NCT01389973

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of ustekinumab in patients with primary biliary cirrhosis who had an inadequate response to ursodeoxycholic acid.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2011

Geographic Reach
2 countries

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 7, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 8, 2011

Completed
2 months until next milestone

Study Start

First participant enrolled

September 1, 2011

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2013

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

October 15, 2014

Completed
Last Updated

June 20, 2016

Status Verified

June 1, 2016

Enrollment Period

1.8 years

First QC Date

July 7, 2011

Results QC Date

August 19, 2014

Last Update Submit

June 17, 2016

Conditions

Primary Biliary Cirrhosis

Keywords

Diseases [C] - Digestive System Diseases [C06]primary biliary cirrhosisursodeoxycholic acid (UDCA)STELARAustekinumab

Outcome Measures

Primary Outcomes (1)

  • Part 1: Number of Participants With Alkaline Phosphatase (ALP) Response at Week 12

    The ALP response was defined as a greater than 40 percent (%) decrease from Baseline in ALP concentration at Week 12.

    Week 12

Secondary Outcomes (4)

  • Part 1: Number of Participants With ALP Response at Week 28

    Week 28

  • Part 1: Number of Participants With ALP Remission at Week 28

    Week 28

  • Part 1: Percent Change From Baseline in ALP Concentration at Week 28

    Baseline and Week 28

  • Part 1: Percent Change From Baseline in Alanine Aminotransferase, Aspartate Aminotransferase, and Bilirubin Concentration at Week 28

    Baseline and Week 28

Study Arms (5)

Open-label: ustekinumab 90 mg

EXPERIMENTAL
Drug: ustekinumab 90 mg

Double-blind: ustekinumab 45 mg

EXPERIMENTAL
Drug: ustekinumab 45 mg

Double-blind: ustekinumab 90 mg

EXPERIMENTAL
Drug: ustekinumab 90 mg

Double-blind: ustekinumb 180 mg

EXPERIMENTAL
Drug: ustekinumab 180 mg

Double-blind: placebo

PLACEBO COMPARATOR
Drug: Placebo

Interventions

ustekinumab 90 mgDRUG

Subcutaneous injections of ustekinumb 90 mg at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.

Double-blind: ustekinumab 90 mgOpen-label: ustekinumab 90 mg
ustekinumab 45 mgDRUG

Subcutaneous injections of ustekinumb 45 mg at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.

Double-blind: ustekinumab 45 mg
ustekinumab 180 mgDRUG

Subcutaneous injections of ustekinumb 180 mg at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.

Double-blind: ustekinumb 180 mg
PlaceboDRUG

Subcutaneous injections of placebo at Weeks 0 and 4 and every 8 weeks until the dose for the Long-Term Extension has been selected then every 8 weeks through Week 196 with the selected dose.

Double-blind: placebo

Eligibility Criteria

Age18 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Have proven or are likely to have Primary Biliary Cirrhosis (PBC)
  • Be on a stable dose of ursodeoxycholic acid for at least 6 months prior to Week 0
  • Have screening alkaline phosphatase (ALP) level \> 1.67 ULN (the upper limit of normal)
  • Have screening laboratory test results within protocol-specified limits
  • Have no history of latent or active tuberculosis (TB) prior to screening and no signs or symptoms suggestive of active TB upon medical history and/or physical examination.

You may not qualify if:

  • Has history of gastrointestinal bleeding, secondary to portal hypertension, hepatic encephalopathy, or ascites requiring treatment with diuretics
  • Has a screening direct bilirubin \> 1.0 mg/dL
  • Has a previous liver histology with a diagnosis of steatohepatitis or has a high risk of nonalcoholic steatohepatitis
  • Has a previous liver histology with a diagnosis of chronic autoimmune hepatitis or has a high risk of autoimmune hepatitis overlap syndrome
  • Testing positive for surface antigen (HBsAg+), regardless of the results of other hepatitis B tests
  • Have used colchicine, methotrexate (MTX), azathioprine (AZA), or systemic corticosteroids within 3 months prior to the first administration of study drug.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Unknown Facility

Jacksonville, Florida, United States

Location

Unknown Facility

Miami, Florida, United States

Location

Unknown Facility

Naples, Florida, United States

Location

Unknown Facility

Tupelo, Mississippi, United States

Location

Unknown Facility

New York, New York, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Vancouver, British Columbia, Canada

Location

Unknown Facility

Halifax, Nova Scotia, Canada

Location

Unknown Facility

Toronto, Ontario, Canada

Location

Unknown Facility

Montreal, Quebec, Canada

Location

Related Publications (1)

  • Mousa HS, Lleo A, Invernizzi P, Bowlus CL, Gershwin ME. Advances in pharmacotherapy for primary biliary cirrhosis. Expert Opin Pharmacother. 2015 Apr;16(5):633-43. doi: 10.1517/14656566.2015.998650. Epub 2014 Dec 29.

    PMID: 25543678DERIVED

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

Ustekinumab

Condition Hierarchy (Ancestors)

Cholestasis, IntrahepaticCholestasisBile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesLiver DiseasesLiver CirrhosisFibrosisPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Limitations and Caveats

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.

Results Point of Contact

Title
Senior Director, Clinical Development, Immunology
Organization
Janssen, R&D, LLC, Spring House, PA
ClinicalTrialDisclosure@its.jnj.com

Study Officials

  • Janssen Research & Development, LLC Clinical Trial

    Janssen Research & Development, LLC

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 7, 2011

First Posted

July 8, 2011

Study Start

September 1, 2011

Primary Completion

June 1, 2013

Study Completion

June 1, 2013

Last Updated

June 20, 2016

Results First Posted

October 15, 2014

Record last verified: 2016-06

Locations

US(6)CA(4)