CSOM230B2410: Non-interventional study for the generation of long term safety and efficacy data of pasireotide s.c. in patients with Cushing's disease (Post-Authorization Safety Study)

J Schopohl; J Aberle; J Fleck; A Pedroncelli; M Droste

doi:10.1055/s-0034-1372144

Experimental and Clinical Endocrinology & Diabetes, Table of Contents

CSOM230B2410: Non-interventional study for the generation of long term safety and efficacy data of pasireotide s.c. in patients with Cushing's disease (Post-Authorization Safety Study)

J Schopohl ¹, J Aberle ², J Fleck ³, A Pedroncelli ³, M Droste ⁴

¹Klinikum der LMU München, Medizinische Klinik und Poliklinik IV, Munich, Germany
²Universtitätsklinikum Hamburg-Eppendorf, III. Medizinische Klinik und Poliklinik, Hamburg, Germany
³Novartis Pharma AG, Basel, Switzerland
⁴Praxis für Endokrinologie und Diabetologie, Oldenburg, Germany

Abstract

Introduction: With an annual incidence of 0.7 – 2.4 per million, Cushing's disease (CD) is a rare but devastating disease caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. CD is associated with severe morbidity and mortality and most commonly affects adults aged 20 – 50, primarily females. Pituitary resection of the adenoma is the current first-line therapy for CD, but surgical failure rates are as high as 25 – 30%. Pasireotide (SOM230), a novel somatostatin analogue, exhibits a unique binding profile with high affinity to four of the five known human somatostatin receptor subtypes (sstr1, 2, 3 and 5) and is approved for pharmacological treatment of CD in the EU and U.S. for those patients for whom surgery has failed or is not an option. A phase III study [CSOM230B2305] showed efficacy of pasireotide s.c. in reducing and normalising hypercortisolism. However, since duration and patient numbers are limited, long-term effects could not be fully addressed.

Objective: The primary objective of this non-interventional study [CSOM230B2410] is therefore to document the long-term safety and tolerability profile of pasireotide s.c. in patients with CD in a real life setting.

Design: This multinational, multi-center post-marketing observational study does not impose a therapy protocol, diagnostic/therapeutic interventions or a visit schedule. Patients ≥18 years with a diagnosis of Cushing's disease and treated with pasireotide s.c. alone or in combination with other therapies will be monitored over a period of 3 years. Treatment is performed according to the investigator's judgment and local prescribing information. All adverse events as well as available demographic, medical history and current medical data will be documented at patients' visits to their site. Additionally, efficacy data will be collected and for documentation of changes in health-related quality of life, patient-reported outcome questionnaires (Cushing QoL, EURO QoL) are available.