The top-line results of a pivotal Phase 3 study investigating the drug recorlevTM (levoketoconazole) in patients with endogenous Cushing’s syndrome have been released. The source article, which contains more detailed information, can be found here, at Globe Newswire.
About Cushing’s Syndrome
Cushing’s syndrome is an uncommon, serious condition that is caused by raised cortisol levels. Cortisol is a steroid hormone that plays a role in a range of body processes, including blood pressure, blood sugar, inflammation, and others. According to the NHS, Cushing’s syndrome usually affects people who are taking medicines that contain cortisol, but in some cases, the condition can be caused by the body over-producing cortisol.
Often, the symptoms of Cushing’s syndrome are varied and not clearly linked to one particular condition, such as skin easily bruising, stretch marks, a build-up of fat in certain areas, low libido, and depression. This can make reaching a diagnosis difficult, and for some patients, this process may take a long time. According to the source article, Cushing’s syndrome most commonly affects people between the ages of twenty and fifty, and the majority (about 70%) of people with the condition are female.
The ‘Sonics’ Study Methodology
The Sonics study is a pivotal Phase 3 study that took place over North America, Europe, and the Middle East. It is an open-label, single-arm study involving 94 patients.
The study comprised of three treatment phases. In the first phase, patients took recorlev twice per day, and the dosage was titrated (adjusted) until a therapeutic dose was reached. In the second phase, patients were given a fixed dose for six months and were then assessed for their urinary free cortisol response rate. In the third and final phase, patients continue for another six months.
The Top-line Results
The Sonics study met its main endpoint, with 30% of participants reaching a statistically significant normalisation rate of urinary free cortisol (UFC) after six months of maintenance treatment at a fixed dosage of recorlev. Urinary free cortisol tests measure the amount of cortisol in a person’s urine and can be used to help diagnose or track Cushing’s syndrome.
Furthermore, secondary endpoints of the study that evaluated markers of cardiovascular risk also showed improvement from baseline. These included measures of total cholesterol, LDL-cholesterol, body weight and BMI, haemoglobin A1C, and fasting blood glucose.
The drug was described as “generally well tolerated”; in total, twelve patients (just over 10%) stopped treatment because of adverse events.