Pasireotide can induce sustained decreases in urinary cortisol and provide clinical benefit in patients with Cushing's disease: results from an open-ended, open-label extension trial.

Schopohl J, Gu F, Rubens R, Van Gaal L, Bertherat J, Ligueros-Saylan M, Trovato A, Hughes G, Salgado LR, Boscaro M, Pivonello R

Pituitary1 Oct 2015Cited by 43 (Europe PMC)

PMID25537481PMCIDPMC4560758DOI10.1007/s11102-014-0618-1PepBankPUB-25537481

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Summary

PurposeReport the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.Methods162 patients entered the core study. 58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension. Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid). Dose titration was permitted according to efficacy or drug-related adverse events.Results40 patients completed 24 months' treatment. Of the patients who entered the extension, 50.0% (29/58) and 34.5% (20/58) had controlled UFC (UFC ≤ ULN) at months 12 and 24, respectively. The mean percentage decrease in UFC was 57.3% (95% CI 40.7-73.9; n = 52) and 62.1% (50.8-73.5; n = 33) after 12 and 24 months' treatment, respectively. Improvements in clinical signs of Cushing's disease were sustained up to month 24. The most frequent drug-related adverse events in patients who received ≥1 dose of pasireotide (n = 162) from core baseline until the 24-month cut-off were diarrhea (55.6%), nausea (48.1%), hyperglycemia (38.9%), and cholelithiasis (31.5%). No new safety issues were identified during the extension.ConclusionsReductions in mean UFC and improvements in clinical signs of Cushing's disease were maintained over 24 months of pasireotide treatment. The safety profile of pasireotide is typical for a somatostatin analogue, except for the frequency and degree of hyperglycemia; patients should be monitored for changes in glucose homeostasis. Pasireotide represents the first approved pituitary-targeted treatment for patients with Cushing's disease.

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

Results from this study10

10 results from studies that gave the peptide. 10 results from people.

