Neoplasms

Nervous System Diseases

Gland and Hormone Related Diseases

All Conditions

Rare Diseases

ACTH-Secreting Pituitary Adenoma

Pituitary ACTH Hypersecretion

Adenoma

Pituitary Diseases

Hypothalamic Diseases

Brain Diseases

Central Nervous System Diseases

Endocrine System Diseases

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Pituitary Neoplasms

Endocrine Gland Neoplasms

ACTH-secreting Pituitary Adenoma

Anti-Inflammatory Agents

All Drugs and Chemicals

Central Nervous System Depressants

Other Dietary Supplements

Hydrocortisone

Hydrocortisone 17-butyrate 21-propionate

Hydrocortisone acetate

Hydrocortisone hemisuccinate

Pasireotide

Benzocaine

Hormones

Hormone Antagonists

Tannic Acid

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.

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

Novartis Pharmaceuticlas

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.

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

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

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.

24-hour urine samples were collected to obtain mean UFC measurements. A negative mean change from baseline indicates improvement.

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.

Blood samples were withdrawn to obtain the serum cortisol levels. A negative change from baseline indicates improvement.

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.

Blood samples were withdrawn to obtain the ACTH levels. A negative change from baseline indicates improvement.

Novartis Pharmaceuticals

Safety Population included participants from ITT Population. ITT Population included participants who completed 15 days of treatment in the Core study, experienced significant clinical benefit without any unacceptable AEs, and received at least 1 dose in the extension period.

Participants received pasireotide 600 μg BID SC, to achieve or maintain UFC normalization.

Pasireotide 600 μg BID SC

Participants received 900 μg BID SC if UFC levels were increased at any time, 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.

Pasireotide 900 μg BID SC

Participants received pasireotide 600 μg BID SC to achieve or maintain 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.

Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC

Age, Continuous

Sex: Female, Male

Intent-to-treat (ITT) population included all participants who completed 15 days of treatment in Core study and experienced significant clinical benefit from treatment and received at least 1 dose in the Extension Period.

Study Director

Primary Efficacy Population included participants from ITT whose mean UFC at core-baseline, based on at least 2 UFC samples, was \>1.0x upper limit of normal (ULN) or for whom the one and only UFC sample available at baseline was \>2.0xULN.

Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits

Core Baseline

Change from Core Baseline to Day 14/Day 15

Change from Core Baseline to Month 6

Change from Core Baseline to Month 12

Change from Core Baseline to Month 24

Change from Core Baseline to Month 102

Primary Efficacy Population included participants from ITT whose mean UFC at core-baseline, based on at least 2 UFC samples, was \>1.0x ULN or for whom the one and only UFC sample available at baseline was \>2.0xULN. Number analyzed is the number of participants with data available for analysis at the given time point.

Change From Baseline in Mean Urinary Free Cortisol (UFC)

Number of Participants Who Had At Least One Adverse Event (AE)

Change from Core Baseline to Day 15

Change from Core Baseline to Month 105

Change From Baseline in Serum Cortisol Levels

Day 15 (Core Study)

Month 6

Pharmacokinetic (PK) Population included participants from ITT who had at least one post dosing PK assessment up to the implementation of Amendment 2 when no further blood samples were collected for PK assessment (24 May 2007).

Plasma Trough Concentrations (Ctrough) of Pasireotide in UFC Responders

Change From Baseline in Plasma Adrenocorticotropic Hormone (ACTH) Levels

We have exhausted all efforts to locate this data and it has since been destroyed, therefore no data is available to report for this outcome measure.

Spots Global Cancer Trial Database for Extension Study to Assess the Safety and Efficacy of Pasireotide in Participants With Cushing's Disease

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Interventions

Study Description

Keywords

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