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A Multi-part, Double Blind Study to Assess Safety, Tolerability and Efficacy of LJN452 in PBC Patients

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ClinicalTrials.gov Identifier: NCT02516605
Recruitment Status : Recruiting
First Posted : August 6, 2015
Last Update Posted : October 10, 2017
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
A multi-part study to assess safety, tolerability and efficacy of LJN452 in patients with primary biliary cholangitis

Condition or disease Intervention/treatment Phase
Primary Biliary Cholangitis Drug: LJN452 Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 95 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Multi-part, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability and Efficacy of LJN452 in Patients With Primary Biliary Cholangitis
Actual Study Start Date : September 9, 2015
Estimated Primary Completion Date : December 27, 2017
Estimated Study Completion Date : May 5, 2019

Arm Intervention/treatment
Experimental: LJN452 Drug: LJN452
LJN452 capsules administered once daily for 28 days
Placebo Comparator: Placebo Drug: Placebo
Matching placebo capsules administered once daily for 28 days

Primary Outcome Measures :
  1. Change in cholestatic markers from baseline [ Time Frame: Baseline, 28 Days ]
    Evaluate the change in cholestatic markers following administration of LJN452

  2. Number of patients with adverse events, serious adverse events and death [ Time Frame: 86 days ]
    Determine safety and tolerability of LJN452

Secondary Outcome Measures :
  1. Change from baseline in disease specific quality of life [ Time Frame: Baseline, 28 days ]
    Evaluate change in quality of life using PBC-40 PRO

  2. Change from baseline in itch as determined by the itch domain in the PBC-40 [ Time Frame: Baseline, 28 days ]
    Evaluate the change in itch using the itch domain in the PBC-40

  3. Change from baseline in itch as determined by 10mm VAS [ Time Frame: Baseline, 28 Days ]
    Evaluate the change in itch from baseline with a 10 mm VAS

  4. Area under the plasma concentration-time profile (AUCtau) [ Time Frame: Day 1, Day 28 ]
    Evalaute AUCtau

  5. Maximum plasma concentration of LJN452 (Cmax) [ Time Frame: Day 1, Day 28 ]
    Evaluate Cmax

  6. Minimum plasma concentration of LJN452 (Cmin) [ Time Frame: Day 28 ]
    Evaluate Cmin

  7. Average steady state plasma concentration following multiple doses of LJN452 (Cav,ss) [ Time Frame: Day 28 ]
    Evaluate Cav,ss

  8. Time to reach maximum concentration after durg administration (Tmax) [ Time Frame: Day 1, Day 28 ]
    Evalute Tmax

Information from the National Library of Medicine

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

Inclusion Criteria:

  • Diagnosis of PBC as demonstrated by the presence of ≥ 2 of the following 3 diagnostic criteria:
  • History of Alkaline Phosphatase elevated above upper limit of normal for at least 6 months
  • Positive anti-mitochondrial antibodies (AMA) titer or if AMA negative or in low titer (<1:80) PBC specific antibodies (anti- GP210 and/or anti-SP100 and/or antibodies against the major M2 components (PDC-E2, 2-oxo-glutaric acid dehydrogenase complex)
  • Previous liver biopsy consistent with PBC
  • At least 1 of the following markers of disease severity:
  • ALP ≥ 1.67× upper limit of normal (ULN)
  • Total bilirubin > ULN but < 2× ULN
  • Subjects must weigh at least 40 kg to participate in the study, and must have a body mass index (BMI) within the range of 18 - 40 kg/m^2. BMI = Body weight (kg) / [Height (m)]^2.
  • Able to communicate well with the investigator, to understand and comply with the requirements of the study.

Exclusion Criteria:

  • 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 5 days following end of treatment.
  • History or presence of other concomitant liver diseases
  • Clinically significant hepatic decompensation.
  • History of any venous thromboembolism, TIA, intracranial hemorrhage, neoplasm, arteriovenous malformation, vasculitis, bleeding disorder, coagulation disorders or screening blood tests that indicate altered coagulability (e.g. platelet count, aPTT, PTT or TT tests).
  • History of conditions that may cause increases in ALP (e.g., Paget's disease).
  • Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment

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 ClinicalTrials.gov identifier (NCT number): NCT02516605

Contact: Novartis Pharmaceuticals 1-888-669-6682 Novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

United States, California
Novartis Investigative Site Recruiting
Rialto, California, United States, 92377
United States, Florida
Novartis Investigative Site Recruiting
Bradenton, Florida, United States, 34211
Novartis Investigative Site Recruiting
Miami, Florida, United States, 33136
United States, Georgia
Novartis Investigative Site Recruiting
Atlanta, Georgia, United States, 30308
Novartis Investigative Site Recruiting
Marietta, Georgia, United States, 30060
United States, Illinois
Novartis Investigative Site Recruiting
Chicago, Illinois, United States, 60612
United States, Kentucky
Novartis Investigative Site Withdrawn
Louisville, Kentucky, United States, 40202
United States, Minnesota
Novartis Investigative Site Recruiting
Rochester, Minnesota, United States, 55905
United States, New York
Novartis Investigative Site Recruiting
Manhasset, New York, United States, 11030
United States, Texas
Novartis Investigative Site Recruiting
San Antonio, Texas, United States, 78215
United States, Washington
Novartis Investigative Site Recruiting
Seattle, Washington, United States, 98104
Canada, Alberta
Novartis Investigative Site Recruiting
Calgary, Alberta, Canada, T2N 4N1
Novartis Investigative Site Recruiting
Edmonton, Alberta, Canada, T6G 2B7
Novartis Investigative Site Recruiting
Hamburg, Germany, 20246
Novartis Investigative Site Recruiting
Hannover, Germany, 30625
Novartis Investigative Site Recruiting
Heidelberg, Germany, 69120
Novartis Investigative Site Recruiting
Munchen, Germany, 81377
Novartis Investigative Site Recruiting
Wurzburg, Germany, 97080
United Kingdom
Novartis Investigative Site Recruiting
Birmingham, West Midlands, United Kingdom, B15 2TH
Novartis Investigative Site Recruiting
Cambridge, United Kingdom, CB2 2QQ
Novartis Investigative Site Recruiting
Hull, United Kingdom, HU3 2JZ
Novartis Investigative Site Recruiting
London, United Kingdom, NW3 2PF
Novartis Investigative Site Recruiting
Newcastle upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Novartis Pharmaceuticals

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02516605     History of Changes
Other Study ID Numbers: CLJN452X2201
2015-001590-41 ( EudraCT Number )
First Posted: August 6, 2015    Key Record Dates
Last Update Posted: October 10, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
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 www.clinicalstudydatarequest.com

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Primary Biliary Cirrhosis, PBC
Primary Biliary Cholangitis

Additional relevant MeSH terms:
Liver Cirrhosis, Biliary
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Liver Diseases
Liver Cirrhosis