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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01331239
• Date of registration: 06/04/2011
• Prospective Registration: No
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Safety and Efficacy of LCI699 in Cushing's Disease Patients.
• Scientific 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
• Date of first enrolment: March 23, 2011
• Target sample size: 33
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01331239
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

France, Italy, Japan, United States

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cushing's Disease

Intervention(s)

Drug: LCI699

Primary Outcome(s)

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

Secondary Outcome(s)

Safety and tolerability of multiple doses of LCI699 [Time Frame: baseline, 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]

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]

Secondary ID(s)

CLCI699C2201

2010-022403-22

Source(s) of Monetary Support

Please refer to primary and secondary sponsors