Obeticholic Acid (OCA) in Primary Sclerosing Cholangitis (PSC) (AESOP)
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|ClinicalTrials.gov Identifier: NCT02177136|
Recruitment Status : Completed
First Posted : June 27, 2014
Results First Posted : May 30, 2018
Last Update Posted : May 30, 2018
|Condition or disease||Intervention/treatment||Phase|
|Primary Sclerosing Cholangitis (PSC)||Drug: OCA Drug: Placebo||Phase 2|
This is a Phase 2, randomized, double-blind, placebo-controlled, dose-finding evaluation of the efficacy and safety of OCA in subjects with PSC. Approximately 75 subjects who provide written informed consent and meet all of the inclusion and none of the exclusion criteria will be randomized to 1 of 3 treatment groups as follows: 1.5 mg titrating to 3 mg OCA, 5 mg titrating to 10 mg OCA, or placebo, in a 1:1:1 ratio. Subjects will administer investigational product (IP) orally, once daily for 2 consecutive 12-week periods.
For the first 12 weeks, the subject's dose will be 1.5 mg OCA, 5 mg OCA, or placebo. After 12 weeks, the subject's dose will be titrated as follows, providing there are no limiting safety or tolerability concerns in the opinion of the investigator, while maintaining the trial blind: the 1.5 mg OCA treatment group will titrate to 3 mg, the 5 mg OCA treatment group will titrate to 10 mg OCA, and the placebo group will remain on placebo. Double-blind treatment will continue for a further 12 weeks at that dose.
Any subjects whose dose is not titrated, due to safety or tolerability concerns, will remain on their starting treatment (1.5 mg OCA, 5 mg OCA, or placebo) for the remainder of the double-blind phase to Week 24.
Randomization will be stratified by the presence or absence of concomitant ursodeoxycholic acid (UDCA) use and total bilirubin level (≤ 1.5x upper limit of normal [ULN] or > 1.5x ULN but < 2.5x ULN).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||77 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding, Clinical Trial Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects With Primary Sclerosing Cholangitis|
|Actual Study Start Date :||February 9, 2015|
|Actual Primary Completion Date :||March 7, 2017|
|Actual Study Completion Date :||March 22, 2018|
Experimental: 1.5 mg OCA titrating to 3 mg OCA
Subjects randomized to 1.5 mg OCA will take 1.5 mg OCA daily for 12 weeks. If tolerated, the dose will be increased to 3 mg OCA daily for an additional 12 weeks.
Experimental: 5 mg OCA titrating to 10 mg OCA
Subjects randomized to 5 mg OCA will take 5 mg OCA daily for 12 weeks. If tolerated, the dose will be increased to 10 mg OCA daily for an additional 12 weeks.
Subjects randomized to placebo will take placebo for 24 weeks.
- Change From Baseline in Serum Alkaline Phosphatase (ALP) [ Time Frame: Baseline and 24 weeks ]The primary efficacy analysis will compare the Week 24 change from Baseline in ALP between OCA treatment group and placebo using an analysis of covariance (ANCOVA) model with fixed effects for treatment group and randomization strata, and Baseline as a covariate.
- Change From Baseline in Serum Alanine Transaminase (ALT) [ Time Frame: Baseline and 24 weeks ]
- Change From Baseline in Serum Aspartate Aminotransferase (AST) [ Time Frame: Baseline and 24 weeks ]
- Change From Baseline in Serum Total Bilirubin [ Time Frame: Baseline and 24 weeks ]
- Change From Baseline in Serum Direct Bilirubin [ Time Frame: Baseline and 24 weeks ]
- Change From Baseline in Serum Gamma-Glutamyl Transferase (GGT) [ Time Frame: Baseline and 24 weeks ]
- Change From Baseline in Plasma Fibroblast Growth Factor-19 (FGF-19) [ Time Frame: Baseline and 24 weeks ]
- Change From Baseline in Plasma C4 [ Time Frame: Baseline and 24 weeks ]
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): NCT02177136
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|Study Director:||David Shapiro, MD||Intercept Pharmaceuticals|