01HumanNon-randomized trialSafety outcome
In Cushing's disease, drug-related diarrhea occurred in 55.6% receiving pasireotide through the 24-month cut-off.
In plain wordsIn Cushing's disease, drug-related diarrhea occurred in 55.6% receiving pasireotide through the 24-month cut-off.
Peptide
Pasireotide
Outcome
Not applicableDiarrhea
Study
n = 162
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated · subcutaneousAs given in this study (human).
Measurements
Drug-related diarrhea
55.6%
patients with >=1 dose (n = 162), core baseline to 24-month cut-off
Source sentences
Summary
The most frequent drug-related adverse events in patients who received ≥1 dose of pasireotide (n = 162) from core baseline until the 24-month cut-off were diarrhea (55.6%), nausea (48.1%), hyperglycemia (38.9%), and cholelithiasis (31.5%).
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748106
02HumanNon-randomized trialSafety outcome
In Cushing's disease, drug-related nausea occurred in 48.1% receiving pasireotide through the 24-month cut-off.
In plain wordsIn Cushing's disease, drug-related nausea occurred in 48.1% receiving pasireotide through the 24-month cut-off.
Peptide
Pasireotide
Outcome
Not applicableNausea
Study
n = 162
Outcome group
Gastrointestinal adverse events · Gastrointestinal
Studied dose
Dose not stated · subcutaneousAs given in this study (human).
Measurements
Drug-related nausea
48.1%
patients with >=1 dose (n = 162), core baseline to 24-month cut-off
Source sentences
Summary
The most frequent drug-related adverse events in patients who received ≥1 dose of pasireotide (n = 162) from core baseline until the 24-month cut-off were diarrhea (55.6%), nausea (48.1%), hyperglycemia (38.9%), and cholelithiasis (31.5%).
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748107
03HumanNon-randomized trialSafety outcome
In Cushing's disease, drug-related hyperglycemia occurred in 38.9% receiving pasireotide through the 24-month cut-off.
In plain wordsIn Cushing's disease, drug-related high blood glucose occurred in 38.9% receiving pasireotide through the 24-month cut-off.
Peptide
Pasireotide
Outcome
Not applicableHyperglycemia
Study
n = 162
Outcome group
Glycemic control · Endocrine and hormones
Studied dose
Dose not stated · subcutaneousAs given in this study (human).
Measurements
Drug-related hyperglycemia
38.9%
patients with >=1 dose (n = 162), core baseline to 24-month cut-off
Source sentences
Summary
The most frequent drug-related adverse events in patients who received ≥1 dose of pasireotide (n = 162) from core baseline until the 24-month cut-off were diarrhea (55.6%), nausea (48.1%), hyperglycemia (38.9%), and cholelithiasis (31.5%).
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748108
04HumanNon-randomized trialSafety outcome
In Cushing's disease, drug-related cholelithiasis occurred in 31.5% receiving pasireotide through the 24-month cut-off.
In plain wordsIn Cushing's disease, drug-related gallstones occurred in 31.5% receiving pasireotide through the 24-month cut-off.
Peptide
Pasireotide
Outcome
Not applicableCholelithiasis
Study
n = 162
Outcome group
Adverse events · Gastrointestinal
Studied dose
Dose not stated · subcutaneousAs given in this study (human).
Measurements
Drug-related cholelithiasis
31.5%
patients with >=1 dose (n = 162), core baseline to 24-month cut-off
Source sentences
Summary
The most frequent drug-related adverse events in patients who received ≥1 dose of pasireotide (n = 162) from core baseline until the 24-month cut-off were diarrhea (55.6%), nausea (48.1%), hyperglycemia (38.9%), and cholelithiasis (31.5%).
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748109
05HumanNon-randomized trialEfficacy outcome
In Cushing's disease extension entrants, 50.0% (29/58) had controlled UFC at month 12 with pasireotide.
In plain wordsAmong Cushing's disease extension entrants, 50.0% had controlled urinary cortisol at month 12 with pasireotide.
Peptide
Pasireotide
Outcome
Not applicableControlled UFC at month 12
Study
n = 58
Outcome group
Cortisol Control · Endocrine and hormones
Studied dose
300-1,200 μg bid · subcutaneousAs given in this study (human).
Measurements
Patients with controlled UFC (UFC <= ULN)
29 patients
month 12; of 58
Patients with controlled UFC (UFC <= ULN)
50%
month 12; of 58
Source sentences
Summary
Of the patients who entered the extension, 50.0% (29/58) and 34.5% (20/58) had controlled UFC (UFC ≤ ULN) at months 12 and 24, respectively.
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748101
06HumanNon-randomized trialEfficacy outcome
In Cushing's disease extension entrants, 34.5% (20/58) had controlled UFC at month 24 with pasireotide.
In plain wordsAmong Cushing's disease extension entrants, 34.5% had controlled urinary cortisol at month 24 with pasireotide.
Peptide
Pasireotide
Outcome
Not applicableControlled UFC at month 24
Study
n = 58
Outcome group
Cortisol Control · Endocrine and hormones
Studied dose
300-1,200 μg bid · subcutaneousAs given in this study (human).
Measurements
Patients with controlled UFC (UFC <= ULN)
20 patients
month 24; of 58
Patients with controlled UFC (UFC <= ULN)
34.5%
month 24; of 58
Source sentences
Summary
Of the patients who entered the extension, 50.0% (29/58) and 34.5% (20/58) had controlled UFC (UFC ≤ ULN) at months 12 and 24, respectively.
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748102
07HumanNon-randomized trialEfficacy outcome
In Cushing's disease, mean UFC decreased 57.3% (95% CI 40.7-73.9) after 12 months of pasireotide.
In plain wordsIn Cushing's disease, urinary cortisol decreased 57.3% after 12 months of pasireotide.
Peptide
Pasireotide
Outcome
DecreaseUFC decrease at month 12
Study
n = 52
Outcome group
Cortisol Control · Endocrine and hormones
Studied dose
300-1,200 μg bid · subcutaneous · 12 monthsAs given in this study (human).
Measurements
Mean percentage decrease in UFC
57.3%
after 12 months of treatment; n = 52
95% CI
40.7 to 73.9%
mean UFC decrease at 12 months
Source sentences
Summary
The mean percentage decrease in UFC was 57.3% (95% CI 40.7-73.9; n = 52) and 62.1% (50.8-73.5; n = 33) after 12 and 24 months' treatment, respectively.
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748103
08HumanNon-randomized trialEfficacy outcome
In Cushing's disease, mean UFC decreased 62.1% (50.8-73.5) after 24 months of pasireotide.
In plain wordsIn Cushing's disease, urinary cortisol decreased 62.1% after 24 months of pasireotide.
Peptide
Pasireotide
Outcome
DecreaseUFC decrease at month 24
Study
n = 33
Outcome group
Cortisol Control · Endocrine and hormones
Studied dose
300-1,200 μg bid · subcutaneous · 24 monthsAs given in this study (human).
Measurements
Mean percentage decrease in UFC
62.1%
after 24 months of treatment; n = 33
Interval reported with mean decrease
50.8 to 73.5%
as written (50.8-73.5)
Source sentences
Summary
The mean percentage decrease in UFC was 57.3% (95% CI 40.7-73.9; n = 52) and 62.1% (50.8-73.5; n = 33) after 12 and 24 months' treatment, respectively.
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748104
09HumanNon-randomized trialEfficacy outcome
In Cushing's disease, improvements in clinical signs with pasireotide persisted to month 24.
In plain wordsIn Cushing's disease, improvements in clinical signs with pasireotide persisted to month 24.
Peptide
Pasireotide
Outcome
Direction not reportedClinical signs
Study
n: not reported in source
Outcome group
Disease Signs And Symptoms · Endocrine and hormones
Studied dose
300-1,200 μg bid · subcutaneousAs given in this study (human).
Source sentences
Summary
Improvements in clinical signs of Cushing's disease were sustained up to month 24.
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748105
10HumanNon-randomized trialSafety outcome
In Cushing's disease, no new safety issues appeared during the pasireotide extension.
In plain wordsIn Cushing's disease, no new safety issues appeared during the pasireotide extension.
Peptide
Pasireotide
Outcome
Not applicableNew safety issues
Study
n: not reported in source
Outcome group
Adverse events · Whole body
Studied dose
300-1,200 μg bid · subcutaneousAs given in this study (human).
Source sentences
Summary
No new safety issues were identified during the extension.
4 context sentences(design, population, supporting numbers)
Summary · context
Report the efficacy and safety of pasireotide sc in patients with Cushing's disease during an open-ended, open-label extension to a randomized, double-blind, 12-month, Phase III study.
Summary · context
58 patients who had mean UFC ≤ ULN at month 12 or were benefiting clinically from pasireotide entered the extension.
Summary · context
Patients received the same dose of pasireotide as at the end of the core study (300-1,200 μg bid).
Summary · context
Dose titration was permitted according to efficacy or drug-related adverse events.
Show in the study text Report a problemFND-2553748110

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials1

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

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

CompletedPhase 3Results postedNCT00434148

Safety and Efficacy of Different Dose Levels of Pasireotide in Patients With de Novo, Persistent or Recurrent Cushing's Disease

Novartis Pharmaceuticalsn = 162Dec 2006 to May 2014

Cushing's Disease

Trial page ClinicalTrials.govUpdated 8 Mar 2016

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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