Long Term Safety and Efficacy of Pasireotide s.c. in Patients With Cushing's Disease

July 19, 2024 updated by: RECORDATI GROUP

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)

This is a non-interventional, multinational, multi-center post-marketing study, to further document the safety and efficacy of pasireotide s.c. administered in routine clinical practice in patients with Cushing's disease. Patients with Cushing's disease and treated with pasireotide s.c. alone and in combination with other therapies will be monitored. For this study, each enrolled patient will be followed up for 3 years after enrollment. Patients who permanently discontinue pasireotide s.c. prior to completing the 3-year observation period will be followed up for 3 months after the last dose of pasireotide s.c.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
152

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Recordati
  • Phone Number: +390248787122

Study Contact Backup

  • Name: Recordati
  • Phone Number: +41612056100

Study Locations

  • Canada
      • Quebec, Canada, G1V 4G2
        • CHUL Centre de recherche du CHU
    • Quebec
      • Montreal, Quebec, Canada, H2W 1T8
        • Centre de recherche du CHUM CRCHUM
      • Sherbrooke, Quebec, Canada, J1H 5N4
        • CHUS - Hôpital Fleurimont
  • Colombia
      • Cali, Colombia
        • Centro Medico Imbanaco de Cali
  • France
      • Amiens Cedex 1, France, 80054
        • CHU Amiens Picardie site Nord
      • Bron Cedex, France, 69677
        • HCL-Groupe Hospitalier Est
      • Lille Cedex, France, 59037
        • Recordati Investigative Site
      • Marseille, France, 13385
        • AP-HM - Hopital de La Conception
      • Montpellier Cedex 5, France, 34295
        • Hopital Lapeyronie
      • Paris, France, 75014
        • Hôpital Cochin
      • Reims, France, 51092
        • Hopital Robert Debre
      • Saint Mande, France, 94160
        • HIA Begin
      • St Priest en Jarez Cedex, France, 42277
        • CHU de Saint Etienne
  • Germany
      • Aachen, Germany, 52074
        • Universitaetsklinikum Aachen
      • Berlin, Germany, 13353
        • Universitaetsmedizin Charite
      • Duesseldorf, Germany, 40225
        • Univ.-Klinikum Düsseldorf
      • Erlangen, Germany, 91054
        • Universitaetsklinikum Erlangen Nuernberg
      • Essen, Germany, 45147
        • Universitaetsklinikum Essen
      • Freiburg, Germany, 79106
        • Universitaetsklinikum Freiburg
      • Halle, Germany, 06108
        • Praxis Dr.med.Frank Ackermann
      • Hamburg, Germany, 20246
        • Universitaetsklinikum Hamburg Eppendorf
      • Leipzig, Germany, 04103
        • Universitaetsklinikum Leipzig Aor
      • Magdeburg, Germany, 39120
        • Universitaetsklinikum Magdeburg AöR
      • Muenchen, Germany, 81377
        • Universitaetsklinikum Muenchen LMU
      • Muenchen, Germany, 81667
        • Medicover Muenchen Ost MVZ
      • Oldenburg, Germany, 26122
        • Praxis Michael Droste
      • Wuerzburg, Germany, 97080
        • Universitaetsklinikum Wuerzburg
  • Israel
      • Petach Tikva, Israel, 49100
        • Rabin Medical Centre Belinson
      • Tel Aviv, Israel, 6423906
        • Tel Aviv Sourasky Medical Centre Ichilov
  • Italy
      • Napoli, Italy, 80131
        • O Universitaria Policlinico Federico II Univ Studi Fed II
    • AN
      • Ancona, AN, Italy, 60126
        • AOU Osp Riuniti Umberto I GM Lancisi G Salesi Univ Studi
    • ME
      • Messina, ME, Italy, 98125
        • Az.Ospe.Universitaria Policlinico G. Martino Univ.di Messina
    • MI
      • Milano, MI, Italy, 20122
        • Fond IRCCS Ca Granda Ospedale Maggiore Policlinico
      • Milano, MI, Italy, 20149
        • Stab.Osp.S.Luca P.O. S.Luca-S.Michele-Ist.Auxol. Italiano
    • PA
      • Palermo, PA, Italy, 90127
        • Az Osp Univ Policlinico P Giaccone Universita Studi Palermo
    • PD
      • Padova, PD, Italy, 35128
        • Azienda Ospedaliera di Padova Università degli Studi
    • PI
      • Pisa, PI, Italy, 56124
        • Az Ospedaliero Universitaria Pisana Pres Osped di Cisanello
    • TO
      • Torino, TO, Italy, 10126
        • P O Molinette AO Citta della Salute e della Scienza Torino
  • Lebanon
      • El Achrafiyé, Lebanon, 166830
        • Hotel Dieu de France Hospital
  • Netherlands
      • Nijmegen, Netherlands, 6500 HB
        • Radboudumc
    • AZ
      • Maastricht, AZ, Netherlands, 5800
        • Maastricht Universitair Medisch Centrum
  • Romania
      • Brasov, Romania, 500365
        • Spitalul Clinic Judetean de Urgenta Brasov
      • Bucuresti, Romania, 011863
        • Institutul de Endocrinologie "I. C. Parhon", Bucuresti
      • Cluj, Romania, 400006
        • Spitalul Clinic Jude Emergency Clinical County Hospital Cluj
      • Târgu-Mureş, Romania, 540061
        • County Hospital Tg. Mures
  • United Kingdom
      • Plymouth, United Kingdom, PL6 8DH
        • Derriford Hospital
      • Southampton, United Kingdom, SO16 6YD
        • Southampton General Hospital
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • St Josephs Hospital and Medical Center
    • California
      • Los Angeles, California, United States, 90033
        • University of Southern California CSOM230B2410 - SC
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital SC - SOM230B2410
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State University SC - SOM230B2410
    • Oregon
      • Bend, Oregon, United States, 97702
        • Endocrinology Services
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15212
        • Allegheny Endocrinology Associates
    • Washington
      • Seattle, Washington, United States, 98122-4379
        • Swedish Medical Center Dept.ofSeattle Neuroscience(2)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The patient population will consist of male and female patients aged 18 years or older with a diagnosis of Cushing's disease for whom surgery has failed or for whom surgery is not an option and who are treated with pasireotide s.c.

