A Study to Evaluate the Safety, and Tolerability, and Efficacy of Seladelpar in Patients With PSC

NCT ID: NCT04024813

Last Updated: 2025-01-16

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 1 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-11-12
• Study Completion Date: 2020-01-09

Brief Summary

The objectives of this study are to evaluate the effect of seladelpar treatment compared to placebo on efficacy, safety, and tolerability in patients with primary sclerosing cholangitis (PSC).

Conditions

Primary Sclerosing Cholangitis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo (N=25)

Placebo for the remainder of the study

Group Type: PLACEBO_COMPARATOR

Placebo to match Seladelpar

Intervention Type: DRUG

Capsule(s) administered orally once daily

Seladelpar 5 mg (N=25)

5 mg seladelpar daily for the remainder of the study

Group Type: EXPERIMENTAL

Seladelpar

Intervention Type: DRUG

Capsule(s) administered orally once daily

Seladelpar 10 mg (N=25)

10 mg seladelpar for the remainder of the study

Group Type: EXPERIMENTAL

Seladelpar

Intervention Type: DRUG

Capsule(s) administered orally once daily

Seladepar 25 mg (N=25)

25 mg seladelpar for the remainder of the study

Group Type: EXPERIMENTAL

Seladelpar

Intervention Type: DRUG

Capsule(s) administered orally once daily

Interventions

Seladelpar

Capsule(s) administered orally once daily

Intervention Type: DRUG

Placebo to match Seladelpar

Capsule(s) administered orally once daily

Intervention Type: DRUG

Other Intervention Names

MBX-8025; Livdelzi®

Eligibility Criteria

Inclusion Criteria

1. Confirmed diagnosis of primary sclerosing cholangitis (PSC) based on any two of the following three criteria:

• Historical evidence of an elevated alkaline phosphatase (AP) > upper limit of normal (ULN) from any prior laboratory result
• Liver biopsy consistent with PSC
• Abnormal cholangiography consistent with PSC as measured by magnetic resonance cholangiopancreatography (MRCP), endoscopic retrograde cholangiopancreatography (ERCP), or percutaneous transhepatic cholangiography (PTC)

2. Individuals must have the following specific additional laboratory parameters measured by the Central Laboratory at Screening:

• AP ≥ 1.5 × ULN and < 8 × ULN
• Total bilirubin ≤ 2 × ULN
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 5 × ULN
• Estimated glomerular filtration rate (eGFR) > 60 mL/min/1.73 m^2
• Platelets ≥ 140 × 10^3/µL
• International Normalized Ratio (INR) ≤ 1.3 (in the absence of warfarin or other anticoagulant therapy)
• Albumin ≥ 3.5 g/dL

3. Patients taking ursodeoxycholic acid (UDCA) will be allowed to enroll if meeting the following criteria:

• Total daily dose of ≤ 20 mg/kg/day
• A minimum of 6 months of stable treatment
• Minimum of 12 weeks off treatment prior to Screening if UDCA is recently discontinued

Exclusion Criteria

1. Clinically significant acute or chronic liver disease of an etiology other than PSC

2. Patients with a diagnosis of overlapping autoimmune hepatitis (AIH) and PSC

3. Secondary or immunoglobulin G4 (IgG4) related sclerosing cholangitis

4. Small duct PSC

5. Presence of a cholangiocarcinoma on cholangiography or MRI at Screening as determined by the central radiologist and the principal investigator (PI) or medical monitor

6. Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening

7. History, evidence, or high suspicion of cholangiocarcinoma or other hepatobiliary malignancy based on imaging, screening laboratory values, and/or clinical symptoms

8. Presumptive or diagnosed acute cholangitis within 12 weeks of Screening and through Day 1

9. Evidence of compensated or decompensated cirrhosis based on histology, relevant medical complications, or laboratory parameters:

• Historical liver biopsy demonstrating cirrhosis (eg, Ludwig Stage 4 or Ishak Stage 5)
• Current or prior history of decompensated liver disease, including ascites, hepatic encephalopathy, variceal bleeding or other clinical conditions consistent with cirrhosis and/or portal hypertension,
• Liver stiffness > 14.4 kPa by FibroScan, or
• Combined low platelet count (< 140 × 10^3/µL ) with one of the following:

• Serum albumin < 3.5 g/dL,
• INR > 1.3 (not due to antithrombotic agent use), or
• Total bilirubin > ULN

10. Prior or actively listed for liver transplantation

11. Prior exposure to seladelpar

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gilead Sciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Gilead Study Director

Role: STUDY_DIRECTOR

Gilead Sciences

Locations

Sutter Pacific Medical Foundation - California Pacific Medical Center

San Francisco, California, United States

University of Colorado Denver and Hospital

Aurora, Colorado, United States

Schiff Center for Liver Diseases/University of Miami

Miami, Florida, United States

Piedmont Atlanta Hospital

Atlanta, Georgia, United States

Henry Ford Health System

Novi, Michigan, United States

New York University

New York, New York, United States

Liver Institute of Virginia

Newport News, Virginia, United States

Bon Secours Liver Institute of Richmond

Richmond, Virginia, United States

Toronto Centre for Liver Disease-Toronto General Hospital

Toronto, Ontario, Canada

ID Clinic

Mysłowice, , Poland

Countries

United States; Canada; Poland

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2019-001760-30

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CB8025-21845

Identifier Type: -

Identifier Source: org_study_id