Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

ClinicalTrials.gov Identifier: NCT02468193

Recruitment Status : Completed

First Posted : June 10, 2015

Last Update Posted : August 26, 2019

Sponsor:

Information provided by (Responsible Party):

Novartis ( Novartis Pharmaceuticals )

Study Description

Brief Summary:

The study aim is to investigate the efficacy and safety of Osilodrostat in patients with Cushing's syndrome due to causes other than Cushing's disease in Japan.

Condition or disease	Intervention/treatment	Phase
Cushing's Syndrome Ectopic Corticotropin Syndrome Adrenal Adenoma Adrenal Carcinoma AIMAH PPNAD	Drug: Osilodrostat	Phase 2

Study Design

Layout table for study information

Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	9 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	A Phase II, Open-label, Dose Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of Osilodrostat in Patients With All Types of Endogenous Cushing's Syndrome Except Cushing's Disease
Actual Study Start Date :	September 24, 2015
Actual Primary Completion Date :	June 7, 2018
Actual Study Completion Date :	October 29, 2018

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Cushing disease Primary macronodular adrenal hyperplasia

MedlinePlus related topics: Cushing's Syndrome

Genetic and Rare Diseases Information Center resources: 21-hydroxylase Deficiency Congenital Adrenal Hyperplasia Cushing's Syndrome ACTH-secreting Pituitary Adenoma Adenoma of the Adrenal Gland Adrenocortical Carcinoma ACTH-independent Macronodular Adrenal Hyperplasia Hyperadrenalism

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: Osilodrostat	Drug: Osilodrostat Osilodrostat will be prescribed by the investigator. Osirodrostat 1 mg, 5 mg, 10 mg in the form of film-coated tablets will be used for oral administration Other Name: LCI699

Outcome Measures

Primary Outcome Measures :

Percent change in the mean Urine Free Cortisol (mUFC) at Week 12 [ Time Frame: 12 weeks ]
Percent change from baseline in the mUFC at the individual patient level

Secondary Outcome Measures :

Percent change in the mUFC at Week 24 and Week 48 [ Time Frame: 24 and 48 weeks ]
Percent change from baseline in the mUFC at the individual patient level
Change from baseline in the mUFC at Week 12, Week 24 and Week 48 [ Time Frame: Baseline, 12, 24 and 48 weeks ]
Absolute and percent change from baseline in the mUFC
Complete, partial, and overall response rate [ Time Frame: 12, 24 and 48 weeks ]
Complete and Partial response rate Overall response rate: proportion of patients with complete or partial responses
Change in serum cortisol [ Time Frame: 12, 24 and 48 weeks ]
Absolute and percent change from baseline in morning serum cortisol at the individual patient level
Change in serum cortisol [ Time Frame: 12, 24 and 48 weeks ]
Absolute and percent change from baseline in morning serum cortisol
Change in steroid hormones [ Time Frame: 12, 24 and 48 weeks ]
Absolute and percent change from baseline in several hormones
Change in cardiovascular-related metabolic parameters associated with Cushing's syndrome [ Time Frame: 12, 24 and 48 weeks ]
Absolute and percent change in metabolic parameters associated with Cushing's syndrome
General safety of LCI699 [ Time Frame: For 72 weeks ]
Adverse events and laboratory abnormalities will be assessed using the National Cancer Institute-Common Toxicology Criteria grading scale
Change from baseline in Patient-Reported Outcomes [ Time Frame: Baseline, 12, 24 and 48 weeks ]
Change from baseline in standardized score of Patient-Reported Outcomes will be assessed
Plasma concentrations of LCI699 [ Time Frame: 48 weeks ]
Exposures of LCI699 in patients with Cushing's syndrome will be evaluated at the scheduled time point

Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information

Ages Eligible for Study:	18 Years to 85 Years (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients confirmed Cushing's syndrome [i.e. ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, ACTH-Independent Macronodular Adrenal Hyperplasia (AIMAH), or Primary Pigmented Nodular Adrenal Dysplasia (PPNAD)]
For patients on medical treatment for hypercortisolism due to Cushing's syndrome, the washout periods must be completed prior to baseline efficacy assessments

Exclusion Criteria:

Patients with Cushing's disease
History of hypersensitivity to osilodrostat or to drugs of similar chemical classes
History of malignancy of any organ system, treated or untreated, within the past 5 years
Patients receiving treatment for within 4 weeks or ≤5 x half-life of the agent (whichever is longer) before first dose of osilodrostat
Patients with risk factors for QTc prolongation or Torsade de Pointes

Other protocol defined Inclusion/Exclusion may apply.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02468193

Locations

Layout table for location information

Japan
Novartis Investigative Site
Fukushima city, Fukushima, Japan, 960 1295
Novartis Investigative Site
Yokohama, Kanagawa, Japan, 245-8575
Novartis Investigative Site
Sendai city, Miyagi, Japan, 980 8574
Novartis Investigative Site
Chiba, Japan, 260 8677

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Layout table for investigator information

Study Director:	Novartis Pharmaceuticals	Novartis Pharmaceuticals

More Information

Layout table for additonal information

Responsible Party:	Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT02468193 History of Changes
Other Study ID Numbers:	CLCI699C1201
First Posted:	June 10, 2015 Key Record Dates
Last Update Posted:	August 26, 2019
Last Verified:	August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	No

Layout table for additional information

Studies a U.S. FDA-regulated Drug Product:	No
Studies a U.S. FDA-regulated Device Product:	No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):


Cushing's syndrome osilodrostat LCI699 ectopic corticotropin syndrome adrenal adenoma	adrenal carcinoma ACTH-Independent Macronodular Adrenal Hyperplasia AIMAH Primary Pigmented Nodular Adrenal Dysplasia PPNAD

Additional relevant MeSH terms:

Layout table for MeSH terms

Adenoma Adrenocortical Adenoma Adrenocortical Carcinoma ACTH Syndrome, Ectopic Cushing Syndrome Syndrome Disease Pathologic Processes Carcinoma Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type Neoplasms	Adrenocortical Hyperfunction Adrenal Gland Diseases Endocrine System Diseases Adrenal Cortex Neoplasms Adrenal Gland Neoplasms Endocrine Gland Neoplasms Neoplasms by Site Adrenal Cortex Diseases Adenocarcinoma Paraneoplastic Endocrine Syndromes Paraneoplastic Syndromes

To Top