ISSN 1470-3947 (print) | ISSN 1479-6848 (online)

Endocrine Abstracts (2017) 49 EP1020 | DOI: 10.1530/endoabs.49.EP1020

European observational study of long-acting pasireotide for uncontrolled acromegaly: ACRONIS study design and rationale

Andrea Giustina1, Gesine Enderle2, Daniela Mesenka3, Debolina Mondal4 & Christof Schöfl5

1Cattedra di Endocrinologia, Universita’ Vita e Salute San Raffaele, Milan, Italy; 2Novartis Farma S.p.A, Origgio, Italy; 3Novartis s.r.o., Prague, Czech Republic; 4Novartis Healthcare Pvt. Ltd., Hyderabad, India; 5Centre of Endocrinology and Metabolism, Obstmarkt 1, 96047 Bamberg, Germany.

Introduction: Acromegaly is a rare, serious disease caused by the presence of a pituitary adenoma secreting an excessive amount of growth hormone (GH), which leads to a consequent increase in circulating insulin-like growth factor-1 (IGF-1) levels, and excessive skeletal growth and soft tissue enlargement. It is usually managed by surgery and/or treatment with somatostatin analogs (SSA). The ACRONIS study (CSOM230CIC05) will provide real-world evidence on the efficacy and safety of the multi-targeted SSA long-acting pasireotide in uncontrolled acromegaly patients.

Methods: This non-interventional study will analyze efficacy and safety data in acromegaly patients (≥18 years) already treated (for ≥6 months) with monthly pasireotide (retrospective dataset) or about to be treated (prospective dataset). About 200 prospective patients (2-year follow-up) and 50 retrospective patients are expected to be enrolled. Outcome measures will include biomarkers (GH and IGF-1), pituitary imaging, symptom- and quality of life (QoL) scores. The primary objective is to document treatment efficacy within the prospective data subset; the endpoint will be the proportion of patients who achieve IGF-1 <1 upper limit of normal (ULN) and GH <1 μg/l after 6 months. Secondary endpoints will include the proportion of patients at 6 months (retrospective) or within 2 years (prospective) who achieve IGF-1 <1 or <1.3 ULN and GH <1 or 2.5 μg/l, change in tumor size, signs and symptoms, QoL and incidence of hyperglycemia. Safety assessments will include adverse events, hematology, clinical chemistry and physical examinations. Interim analyses will be performed once at least 50 retrospective patients have been exposed to 6 months of treatment and 100 prospective patients have been treated for at least 6 months, respectively. Descriptive statistics, as well as, absolute and relative frequencies will be reported.

Conclusion: ACRONIS will provide European real-world data to increase our understanding of the long-term efficacy and safety of pasireotide in uncontrolled acromegaly.

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