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A study evaluating the safety and efficacy of MBX-8025 in subjects with Primary Biliary Cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA)

Phase 1
Conditions
Primary Biliary Cholangitis
MedDRA version: 20.1 Level: LLT Classification code 10036680 Term: Primary biliary cirrhosis System Organ Class: 100000004871
Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Registration Number
EUCTR2016-002996-91-DE
Lead Sponsor
CymaBay Therapeutics, Inc.
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
Not specified
Target Recruitment
128
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

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

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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