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

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ClinicalTrials.gov Identifier: NCT02609048
Recruitment Status : Terminated (Study stopped early after review of safety and efficacy demonstrated efficacy proof-of-concept and need for dose reduction.)
First Posted : November 20, 2015
Last Update Posted : January 29, 2018
Sponsor:
Information provided by (Responsible Party):
CymaBay Therapeutics, Inc.

Brief Summary:
A 12-week, double-blind, randomized, placebo-controlled, Phase 2 study, to evaluate the effects of two doses of seladelpar/MBX-8025 in subjects with Primary Biliary Cirrhosis (PBC) and an inadequate response to ursodeoxycholic acid (UDCA)

Condition or disease Intervention/treatment Phase
Primary Biliary Cirrhosis (PBC) Drug: Placebo Comparator Drug: Experimental: Seladelpar / MBX-8025 50 mg Drug: Experimental: Seladelpar / MBX-8025 200 mg Phase 2

Detailed Description:

Primary:

To evaluate the effect of MBX-8025 on Alkaline Phosphatase (AP) levels

Secondary:

To evaluate the safety and tolerability of MBX-8025 in subjects with Primary Biliary Cirrhosis (PBC) To evaluate the effects of MBX-8025 on Primary Biliary Cirrhosis (PBC) response criteria To evaluate the effects of MBX-8025 on other markers of liver function, lipids, pruritus and Quality of Life (QoL)

Exploratory:

To evaluate the effect of MBX-8025 on liver imaging and other biochemical markers that may be relevant to the pathophysiology of PBC or the mechanism of action of the drug

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : November 2015
Actual Primary Completion Date : July 2016
Actual Study Completion Date : July 2016


Arm Intervention/treatment
Placebo Comparator: Placebo Dose
Placebo Capsule Two Capsules Daily
Drug: Placebo Comparator
Placebo Capsule (2 taken once daily)
Other Name: Placebo

Experimental: Seladelpar / MBX-8025 50 mg Dose
MBX-8025 50 mg capsule One Capsule Daily
Drug: Experimental: Seladelpar / MBX-8025 50 mg
MBX-8025 50 mg capsule (1 taken once daily)
Other Name: MBX-8025 50 mg dose

Experimental: Seladelpar / MBX-8025 200 mg Dose
MBX-8025 100 mg capsules (2 taken once daily)
Drug: Experimental: Seladelpar / MBX-8025 200 mg
MBX-8025 100 mg capsules (2 taken once daily)
Other Name: MBX-8025 100 mg capsules




Primary Outcome Measures :
  1. Serum Alkaline Phosphatase (AP) [ Time Frame: 12-Weeks ]

Secondary Outcome Measures :
  1. Composite Endpoint of AP and Bilirubin [ Time Frame: 12-Weeks ]
    • AP < 1.67 × upper limit of normal (ULN) and Total Bilirubin within normal limit and
    • > 15% decrease in AP

  2. Laboratory Values [ Time Frame: 12-Weeks ]
    Aspartate aminotransferase (AST)

  3. Laboratory Values [ Time Frame: 12-Weeks ]
    Alanine aminotransferase (ALT)

  4. Laboratory Values [ Time Frame: 12-Weeks ]
    Gamma-glutamyl transferase (GGT)

  5. Laboratory Values [ Time Frame: 12-Weeks ]
    5'nucleotidase

  6. Laboratory Values [ Time Frame: 12-Weeks ]
    Bilirubin (Total, Conjugated, Unconjugated)

  7. Laboratory Values [ Time Frame: 12-Weeks ]
    Bone-specific AP

  8. Laboratory Values [ Time Frame: 12-Weeks ]
    Triglycerides (TG)

  9. Laboratory Values [ Time Frame: 12-Weeks ]
    Total Cholesterol (TC)

  10. Laboratory Values [ Time Frame: 12-Weeks ]
    High Density Lipoprotein Cholesterol (HDL-C)

  11. Laboratory Values [ Time Frame: 12-Weeks ]
    Low Density Lipoprotein Cholesterol (LDL-C)

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

  13. Published PBC response criteria [ Time Frame: 12-Weeks ]
    Paris II

  14. Published PBC response criteria [ Time Frame: 12-Weeks ]
    Toronto I

  15. Published PBC response criteria [ Time Frame: 12-Weeks ]
    Toronto II

  16. Published PBC response criteria [ Time Frame: 12-Weeks ]
    United Kingdom (UK) UK-PBC risk score

  17. 5D-itch scale [ Time Frame: 12-Weeks ]
  18. Visual Analog Score (VAS) [ Time Frame: 12-Weeks ]
  19. PBC-40 Quality of Life (QoL) [ Time Frame: 12-Weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02609048


Locations
Show Show 49 study locations
Sponsors and Collaborators
CymaBay Therapeutics, Inc.
Investigators
Layout table for investigator information
Study Chair: Pol F Boudes, M.D. CymaBay Therapeutics, Inc.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: CymaBay Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02609048    
Other Study ID Numbers: CB8025-21528
First Posted: November 20, 2015    Key Record Dates
Last Update Posted: January 29, 2018
Last Verified: January 2018
Keywords provided by CymaBay Therapeutics, Inc.:
PBC
Additional relevant MeSH terms:
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Liver Cirrhosis
Liver Cirrhosis, Biliary
Fibrosis
Pathologic Processes
Liver Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases