Soleno Therapeutics has started a Phase III clinical trial of Diazoxide Choline Controlled-Release (DCCR) tablet for the treatment of patients with Prader-Willi Syndrome (PWS).
The randomised, placebo-controlled trial will enrol around 100 PWS patients at 10-15 sites in the US.
Seattle Children’s Hospital in the US is the first site to be activated for the trial, which is expected to be completed over a period of nine to 12 months.
Soleno has appointed Dr Parisa Salehi as the Principal Investigator for the trial at the Seattle Children’s Hospital site.
Salehi said: “Prader-Willi Syndrome leads to hyperphagia that can cause life-threatening obesity if left uncontrolled.
“This excessive hunger can cause significant harm to the lives of these individuals and their families. There is a lack of effective medical therapy targeting hunger in this population, and such a drug would be life-altering.
“Based on the data generated to date, DCCR has the potential to address this treatment void.”
DCCR is a new, extended-release, crystalline salt formulation of diazoxide, which is given once-daily to the patients and has been used for ages for treating patients suffering from a few rare diseases in neonates, infants, children and adults.
The tablet, however, has not been approved for use in PWS.
According to Prader-Willi Syndrome Association USA, one in 12,000 to 15,000 people in the US is estimated to have PWS, which leads to hyperphagia, a chronic feeling of insatiable hunger that significantly reduces the quality of life for PWS patients and their families.