Safety and Efficacy of LCI699 in Cushing's Disease Patients

• Status: Completed
• Trial end date: 2019-10-22
• Target enrollment: 0
• Participant gender: All

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 evaluated 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 provided patients who were clinically benefitting from LCI699 an opportunity to continue to have access to the drug until LCI699 was commercially available and reimbursed or through the availability of a local access program.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

Criteria

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.