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Study · Non-randomized trial · n = 18 · 2014

6 results

Extended treatment of Cushing's disease with pasireotide: results from a 2-year, Phase II study.

Boscaro M, Bertherat J, Findling J, Fleseriu M, Atkinson AB, Petersenn S, Schopohl J, Snyder P, Hughes G, Trovato A, Hu K, Maldonado M, Biller BM

Pituitary1 Aug 2014Cited by 38 (Europe PMC)

PMID23943009 (opens in a new tab)PMCIDPMC4085509 (opens in a new tab)DOI10.1007/s11102-013-0503-3 (opens in a new tab)PepBankPUB-23943009
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Summary

In a previous 15-day, Phase II study of patients with de novo or persistent/recurrent Cushing's disease (core study), treatment with pasireotide 600 μg sc bid reduced urinary free cortisol (UFC) levels in 76% of patients and normalized UFC in 17%. The objective of this study was to evaluate the efficacy and safety of extended treatment with pasireotide. This was a planned, open-ended, single-arm, multicenter extension study (primary endpoint: 6 months). Patients aged ≥18 years with Cushing's disease who completed the core study could enter the extension if they achieved UFC normalization at core study end and/or obtained significant clinical benefit. Of the 38 patients who completed the core study, 19 entered the extension and 18 were included in the efficacy analyses (three responders, 11 reducers, four non-reducers in the core study). At data cut-off, median treatment duration in the extension was 9.7 months (range: 2 months to 4.8 years). At extension month 6, 56% of the 18 patients had lower UFC than at core baseline and 22% had normalized UFC. Of the four patients who remained on study drug at month 24, one had normalized UFC. Reductions in serum cortisol, plasma adrenocorticotropic hormone, body weight and diastolic blood pressure were observed. The most common adverse events were mild-to-moderate gastrointestinal disorders and hyperglycemia. Pasireotide offers a tumor-directed medical therapy that may be effective for the extended treatment of some patients with Cushing's disease.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study6

6 results from studies that gave the peptide. 6 results from people.

  1. 01HumanNon-randomized trialEfficacy outcome

    In Cushing's disease, 56% of patients had lower UFC than core baseline at extension month 6 with pasireotide.

    In plain wordsIn Cushing's disease, 56% had lower urinary cortisol than core baseline at extension month 6 with pasireotide.

    Peptide
    Pasireotide
    Outcome
    DecreaseLower UFC at extension month 6
    Study

    n = 18

    Comparator
    Core baseline
    Outcome group
    Cortisol Control · Endocrine and hormones
    Studied dose
    Dose not stated

    Measurements

    Patients with lower UFC than core baseline
    56%
    extension month 6, 18 patients

    Source sentences

    Summary
    At extension month 6, 56% of the 18 patients had lower UFC than at core baseline and 22% had normalized UFC.
    3 context sentences
    Summary · context
    This was a planned, open-ended, single-arm, multicenter extension study (primary endpoint: 6 months).
    Summary · context
    Patients aged ≥18 years with Cushing's disease who completed the core study could enter the extension if they achieved UFC normalization at core study end and/or obtained significant clinical benefit.
    Summary · context
    Of the 38 patients who completed the core study, 19 entered the extension and 18 were included in the efficacy analyses (three responders, 11 reducers, four non-reducers in the core study).
  2. 02HumanNon-randomized trialEfficacy outcome

    In Cushing's disease, 22% had normalized UFC at extension month 6 with pasireotide.

    In plain wordsIn Cushing's disease, 22% had normal urinary cortisol at extension month 6 with pasireotide.

    Peptide
    Pasireotide
    Outcome
    Not applicableNormalized UFC at extension month 6
    Study

    n = 18

    Outcome group
    Cortisol Control · Endocrine and hormones
    Studied dose
    Dose not stated

    Measurements

    Patients with normalized UFC
    22%
    extension month 6, 18 patients

    Source sentences

    Summary
    At extension month 6, 56% of the 18 patients had lower UFC than at core baseline and 22% had normalized UFC.
    3 context sentences
    Summary · context
    This was a planned, open-ended, single-arm, multicenter extension study (primary endpoint: 6 months).
    Summary · context
    Patients aged ≥18 years with Cushing's disease who completed the core study could enter the extension if they achieved UFC normalization at core study end and/or obtained significant clinical benefit.
    Summary · context
    Of the 38 patients who completed the core study, 19 entered the extension and 18 were included in the efficacy analyses (three responders, 11 reducers, four non-reducers in the core study).
  3. 03HumanNon-randomized trialEfficacy outcome

    In Cushing's disease, one of four patients remaining on pasireotide at month 24 had normalized UFC.

