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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01331239
Recruitment Status : Completed
First Posted : April 8, 2011
Last Update Posted : September 7, 2020
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:

This exploratory study was a proof of concept study to determine whether LCI699 could 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.

Condition or disease Intervention/treatment Phase
Cushing's Disease Drug: LCI699 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 33 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
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
Actual Study Start Date : March 23, 2011
Actual Primary Completion Date : October 22, 2019
Actual Study Completion Date : October 22, 2019

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: LCI699
Participants too an ascending dose from 2mg bid or 5 mg bid, up to 30 mg bid
Drug: LCI699

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

Secondary Outcome Measures :
  1. Changes on steroid hormones of the HPA-axis in plasma, urine and saliva [ Time Frame: baseline, 10 weeks, 22 weeks ]
  2. Changes in metabolic abnormalities, e.g. insulin, Hemoglobin A1C (HbA1C) [ Time Frame: baseline, 10 weeks, 22 weeks ]
  3. Safety and tolerability of multiple doses of LCI699 [ Time Frame: baseline, 10 weeks ]
  4. Change in 24 hour urine free cortisol concentration [ Time Frame: baseline, 22 weeks ]
  5. Safety and tolerability of multiple doses of LCI699 [ Time Frame: baseline, 22 weeks ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01331239

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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 and 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
Ancona, Italy, L60020
Novartis Investigative Site
Napoli, Italy, 80131
Novartis Investigative Site
Sapporo city, Hokkaido, Japan, 060 8648
Novartis Investigative Site
Chiba, Japan, 260 8677
Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01331239    
Other Study ID Numbers: CLCI699C2201
2010-022403-22 ( EudraCT Number )
First Posted: April 8, 2011    Key Record Dates
Last Update Posted: September 7, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Cushing Disease
Pituitary Gland
Adrenocorticotropic Hormone
Additional relevant MeSH terms:
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ACTH-Secreting Pituitary Adenoma
Pituitary ACTH Hypersecretion
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