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Safety and Efficacy of LCI699 in Cushing's Disease Patients

This study is currently recruiting participants.
Verified April 2013 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01331239
First received: April 6, 2011
Last updated: April 5, 2013
Last verified: April 2013
History of Changes
  Purpose

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.


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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Change in 24 hour urine free cortisol concentration [ Time Frame: baseline, 10 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Changes on steroid hormones of the HPA-axis in plasma, urine and saliva [ Time Frame: baseline, 10 weeks ] [ Designated as safety issue: No ]
  • Changes in metabolic abnormalities, e.g. insulin, Hemoglobin A1C (HbA1C) [ Time Frame: baseline, 10 weeks ] [ Designated as safety issue: No ]
  • Safety and tolerability of multiple doses of LCI699 [ Time Frame: baseline, 10 weeks ] [ Designated as safety issue: Yes ]
  • Change in 24 hour urine free cortisol concentration [ Time Frame: baseline, 22 weeks ] [ Designated as safety issue: No ]
  • Safety and tolerability of multiple doses of LCI699 [ Time Frame: baseline, 22 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 39
Study Start Date: March 2011
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LCI699
Ascending dose from 2mg bid or 5 mg bid, up to 50 mg bid
 Drug: LCI699

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01331239

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals +41-61-324-1111 ext +41613241111

Locations
United States, Illinois
Northwestern University Endo, Metabolism and Molecular Recruiting
Chicago, Illinois, United States, 60611-3308
Contact     312-503-4130        
Principal Investigator: Mark Molitch            
United States, Massachusetts
Massachusetts General Hospital Neuroendocrine Unit Recruiting
Boston, Massachusetts, United States, 02114
Principal Investigator: Beverly M. K. Biller            
Brigham and Women's Hospital SC Not yet recruiting
Boston, Massachusetts, United States, 02115
Contact: Charles Kelly     617-732-6940     ckellyIII@partners.org    
Principal Investigator: Whitney Woodmansee            
United States, Michigan
University of Michigan Health System Dept of Oncology Not yet recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Carole Ramm     734-615-8914     cmcr@umich.edu    
Principal Investigator: Ariel Barkan            
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Principal Investigator: Amir Hamrahian            
United States, Oregon
Oregon Health & Science University SC Recruiting
Portland, Oregon, United States, 97239-3098
Contact: Rian Vaught     503-494-9546     vaughtr@ohsu.edu    
Principal Investigator: Maria Fleseriu            
United States, Texas
Baylor College of Medicine SC-2 Not yet recruiting
Dallas, Texas, United States, 75246
Contact: Maribeth Staudinger     713-873-8772     stauding@bcm.edu    
Principal Investigator: Susan L. Samson            
France
Novartis Investigative Site Recruiting
Le Kremlin Bicetre, France, 94275
Novartis Investigative Site Recruiting
Paris, France, 75006
Italy
Novartis Investigative Site Not yet recruiting
Padova, PD, Italy, 35128
Novartis Investigative Site Recruiting
Ancona, Italy, l60020
Novartis Investigative Site Recruiting
Napoli, Italy, 80131
Japan
Novartis Investigative Site Recruiting
Sapporo-city, Hokkaido, Japan, 060-8648
Novartis Investigative Site Not yet recruiting
Kyoto-city, Kyoto, Japan, 612-8555
Novartis Investigative Site Withdrawn
Hamamatsu, Shizuoka, Japan, 431-3192
Novartis Investigative Site Recruiting
Minato-ku, Tokyo, Japan, 105-8470
Novartis Investigative Site Recruiting
Chiba, Japan, 260-8677
Novartis Investigative Site Recruiting
Yamagata, Japan, 990-9585
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01331239     History of Changes
Other Study ID Numbers: CLCI699C2201, 2010-022403-22
Study First Received: April 6, 2011
Last Updated: April 5, 2013
Health Authority: United States: Food and Drug Administration
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Italy: Ethics Committee
Japan: Pharmaceuticals and Medical Devices Agency

Keywords provided by Novartis:
Cushing Disease
LCI699
Pituitary Gland
Adrenocorticotropic Hormone

Additional relevant MeSH terms:
Cushing Syndrome
Pituitary ACTH Hypersecretion
Adrenocortical Hyperfunction
Adrenal Gland Diseases
Endocrine System Diseases
Hyperpituitarism
Pituitary Diseases
Hypothalamic Diseases
 Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Adrenocorticotropic Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 23, 2013