Study of INT 747 in Combination With URSO in Patients With Primay Biliary Cirrhosis (PBC)

Intercept Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Liver Cirrhosis, Biliary

Treatments: Drug: INT-747; Drug: Ursodeoxycholic Acid (URSO); Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00550862

747-202

Details and patient eligibility

About

The primary hypothesis is that INT-747 will cause a reduction in alkaline phosphatase levels in Primary Biliary Cirrhosis patients, over a 12 week treatment period, as compared to placebo.

Full description

None provided

Enrollment: 165 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female age 18 to 70 years.

• Stable dose of ursodeoxycholic acid (URSO, UDCA) for at least 6 months prior to screening.

• Female patients must be postmenopausal, surgically sterile, or prepared to use 2 methods of contraception with all sexual partners during the study and for 14 days after the end of dosing.

• Male patients must be prepared to use 2 methods of contraception with all sexual partners during the study and for 14 days after the end of the dosing.

• Proven or likely PBC, as demonstrated by the patient presenting with at least 2 of the following 3 diagnostic factors:

  1. History of increased AP levels for at least 6 months prior to Day 0
  2. Positive AMA titer (>1:40 titer on immunofluorescence or M2 positive by ELISA) or PBC-specific antinuclear antibodies (antinuclear dot and nuclear rim positive)
  3. Liver biopsy consistent with PBC.

• Screening AP value between 1.5 and 10 × ULN.

Exclusion criteria

• Administration of the following drugs at any time during the 3 months prior to screening for the study: colchicine, methotrexate, azathioprine, or systemic corticosteroids.
• Screening conjugated (direct) bilirubin >2 × ULN.
• Screening ALT or AST >5 × ULN.
• Screening serum creatinine >1.5 mg/dL (133 mol/L).
• History or presence of hepatic decompensation (e.g., variceal bleeds, encephalopathy, or poorly controlled ascites).
• History or presence of other concomitant liver diseases including hepatitis due to hepatitis B or C virus (HCV, HBV) infection, primary sclerosing cholangitis (PSC), alcoholic liver disease, definite autoimmune liver disease or biopsy proven nonalcoholic steatohepatitis (NASH).
• Pregnancy.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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165 participants in 4 patient groups, including a placebo group

INT-747 10 mg

Experimental group

Description:

INT-747 10 mg once daily in combination with URSO for 12 weeks.

Treatment:

Drug: Ursodeoxycholic Acid (URSO)

Drug: INT-747

INT-747 25 mg

Experimental group

Description:

INT-747 25 mg once daily in combination with URSO for 12 weeks.

Treatment:

Drug: Ursodeoxycholic Acid (URSO)

Drug: INT-747

INT-747 50 mg

Experimental group

Description:

INT-747 50 mg once daily in combination with URSO for 12 weeks.

Treatment:

Drug: Ursodeoxycholic Acid (URSO)

Drug: INT-747

Placebo

Placebo Comparator group

Description:

Placebo once daily in combination with URSO for 12 weeks.

Treatment:

Drug: Placebo

Drug: Ursodeoxycholic Acid (URSO)

Drug: INT-747