    In plain wordsIn Cushing's disease, one of four patients remaining on pasireotide at month 24 had normal urinary cortisol.

    Peptide
    Pasireotide
    Outcome
    Not applicableNormalized UFC at month 24
    Study

    n = 4

    Outcome group
    Cortisol Control · Endocrine and hormones
    Studied dose
    Dose not stated

    Measurements

    Patients with normalized UFC
    1 patients
    one of four patients remaining on study drug at month 24

    Source sentences

    Summary
    Of the four patients who remained on study drug at month 24, one had normalized UFC.
    3 context sentences
    Summary · context
    This was a planned, open-ended, single-arm, multicenter extension study (primary endpoint: 6 months).
    Summary · context
    Patients aged ≥18 years with Cushing's disease who completed the core study could enter the extension if they achieved UFC normalization at core study end and/or obtained significant clinical benefit.
    Summary · context
    Of the 38 patients who completed the core study, 19 entered the extension and 18 were included in the efficacy analyses (three responders, 11 reducers, four non-reducers in the core study).
  4. 04HumanNon-randomized trialEfficacy outcome

    In Cushing's disease, pasireotide treatment accompanied reductions in serum cortisol, plasma ACTH, body weight and diastolic blood pressure.

    In plain wordsIn Cushing's disease, cortisol, ACTH, weight and diastolic blood pressure decreased during pasireotide treatment.

    Peptide
    Pasireotide
    Outcome
    DecreaseSerum cortisol, ACTH, body weight and diastolic pressure
    Study

    n: not reported in source

    Outcome group
    Disease Signs And Symptoms · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Reductions in serum cortisol, plasma adrenocorticotropic hormone, body weight and diastolic blood pressure were observed.
    3 context sentences
    Summary · context
    This was a planned, open-ended, single-arm, multicenter extension study (primary endpoint: 6 months).
    Summary · context
    Patients aged ≥18 years with Cushing's disease who completed the core study could enter the extension if they achieved UFC normalization at core study end and/or obtained significant clinical benefit.
    Summary · context
    Of the 38 patients who completed the core study, 19 entered the extension and 18 were included in the efficacy analyses (three responders, 11 reducers, four non-reducers in the core study).
  5. 05HumanNon-randomized trialSafety outcome

    In Cushing's disease, mild-to-moderate gastrointestinal disorders were among the most common adverse events with extended pasireotide.

    In plain wordsIn Cushing's disease, mild-to-moderate digestive disorders were among the most common adverse events with extended pasireotide.

    Peptide
    Pasireotide
    Outcome
    Not applicableGastrointestinal disorders
    Study

    n: not reported in source

    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    The most common adverse events were mild-to-moderate gastrointestinal disorders and hyperglycemia.
    3 context sentences
    Summary · context
    This was a planned, open-ended, single-arm, multicenter extension study (primary endpoint: 6 months).
    Summary · context
    Patients aged ≥18 years with Cushing's disease who completed the core study could enter the extension if they achieved UFC normalization at core study end and/or obtained significant clinical benefit.
    Summary · context
    Of the 38 patients who completed the core study, 19 entered the extension and 18 were included in the efficacy analyses (three responders, 11 reducers, four non-reducers in the core study).
  6. 06HumanNon-randomized trialSafety outcome

    In Cushing's disease, hyperglycemia was among the most common adverse events with extended pasireotide.

    In plain wordsIn Cushing's disease, high blood glucose was among the most common adverse events with extended pasireotide.

    Peptide
    Pasireotide
    Outcome
    Not applicableHyperglycemia
    Study

    n: not reported in source

    Outcome group
    Adverse events · Endocrine and hormones
    Studied dose
    Dose not stated

    Source sentences

    Summary
    The most common adverse events were mild-to-moderate gastrointestinal disorders and hyperglycemia.
    3 context sentences
    Summary · context
    This was a planned, open-ended, single-arm, multicenter extension study (primary endpoint: 6 months).
    Summary · context
    Patients aged ≥18 years with Cushing's disease who completed the core study could enter the extension if they achieved UFC normalization at core study end and/or obtained significant clinical benefit.
    Summary · context
    Of the 38 patients who completed the core study, 19 entered the extension and 18 were included in the efficacy analyses (three responders, 11 reducers, four non-reducers in the core study).

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 2Results postedNCT00171951

Extension Study to Assess the Safety and Efficacy of Pasireotide in Participants With Cushing's Disease

Novartis Pharmaceuticalsn = 1913 Aug 2004 to 8 Jul 2013

Cushing Disease

Trial pageClinicalTrials.govUpdated 2 Jun 2021

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.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:34 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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