Efficacy and safety of long-acting pasireotide in Japanese patients with acromegaly or pituitary gigantism: results from a multicenter, open-label, randomized, phase 2 study

Endocrine journal

Tomomi Kaneko¹  Shigeyuki Tahara³  Mami Murakami⁴  Akira Shimatsu⁴ 

[1] Medical affairs, Novartis Pharma K.K., Tokyo 105-6333, Japan;Clinical Research Institute, National Hospital Organization Kyoto Medical Center, Kyoto 612-8555, Japan;Department of Neurosurgery, Nippon Medical School, Tokyo 113-8602, Japan;Oncology development &

关键词: Pasireotide;    Acromegaly;    Growth hormone;    Insulin-like growth factor-1;    Japanese;   

DOI  :  10.1507/endocrj.EJ16-0624

学科分类：内分泌与代谢学

来源: Japan Endocrine Society

【 摘 要 】

A multicenter, open-label, phase 2 study was conducted to investigate the efficacy and safety of long-acting pasireotide formulation in Japanese patients with acromegaly or pituitary gigantism.Medically naïve or inadequately controlled patients (on somatostatin analogues or dopamine agonists) were included.Primary end point was the proportion of all patients who achieved biochemical control (mean growth hormone [GH] levels<2.5μg/L and normalized insulin-like growth factor-1 [IGF-1]) at month 3.Thirty-three patients (acromegaly, n=32; pituitary gigantism, n=1) were enrolled and randomized 111 to receive open-label pasireotide 20mg, 40mg, or 60mg.The median age was 52 years (range, 31-79) and 20 patients were males.At month 3, 18.2% of patients (6/33; 90% confidence interval 8.2%, 32.8%) had biochemical control (21.2% [7/33] when including a patient with mean GH<2.5μg/L and IGF-1< lower limit of normal).Reductions in the median GH and IGF-1 levels observed at month 3 were maintained up to month 12; the median percent change from baseline to month 12 in GH and IGF-1 levels were -74.71% and -59.33%, respectively.Twenty-nine patients completed the 12-month core phase, 1 withdrew consent, and 3 discontinued treatment due to adverse events (AEs; diabetes mellitus, hyperglycemia, liver function abnormality, n=1 each).Almost all patients (97%; 32/33) experienced AEs; the most common AEs were nasopharyngitis (48.5%), hyperglycemia (42.4%), diabetes mellitus (24.2%), constipation (18.2%), and hypoglycemia (15.2%).Serious AEs were reported in 7 patients with the most common being hyperglycemia (n=2).Long-acting pasireotide demonstrated clinically relevant efficacy and was well tolerated in Japanese patients with acromegaly or pituitary gigantism.

【 授权许可】

Unknown