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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03301506




Registration number
NCT03301506
Ethics application status
Date submitted
26/09/2017
Date registered
4/10/2017
Date last updated
15/02/2024

Titles & IDs
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)
Secondary ID [1] 0 0
CB8025-31731
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Biliary Cirrhosis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Infection 0 0 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Seladelpar 5 mg Capsule
Treatment: Drugs - Seladelpar 10 mg Capsule

Experimental: Seladelpar 5 mg Capsules -

Experimental: Seladelpar 10 mg Capsule -


Treatment: Drugs: Seladelpar 5 mg Capsule
Subjects will be assigned to a treatment group if tolerability issues noted in the previous study.

Treatment: Drugs: Seladelpar 10 mg Capsule
Subjects will be assigned to a treatment group unless there are tolerability issues.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Treatment emergent adverse events (TEAEs) (National Cancer Institute {NCI} Common Terminology Criteria for Adverse Events {CTCAE} Version 5.0), biochemistry and hematology results
Timepoint [1] 0 0
Through study completion, up to 60 Months
Secondary outcome [1] 0 0
Death
Timepoint [1] 0 0
60 Months
Secondary outcome [2] 0 0
Liver transplantation
Timepoint [2] 0 0
60 Months
Secondary outcome [3] 0 0
Change in MELD
Timepoint [3] 0 0
60 Months
Secondary outcome [4] 0 0
Ascites
Timepoint [4] 0 0
60 Months
Secondary outcome [5] 0 0
Hospitalization for variceal bleeding
Timepoint [5] 0 0
60 Months
Secondary outcome [6] 0 0
Hospitalization for hepatic encephalopathy
Timepoint [6] 0 0
60 Months
Secondary outcome [7] 0 0
Hospitalization for spontaneous bacterial peritonitis
Timepoint [7] 0 0
60 Months
Secondary outcome [8] 0 0
Response on composite endpoint
Timepoint [8] 0 0
60 Months
Secondary outcome [9] 0 0
Response on composite endpoint
Timepoint [9] 0 0
60 Months
Secondary outcome [10] 0 0
Normalization of ALP
Timepoint [10] 0 0
60 Months
Secondary outcome [11] 0 0
Laboratory Value: Serum Alkaline Phosphatase (ALP)
Timepoint [11] 0 0
Through study completion, up to 60 Months
Secondary outcome [12] 0 0
Laboratory Value: Aspartate Aminotransferase (AST)
Timepoint [12] 0 0
Through study completion, up to 60 Months
Secondary outcome [13] 0 0
Laboratory Value: Alanine Aminotransferase (ALT)
Timepoint [13] 0 0
Through study completion, up to 60 Months
Secondary outcome [14] 0 0
Laboratory Value: Gamma-glutamyl Transferase (GGT)
Timepoint [14] 0 0
Through study completion, up to 60 Months
Secondary outcome [15] 0 0
Laboratory Value: Bilirubin - Total Bilirubin
Timepoint [15] 0 0
Through study completion, up to 60 Months
Secondary outcome [16] 0 0
Laboratory Value: Bilirubin - Conjugated Bilirubin
Timepoint [16] 0 0
Through study completion, up to 60 Months
Secondary outcome [17] 0 0
Laboratory Value: Bilirubin - Unconjugated Bilirubin
Timepoint [17] 0 0
Through study completion, up to 60 Months

