Long-term efficacy and safety of subcutaneous pasireotide alone or in combination with cabergoline in Cushing's disease.

Feelders RA, Fleseriu M, Kadioglu P, Bex M, González-Devia D, Boguszewski CL, Yavuz DG, Patino H, Pedroncelli AM, Maamari R, Chattopadhyay A, Biller BMK, Pivonello R

Frontiers in endocrinology9 Oct 2023Cited by 16 (Europe PMC)

PMID37876540PMCIDPMC10593462DOI10.3389/fendo.2023.1165681PepBankPUB-37876540

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Summary

ObjectiveThis study evaluated short- and long-term efficacy and safety of the second-generation somatostatin receptor ligand pasireotide alone or in combination with dopamine agonist cabergoline in patients with Cushing's disease (CD).Study designThis is an open-label, multicenter, non-comparative, Phase II study comprising 35-week core phase and an optional extension phase. All patients started with pasireotide, and cabergoline was added if cortisol remained elevated. Eligible patients had active CD, with or without prior surgery, were pasireotide naïve at screening or had discontinued pasireotide for reasons other than safety. Primary endpoint was proportion of patients with a mean urinary free cortisol (mUFC) level not exceeding the upper limit of normal (ULN) at week 35 with missing data imputed using last available post-baseline assessments.ResultsOf 68 patients enrolled, 26 (38.2%) received pasireotide monotherapy and 42 (61.8%) received pasireotide plus cabergoline during the core phase. Thirty-four patients (50.0%; 95% CI 37.6-62.4) achieved the primary endpoint, of whom 17 (50.0%) received pasireotide monotherapy and 17 (50.0%) received combination therapy. Proportion of patients with mUFC control remained stable during the extension phase up to week 99. Treatment with either mono or combination therapy provided sustained improvements in clinical symptoms of hypercortisolism up to week 99. Hyperglycemia and nausea (51.5% each), diarrhea (44.1%) and cholelithiasis (33.8%) were the most frequent adverse events.ConclusionAddition of cabergoline in patients with persistently elevated mUFC on maximum tolerated doses of pasireotide is an effective and well-tolerated long-term strategy for enhancing control of hypercortisolism in some CD patients.Clinical trial registrationhttps://clinicaltrials.gov/ct2/show/NCT01915303, identifier NCT01915303.

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study2

2 results from studies that gave the peptide. 2 results from people.

01HumanNon-randomized trialEfficacy outcome
In Cushing's disease, 17 patients receiving pasireotide monotherapy achieved mUFC not exceeding ULN at week 35, representing 50.0% of primary-endpoint responders.
In plain wordsIn Cushing's disease, 17 pasireotide monotherapy patients achieved urinary cortisol control at week 35.
Peptide
Pasireotide
Outcome
Not applicablemUFC control at week 35
Study
n = 26
Analysis
Primary
Outcome group
Disease control · Endocrine and hormones
Studied dose
Dose not stated · subcutaneousAs given in this study (human).
Measurements
Monotherapy responders
17 patients
primary endpoint
Share of all responders
50%
pasireotide monotherapy
Source sentences
Summary
Thirty-four patients (50.0%; 95% CI 37.6-62.4) achieved the primary endpoint, of whom 17 (50.0%) received pasireotide monotherapy and 17 (50.0%) received combination therapy.
3 context sentences(design, population, supporting numbers)
Summary · context
This is an open-label, multicenter, non-comparative, Phase II study comprising 35-week core phase and an optional extension phase.
Summary · context
All patients started with pasireotide, and cabergoline was added if cortisol remained elevated.
Summary · context
Primary endpoint was proportion of patients with a mean urinary free cortisol (mUFC) level not exceeding the upper limit of normal (ULN) at week 35 with missing data imputed using last available post-baseline assessments.
Show in the study text Report a problemFND-3787654001
02HumanNon-randomized trialEfficacy outcome
In Cushing's disease, pasireotide monotherapy provided sustained improvements in clinical symptoms of hypercortisolism up to week 99.
In plain wordsIn Cushing's disease, symptoms of excess cortisol improved during pasireotide monotherapy, with sustained improvements up to week 99.
Peptide
Pasireotide
Outcome
Direction not reportedClinical symptoms of hypercortisolism during monotherapy
Study
n: not reported in source
Outcome group
Disease control · Endocrine and hormones
Studied dose
Dose not stated · subcutaneousAs given in this study (human).
Source sentences
Summary
Treatment with either mono or combination therapy provided sustained improvements in clinical symptoms of hypercortisolism up to week 99.
2 context sentences(design, population, supporting numbers)
Summary · context
This is an open-label, multicenter, non-comparative, Phase II study comprising 35-week core phase and an optional extension phase.
Summary · context
All patients started with pasireotide, and cabergoline was added if cortisol remained elevated.
Show in the study text Report a problemFND-3787654002

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Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 stopped early · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

TerminatedPhase 2Results postedNCT01915303

Study of the Efficacy and Safety of Pasireotide s.c. +/- Cabergoline in Patients With Cushing's Disease

Novartis Pharmaceuticalsn = 686 Mar 2014 to 4 Sept 2019

Cushings Disease

Trial page ClinicalTrials.govUpdated 18 Sept 2020

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:34 UTC
First added · source update from Europe PMC

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