Primary Biliary Cirrhosis MedDRA version: 18.1 Level: LLT Classification code 10036680 Term: Primary biliary cirrhosis System Organ Class: 100000004871
Conditions
Interventions
Product Name: MBX-8025
Product Code: MBX-8025
Pharmaceutical Form: Capsule, hard
CAS Number: 928821-40-3
Current Sponsor code: MBX-8025
Other descriptive name: MBX-8025
Concentration unit: mg millig
Sponsors
CymaBay Therapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion 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 AMA (Anti-Mitochondrial Antibodies) titers (>1/40 on immunofluorescence or M2 positive by 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 six 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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 25
Exclusion criteria
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 = 2 times ULN 3. Total bilirubin >2 times ULN 4. Auto-immune hepatitis 5. Primary sclerosing cholangitis 6. Known histroy of alpha-1-Antitrypsin deficiency 7. Known history of chronic viral hepatitis 8. Creatine kinase above ULN 9. For females, pregnancy or breast-feeding 10. Use of colchicine, methotrexate, azathioprine or systemic steroids in the two months preceding screening 11. Use of an experimental treatment for PBC 12. Use of an experimental or unapproved immunosuppressant 13. 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the effect of MBX-8025 on AP levels.;Secondary Objective: The secondary objectives of this study are: 1. To evaluate the safety and tolerability of MBX-8025 in subjects with PBC 2. To evaluate the effects of MBX-8025 on PBC response criteria 3. To evaluate the effects of MBX-8025 on other markers of liver function, lipids, pruritus and Quality of Life (QoL) ;Primary end point(s): To evaluate the effect of MBX-8025 on Alkaline Phosphatase (AP) Levels ;Timepoint(s) of evaluation of this end point: End of Treatment - Week 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Composite endpoint of AP and Total Bilirubin: • AP 15% decrease in AP 2. Aspartate aminotransferase (AST), Alanine aminotransferase (ALT), Gamma-Glutamyl Transpeptidase (GGT), 5’nucleotidase, Bilirubin (Total, Conjugated, Unconjugated), and bone-specific AP Triglycerides (TG), Total Cholesterol (TC), High Density Lipoprotein Cholesterol (HDL-C), and Low Density Lipoprotein Cholesterol (LDL-C). 3. Published PBC response criteria (Paris I and II, Toronto I and II), UK-PBC risk score 4. 5D-itch scale, pruritus VAS 5. PBC-40 QoL ;Timepoint(s) of evaluation of this end point: Visit 2 (Week 0), Visit 3 (Week 2), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 12). | — |
Countries
Canada, Germany, Poland, United Kingdom, United States
Contacts
Public ContactKirk Rosemark
CymaBay Therapeutics, Inc.
Outcome results
None listed