Eligibility
Key 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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/12/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
500
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Royal Brisbane & Women's Hospital - Herston
Recruitment hospital [2] 0 0
Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Georgia
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Louisiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Maryland
Country [10] 0 0
United States of America
State/province [10] 0 0
Massachusetts
Country [11] 0 0
United States of America
State/province [11] 0 0
Michigan
Country [12] 0 0
United States of America
State/province [12] 0 0
Minnesota
Country [13] 0 0
United States of America
State/province [13] 0 0
Mississippi
Country [14] 0 0
United States of America
State/province [14] 0 0
Missouri
Country [15] 0 0
United States of America
State/province [15] 0 0
New York
Country [16] 0 0
United States of America
State/province [16] 0 0
North Carolina
Country [17] 0 0
United States of America
State/province [17] 0 0
Pennsylvania
Country [18] 0 0
United States of America
State/province [18] 0 0
Tennessee
Country [19] 0 0
United States of America
State/province [19] 0 0
Texas
Country [20] 0 0
United States of America
State/province [20] 0 0
Virginia
Country [21] 0 0
United States of America
State/province [21] 0 0
Washington
Country [22] 0 0
Argentina
State/province [22] 0 0
Buenos Aires
Country [23] 0 0
Argentina
State/province [23] 0 0
Buenos Aries
Country [24] 0 0
Austria
State/province [24] 0 0
Wels
Country [25] 0 0
Belgium
State/province [25] 0 0
Oost-Vlaanderen
Country [26] 0 0
Belgium
State/province [26] 0 0
Vlaams-Brabant
Country [27] 0 0
Canada
State/province [27] 0 0
Alberta
Country [28] 0 0
Canada
State/province [28] 0 0
Ontario
Country [29] 0 0
Chile
State/province [29] 0 0
Coquimbo
Country [30] 0 0
Czechia
State/province [30] 0 0
Ostrava
Country [31] 0 0
France
State/province [31] 0 0
Lyon
Country [32] 0 0
France
State/province [32] 0 0
Paris
Country [33] 0 0
Germany
State/province [33] 0 0
Baden-Wurttemberg
Country [34] 0 0
Germany
State/province [34] 0 0
Bayern
Country [35] 0 0
Germany
State/province [35] 0 0
Nordrhein-Westfalen
Country [36] 0 0
Germany
State/province [36] 0 0
Schleswig-Holstein
Country [37] 0 0
Germany
State/province [37] 0 0
Berlin
Country [38] 0 0
Germany
State/province [38] 0 0
Hamburg
Country [39] 0 0
Greece
State/province [39] 0 0
Thessaly
Country [40] 0 0
Hungary
State/province [40] 0 0
Ulloi
Country [41] 0 0
Hungary
State/province [41] 0 0
Kaposvár
Country [42] 0 0
Israel
State/province [42] 0 0
Haifa
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Israel
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Jerusalem
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Israel
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Ramat Gan
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Israel
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Tel Aviv
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Italy
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MB
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Italy
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Modena
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Italy
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Palermo
Country [49] 0 0
Korea, Republic of
State/province [49] 0 0
Gyeonggi-do
Country [50] 0 0
Korea, Republic of
State/province [50] 0 0
Seoul
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Korea, Republic of
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Busan
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Korea, Republic of
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Daegu
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Mexico
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Ciudad de Mexico
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Mexico
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Metepec
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Netherlands
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Nijmegen
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New Zealand
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Canterbury
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New Zealand
State/province [57] 0 0
Otago
Country [58] 0 0
Poland
State/province [58] 0 0
Katowice
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Poland
State/province [59] 0 0
Myslowice
Country [60] 0 0
Romania
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Bucharest
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Russian Federation
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Moscow
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Russian Federation
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Saint Petersburg
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Russian Federation
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Stavropol
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Russian Federation
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Ulyanovsk
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Spain
State/province [65] 0 0
Barcelona
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Spain
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Cantabria
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Spain
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Madrid
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Spain
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Málaga
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Switzerland
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Zürich
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Turkey
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Ankara
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Turkey
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Fatih
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Turkey
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Istanbul
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Turkey
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Izmir
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United Kingdom
State/province [74] 0 0
Devon
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United Kingdom
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Hampshire
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United Kingdom
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Nottinghamshire
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United Kingdom
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Birmingham
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United Kingdom
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Hull
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United Kingdom
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London
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United Kingdom
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Newcastle Upon Tyne
Country [81] 0 0
United Kingdom
State/province [81] 0 0
Newcastle

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
CymaBay Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Seladelpar in
Subjects with Primary Biliary Cholangitis (PBC)
Trial website
https://clinicaltrials.gov/ct2/show/NCT03301506
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Barry Crittenden, MD
Address 0 0
Country 0 0
Phone 0 0
510-293-8800
Fax 0 0
Email 0 0
medinfo@cymabay.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03301506