Study to Evaluate the Effects of Two Doses of MBX-8025 in Subjects With Primary Biliary Cirrhosis (PBC)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02609048
• Date of registration: 13/11/2015
• Prospective Registration: No
• Primary sponsor: CymaBay Therapeutics, Inc.
• Public title: Study to Evaluate the Effects of Two Doses of MBX-8025 in Subjects With Primary Biliary Cirrhosis (PBC)
• Scientific title: A 12-week, Double-blind, Randomized, Placebo-controlled, Phase 2 Study, to Evaluate the Effects of Two Doses of MBX-8025 in Subjects With Primary Biliary Cirrhosis (PBC) and an Inadequate Response to Ursodeoxycholic Acid (UDCA)
• Date of first enrolment: November 2015
• Target sample size: 41
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02609048
• 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, Germany, Poland, United Kingdom, United States

Contacts

• Name: Pol F Boudes, M.D.
• Affiliation: CymaBay Therapeutics, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Must have given written informed consent (signed and dated) and any authorizations
required by local law

2. 18 to 75 years old (inclusive)

3. Male or female with a diagnosis of PBC, by at least two of the following criteria:

- History of AP above ULN for at least six months

- Positive Anti-Mitochondrial Antibodies (AMA) titers (>1/40 on immunofluorescence
or M2 positive by enzyme linked immunosorbent assay (ELISA) or positive
PBC-specific antinuclear antibodies

- Documented liver biopsy result consistent with PBC

4. On a stable and recommended dose of UDCA for the past twelve months

5. AP = 1.67 × ULN

6. For females of reproductive potential, use of at least one barrier contraceptive and a
second effective birth control method during the study and for at least two weeks
after the last dose. For male subjects, use of appropriate contraception (e.g.,
condoms), so their female partners of reproductive potential do not become pregnant
during the study and for at least two weeks after the last dose

Exclusion Criteria:

1. A medical condition, other than PBC, that in the investigator's opinion would preclude
full participation in the study or confound its results (e.g., cancer on active
treatment)

2. AST or ALT > 3 × ULN

3. Total bilirubin > 2 × ULN

4. Auto-immune hepatitis

5. Primary sclerosing cholangitis

6. Known history of alpha-1-Antitrypsin deficiency

7. Known history of chronic viral hepatitis

8. Creatine kinase above ULN

9. Serum creatinine above ULN

10. For females, pregnancy or breast-feeding

11. Use of colchicine, methotrexate, azathioprine, or systemic steroids in the two months
preceding screening

12. Current use of fibrates, including fenofibrates, or simvastatin

13. Use of an experimental treatment for PBC

14. Use of experimental or unapproved immunosuppressant

15. Any other condition(s) that would compromise the safety of the subject or compromise
the quality of the clinical study, as judged by the Investigator



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Biliary Cirrhosis (PBC)

Intervention(s)

Drug: Placebo Comparator

Drug: Experimental: Seladelpar / MBX-8025 50 mg

Drug: Experimental: Seladelpar / MBX-8025 200 mg

Primary Outcome(s)

Serum Alkaline Phosphatase (AP) [Time Frame: 12-Weeks]

Secondary Outcome(s)

Composite Endpoint of AP and Bilirubin [Time Frame: 12-Weeks]

Laboratory Values [Time Frame: 12-Weeks]

5D-itch scale [Time Frame: 12-Weeks]

PBC-40 Quality of Life (QoL) [Time Frame: 12-Weeks]

Visual Analog Score (VAS) [Time Frame: 12-Weeks]

Published PBC response criteria [Time Frame: 12-Weeks]

Secondary ID(s)

CB8025-21528

Source(s) of Monetary Support

Please refer to primary and secondary sponsors