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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 28, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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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
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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


Arms and Interventions
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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
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Primary Outcome Measures :
  1. 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 :
  1. 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

  2. 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

  3. 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

  4. 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

  5. Change in serum cortisol [ Time Frame: 12, 24 and 48 weeks ]
    Absolute and percent change from baseline in morning serum cortisol

  6. Change in steroid hormones [ Time Frame: 12, 24 and 48 weeks ]
    Absolute and percent change from baseline in several hormones

  7. 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

  8. 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

  9. 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

  10. 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
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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
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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
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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
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02468193    
Other Study ID Numbers: CLCI699C1201
First Posted: June 10, 2015    Key Record Dates
Last Update Posted: August 28, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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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