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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03890120




Registration number
NCT03890120
Ethics application status
Date submitted
25/03/2019
Date registered
26/03/2019

Titles & IDs
Public title
Study of Cilofexor in Adults With Primary Sclerosing Cholangitis
Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety, Tolerability, and Efficacy of Cilofexor in Non-Cirrhotic Subjects With Primary Sclerosing Cholangitis
Secondary ID [1] 0 0
2019-000204-14
Secondary ID [2] 0 0
GS-US-428-4194
Universal Trial Number (UTN)
Trial acronym
PRIMIS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Sclerosing Cholangitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Infection 0 0 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Cilofexor
Treatment: Drugs - Placebo

Experimental: Cilofexor 100 mg (Blinded Phase) - Participants received cilofexor 100 mg tablet, orally, once daily for up to 100.3 weeks.

Placebo comparator: Placebo (Blinded Phase) - Participants received placebo to match cilofexor 100 mg tablet, orally, once daily for up to 98.1 weeks.

Experimental: Cilofexor From Cilofexor 100 mg (OLE Phase) - Participants who received cilofexor in blinded phase and had entered the open-label extension (OLE) phase received open-label cilofexor 100 mg tablet, orally, once daily for up to 44.7 weeks.

Experimental: Cilofexor From Placebo (OLE Phase) - Participants who received placebo in blinded phase and had entered the OLE phase received open-label cilofexor 100 mg tablet, orally, once daily for up to 45.0 weeks.


Treatment: Drugs: Cilofexor
100 mg tablet administered orally once daily

Treatment: Drugs: Placebo
Tablet administered orally once daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Progression of Liver Fibrosis at Blinded Phase Week 96
Timepoint [1] 0 0
Blinded Phase Week 96
Secondary outcome [1] 0 0
Percentage of Participants Who Experienced Treatment-emergent Adverse Events (TEAEs) in The Blinded Phase
Timepoint [1] 0 0
First dose date in the Blinded Phase up to 100.3 weeks plus 30 days
Secondary outcome [2] 0 0
Percentage of Participants Who Experienced TEAEs in The OLE Phase
Timepoint [2] 0 0
First dose date in the OLE Phase up to 45 weeks plus 30 days
Secondary outcome [3] 0 0
Percentage of Participants Who Experienced Treatment-emergent Serious Adverse Events (SAEs) in the Blinded Phase
Timepoint [3] 0 0
First dose date in the Blinded Phase up to 100.3 weeks plus 30 days
Secondary outcome [4] 0 0
Percentage of Participants Who Experienced Treatment-emergent SAEs in the OLE Phase
Timepoint [4] 0 0
First dose date in the OLE Phase up to 45 weeks plus 30 days
Secondary outcome [5] 0 0
Change From Baseline in Serum Concentrations of Alkaline Phosphatase (ALP) at Blinded Phase Week 96
Timepoint [5] 0 0
Baseline, Blinded Phase Week 96
Secondary outcome [6] 0 0
Change From Baseline in Serum Concentrations of Alanine Aminotransferase (ALT) at Blinded Phase Week 96
Timepoint [6] 0 0
Baseline, Blinded Phase Week 96
Secondary outcome [7] 0 0
Change From Baseline in Serum Concentrations of Fasting Total Bile Acids at Blinded Phase Week 96
Timepoint [7] 0 0
Baseline, Blinded Phase Week 96
Secondary outcome [8] 0 0
Percentage of Participants With = 25% Relative Reduction in Serum ALP Concentration From Baseline and No Worsening of Fibrosis According to the Ludwig Classification at Blinded Phase Week 96
Timepoint [8] 0 0
Baseline, Blinded Phase Week 96
Secondary outcome [9] 0 0
Percentage of Participants With Fibrosis Improvement According to the Ludwig Classification at Blinded Phase Week 96
Timepoint [9] 0 0
Blinded Phase Week 96
Secondary outcome [10] 0 0
Change From Baseline in Primary Sclerosing Cholangitis (PSC) Symptoms - Module 1 Based on Disease-specific Patient Reported Outcome (PSC-PRO) at Blinded Phase Week 96
Timepoint [10] 0 0
Baseline, Blinded Phase Week 96
Secondary outcome [11] 0 0
Change From Baseline in Enhanced Liver Fibrosis (ELF™ ) Test Score at Blinded Phase Week 96
Timepoint [11] 0 0
Baseline, Blinded Phase Week 96
Secondary outcome [12] 0 0
Change From Baseline in Liver Stiffness by FibroScan® at Blinded Phase Week 96
Timepoint [12] 0 0
Baseline, Blinded Phase Week 96

