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

This study is currently recruiting participants.
Verified November 2017 by Novartis ( Novartis Pharmaceuticals )
Sponsor:
ClinicalTrials.gov Identifier:
NCT02468193
First Posted: June 10, 2015
Last Update Posted: November 14, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
  Purpose
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 Intervention Phase
Cushing's Syndrome Ectopic Corticotropin Syndrome Adrenal Adenoma Adrenal Carcinoma AIMAH PPNAD Drug: Osilodrostat Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

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


Estimated Enrollment: 10
Actual Study Start Date: September 24, 2015
Estimated Study Completion Date: October 31, 2019
Estimated Primary Completion Date: June 21, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

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.


Ages Eligible for Study:   18 Years to 85 Years   (Adult, Senior)
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


Contacts
Contact: Novartis Pharmaceuticals +81337978748 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals novartis.email@novartis.com

Locations
Japan
Novartis Investigative Site Recruiting
Fukuoka city, Fukuoka, Japan, 812-8582
Novartis Investigative Site Recruiting
Fukushima-city, Fukushima, Japan, 960-1295
Novartis Investigative Site Recruiting
Kobe-city, Hyogo, Japan, 650-0017
Novartis Investigative Site Recruiting
Nishinomiya-city, Hyogo, Japan, 663 8501
Novartis Investigative Site Recruiting
Yokohama, Kanagawa, Japan, 245-8575
Novartis Investigative Site Recruiting
Sendai-city, Miyagi, Japan, 980-8574
Novartis Investigative Site Recruiting
Chiba, Japan, 260-8677
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02468193     History of Changes
Other Study ID Numbers: CLCI699C1201
First Submitted: June 8, 2015
First Posted: June 10, 2015
Last Update Posted: November 14, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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