This study is the first Phase III trial to demonstrate the efficacy of a medical therapy for Cushing’s disease, which is a debilitating endocrine disorder caused by excess cortisol in the body due to the presence of a non-cancerous pituitary tumor.
Patients were randomized to receive pasireotide subcutaneous (sc) injection in doses of 900 micrograms or 600 micrograms twice daily.
For the 900 microgram group, the study met the primary endpoint of normalizing urinary-free cortisol (UFC) levels, the key measure of biochemical control of the disease.
UFC levels were normalized in 26.3% and 14.6% of patients with Cushing’s disease randomized to receive pasireotide 900 micrograms and 600 micrograms twice daily, respectively, at six months of treatment. After 12 months of treatment, results confirmed the durability of the effect.
lead study investigator and chief of the Neuroendocrine Unit at the Department of Molecular and Clinical Endocrinology and Oncology, Federico II University of Naples Annamaria Colao said, "These data on pasireotide are the first to show a therapeutic treatment can help patients achieve biochemical control of their Cushing’s disease, while improving associated symptoms."
Study results also showed that cortisol levels decreased quickly in the majority of patients, with a median decrease of approximately 50% by month two, and remained stable in both groups through the end of the study.
Investigators listed diarrhea, nausea, hyperglycemia, cholelithiasis, abdominal pain, diabetes mellitus, injection site reactions, fatigue and increased glycosylated hemoglobin (HbA1c), with most events being Grade 1-2 as the most frequently reported adverse events (>10%) for pasireotide.
The trial is the basis for regulatory submissions for pasireotide under way worldwide for the treatment of this condition, and if approved, the brand name for pasireotide will be Signifor.