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

• Register: ClinicalTrials.gov
• Last refreshed on: 14 June 2021
• Main ID: NCT04024813
• Date of registration: 20/06/2019
• Prospective Registration: Yes
• Primary sponsor: CymaBay Therapeutics, Inc.
• Public title: A Study to Evaluate the Safety, and Tolerability, and Efficacy of Seladelpar in Patients With PSC
• Scientific 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)
• Date of first enrolment: November 12, 2019
• Target sample size: 1
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04024813
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada, Poland, United States

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Sclerosing Cholangitis

Intervention(s)

Drug: Placebo to match Seladelpar

Drug: Seladelpar

Primary Outcome(s)

Relative change in Baseline serum alkaline phosphatase (AP) at Week 24 [Time Frame: 24 weeks]

Secondary Outcome(s)

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]

Incidence and severity of PSC-related symptoms or procedures [Time Frame: Up to 24 weeks]

Incidence of treatment-emergent adverse events (TEAEs), as well as biochemistry, hematology, and urinalysis [Time Frame: Up to 24 weeks]

Secondary ID(s)

2019-001760-30

CB8025-21845

Source(s) of Monetary Support

Please refer to primary and secondary sponsors