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

behalf of SOM230C1202 study group

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

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 1:1:1 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.

Original languageEnglish
Pages (from-to)735-747
Number of pages13
JournalEndocrine Journal
Volume64
Issue number7
DOIs
Publication statusPublished - 01-01-2017

Fingerprint

Gigantism
Acromegaly
Safety
Somatomedins
Growth Hormone
Hyperglycemia
Diabetes Mellitus
pasireotide
Nasopharyngitis
Dopamine Agonists
Constipation
Somatostatin
Hypoglycemia

All Science Journal Classification (ASJC) codes

  • Endocrinology, Diabetes and Metabolism
  • Endocrinology

Cite this

@article{0f716c40f6454e5b925ae029ed74bdd6,
title = "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",
abstract = "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 na{\"i}ve 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 1:1:1 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.",
author = "{behalf of SOM230C1202 study group} and Shigeyuki Tahara and Mami Murakami and Tomomi Kaneko and Akira Shimatsu and Masanobu Yamada and Nobuaki Ito and Atsuhiro Ichihara and Shozo Yamada and Hiroshi Arima and Chikara Shimizu and Michio Otsuki and Yutaka Takahashi and Tatsuhide Inoue and Tomoaki Tanaka and Wataru Kameda and Fumio Otsuka and Kazunori Arita and Atsushi Suzuki and Masafumi Fukagawa and Jun Saito and Shigeru Nishizawa and Kuniaki Ogasawara and Yuji Tanaka and Fumitoshi Satoh and Atsushi Suzuki and Masayuki Hosoi and Sachiko Honjo and Sanae Midorikawa and Akira Haketa and Masatoshi Nomura and Hidenori Fukuoka and Masataka Kudo and Koutaro Kurasaki and Ryo Morimoto and Yosuke Ono and Toshiro Seki and Kishio Touma and Tsukasa Wada and Keiko Yamagami and Chiho Yamamoto and Kohei Yamamoto",
year = "2017",
month = "1",
day = "1",
doi = "10.1507/endocrj.EJ16-0624",
language = "English",
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pages = "735--747",
journal = "Endocrine Journal",
issn = "0918-8959",
publisher = "Japan Endocrine Society",
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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. / behalf of SOM230C1202 study group.

In: Endocrine Journal, Vol. 64, No. 7, 01.01.2017, p. 735-747.

Research output: Contribution to journalArticle

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T1 - Efficacy and safety of long-acting pasireotide in japanese patients with acromegaly or pituitary gigantism

T2 - Results from a multicenter, open-label, randomized, phase 2 study

AU - behalf of SOM230C1202 study group

AU - Tahara, Shigeyuki

AU - Murakami, Mami

AU - Kaneko, Tomomi

AU - Shimatsu, Akira

AU - Yamada, Masanobu

AU - Ito, Nobuaki

AU - Ichihara, Atsuhiro

AU - Yamada, Shozo

AU - Arima, Hiroshi

AU - Shimizu, Chikara

AU - Otsuki, Michio

AU - Takahashi, Yutaka

AU - Inoue, Tatsuhide

AU - Tanaka, Tomoaki

AU - Kameda, Wataru

AU - Otsuka, Fumio

AU - Arita, Kazunori

AU - Suzuki, Atsushi

AU - Fukagawa, Masafumi

AU - Saito, Jun

AU - Nishizawa, Shigeru

AU - Ogasawara, Kuniaki

AU - Tanaka, Yuji

AU - Satoh, Fumitoshi

AU - Suzuki, Atsushi

AU - Hosoi, Masayuki

AU - Honjo, Sachiko

AU - Midorikawa, Sanae

AU - Haketa, Akira

AU - Nomura, Masatoshi

AU - Fukuoka, Hidenori

AU - Kudo, Masataka

AU - Kurasaki, Koutaro

AU - Morimoto, Ryo

AU - Ono, Yosuke

AU - Seki, Toshiro

AU - Touma, Kishio

AU - Wada, Tsukasa

AU - Yamagami, Keiko

AU - Yamamoto, Chiho

AU - Yamamoto, Kohei

PY - 2017/1/1

Y1 - 2017/1/1

N2 - 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 1:1:1 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.

AB - 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 1:1:1 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.

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