Clinical Trial: Safety and Efficacy of LCI699 in Cushing's Disease Patients.

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Proof of Concept, Open-label, Forced Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of 10-weeks Treatment of LCI699 Followed by a 12 - Week Treatment Period of LCI699 i

Brief Summary: This exploratory study is a proof of concept study to determine whether LCI699 can safely reduce the level of urinary free cortisol in patients with Cushing's disease. In addition, this study will evaluate the long term efficacy and safety of LCI699 including an additional 12 week of treatment followed by a 12 month long term optional extension. A second extension will provide patients who are clinically benefitting from LCI699 an opportunity to continue to have access to the drug until LCI699 is commercially available and reimbursed or through the availability of a local access program.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Change in 24 hour urine free cortisol concentration [ Time Frame: baseline, 10 weeks ]

Original Primary Outcome: Change in 24 hour urine free cortisol concentration [ Time Frame: 10 weeks ]

Current Secondary Outcome:

  • Changes on steroid hormones of the HPA-axis in plasma, urine and saliva [ Time Frame: baseline, 10 weeks, 22 weeks ]
  • Changes in metabolic abnormalities, e.g. insulin, Hemoglobin A1C (HbA1C) [ Time Frame: baseline, 10 weeks, 22 weeks ]
  • Safety and tolerability of multiple doses of LCI699 [ Time Frame: baseline, 10 weeks ]
  • Change in 24 hour urine free cortisol concentration [ Time Frame: baseline, 22 weeks ]
  • Safety and tolerability of multiple doses of LCI699 [ Time Frame: baseline, 22 weeks ]


Original Secondary Outcome:

  • Changes in hormone levels, e.g. testosterone, estradiol [ Time Frame: 10 weeks ]
  • Changes in metabolic abnormalities, e.g. insulin, Hemoglobin A1C (HbA1C) [ Time Frame: 10 weeks ]


Information By: Novartis

Dates:
Date Received: April 6, 2011
Date Started: March 23, 2011
Date Completion: December 21, 2018
Last Updated: March 8, 2017
Last Verified: March 2017