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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

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

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome:

• Change in cholestatic markers from baseline [ Time Frame: Baseline, 28 Days ]
  Evaluate the change in cholestatic markers following administration of LJN452
• Number of patients with adverse events, serious adverse events and death [ Time Frame: 86 days ]
  Determine safety and tolerability of LJN452

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Change from baseline in disease specific quality of life [ Time Frame: Baseline, 28 days ]
  Evaluate change in quality of life using PBC-40 PRO
• 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
• 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
• Area under the plasma concentration-time profile (AUCtau) [ Time Frame: Day 1, Day 28 ]
  Evalaute AUCtau
• Maximum plasma concentration of LJN452 (Cmax) [ Time Frame: Day 1, Day 28 ]
  Evaluate Cmax
• Minimum plasma concentration of LJN452 (Cmin) [ Time Frame: Day 28 ]
  Evaluate Cmin
• Average steady state plasma concentration following multiple doses of LJN452 (Cav,ss) [ Time Frame: Day 28 ]
  Evaluate Cav,ss
• Time to reach maximum concentration after durg administration (Tmax) [ Time Frame: Day 1, Day 28 ]
  Evalute Tmax

Original Secondary Outcome:

• Change from baseline in disease specific quality of life [ Time Frame: Baseline, 28 days ]
  Evaluate change in quality of life using PBC-40 PRO
• 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
• 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
• Area under the plasma concentration-time profile (AUCtau) [ Time Frame: Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56 ]
  Evalaute AUCtau
• Maximum plasma concentration of LJN452 (Cmax) [ Time Frame: Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56 ]
  Evaluate Cmax
• Minimum plasma concentration of LJN452 (Cmin) [ Time Frame: Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56 ]
  Evaluate Cmin
• Average steady state plasma concentration following multiple doses of LJN452 (Cav,ss) [ Time Frame: Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56 ]
  Evaluate Cav,ss
• Time to reach maximum concentration after durg administration (Tmax) [ Time Frame: Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56 ]
  Evalute Tmax
• Apparent systemic clearance from plasma (CL/F) [ Time Frame: Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56 ]
  Evaluate CL/F
• Accumulation ratio of LJN452 (Racc) [ Time Frame: Day 1, Day 7, Day 14, Day 21, Day 28, Day 29, Day 56 ]
  Evaluate Racc

Information By: Novartis

Dates:
Date Received: August 4, 2015
Date Started: September 9, 2015
Date Completion: December 27, 2017
Last Updated: March 15, 2017
Last Verified: March 2017