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Completed

NCT Number: NCT00171951

Extension Study to Assess the Safety and Efficacy of Pasireotide in Participants 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.

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Key information

Conditions

Cushing Disease Brain Diseases Central Nervous System Diseases Endocrine System Diseases Hyperpituitarism Hypothalamic Diseases Nervous System Diseases Pituitary ACTH Hypersecretion Pituitary Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Novartis Investigative Site, Paris, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Pasireotide

Drug

Pasireotide 600 μg or 900 μg was administered as an SC injection.

Other names: SOM230

Primary outcomes

  1. 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.

  2. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. Change From Baseline in Gene-expression and Protein in Blood and Urine for Biomarker Development

    Time frame: Baseline to end of the study

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2004
Primary completion
2013
Study completion
2013
First posted
Sep 15, 2005
Registry last updated
Jun 2, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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