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

Primary Purpose

Primary Biliary Cholangitis

Status
Recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Seladelpar 10 mg
Placebo
Sponsored by
CymaBay Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Biliary Cholangitis focused on measuring Primary Biliary Cholangitis (PBC), PBC

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Subjects must meet the following criteria to be eligible for study participation: Must be 18 to 75 years old (inclusive) Must have a confirmed prior diagnosis of PBC Evidence of cirrhosis CP Score A or B 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 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: Prior exposure to seladelpar A medical condition other than PBC that, in the Investigator's opinion, would preclude full participation in the study History of liver transplantation or actively listed for cadaveric or planned living donor transplant. Decompensated cirrhosis Evidence of portal vein thrombosis based on imaging at time of Screening by Doppler ultrasound or prior evidence by CT or MRI Hospitalization for liver-related complication within 12 weeks of Screening Laboratory parameters at Screening: ALP ≥10×ULN ALT or AST ≥5×ULN TB ≥5×ULN Platelet count ≤75×10^3/µL Albumin ≤2.8 g/dL Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m^2 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. Serum alpha-fetoprotein (AFP) >20 ng/mL INR >1.7 CP-C cirrhosis History or presence of other concomitant liver diseases

Sites / Locations

  • Om Research LLCRecruiting
  • California Liver Research CenterRecruiting
  • Florida Research Institute, LLCRecruiting
  • Mercy Medical CenterRecruiting
  • University Hospitals Cleveland Medical CenterRecruiting
  • The Liver Institute at Methodist Dallas Medical CenterRecruiting
  • VCU Health Clinical Research Services Unit (CRSU)Recruiting
  • Liver Institute NorthwestRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Seladelpar 10 mg

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Overall survival
Time from start of treatment to death from any cause.
Liver transplant-free survival
Time from start of treatment to the first occurrence of liver-related death or liver liver transplantation.
Time to hospitalization
Time from start of treatment for esophagus or stomach varices
Time to Event Free Survival (EFS)
MELD score ≥15 Ascites requiring treatment progression to Child-Pugh-C (advanced hepatic dysfunction)

Full Information

First Posted
September 18, 2023
Last Updated
October 19, 2023
Sponsor
CymaBay Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT06051617
Brief Title
Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 7, 2023 (Actual)
Primary Completion Date
July 2029 (Anticipated)
Study Completion Date
July 2029 (Anticipated)

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Biliary Cholangitis
Keywords
Primary Biliary Cholangitis (PBC), PBC

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
192 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Seladelpar 10 mg
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Seladelpar 10 mg
Intervention Description
Seladelpar 10 mg one capsule daily for up to 36 months.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
One capsule daily for up to 36 months.
Primary Outcome Measure Information:
Title
Event Free Survival (EFS)
Description
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
Time Frame
36 months
Secondary Outcome Measure Information:
Title
Overall survival
Description
Time from start of treatment to death from any cause.
Time Frame
36 months
Title
Liver transplant-free survival
Description
Time from start of treatment to the first occurrence of liver-related death or liver liver transplantation.
Time Frame
36 months
Title
Time to hospitalization
Description
Time from start of treatment for esophagus or stomach varices
Time Frame
36 months
Title
Time to Event Free Survival (EFS)
Description
MELD score ≥15 Ascites requiring treatment progression to Child-Pugh-C (advanced hepatic dysfunction)
Time Frame
36 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subjects must meet the following criteria to be eligible for study participation: Must be 18 to 75 years old (inclusive) Must have a confirmed prior diagnosis of PBC Evidence of cirrhosis CP Score A or B 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 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: Prior exposure to seladelpar A medical condition other than PBC that, in the Investigator's opinion, would preclude full participation in the study History of liver transplantation or actively listed for cadaveric or planned living donor transplant. Decompensated cirrhosis Evidence of portal vein thrombosis based on imaging at time of Screening by Doppler ultrasound or prior evidence by CT or MRI Hospitalization for liver-related complication within 12 weeks of Screening Laboratory parameters at Screening: ALP ≥10×ULN ALT or AST ≥5×ULN TB ≥5×ULN Platelet count ≤75×10^3/µL Albumin ≤2.8 g/dL Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m^2 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. Serum alpha-fetoprotein (AFP) >20 ng/mL INR >1.7 CP-C cirrhosis History or presence of other concomitant liver diseases
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Barry Crittenden, MD
Phone
510-293-8800
Email
medinfo@cymabay.com
Facility Information:
Facility Name
Om Research LLC
City
Lancaster
State/Province
California
ZIP/Postal Code
93534
Country
United States
Individual Site Status
Recruiting
Facility Name
California Liver Research Center
City
Pasadena
State/Province
California
ZIP/Postal Code
91105
Country
United States
Individual Site Status
Recruiting
Facility Name
Florida Research Institute, LLC
City
Lakewood Ranch
State/Province
Florida
ZIP/Postal Code
34211
Country
United States
Individual Site Status
Recruiting
Facility Name
Mercy Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21202
Country
United States
Individual Site Status
Recruiting
Facility Name
University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Individual Site Status
Recruiting
Facility Name
The Liver Institute at Methodist Dallas Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75203
Country
United States
Individual Site Status
Recruiting
Facility Name
VCU Health Clinical Research Services Unit (CRSU)
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Individual Site Status
Recruiting
Facility Name
Liver Institute Northwest
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

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

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