study saw a 45 percent reduction in triglycerides

DCCR’s Safety Profile

  • Safety profile is well understood
  • Drug has been used in eight trials
  • The parent molecule has been used frequently
  • There 120,000 patient years of data at doses above the dose they’re planning to use in PWS
  • No new safety issues have emerged

Audience Q&A

  • After the Phase 3 study, there will be an open-label treatment trial that patients can enroll in
  • A study in Australia found that if gamma aminobutyric acid (GABA), a neurotransmitter, was low, there was aggressive behavior in PWS. Researchers think they amplify GABA signalling when improving the behavior.

For updated information on PWS clinical trial opportunities and to sign up for a monthly PWS Clinical Trial Alert, visit our PWS Clinical Trials page.

PWS Clinical Trials

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 natural history of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she hosted her first One SMALL Step walk in 2010 and began the development of the One SMALL Step walk program which now raises over $1.5 million a year for PWS research.