Eligibility
Key inclusion criteria
Key

* Diagnosis of large duct PSC
* Liver biopsy at screening that is deemed acceptable for interpretation and demonstrates stage F0 - F3 fibrosis in the opinion of the central reader
* Individual has the following laboratory parameters at the screening visit, as determined by the central laboratory:

* Platelet count = 150,000/mm^3
* Estimated glomerular filtration rate (eGFR) = 30 milliliter/minute (mL/min), as calculated by the Cockcroft-Gault equation
* Alanine transaminase (ALT) = 8 x upper limit of the normal range (ULN)
* Total bilirubin < 2 mg/dL, unless the individual is known to have Gilbert's syndrome or hemolytic anemia
* International normalized ratio (INR) = 1.4, unless due to therapeutic anticoagulation
* Negative anti-mitochondrial antibody

Key
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Current or prior history of any of the following:

* Cirrhosis
* Liver transplantation
* Cholangiocarcinoma or hepatocellular carcinoma (HCC)
* Ascending cholangitis within 30 days of screening
* Presence of a percutaneous drain or biliary stent
* Other causes of liver disease
* Current or prior history of unstable cardiovascular disease
* Current moderate to severe inflammatory bowel disease (IBD) (including ulcerative colitis, Crohn's disease, and indeterminate colitis)

Note: Other protocol defined Inclusion/Exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/03/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/12/2022
Sample size
Target
Accrual to date
Final
419
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
St. Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [3] 0 0
Nepean Hospital - Penrith
Recruitment hospital [4] 0 0
Westmead Hospital - Westmead
Recruitment hospital [5] 0 0
Coral Sea Clinical Research Institute - Mackay - Auchenflower
Recruitment hospital [6] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [7] 0 0
Mater Misericordiae Ltd - South Brisbane
Recruitment hospital [8] 0 0
Monash Health - Clayton
Recruitment hospital [9] 0 0
Austin Health - Heidelberg
Recruitment hospital [10] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [11] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment hospital [12] 0 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [13] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [3] 0 0
2751 - Penrith
Recruitment postcode(s) [4] 0 0
2145 - Westmead
Recruitment postcode(s) [5] 0 0
4066 - Auchenflower
Recruitment postcode(s) [6] 0 0
4029 - Herston
Recruitment postcode(s) [7] 0 0
4101 - South Brisbane
Recruitment postcode(s) [8] 0 0
3168 - Clayton
Recruitment postcode(s) [9] 0 0
3084 - Heidelberg
Recruitment postcode(s) [10] 0 0
3004 - Melbourne
Recruitment postcode(s) [11] 0 0
3050 - Parkville
Recruitment postcode(s) [12] 0 0
6009 - Nedlands
Recruitment postcode(s) [13] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
State/province [2] 0 0
California
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United States of America
State/province [3] 0 0
Colorado
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United States of America
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Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
District of Columbia
Country [6] 0 0
United States of America
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Florida
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United States of America
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Georgia
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Illinois
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Indiana
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Kansas
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Louisiana
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Mississippi
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Austria
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Klagenfurt am Wörthersee
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Yamagata
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Auckland
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Christchurch
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Hamilton
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Spain
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Alicante
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Spain
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Barcelona
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Madrid
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Majadahonda
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Pontevedra
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Valencia
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Zaragoza
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Bern
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Lugano
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Zurich
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Birmingham
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Cambridge
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Glasgow
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Nottingham
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Oxford
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Portsmouth

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Gilead Sciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Gilead Study Director
Address 0 0
Gilead Sciences
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03890120