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

Status:	Active, not recruiting
Conditions:	Endocrine
Therapuetic Areas:	Endocrinology
Healthy:	No
Age Range:	18 - 75
Updated:	11/9/2018
Start Date:	March 23, 2011
End Date:	December 21, 2018

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

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.

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

We found this trial at

sites

Northwestern University Endo, Metabolism and Molecular

Chicago, Illinois 60611

from

Chicago, IL