Children with Prader-Willi syndrome (PWS) as young as 4 are now eligible to participate in the ongoing Phase 3 trial testing diazoxide choline controlled-release (DCCR) tablets for PWS initiated by Soleno Therapeutics earlier this year.
The original minimum age limit for enrollment was 8.
Feedback from the U.S. Food and Drug Administration on Soleno’s initial plan contributed to this protocol amendment to better reflect the demographics of the Prader-Willi syndrome population.
“This important protocol change to the ongoing Phase III trial of DCCR in PWS allows enrollment of hyperphagic [Prader-Willi] patients as young as four years of age, permitting inclusion of those children with an earlier age of hyperphagia onset,” Anish Bhatnagar, MD, the CEO of Soleno, said in a press release.
Hyperphagia, or increased appetite, is a classic symptom of PWS and can occur in children as young as 4.
“Managing hyperphagia in PWS patients is a critical unmet need for which there are no currently approved treatments,” said Jennifer L. Miller, MD, lead investigator of the study and associate professor in the division of pediatric endocrinology at the University of Florida.
Under this amendment, a total of up to 105 Prader-Willi patients, ages 4 and older, will be enrolled in the DESTINY PWS Phase 3 study (NCT03440814).
Patients will be randomly selected to receive either the placebo or oral DCCR once daily. The main outcome (primary endpoint) of the study is a change in hyperphagia severity after 13 weeks of treatment.
About 15 different centers across the United States are involved in the trial. Soleno hopes to complete the study in the first half of 2019.
DCCR is a potassium channel opener or activator. Open potassium channels promote fat metabolism in the body and can lower the secretion of appetite-stimulating hormones. DCCR received FDA fast-track designation as a PWS therapy in July 2018.
Recently, Soleno was awarded a new U.S. patent for the formulation of DCCR tablets (formulations of diazoxide and diazoxide choline) for the treatment of Prader-Willi syndrome.
Diazoxide choline has also been granted orphan drug designation for the treatment of PWS in the U.S. and EU.
Diazoxide has been approved for the treatment of conditions such as secondary hyperinsulinemia (high levels of insulin in the blood) under the brand name Proglycem, but it has not been approved for Prader-Willi syndrome.
“Because the parent molecule in DCCR has been approved for use in neonates through adults for other conditions, and at much higher daily doses than are being used in this study, its safety profile is well understood,” Miller said. “We are pleased to continue evaluating this promising product candidate in PWS patients with hyperphagia in all ages in the ongoing Phase III trial.”
Bhatnagar added that Soleno will “continue to work with our investigators, as well as the PWS community, to complete enrollment and advance this trial through the clinic as expeditiously as possible.”
Patients who successfully complete the trial will be eligible to participate in an extended, open-label study testing the continual safety of DCCR. The extended study will last for nine months.