Description

Inclusion Criteria:

  • Written informed consent or equivalent document (e.g., written information) as per country regulation prior to registration of any patient data
  • Male or female patients aged 18 years or older with a diagnosis of Cushing's disease for whom surgery has failed or for whom surgery is not an option
  • Patients must be treated with pasireotide s.c. started either at the first visit for this study or prior to study entry

Exclusion Criteria:

  • Patients with ectopic ACTH-dependent Cushing's syndrome
  • Patients with adrenal Cushing's syndrome
  • Patients with Pseudo Cushing's syndrome

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Cohort 1/new use cohort
Subjects who initiated pasireotide s.c. at time of study entry (on or after the signing of the informed consent or acknowledging an equivalent document -for example, written information- as per country regulation).
Drug: Pasireotide
Other Names:
  • Signifor
Cohort 2/prior use cohort
Subjects who initiated pasireotide s.c. prior to study entry.
Drug: Pasireotide
Other Names:
  • Signifor

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events as a Measure of Safety and Tolerability Profile of Pasireotide s.c.
Time Frame: 3-year follow-up
Number of pasireotide s.c related adverse events and serious adverse events, when administered as monotherapy or in combination with other therapies in patients with Cushing's disease
3-year follow-up

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
The proportion of patients that reach mean UFC ≤ ULN as short and long-term efficacy measure
Time Frame: At 1, 3, 6, 12, 24 and 36 months after enrolling into the study
The proportion of patients with a mean UFC ≤ ULN at 1, 3, 6, 12, 24 and 36 months after enrolling into the study
At 1, 3, 6, 12, 24 and 36 months after enrolling into the study
Changes of mean UFC measures of disease activity
Time Frame: 3-year follow-up
The absolute and percentage change from baseline of mean UFC measures of disease activity after enrolling into the study.
3-year follow-up
Proportion of patients achieving serum cortisol Normalization of disease activity
Time Frame: 3-year follow-up
Proportion of patients achieving serum cortisol Normalization of disease activity where normalization refers to being within the upper and lower limit of normal ranges
3-year follow-up
The absolute and percentage change of blood pressure from baseline
Time Frame: 3-year follow-up
The absolute and percentage change of blood pressure from baseline after enrolling into the study
3-year follow-up
The proportion of patients with favorable shift of blood pressure from baseline
Time Frame: 3-year follow-up
The proportion of patients with favorable shift of blood pressure from baseline after enrolling into the study
3-year follow-up
Changes in safety and efficacy parameters over a period of 3 months
Time Frame: 3 months after patient's discontinuation
Safety parameters include: Adverse events , Serious Adverse Events, vital signs, blood pressure, heart rate, body temperature, blood glucose (fasting plasma glucose, HemoglobinA1c), hormones (IGF-1, GH, TSH/free T4), liver enzymes (AST, ALT, alkaline phosphatase, γGT, total bilirubin), hematology, electrolytes, immunological events (e.g., allergic reactions: rash, pruritus, injection site reactions), gallbladder ultrasound and ECGs. Efficacy parameters include: Urinary Free Cortisol, serum cortisol, serum cortisol after dexamethasone testing, salivary cortisol, plasma ACTH, fasting serum lipid profile, Blood pressure, body weight, body mass index, waist circumference, tumor size and quality of life.
3 months after patient's discontinuation
The absolute and percentage change in tumor size
Time Frame: 3-year follow-up
The absolute and percentage change in tumor size after enrolling into the study
3-year follow-up
The absolute and percentage change from baseline in patient-reported outcome questionnaires
Time Frame: 3-year follow-up
The absolute and percentage change from baseline in patient -reported outcome questionnaires (Cushing QoL and EURO QoL) after enrolling into the study
3-year follow-up
Change of serum cortisol measures of disease activity
