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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: April 6, 2011
Last updated: March 8, 2017
Last verified: March 2017
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.

Condition Intervention Phase
Cushing's Disease
Drug: LCI699
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Novartis:

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

Secondary Outcome Measures:
  • 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 ]

Enrollment: 33
Actual Study Start Date: March 23, 2011
Estimated Study Completion Date: December 21, 2018
Estimated Primary Completion Date: December 21, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LCI699
Ascending dose from 2mg bid or 5 mg bid, up to 30 mg bid
Drug: LCI699


Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01331239

United States, Illinois
Northwestern University Endo, Metabolism and Molecular
Chicago, Illinois, United States, 60611-3308
United States, Massachusetts
Massachusetts General Hospital Neuroendocrine Unit
Boston, Massachusetts, United States, 02114
United States, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
United States, Oregon
Oregon Health & Science University SC
Portland, Oregon, United States, 97239-3098
Novartis Investigative Site
Le Kremlin Bicetre, France, 94275
Novartis Investigative Site
Paris, France, 75014
Novartis Investigative Site
Padova, PD, Italy, 35128
Novartis Investigative Site
Ancona, Italy, l60020
Novartis Investigative Site
Napoli, Italy, 80131
Novartis Investigative Site
Chiba-city, Chiba, Japan, 260-8677
Novartis Investigative Site
Sapporo-city, Hokkaido, Japan, 060-8648
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Novartis Pharmaceuticals Identifier: NCT01331239     History of Changes
Other Study ID Numbers: CLCI699C2201
2010-022403-22 ( EudraCT Number )
Study First Received: April 6, 2011
Last Updated: March 8, 2017

Keywords provided by Novartis:
Cushing Disease
Pituitary Gland
Adrenocorticotropic Hormone

Additional relevant MeSH terms:
Pituitary ACTH Hypersecretion
ACTH-Secreting Pituitary Adenoma
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Endocrine System Diseases
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Pituitary Neoplasms
Endocrine Gland Neoplasms
Neoplasms by Site processed this record on April 21, 2017