Soleno Therapeutics, Inc. of Capnia reported in early July of this year that the company plans to start a Phase 3 clinical trial for evaluating diazoxide choline controlled-release (DCCR) in treating children affected with Prader-Willi syndrome. The company’s timeline is to begin this study by year’s end.
For those of you that don’t know, Prader-Willi syndrome is a rare genetic disorder that causes a constant and debilitating sense of hunger, resulting in a series of mental, physical, and behavioral complications. Currently, there is no specific treatment for the condition, only symptom management. To learn more about Prader-Willi syndrome, click here.
Soleno’s announcement comes after a successful meeting with the U.S. FDA, in which the company received constructive guidance for designing the study.
Officially, this Phase 3 clinical trial will be a double-blind, randomized and placebo-controlled study of about 100 Prader-Willi syndrome patients. The goal of the clinical trial (i.e. the primary endpoint) is for patients to witness an improvement in a hyperphagia score (even in the absence of a change of weight) compared to the placebo.
To clarify, hyperphagia is defined as the abnormally great desire for food and excessive eating, which is the characteristic symptom of Prader-Willi syndrome. Thus, researchers hope to determine that this new treatment will reduce the participants’ overall sense of hunger.
Furthermore, even after the study’s conclusion after a short quarter, the drug’s safety information will be attained in an ongoing, long-term manner.
So how does DCCR work?
DCCR is a tablet of a potassium channel activator that has been shown to be effective in inhibiting the secretion of insulin. It overall works to treat the root cause of Prader-Willi syndrome, instead of just merely suppressing the symptoms of the disease.
For this reason, the CEO of Soleno, Dr. Anish Bhatnagar, stated that he believes DCCR, if eventually approved, has the potential to be an urgently-needed, effective, and safe treatment for Prader-Willi syndrome.
Hopefully, following the conclusion of this study by early next year, the results will prove the Dr. Bhatnagar’s optimism, and children with Prader-Willi syndrome will finally feel relief from their symptoms.
If you want to read more about this exciting announcement from Rare Disease Report, click here and if you want read the original release in Global Newswire, click here.
