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

Primary Sclerosing Cholangitis Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04024813
• Other study ID #: CB8025-21845
• Secondary ID: 2019-001760-30
• Status: Completed
• Phase: Phase 2
• Start date: November 12, 2019
• Est. completion date: January 9, 2020

Study information

• Verified date: June 2021
• Source: CymaBay Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: January 9, 2020
• Est. primary completion date: January 9, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cholangitis
• Cholangitis, Sclerosing
• Primary Sclerosing Cholangitis

Intervention

Drug:
• Seladelpar
Capsule(s) administered orally once daily
• Placebo to match Seladelpar
Capsule(s) administered orally once daily

Locations

Country	Name	City	State
Canada	Toronto Centre for Liver Disease-Toronto General Hospital	Toronto	Ontario
Poland	ID Clinic	Myslowice
United States	Piedmont Atlanta Hospital	Atlanta	Georgia
United States	University of Colorado Denver and Hospital	Aurora	Colorado
United States	Schiff Center for Liver Diseases/University of Miami	Miami	Florida
United States	New York University	New York	New York
United States	Liver Institute of Virginia	Newport News	Virginia
United States	Henry Ford Health System	Novi	Michigan
United States	Bon Secours Liver Institute of Richmond	Richmond	Virginia
United States	Sutter Pacific Medical Foundation - California Pacific Medical Center	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
CymaBay Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Relative change in Baseline serum alkaline phosphatase (AP) at Week 24		24 weeks
Secondary	Incidence of treatment-emergent adverse events (TEAEs), as well as biochemistry, hematology, and urinalysis		Up to 24 weeks
Secondary	Incidence and severity of PSC-related symptoms or procedures		Up to 24 weeks
Secondary	Incidence of Hepatic disease progression events, defined by the occurrence of liver transplantation, MELD score, hepatic decompensation events, and/or hepatocellular carcinoma		Up to 24 weeks