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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 1 February 2020
Main ID:  EUCTR2018-001171-20-FR
Date of registration: 18/10/2018
Prospective Registration: Yes
Primary sponsor: CymaBay Therapeutics, Inc.
Public title: A clinical trial to assess the safety and efficacy of seladelpar in patients with primary biliary cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA).
Scientific title: A 52-week, placebo-controlled, randomized, Phase 3 study to evaluate the safety and efficacy of seladelpar in subjects with primary biliary cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA).
Date of first enrolment: 23/01/2019
Target sample size: 240
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-001171-20
Study type:  Interventional clinical trial of medicinal product
Study design: 
Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: yes
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: no
Number of treatment arms in the trial: 3
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Argentina Austria Belgium Brazil Canada France Germany Greece
Hungary Israel Italy Korea, Republic of Mexico Netherlands Poland Romania
Russian Federation Serbia Spain United Kingdom United States
Contacts
Name: Senior VP, RA&QA - Klara Dickinson   
Address:  7575 Gateway Boulevard Suite 110 94560 Newark United States
Telephone: +15102938836
Email: kdickinson@cymabay.com
Affiliation:  CymaBay Therapeutics, Inc.
Name: Senior VP, RA&QA - Klara Dickinson   
Address:  7575 Gateway Boulevard Suite 110 94560 Newark United States
Telephone: +15102938836
Email: kdickinson@cymabay.com
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 6 months
• Positive anti-mitochondrial antibody (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 12 months OR intolerant to UDCA (last dose of UDCA > 3 months prior to Screening)
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 200
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

Exclusion criteria:
1. Previous exposure to seladelpar (MBX-8025)
2. 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)
3. AST above 3 × ULN
4. ALT above 3 × ULN
5. Total bilirubin above 2.0 × ULN
6. Advanced PBC as defined by the Rotterdam criteria (albumin below LLN AND total bilirubin above 1 × ULN
7. Creatine kinase (CK) above 1.0 × ULN
8. eGFR below 60 mL/min/1.73 m2 (calculated by MDRD formula)
7. Serum creatinine above 1.0 × ULN
9. International normalized ratio (INR) above 1.0 × ULN
10. Platelet count below 100 × 103/µL
11. Presence of clinically significant hepatic decompensation, including:
- History of liver transplantation, current placement on liver transplantation list, or current Model for End-Stage Liver Disease (MELD) score = 15
- Complications of portal hypertension, including known esophageal varices, history of variceal bleeds or related interventions (e.g., transjugular intrahepatic portosystemic shunt placement), relevant ascites, hepatic encephalopathy
- Cirrhosis with complications, including history or presence of spontaneous bacterial peritonitis
12. Other chronic liver diseases:
a.Current features of auto-immune hepatitis as determined by the investigator based on immunoserology, liver biochemistry and histology
b.Primary sclerosing cholangitis determined by presence of diagnostic cholangiographic findings
c.History or clinical evidence of alcoholic liver disease
d.History or clinical evidence of alpha-1-antitrypsin deficiency
e.Biopsy confirmed nonalcoholic steatohepatitis
f.History or evidence of Gilbert’ Syndrome with elevated total bilirubin
g.History or evidence of hemochromatosis
h.Hepatitis B defined as presence of hepatitis B surface antigen (HBsAg)
i.Hepatitis C defined as presence of HCV RNA
13. Known history of HIV
14. Evidence of significant alcohol consumption
15. Evidence of drug abuse
16. Subjects with inadequate response to obeticholic acid (OCA) or intolerance to OCA: OCA must be discontinued 30 days prior to Screening
17. Use of colchicine, methotrexate, azathioprine, or long-term systemic corticosteroids (> 2 weeks) within two months prior to Screening
18. Use of fibrates within 30 days prior to Screening
19. Use of simvastatin within 7 days prior to Screening
20. Use of an experimental or unapproved treatment for PBC within 30 days prior to Screening
21. Use of experimental or unapproved immunosuppressant within 30 days prior to Screening
22. Treatment with any other investigational therapy or device within 30 days or within five half-lives, whatever is longer, prior to Screening
23. For females, pregnancy or breast-feeding
24. 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:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Primary biliary cholangitis (PBC, formerly known as primary biliary cirrhosis) is a serious and potentially life threatening autoimmune disease of the liver characterized by impaired bile flow (cholestasis) and accumulation of toxic bile acids (BA).
Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Intervention(s)

Product Name: Seladelpar
Product Code: MBX-8025
Pharmaceutical Form: Capsule
INN or Proposed INN: SELADELPAR
CAS Number: 928821-40-3
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Product Name: Seladelpar
Product Code: MBX-8025
Pharmaceutical Form: Capsule
INN or Proposed INN: SELADELPAR
CAS Number: 928821-40-3
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Primary Outcome(s)

Primary end point(s): 1 - Response on the composite endpoint of AP and total bilirubin at 12 months:
o AP < 1.67 × ULN,
o = 15% decrease in AP, and
o Total bilirubin = ULN
2 - Assessment of treatment-emergent AEs (TEAEs) (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE] Version 4.0), biochemistry and hematology

Secondary Objective: Evaluate the effect of seladelpar on normalization of alkaline phosphate (AP) levels and to evaluate the effect of seladelpar on pruritus.
Evaluate the effect of seladelpar on quality of life (QoL), to evaluate the effect of seladelpar on other measures of cholestasis, metabolic outcomes, PBC prognosis criteria, and the effect of seladelpar on PBC clinical outcomes.
Main Objective: Evaluate the safety and effect on cholestasis of two seladelpar regimens (5 mg/day titrated to 10 mg/day and 10 mg/day) over 52 weeks of treatment compared to placebo.

Timepoint(s) of evaluation of this end point: 1- 12 months
2 - Throughout the study
Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1 - 12 months
2 - 6 months

Secondary end point(s): 1 - Proportion of patients with AP =1.0 × ULN at 12 months
2 - Change from baseline in pruritus NRS at 6 months
Secondary ID(s)
NCT03602560
CB8025-31735
2018-001171-20-GR
Source(s) of Monetary Support
CymaBay Therapeutics, Inc.
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 23/01/2019
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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