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

Primary Purpose

Primary Sclerosing Cholangitis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Seladelpar
Placebo to match Seladelpar
Sponsored by
CymaBay Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Sclerosing Cholangitis focused on measuring PSC, Cholangitis, Sclerosing, Cholangitis, Bile Duct Diseases, Biliary Tract Diseases, Digestive System Diseases

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  1. Confirmed diagnosis of PSC based on any two of the following three criteria:

    • Historical evidence of an elevated AP > ULN from any prior laboratory result
    • Liver biopsy consistent with PSC
    • Abnormal cholangiography consistent with PSC as measured by MRCP, ERCP, or percutaneous transhepatic cholangiography (PTC)
  2. Subjects 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
    • ALT and AST ≤ 5 × ULN
    • 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 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

Key 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 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

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • Sutter Pacific Medical Foundation - California Pacific Medical Center
  • University of Colorado Denver and Hospital
  • Schiff Center for Liver Diseases/University of Miami
  • Piedmont Atlanta Hospital
  • Henry Ford Health System
  • New York University
  • Liver Institute of Virginia
  • Bon Secours Liver Institute of Richmond
  • Toronto Centre for Liver Disease-Toronto General Hospital
  • ID Clinic

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Placebo Comparator

Experimental

Experimental

Experimental

Arm Label

Placebo (N=25)

Seladelpar 5 mg (N=25)

Seladelpar 10 mg (N=25)

Seladepar 25 mg (N=25)

Arm Description

Placebo for the remainder of the study

5 mg seladelpar daily for the remainder of the study

10 mg seladelpar for the remainder of the study

25 mg seladelpar for the remainder of the study

Outcomes

Primary Outcome Measures

Relative change in Baseline serum alkaline phosphatase (AP) at Week 24

Secondary Outcome Measures

Incidence of treatment-emergent adverse events (TEAEs), as well as biochemistry, hematology, and urinalysis
Incidence and severity of PSC-related symptoms or procedures
Incidence of Hepatic disease progression events, defined by the occurrence of liver transplantation, MELD score, hepatic decompensation events, and/or hepatocellular carcinoma

Full Information

First Posted
June 20, 2019
Last Updated
June 1, 2021
Sponsor
CymaBay Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04024813
Brief Title
A Study to Evaluate the Safety, and Tolerability, and Efficacy of Seladelpar in Patients With PSC
Official Title
A Phase 2, Randomized, Double Blind, Placebo Controlled, Multiple Center Study to Evaluate the Safety, Tolerability, and Efficacy of Seladelpar Administered for 24 Weeks in Adult Patients With Primary Sclerosing Cholangitis (PSC)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2021
Overall Recruitment Status
Completed
Study Start Date
November 12, 2019 (Actual)
Primary Completion Date
January 9, 2020 (Actual)
Study Completion Date
January 9, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CymaBay Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Sclerosing Cholangitis
Keywords
PSC, Cholangitis, Sclerosing, Cholangitis, Bile Duct Diseases, Biliary Tract Diseases, Digestive System Diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Dose masking
Allocation
Randomized
Enrollment
1 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo (N=25)
Arm Type
Placebo Comparator
Arm Description
Placebo for the remainder of the study
Arm Title
Seladelpar 5 mg (N=25)
Arm Type
Experimental
Arm Description
5 mg seladelpar daily for the remainder of the study
Arm Title
Seladelpar 10 mg (N=25)
Arm Type
Experimental
Arm Description
10 mg seladelpar for the remainder of the study
Arm Title
Seladepar 25 mg (N=25)
Arm Type
Experimental
Arm Description
25 mg seladelpar for the remainder of the study
Intervention Type
Drug
Intervention Name(s)
Seladelpar
Other Intervention Name(s)
MBX-8025
Intervention Description
Capsule(s) administered orally once daily
Intervention Type
Drug
Intervention Name(s)
Placebo to match Seladelpar
Intervention Description
Capsule(s) administered orally once daily
Primary Outcome Measure Information:
Title
Relative change in Baseline serum alkaline phosphatase (AP) at Week 24
Time Frame
24 weeks
Secondary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (TEAEs), as well as biochemistry, hematology, and urinalysis
Time Frame
Up to 24 weeks
Title
Incidence and severity of PSC-related symptoms or procedures
Time Frame
Up to 24 weeks
Title
Incidence of Hepatic disease progression events, defined by the occurrence of liver transplantation, MELD score, hepatic decompensation events, and/or hepatocellular carcinoma
Time Frame
Up to 24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Confirmed diagnosis of PSC based on any two of the following three criteria: Historical evidence of an elevated AP > ULN from any prior laboratory result Liver biopsy consistent with PSC Abnormal cholangiography consistent with PSC as measured by MRCP, ERCP, or percutaneous transhepatic cholangiography (PTC) Subjects 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 ALT and AST ≤ 5 × ULN 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 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 Key Exclusion Criteria: 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 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 Total bilirubin > ULN Prior or actively listed for liver transplantation Prior exposure to seladelpar Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Facility Information:
Facility Name
Sutter Pacific Medical Foundation - California Pacific Medical Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94109
Country
United States
Facility Name
University of Colorado Denver and Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Schiff Center for Liver Diseases/University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Piedmont Atlanta Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30309
Country
United States
Facility Name
Henry Ford Health System
City
Novi
State/Province
Michigan
ZIP/Postal Code
48377
Country
United States
Facility Name
New York University
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Liver Institute of Virginia
City
Newport News
State/Province
Virginia
ZIP/Postal Code
23602
Country
United States
Facility Name
Bon Secours Liver Institute of Richmond
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23226
Country
United States
Facility Name
Toronto Centre for Liver Disease-Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Facility Name
ID Clinic
City
Mysłowice
ZIP/Postal Code
41-400
Country
Poland

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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