Time Frame: 3-year follow-up
The absolute and percentage change from baseline of serum cortisol measures of disease activity after enrolling into the study
3-year follow-up
Change of serum cortisol after dexamathasone testing measures of disease activity
Time Frame: 3-year followup
The absolute and percentage change from baseline of serum cortisol after dexamathasone testing measures of disease activity after enrolling into the study
3-year followup
Changes of salivary cortisol measures of disease activity
Time Frame: 3-year follow-up
The absolute and percentage change from baseline of salivary cortisol measures of disease activity after enrolling into the study
3-year follow-up
Changes of ACTH measures of disease activtity
Time Frame: 3-year follow-up
The absolute and percentage change from baseline of ACTH measures of disease activity after enrolling into the study
3-year follow-up
Proportion of patients achieving serum cortisol after dexamethasone testing Normalization of disease activity
Time Frame: 3-year follow-up
Proportion of patients achieving serum cortisol after dexamethasone testing Normalization of disease activity where normalization refers to being within the upper and lower limit of normal ranges
3-year follow-up
Proportion of patients achieving salivary cortisol Normalization of disease activity
Time Frame: 3-year follow-up
Proportion of patients achieving salivary cortisol of disease activity where normalization refers to being within the upper and lower limit of normal ranges
3-year follow-up
Proportion of patients achieving ACTH Normalization of disease activity
Time Frame: 3-year follow-up
Proportion of patients achieving ACTH of disease activity where normalization refers to being within the upper and lower limit of normal ranges
3-year follow-up
Changes of fasting serum lipid profile measures of disease activtity
Time Frame: 3-year follow-up
The absolute and percentage change from baseline of fasting serum lipid profile measures of disease activity after enrolling into the study
3-year follow-up
The absolute and percentage change of body weight from baseline
Time Frame: 3-year follow-up
The absolute and percentage change of body weight from baseline after enrolling into the study
3-year follow-up
The absolute and percentage change of body mass index from baseline
Time Frame: 3-year follow-up
The absolute and percentage change of body mass index from baseline after enrolling into the study
3-year follow-up
The absolute and percentage change of waist circumference from baseline
Time Frame: 3-year follow-up
The absolute and percentage change of waist circumference from baseline after enrolling into the study
3-year follow-up
The proportion of patients with favorable shift of body weight from baseline
Time Frame: 3-year follow-up
The proportion of patients with favorable shift of body weight from baseline after enrolling into the study
3-year follow-up
The proportion of patients with favorable shift of body mass index from baseline
Time Frame: 3-year follow-up
The proportion of patients with favorable shift of body mass index from baseline after enrolling into the study
3-year follow-up
The proportion of patients with favorable shift of waist circumference from baseline
Time Frame: 3-year follow-up
The proportion of patients with favorable shift of waist circumference from baseline after enrolling into the study
3-year follow-up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
RECORDATI GROUP

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Mario MALDONADO, MD, Recordati AG

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 28, 2013

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 10, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 10, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 26, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 5, 2014

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 8, 2014

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 15, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 19, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CSOM230B2410

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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