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Phase 2a Evaluation of Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Patients With Primary Sclerosing Cholangitis (PSC)

Phase 2
Completed
Conditions
Primary Sclerosing Cholangitis
Interventions
Drug: Placebo
Registration Number
NCT04480840
Lead Sponsor
Pliant Therapeutics, Inc.
Brief Summary

A Phase 2a, multicenter, randomized, double-blind, dose-ranging, placebo-controlled, study to evaluate the safety, tolerability, and PK of PLN-74809 in participants with primary sclerosing cholangitis and suspected liver fibrosis

Detailed Description

Three-part study:

Part 1 - 12-week treatment period evaluating 40 mg of PLN-74809 or matching placebo \[Complete\] Part 2 - 12-week treatment period evaluating two dose groups, 80 mg and 160 mg of PLN-74809 or matching placebo Part 3 - minimum 24-week, up to 48-week treatment period evaluating 320 mg of PLN-74809 or matching placebo

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
121
Inclusion Criteria
  • Established clinical diagnosis of large duct PSC based on an abnormal cholangiography as assessed by magnetic resonance cholangiopancreatography (MRCP), endoscopic retrograde cholangiopancreatography (ERCP), and/or percutaneous transhepatic cholangiopancreatography (PTC) in the context of cholestatic liver chemistry
  • Suspected liver fibrosis, as defined by liver stiffness measurement (LSM), assessed by ultrasound-based transient elastography (TE, FibroScan®) OR Enhanced Liver Fibrosis (ELF) Score OR Historical liver biopsy showing fibrosis without cirrhosis (by any scoring system) OR Magnetic resonance elastography (MRE)
  • Serum ALP concentration within normal limits or > 1 times the upper limit of normal (ULN)
  • Participants receiving treatment for IBD are allowed, if on a stable dose from screening and expected to remain stable for the duration of the study
  • Serum AST and ALT concentration ≤ 5 times the upper limit of normal
  • If receiving treatment with UDCA, therapy is at a dose of < 25 mg/kg/day, has been stable for at least 3 months before screening.
Exclusion Criteria
  • Other causes of liver disease, including secondary sclerosing cholangitis or viral, metabolic, or alcoholic liver disease, as assessed clinically
  • Known or suspected overlapping clinical and histologic diagnosis of autoimmune hepatitis
  • Small duct PSC with no evidence of large duct involvement (evidence of PSC on historical liver histology, with normal bile ducts on cholangiography)
  • Presence of liver cirrhosis as assessed by liver histology, ultrasound-based liver stiffness measurement, ELF score, MRE, and/or signs and symptoms of hepatic decompensation (including but not limited to, jaundice, ascites, variceal hemorrhage, and/or hepatic encephalopathy.
  • Serum ALP concentration > 10 times the upper limit of normal.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebo-
PLN-74809 Dose Level 4PLN-74809Dose: 320 mg; PLN-74809 Dose Level 4 following PLN-74809 Dose Level 3
PLN-74809 Dose Level 1PLN-74809Dose: 40 mg;
PLN-74809 Dose Level 3PLN-74809Dose: 160 mg; PLN-74809 Dose Level 3 following PLN-74809 Dose Level 2
PLN-74809 Dose Level 2PLN-74809Dose: 80 mg; PLN-74809 Dose Level 2 following PLN-74809 Dose Level 1
Primary Outcome Measures
NameTimeMethod
Number of participants with treatment-related adverse events and laboratory abnormalities, as assessed by CTCAE v5.012-48 weeks

Nature and proportion of AEs between PLN-74809 and placebo groups

Secondary Outcome Measures
NameTimeMethod
Assessment of PLN-74809 plasma concentrations12-48 weeks

Plasma PLN-74809 concentrations (total and unbound concentrations) at each sampling timepoint

Trial Locations

Locations (60)

California Liver Research Institute

🇺🇸

Pasadena, California, United States

Stanford University School of Medicine

🇺🇸

Redwood City, California, United States

University of California, Davis Medical Center

🇺🇸

Sacramento, California, United States

California Pacific Medical Center Research Institute

🇺🇸

San Francisco, California, United States

University of California San Francisco

🇺🇸

San Francisco, California, United States

Yale School of Medicine

🇺🇸

New Haven, Connecticut, United States

Florida Research Institute

🇺🇸

Lakewood Ranch, Florida, United States

Schiff Center of Liver Diseases/University of Miami

🇺🇸

Miami, Florida, United States

Piedmont Atlanta Hospital

🇺🇸

Atlanta, Georgia, United States

University of Chicago Medical Center

🇺🇸

Chicago, Illinois, United States

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California Liver Research Institute
🇺🇸Pasadena, California, United States

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