Clinical Trial: Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 D

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.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: 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


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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: every visit for 96 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


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: June 8, 2015
Date Started: September 24, 2015
Date Completion: September 5, 2019
Last Updated: May 15, 2017
Last Verified: May 2017