Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis

Primary Biliary Cholangitis Clinical Trial

— AFFIRM  

Official title:

AFFIRM: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06051617
• Other study ID #: CB8025-41837
• Status: Recruiting
• Phase: Phase 3
• Start date: September 7, 2023
• Est. completion date: July 2029

Study information

• Verified date: April 2024
• Source: CymaBay Therapeutics, Inc.
• Contact: Barry Crittenden, MD
• Phone: 510-293-8800
• Email: medinfo[@]cymabay.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients with Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis.

Description:

To Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients with Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 192
• Est. completion date: July 2029
• Est. primary completion date: July 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

Subjects must meet the following criteria to be eligible for study participation:

1. Must be 18 to 75 years old (inclusive)

2. Must have a confirmed prior diagnosis of PBC

3. Evidence of cirrhosis

4. CP Score A or B

5. Females of reproductive potential must use at least 1 barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male subjects who are sexually active with female partners of reproductive potential must use barrier contraception, and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose

6. Subjects must be able to comply with the instructions for study drug administration and be able to complete the study schedule of assessments (SOA)

Exclusion Criteria:

Subjects must not meet any of the following criteria to be eligible for study participation:

1. Prior exposure to seladelpar

2. A medical condition other than PBC that, in the Investigator's opinion, would preclude full participation in the study

3. History of liver transplantation or actively listed for cadaveric or planned living donor transplant.

4. Decompensated cirrhosis

5. Evidence of portal vein thrombosis based on imaging at time of Screening by Doppler ultrasound or prior evidence by CT or MRI

6. Hospitalization for liver-related complication within 12 weeks of Screening

7. Laboratory parameters at Screening:

1. ALP =10×ULN

2. ALT or AST =5×ULN

3. TB =5×ULN

4. Platelet count =75×10^3/µL

5. Albumin =2.8 g/dL

6. Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m^2

7. MELD score >12. For subjects on anticoagulation medication, baseline INR determination for MELD score calculation should take anticoagulant use into account, in consultation with the Medical Monitor.

8. Serum alpha-fetoprotein (AFP) >20 ng/mL

9. INR >1.7

8. CP-C cirrhosis

9. History or presence of other concomitant liver diseases

Related Conditions & MeSH terms

• Cholangitis
• Liver Cirrhosis, Biliary
• Primary Biliary Cholangitis

Intervention

Drug:
• Seladelpar 10 mg
Seladelpar 10 mg one capsule daily for up to 36 months.
• Placebo
One capsule daily for up to 36 months.

Locations

Country	Name	City	State
Korea, Republic of	Pusan National University Hospital	Busan
Korea, Republic of	Kyungpook National University Hospital	Daegu
Korea, Republic of	Seoul National University Bundang Hospital	Seongnam-si	Gyeonggi-do
Turkey	Uludag University Medical Faculty Gorkule Kampusu	Bursa
Turkey	Marmara University Pendik Training and Research Hospital	Istanbul
Turkey	Ege University, Department of Gastroenterology	Izmir
Turkey	Kocaeli University Medical Faculty Hospital	Kocaeli
Turkey	Mersin Universitesi Tip Fakultesi Hastanesi	Mersin	Yenisehir
Turkey	Rize Recep Tayyip Erdogan University Training and Research Hospital	Rize
United States	Piedmont Atlanta Hospital/Piedmont Transplant Institute	Atlanta	Georgia
United States	Mercy Medical Center	Baltimore	Maryland
United States	Beth Israel Deconess Medical Center	Boston	Massachusetts
United States	University of Chicago Medical Center	Chicago	Illinois
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	The Liver Institute at Methodist Dallas Medical Center	Dallas	Texas
United States	University of Texas Southwestern Medical Center, Professional Office Building One	Dallas	Texas
United States	Medstar Health Research Institute/MedStar Georgetown Transplant	Fairfax	Virginia
United States	Covenant Metabolic Specialists, LLC	Fort Myers	Florida
United States	University of Iowa Hospitals & Clinics	Iowa City	Iowa
United States	Florida Research Institute, LLC	Lakewood Ranch	Florida
United States	Om Research LLC	Lancaster	California
United States	SCPMG/ Kaise Permanente Los Angeles Medical Center	Los Angeles	California
United States	University of Louisville	Louisville	Kentucky
United States	Northwell Health Center for Liver Disease and Transplantation	Manhasset	New York
United States	University Healthcare System East Jefferson General Hospital Campus	Metairie	Louisiana
United States	University of Miami Hospital	Miami	Florida
United States	Ochsner Clinic Foundation	New Orleans	Louisiana
United States	Mount Sinai	New York	New York
United States	NYU Langone Health	New York	New York
United States	Mayview Hospital LLC d/b/a Bon Secours Liver Institute of Hampton Roads	Newport News	Virginia
United States	California Liver Research Center	Pasadena	California
United States	UPMC Center for Liver Diseases	Pittsburgh	Pennsylvania
United States	VCU Health Clinical Research Services Unit (CRSU)	Richmond	Virginia
United States	University of California, Davis Medical Center (Study Visits)	Sacramento	California
United States	Pinnacle Clinical Research, LLC	San Antonio	Texas
United States	California Pacific Medical Center - Sutter Pacific Medical Foundation	San Francisco	California
United States	Connie Frank Transplant center at UCSF	San Francisco	California
United States	Covenant Metabolic Specialists, LLC	Sarasota	Florida
United States	Liver Institute Northwest	Seattle	Washington
United States	Wake Forest Baptist Medical Center	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
CymaBay Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Korea, Republic of,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Event Free Survival (EFS)	EFS as measured by the time from start of treatment to the first occurrence of any of the following clinical events: Death by any cause; Liver transplantation; MELD score =15; Ascites requiring treatment; Hospitalization for esophagus or stomach varices	36 months
Secondary	Overall survival	Time from start of treatment to death from any cause.	36 months
Secondary	Liver transplant-free survival	Time from start of treatment to the first occurrence of liver-related death or liver liver transplantation.	36 months
Secondary	Time to hospitalization	Time from start of treatment for esophagus or stomach varices	36 months
Secondary	Time to Event Free Survival (EFS)	MELD score =15; Ascites requiring treatment; progression to Child-Pugh-C (advanced hepatic dysfunction)	36 months