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

May 6, 2021 updated by: Novartis Pharmaceuticals

Extension to a Multicenter, Open-label Study to Assess the Safety and Efficacy of 600 μg SOM230, Administered Subcutaneously, Bid in Patients With Cushing's Disease

Cushing's disease is a rare serious condition that is caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. This study assessed the long-term safety and efficacy of pasireotide in participants with Cushing's disease.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

19

Phase

  • Phase 2

Contacts and Locations

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

Study Locations

  • France
      • Paris, France, 75006
        • Novartis Investigative Site
  • Germany
      • Essen, Germany, 45122
        • Novartis Investigative Site
      • Muenchen, Germany, 80336
        • Novartis Investigative Site
  • Italy
    • AN
      • Ancona, AN, Italy, 60126
        • Novartis Investigative Site
  • United Kingdom
      • Belfast, United Kingdom, BT12 6BA
        • Novartis Investigative Site
  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Sciences University Dept.ofOregonHealth&SciencesU.
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104-6149
        • University of Pennsylvania Medical Center

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

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Participants who have completed the 15 days of Pasireotide treatment in the CSOM230B2208 study and have achieved normalization of 24-hour urinary free cortisol. Participants who did not achieve normalization of 24 -hour urinary free cortisol may be enrolled if in the opinion of the investigator the participant is getting significant clinical benefits from treatment with Pasireotide .
  • The participant did not experience any unacceptable adverse events of tolerability issues during the original 15 day treatment.
  • Female participants of childbearing potential who have not undergone clinically documented total hysterectomy and/or ovariectomy or tubal ligation must agree to use barrier contraception throughout the course of the extension study, and for one month after the study has ended.

Exclusion Criteria:

  • Participant who have developed poorly controlled diabetes mellitus as indicated by ketoacidosis or hemoglobin (Hgb) A1C (HgbA1C) > 10 since starting [study CSOM230B2208].
  • Participant with persistent alanine aminotransferase (ALT)/ aspartate transaminase (AST) or alkaline phosphatase levels more than 2.5X upper limit of normal (ULN), serum creatinine > 2.0 X ULN, serum bilirubin > 2 X ULN.
  • Participant with abnormal coagulation (Prothrombin time (PT) and partial thromboplastin time (PTT) elevated by 30% above normal limits), white blood cells (WBC) <3.0x1'000'000'000/L; Hgb <12.0g/dL for females, Hgb <13.0g/dL for males; PLT <100x1'000'000'000/L.

Other protocol-defined inclusion / exclusion criteria may apply.

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

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC
Participants received pasireotide 600 micrograms (μg) twice daily (BID) subcutaneously (SC) to achieve or maintain urinary free cortisol (UFC) normalization. If UFC levels were increased at any time, participants received 900 μg BID SC, until no safety or tolerability concerns were observed as per investigators assessment. If the participant was unable to tolerate the 900 μg BID, dosing of 600 μg three times a day was given.
Drug: Pasireotide
Pasireotide 600 μg or 900 μg was administered as an SC injection.
Other Names:
  • SOM230

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits
Time Frame: Month 6
A participant was considered a responder if the mean UFC from the two 24-hour urine samples collected at Month 6 was within normal limits. The normal range for UFC is 55 to 276 nmol/day.
Month 6
Change From Baseline in Mean Urinary Free Cortisol (UFC)
Time Frame: Core Baseline, Days 14/15 (Core study), Months 6, 12, 24 and 102
24-hour urine samples were collected to obtain mean UFC measurements. A negative mean change from baseline indicates improvement.
Core Baseline, Days 14/15 (Core study), Months 6, 12, 24 and 102

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants Who Had At Least One Adverse Event (AE)
Time Frame: Up to approximately 106 months
An AE was any undesirable sign, symptom or medical condition occurring after starting study drug even if the event is not considered to be related to study drug. AEs were assessed according to incident dose group: Pasireotide 1200 μg sc total daily dose (TDD), Pasireotide 1800 μg SC TDD and Pasireotide SC Any Dose. The incident dose for an AE was the last total daily dose administered on or prior to the AE onset date.
Up to approximately 106 months
Change From Baseline in Serum Cortisol Levels
Time Frame: Core Baseline, Day 15 (Core study), Months 6, 12, 24, and Month 105 (end of the study)
Blood samples were withdrawn to obtain the serum cortisol levels. A negative change from baseline indicates improvement.
Core Baseline, Day 15 (Core study), Months 6, 12, 24, and Month 105 (end of the study)
Plasma Trough Concentrations (Ctrough) of Pasireotide in UFC Responders
Time Frame: Day 15 (Core study) and Month 6
Participants with Cushing's disease were considered responders if mean UFC levels from the 24-hour urine collections at Day 15 (Core study) and at extension Month 6, were within normal limits. Ctrough levels of pasireotide were measured at Day 15 of Core study and Month 6.
Day 15 (Core study) and Month 6
Change From Baseline in Plasma Adrenocorticotropic Hormone (ACTH) Levels
Time Frame: Core Baseline, Day 15 (Core study), Months 6, 12, 24 and Month 105 (end of the study)
Blood samples were withdrawn to obtain the ACTH levels. A negative change from baseline indicates improvement.
Core Baseline, Day 15 (Core study), Months 6, 12, 24 and Month 105 (end of the study)
Change From Baseline in Gene-expression and Protein in Blood and Urine for Biomarker Development
Time Frame: Baseline to end of the study
Baseline to end of the study

Collaborators and Investigators

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

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticlas, Novartis Pharmaceuticals

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)

August 13, 2004

Primary Completion (Actual)

July 8, 2013

Study Completion (Actual)

July 8, 2013

Study Registration Dates

First Submitted

September 13, 2005

First Submitted That Met QC Criteria

September 13, 2005

First Posted (Estimate)

September 15, 2005

Study Record Updates

Last Update Posted (Actual)

June 2, 2021

Last Update Submitted That Met QC Criteria

May 6, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CSOM230B2208E1
  • 2004-002407-32 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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.

Clinical Trials on Cushing Disease

Clinical Trials on Pasireotide

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Dominican Republic

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe