This trial is active, not recruiting.

Condition cushing's disease
Treatment lci699
Phase phase 2
Sponsor Novartis Pharmaceuticals
Start date March 2011
End date December 2018
Trial size 33 participants
Trial identifier NCT01331239, 2010-022403-22, CLCI699C2201


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose treatment
Ascending dose from 2mg bid or 5 mg bid, up to 30 mg bid

Primary Outcomes

Change in 24 hour urine free cortisol concentration
time frame: baseline, 10 weeks

Secondary Outcomes

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

Eligibility Criteria

Male or female participants from 18 years up to 75 years old.

Inclusion Criteria: - Patients with a confirmed diagnosis of Cushing's Disease (persistent or recurrent) as evidenced by increased 24-hour urine free cortisol (UFC), normal or increased morning plasma Adrenocorticotropic Hormone (ACTH), and pituitary origin of excess ACTH. - Patients with de novo Cushing's disease can be included only if they are not considered candidate for surgery Exclusion Criteria: - Patients treated with mitotane 6 months prior to Visit 1 - Patients with compression of the optic chiasm - Patients with a known inherited syndrome as the cause for hormone over secretion - Patients with Cushing's syndrome due to ectopic ACTH secretion or adrenal Cushing's syndrome - Patients with pseudo-Cushing's syndrome - Patients who are not biochemically euthyroid - Diabetic patients with poorly controlled diabetes (HbA1c >9%) - Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 week after completion of dosing. - Patients who have received pituitary irradiation within five years prior to Visit 1. - Patients with risk factors for QTc prolongation or Torsade de Pointes. Other protocol-defined inclusion/exclusion criteria may apply

Additional Information

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 in Patients With Cushing's Disease
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Novartis.