Primary Biliary Cholangitis MedDRA version: 20.1 Level: LLT Classification code 10036680 Term: Primary biliary cirrhosis System Organ Class: 100000004871
Conditions
Interventions
Product Name: Seladelpar
Product Code: MBX-8025
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Seladelpar
CAS Number: 928821-40
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 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 or intolerant to UDCA 5.AP = 1.67 × ULN 6.Females of reproductive potential must use at least one 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 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 96 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 32
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 > 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 or simvastatin 13.Current use of obeticholic acid 14.Use of an experimental or unapproved treatment for PBC 15.Use of experimental or unapproved immunosuppressant 16.Adverse event leading to MBX-8025 discontinuation from CymaBay’s phase 2 PBC study CB8025-21528 17.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: To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, 10 mg, and 20 mg over 8 weeks of treatment ; Primary end point(s): 1.Serum alkaline phosphatase (AP) 2.Adverse events (AE) and Treatment Emergent Adverse Events (TEAEs), ECG, biochemistry ,hematology, and urinalysis(NCI CTCAE Version 4.0) ;Timepoint(s) of evaluation of this end point: End of Treatment - Week 8; Secondary Objective: To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, 10 mg, and 20 mg over 12 and 26 weeks of treatment To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, 10 mg, and 20 mg over 52 weeks of treatment To evaluate the pharmacokinetics (PK) of MBX-8025 | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Baseline to the end of extension (52-week treatment); Secondary end point(s): 1. Aspartate aminotransferase (AST), Alanine aminotransferase (ALT), Gamma-glutamyl transferase (GGT), 5’nucleotidase, Bilirubin (Total, Conjugated, Unconjugated), Bone-specific AP, Triglycerides (TG), Total Cholesterol (TC), High Density Lipoprotein Cholesterol (HDL-C), and Low Density Lipoprotein Cholesterol (LDL-C) 2.Composite endpoint of AP and Total Bilirubin: •AP 15% decrease in AP 3.Published PBC response criteria (Paris I and II, Toronto I and II), UK-PBC risk score 4.5D-itch scale and pruritus Visual Analog Score (VAS) 5.PBC-40 QoL 6.PK of MBX-8025 and metabolites (at 0, 0.5, 1, 2, 4, 6 and 24 hours for Cmax, Tmax, T1/2 and AUC; trough level) | — |
Countries
Canada, Germany, United Kingdom, United States
Contacts
Public ContactKlara Dickinson
CymaBay Therapeutics, Inc.
Outcome results
None listed