Rhythm Pharmaceuticals has enrolled the first patient in a pivotal Phase lll clinical trial examining setmelanotide for the treatment of patients with Bardet-Biedl Syndrome (BBS) and Alström Syndrome.
Both BBS and Alström Syndrome are rare genetic disorders and have multiple biological similarities. People suffering from these diseases experience an insatiable hunger and severe obesity early in life.
The pivotal Phase lll trial includes a multinational, open-label, single-arm study design.
It is expected to enrol 30 patients, including around 20 BBS patients and approximately six Alström Syndrome patients, aged six years and older.
During the trial’s 52 weeks of the treatment period, patients will receive a daily subcutaneous injection of setmelanotide. The trial will also comprise a 14-week placebo-controlled period.
The trial’s primary objective is a responder analysis after nearly 52 weeks of therapy.
Following the completion of enrolment, Rhythm plans to continue enrolling supplemental patients in order to gather additional safety and efficacy data in people living with BBS and Alström Syndrome.
The trial’s principal investigator Robert Haws said: “Many of our BBS patients struggle with insatiable hunger and severe obesity, which can be a challenging burden while also managing the disorder’s potential effects on vision, kidney function and other organs.”
Rhythm’s setmelanotide is an investigational potent, melanocortin-4 receptor (MC4R) agonist designed to treat rare genetic disorders of obesity.
Setmelanotide is developed to activate MC4R, which is a major biological pathway that independently controls energy expenditure and appetite.