Seladelpar in Subjects With Primary Biliary Cholangitis (PBC)

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2024
• Main ID: NCT03301506
• Date of registration: 26/09/2017
• Prospective Registration: Yes
• Primary sponsor: CymaBay Therapeutics, Inc.
• Public title: Seladelpar in Subjects With Primary Biliary Cholangitis (PBC)
• Scientific title: ASSURE: An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Seladelpar in Subjects With Primary Biliary Cholangitis (PBC)
• Date of first enrolment: December 12, 2017
• Target sample size: 500
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03301506
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belgium, Canada, Chile, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Korea, Republic of, Mexico, Netherlands, New Zealand, Poland, Romania, Russian Federation, Spain, Switzerland, Turkey, United Kingdom, United States

Contacts

• Name: Barry Crittenden, MD

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Must have given written informed consent (signed and dated)

2. Participated in a PBC study with seladelpar

3. 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

Exclusion Criteria:

Exclusion criteria are only applicable for subjects with a seladelpar interruption greater
than 4 weeks prior to Day 1 of this study and for subjects who participated in CB8025-21838
irrespective of seladelpar interruption.

1. Treatment-related adverse event (AE) leading to seladelpar discontinuation in a
previous PBC study with 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 or ALT above 3 × the upper limit of normal (ULN)

4. Total bilirubin above 2 × ULN

5. MELD score = 12. For subjects on anticoagulation medication, evaluation of the
baseline INR, in concert with any current dose adjustments in anti-coagulant
medications, will be taken into account when calculating this score. This will be done
in consultation with the medical monitor.

6. Evidence of advanced PBC as defined by the Rotterdam criteria: albumin below 1× the
lower limit of normal (LLN) AND total bilirubin above 1 × ULN)

7. eGFR =45 mL/min/1.73 m2 (calculated by MDRD formula)

8. Auto-immune hepatitis

9. Primary sclerosing cholangitis

10. Known history of alpha-1-antitrypsin deficiency

11. Known history of chronic viral hepatitis

12. For females, pregnancy or breast-feeding

13. Use of colchicine, methotrexate, azathioprine, or long-term use of systemic steroids
(e.g. prednisone, prednisolone, budesonide) (>2 weeks) within 2 months prior to
Screening

14. Current use of fibrates or use of fibrates within 3 months prior to Screening

15. Current use of obeticholic acid or use of obeticholic acid within 3 months prior to
Screening

16. Use of an experimental or unapproved treatment for PBC within 3 months prior to
Screening

17. History of malignancy diagnosed or treated, actively or within 2 years, or active
evaluation for malignancy; localized treatment of squamous or non-invasive basal cell
skin cancers and cervical carcinoma in-situ is allowed if appropriately treated prior
to Screening

18. Treatment with any other investigational therapy or medical device within 30 days or
within 5 half-lives, whatever is longer, prior to Screening

19. 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

20. Immunosuppressant therapies (e.g., cyclosporine, tacrolimus, anti-TNF or other
immunosuppressive biologics)

21. Other medications that effect liver or GI functions such as absorption of medications
or the roux-en-y gastric bypass procedure may be prohibited and should be discussed
with the medical monitor on a case-by-case basis

22. Positive for:

1. Hepatitis B, defined as the presence of hepatitis B surface antigen

2. Hepatitis C, defined as the presence of hepatitis C virus ribonucleic acid (RNA)

3. Human immunodeficiency virus (HIV) antibody

23. Active COVID-19 infection during screening



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Biliary Cirrhosis

Intervention(s)

Drug: Seladelpar 10 mg Capsule

Drug: Seladelpar 5 mg Capsule

Primary Outcome(s)

Treatment emergent adverse events (TEAEs) (National Cancer Institute {NCI} Common Terminology Criteria for Adverse Events {CTCAE} Version 5.0), biochemistry and hematology results [Time Frame: Through study completion, up to 60 Months]

Secondary Outcome(s)

Change in MELD [Time Frame: 60 Months]

Hospitalization for spontaneous bacterial peritonitis [Time Frame: 60 Months]

Ascites [Time Frame: 60 Months]

Laboratory Value: Bilirubin - Total Bilirubin [Time Frame: Through study completion, up to 60 Months]

Laboratory Value: Bilirubin - Conjugated Bilirubin [Time Frame: Through study completion, up to 60 Months]

Laboratory Value: Bilirubin - Unconjugated Bilirubin [Time Frame: Through study completion, up to 60 Months]

Laboratory Value: Gamma-glutamyl Transferase (GGT) [Time Frame: Through study completion, up to 60 Months]

Hospitalization for variceal bleeding [Time Frame: 60 Months]

Laboratory Value: Aspartate Aminotransferase (AST) [Time Frame: Through study completion, up to 60 Months]

Liver transplantation [Time Frame: 60 Months]

Response on composite endpoint [Time Frame: 60 Months]

Normalization of ALP [Time Frame: 60 Months]

Death [Time Frame: 60 Months]

Hospitalization for hepatic encephalopathy [Time Frame: 60 Months]

Laboratory Value: Alanine Aminotransferase (ALT) [Time Frame: Through study completion, up to 60 Months]

Laboratory Value: Serum Alkaline Phosphatase (ALP) [Time Frame: Through study completion, up to 60 Months]

Secondary ID(s)

CB8025-31731

Source(s) of Monetary Support

Please refer to primary and secondary sponsors