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

NCT04024813 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: CB8025-218452019-001760-30
• Study Type: interventional
• Target: 1
• Locations: 3 countries
• Sites: 10

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

Keywords

PSC; Cholangitis, Sclerosing; Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Outcome Measures

Primary Outcomes (1)

• Relative Change in Baseline Serum Alkaline Phosphatase (AP) at Week 24

  Baseline, Week 24

Secondary Outcomes (3)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  Up to Day 59

• Incidence of Primary Sclerosing Cholangitis (PSC)-Related Symptoms or Procedures

  Up to 24 weeks

• Incidence of Hepatic Disease Progression Events

  Up to 24 weeks

Study Arms (4)

Placebo (N=25)

PLACEBO COMPARATOR

Placebo for the remainder of the study

Drug: Placebo to match Seladelpar

Seladelpar 5 mg (N=25)

EXPERIMENTAL

5 mg seladelpar daily for the remainder of the study

Drug: Seladelpar

Seladelpar 10 mg (N=25)

EXPERIMENTAL

10 mg seladelpar for the remainder of the study

Drug: Seladelpar

Seladepar 25 mg (N=25)

EXPERIMENTAL

25 mg seladelpar for the remainder of the study

Drug: Seladelpar

Interventions

Seladelpar DRUG

Capsule(s) administered orally once daily

Also known as: MBX-8025, Livdelzi®

Seladelpar 10 mg (N=25); Seladelpar 5 mg (N=25); Seladepar 25 mg (N=25)

Placebo to match Seladelpar DRUG

Capsule(s) administered orally once daily

Placebo (N=25)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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
• 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

You may not qualify if:

• Clinically significant acute or chronic liver disease of an etiology other than PSC
• Patients with a diagnosis of overlapping autoimmune hepatitis (AIH) and PSC
• Secondary or immunoglobulin G4 (IgG4) related sclerosing cholangitis
• Small duct PSC
• Presence of a cholangiocarcinoma on cholangiography or MRI at Screening as determined by the central radiologist and the principal investigator (PI) or medical monitor
• Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
• History, evidence, or high suspicion of cholangiocarcinoma or other hepatobiliary malignancy based on imaging, screening laboratory values, and/or clinical symptoms
• Presumptive or diagnosed acute cholangitis within 12 weeks of Screening and through Day 1
• 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

Sponsors & Collaborators

• Gilead Sciences: lead

Study Sites (10)

Sutter Pacific Medical Foundation - California Pacific Medical Center

San Francisco, California, 94109, United States

Location

University of Colorado Denver and Hospital

Aurora, Colorado, 80045, United States

Location

Schiff Center for Liver Diseases/University of Miami

Miami, Florida, 33136, United States

Location

Piedmont Atlanta Hospital

Atlanta, Georgia, 30309, United States

Location

Henry Ford Health System

Novi, Michigan, 48377, United States

Location

New York University

New York, New York, 10016, United States

Location

Liver Institute of Virginia

Newport News, Virginia, 23602, United States

Location

Bon Secours Liver Institute of Richmond

Richmond, Virginia, 23226, United States

Location

Toronto Centre for Liver Disease-Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

ID Clinic

Mysłowice, 41-400, Poland

Location

MeSH Terms

Conditions

Cholangitis, Sclerosing; Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Interventions

seladelpar; (2-methyl-4-(5-methyl-2-(4-trifluoromethyl-phenyl)-2H-(1,2,3)triazol-4-ylmethylsulfanyl)phenoxy)acetic acid

Limitations and Caveats

The study was terminated early and only 1 participant enrolled in the placebo arm and received placebo up to Day 14. The pre-specified analyses were not conducted due to study termination.

Results Point of Contact

• Title: Gilead Clinical Study Information Center
• Organization: Gilead Sciences

GileadClinicalTrials@gilead.com

1-833-445-3230 (GILEAD-0)

Study Officials

• Gilead Study Director

  Gilead Sciences

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Dose masking
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 20, 2019
• First Posted: July 18, 2019
• Study Start: November 12, 2019
• Primary Completion: January 9, 2020
• Study Completion: January 9, 2020
• Last Updated: January 16, 2025
• Results First Posted: January 16, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1); US (8); CA (1)