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Trial record 23 of 34 for:    Biliary Cirrhosis, Primary, 4

A Study of Efficacy and Safety of Ustekinumab in Patients With Primary Biliary Cirrhosis (PBC) Who Had an Inadequate Response to Ursodeoxycholic Acid

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ClinicalTrials.gov Identifier: NCT01389973
Recruitment Status : Completed
First Posted : July 8, 2011
Results First Posted : October 15, 2014
Last Update Posted : June 20, 2016
Sponsor:
Information provided by (Responsible Party):
Janssen Research & Development, LLC

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Primary Biliary Cirrhosis
Interventions Drug: ustekinumab 90 mg
Drug: ustekinumab 45 mg
Drug: ustekinumab 180 mg
Drug: Placebo
Enrollment 20
Recruitment Details The study had 2 parts, Part 1 (proof of concept) and Part 2. As per the study design, the study was considered completed after part 1 and part 2 was not initiated. Results shown below are for Part 1 of the study.
Pre-assignment Details  
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
Period Title: Overall Study
Started 20
Completed 0
Not Completed 20
Reason Not Completed
Lost to Follow-up             1
Trial Stopped by Sponsor             9
Other             10
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
Efficacy analysis set included all participants who received at least 1 administration of ustekinumab (full or partial).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants
48.8  (10.13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Female
19
  95.0%
Male
1
   5.0%
1.Primary Outcome
Title Part 1: Number of Participants With Alkaline Phosphatase (ALP) Response at Week 12
Hide Description The ALP response was defined as a greater than 40 percent (%) decrease from Baseline in ALP concentration at Week 12.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 administration of ustekinumab (full or partial).
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description:
In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
0
2.Secondary Outcome
Title Part 1: Number of Participants With ALP Response at Week 28
Hide Description [Not Specified]
Time Frame Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 administration of ustekinumab (full or partial).
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description:
In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
0
3.Secondary Outcome
Title Part 1: Number of Participants With ALP Remission at Week 28
Hide Description ALP remission is defined as either normalization of ALP (for participants with baseline ALP between 1.67*and 2.8* upper limit of normal [ULN] or an ALP less than [˂]1.67*ULN [for participants with baseline ALP greater than {˃} 2.8* ULN]). ALP levels above 1.67* ULN level were associated with an increased rate of disease progression.
Time Frame Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 administration of ustekinumab (full or partial).
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description:
In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
0
4.Secondary Outcome
Title Part 1: Percent Change From Baseline in ALP Concentration at Week 28
Hide Description [Not Specified]
Time Frame Baseline and Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 administration of ustekinumab (full or partial).
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description:
In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: percent change
-11.25  (17.462)
5.Secondary Outcome
Title Part 1: Percent Change From Baseline in Alanine Aminotransferase, Aspartate Aminotransferase, and Bilirubin Concentration at Week 28
Hide Description [Not Specified]
Time Frame Baseline and Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy analysis set included all participants who received at least 1 administration of ustekinumab (full or partial). "N" (number of participants analyzed) signifies participants who were evalubale for this outcome measure. "n" signifies participants who were evalubale for each specified category.
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description:
In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: percent change
Alanine Aminotransferase (n=18) -9.21  (32.362)
