Phase 2 N=60 Randomized Double-blind Treatment

Study of INT-747 as Monotherapy in Participants With Primary Biliary Cirrhosis (PBC)

Liver Cirrhosis, Biliary

Bottom Line

View on ClinicalTrials.gov: NCT00570765 ↗

Enrolled (actual)

Serious AEs

11.5%

Results posted

Jul 2011

Primary outcome: Primary: DB Phase: Mean Percent Change In Serum Alkaline Phosphatase (ALP) From Baseline To Day 85 — -44.5; -37.6; 0.4 Percent change — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Placebo (Drug); Obeticholic Acid (OCA) (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Intercept Pharmaceuticals
Primary completion: Sep 2010

Outcome Measures

Outcome	Result	p-value
PRIMARY DB Phase: Mean Percent Change In Serum Alkaline Phosphatase (ALP) From Baseline To Day 85	-44.5; -37.6; 0.4	<0.0001 sig
SECONDARY DB Phase: Mean Percent Change In Gamma-glutamyl Transferase (GGT) From Baseline To Day 85	-73; -65; -3	<0.0001 sig
SECONDARY DB Phase: Mean Percent Change In Alanine Transaminase (ALT) From Baseline To Day 85	-37; -35; -4	<0.01 sig
SECONDARY DB: Plasma Trough Concentrations Of OCA And Its Major, Known Metabolites	—	—
SECONDARY DB Phase: Mean Percent Change In Conjugated Bilirubin From Baseline To Day 85	0.7; -1.7; 30.3	—
SECONDARY LTSE Phase: Median Percent Change In Serum ALP From Baseline To Month 24, Month 48, Month 72, And Last Available Visit	-43.1; -44.4; -33.4; -31.8	—
SECONDARY LTSE Phase: Mean Percent Change In Serum ALP From Baseline To Month 24, Month 48, Month 72, And Last Available Visit	-38.8; -39.3; -31.7; -30.4	—
SECONDARY LTSE: Median Percent Change In GGT From Baseline To Last Available Visit	-71.1	—
SECONDARY LTSE: Mean Percent Change In GGT From Baseline To Last Available Visit	-55.6	—
SECONDARY LTSE: Median Percent Change In ALT From Baseline To Last Available Visit	-52.2	—
SECONDARY LTSE: Mean Percent Change In ALT From Baseline To Last Available Visit	-39.6	—
SECONDARY LTSE: Median Percent Change In Conjugated Bilirubin From Baseline To Last Available Visit	33.3	—
SECONDARY LTSE: Mean Percent Change In Conjugated Bilirubin From Baseline To Last Available Visit	57.8	—
SECONDARY LTSE: Median Percent Change In Total Bilirubin From Baseline To Last Available Visit	5.2	—
SECONDARY LTSE: Mean Percent Change In Total Bilirubin From Baseline To Last Available Visit	2.2	—

Summary

The primary hypothesis was that obeticholic acid (OCA) will cause a reduction in alkaline phosphatase levels in PBC participants, over a 12-week treatment period, as compared to placebo.

Eligibility Criteria

Inclusion Criteria

Female participants must be postmenopausal, surgically sterile, or if premenopausal, be prepared to use 1 effective method of contraception with all sexual partners during the study and for 14 days after the end of dosing.
Male participants must be prepared to use 1 effective method of contraception with all sexual partners during the study during the study unless they had a prior vasectomy.
Proven or likely PBC, as demonstrated by the participant presenting with at least 2 of the following 3 diagnostic factors:
History of increased alkaline phosphatase (ALP) levels for at least 6 months;
Positive antimitochondrial antibody titer (>1: 40 titer on immunofluorescence or M2 positive by enzyme-linked immunosorbent assay) or PBC-specific antinuclear antibodies (antinuclear dot and nuclear rim positive);
Liver biopsy consistent with PBC
Screening ALP level between 1.5 and 10 × upper limit of normal (ULN).

Exclusion Criteria

Administration of the following drugs at any time during the 3 months prior to screening for the study: ursodeoxycholic acid, colchicine, methotrexate, azathioprine, or systemic corticosteroids.
Screening conjugated (direct) bilirubin >2 × ULN.
Screening alanine aminotransferase or aspartate aminotransferase >5 × ULN.
Screening serum creatinine >133 micromoles/liter (1.5 mg/deciliter).

History or presence of hepatic decompensation (for example, variceal bleeds, encephalopathy, or poorly controlled ascites).

History or presence of other concomitant liver diseases including hepatitis due to hepatitis B or C virus infection, primary sclerosing cholangitis, alcoholic liver disease, definite autoimmune liver disease or biopsy proven nonalcoholic steatohepatitis.
Pregnancy.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00570765). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.