Aspartate Aminotransferase (n=18) -7.22  (27.188)
Bilirubin (n=3) -4.61  (14.183)
Time Frame Week 0 up to final safety visit in Part 1
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Open-label: Ustekinumab 90 mg
Hide Arm/Group Description In proof of concept phase of Part 1, participants received ustekinumab 90 milligram (mg) subcutaneously at Week 0, 4 and thereafter every 8 weeks through Week 20. Eligible participants entered long-term extension which began at Week 28 and continued through Week 216.
All-Cause Mortality
Open-label: Ustekinumab 90 mg
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Open-label: Ustekinumab 90 mg
Affected / at Risk (%)
Total   1/20 (5.00%) 
Gastrointestinal disorders   
Upper gastrointestinal haemorrhage *  1/20 (5.00%) 
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Open-label: Ustekinumab 90 mg
Affected / at Risk (%)
Total   18/20 (90.00%) 
Blood and lymphatic system disorders   
Anaemia *  1/20 (5.00%) 
Eye disorders   
Conjunctivitis *  1/20 (5.00%) 
Dry eye *  1/20 (5.00%) 
Gastrointestinal disorders   
Nausea *  3/20 (15.00%) 
Abdominal pain *  2/20 (10.00%) 
Abdominal pain upper *  2/20 (10.00%) 
Diarrhoea *  2/20 (10.00%) 
Dyspepsia *  1/20 (5.00%) 
Hypoaesthesia oral *  1/20 (5.00%) 
Inguinal hernia *  1/20 (5.00%) 
Paraesthesia oral *  1/20 (5.00%) 
Rectal haemorrhage *  1/20 (5.00%) 
Salivary gland enlargement *  1/20 (5.00%) 
Toothache *  1/20 (5.00%) 
General disorders   
Fatigue *  8/20 (40.00%) 
Feeling abnormal *  1/20 (5.00%) 
Injection site bruising *  1/20 (5.00%) 
Injection site erythema *  1/20 (5.00%) 
Pyrexia *  1/20 (5.00%) 
Hepatobiliary disorders   
Hepatic pain *  1/20 (5.00%) 
Immune system disorders   
Seasonal allergy *  1/20 (5.00%) 
Infections and infestations   
Urinary tract infection *  4/20 (20.00%) 
Influenza *  2/20 (10.00%) 
Nasopharyngitis *  2/20 (10.00%) 
Oral herpes *  2/20 (10.00%) 
Sinusitis *  2/20 (10.00%) 
Upper respiratory tract infection *  2/20 (10.00%) 
Bronchitis *  1/20 (5.00%) 
Ear infection *  1/20 (5.00%) 
Vaginal infection *  1/20 (5.00%) 
Injury, poisoning and procedural complications   
Laceration *  1/20 (5.00%) 
Rib fracture *  1/20 (5.00%) 
Investigations   
Weight decreased *  1/20 (5.00%) 
Musculoskeletal and connective tissue disorders   
Back pain *  3/20 (15.00%) 
Arthralgia *  2/20 (10.00%) 
Bursitis *  1/20 (5.00%) 
Musculoskeletal stiffness *  1/20 (5.00%) 
Pain in extremity *  1/20 (5.00%) 
Nervous system disorders   
Headache *  5/20 (25.00%) 
Migraine *  1/20 (5.00%) 
Paraesthesia *  1/20 (5.00%) 
Radiculopathy *  1/20 (5.00%) 
Psychiatric disorders   
Insomnia *  1/20 (5.00%) 
Renal and urinary disorders   
Dysuria *  1/20 (5.00%) 
Reproductive system and breast disorders   
Menorrhagia *  2/20 (10.00%) 
Dysmenorrhoea *  1/20 (5.00%) 
Vulvovaginal dryness *  1/20 (5.00%) 
Vulvovaginal pruritus *  1/20 (5.00%) 
Respiratory, thoracic and mediastinal disorders   
Allergic sinusitis *  1/20 (5.00%) 
Asthma *  1/20 (5.00%) 
Dyspnoea *  1/20 (5.00%) 
Sinus congestion *  1/20 (5.00%) 
Epistaxis *  1/20 (5.00%) 
Oropharyngeal pain *  1/20 (5.00%) 
Skin and subcutaneous tissue disorders   
Alopecia *  2/20 (10.00%) 
Pruritus *  2/20 (10.00%) 
Rash *  2/20 (10.00%) 
Dry skin *  1/20 (5.00%) 
Skin lesion *  1/20 (5.00%) 
Vascular disorders   
Raynaud's phenomenon *  1/20 (5.00%) 
*
Indicates events were collected by non-systematic assessment
Based on the efficacy results of Part 1, Part 2 of the study was not initiated and as per the study design, the study was considered completed after part 1.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Senior Director, Clinical Development, Immunology
Organization: Janssen, R&D, LLC, Spring House, PA
EMail: ClinicalTrialDisclosure@its.jnj.com
Layout table for additonal information
Responsible Party: Janssen Research & Development, LLC
ClinicalTrials.gov Identifier: NCT01389973     History of Changes
Other Study ID Numbers: CR018748
CNTO1275PBC2001 ( Other Identifier: Janssen Research & Development, LLC )
2011-000554-31 ( EudraCT Number )
First Submitted: July 7, 2011
First Posted: July 8, 2011
Results First Submitted: August 19, 2014
Results First Posted: October 15, 2014
Last Update Posted: June 20, 2016