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    Summary
    EudraCT Number:2020-004348-27
    Sponsor's Protocol Code Number:CB8025-32048
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-02-02
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-004348-27
    A.3Full title of the trial
    RESPONSE: A Placebo-controlled, Randomized, Phase 3 Study to Evaluate the Efficacy and Safety of Seladelpar in Patients With Primary Biliary Cholangitis (PBC) and an Inadequate Response to or an Intolerance to Ursodeoxycholic Acid (UDCA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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).
    A.3.2Name or abbreviated title of the trial where available
    RESPONSE
    A.4.1Sponsor's protocol code numberCB8025-32048
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04620733
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCymaBay Therapeutics, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCymaBay Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCymaBay Therapeutics, Inc.
    B.5.2Functional name of contact pointCaroline Cooper
    B.5.3 Address:
    B.5.3.1Street Address7575 Gateway Boulevard Suite 110
    B.5.3.2Town/ cityNewark
    B.5.3.3Post code94560
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1510293-8819
    B.5.5Fax number+15102938133
    B.5.6E-mailccooper@cymabay.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1930
    D.3 Description of the IMP
    D.3.1Product nameSeladelpar
    D.3.2Product code MBX-8025
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSELADELPAR
    D.3.9.1CAS number 928821-40-3
    D.3.9.2Current sponsor codeMBX-8025
    D.3.9.4EV Substance CodeSUB192392
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1930
    D.3 Description of the IMP
    D.3.1Product nameSeladelpar
    D.3.2Product code MBX-8025
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSELADELPAR
    D.3.9.1CAS number 928821-40-3
    D.3.9.2Current sponsor codeMBX-8025
    D.3.9.4EV Substance CodeSUB192392
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    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).
    E.1.1.1Medical condition in easily understood language
    Disease of the liver
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the treatment effect of seladelpar on composite biochemical improvement in cholestasis markers based on ALP and total bilirubin and to evaluate the safety of seladelpar over 12 months of treatment compared to placebo.
    E.2.2Secondary objectives of the trial
    The key secondary objectives of this study are to evaluate the effect of seladelpar on the normalization of ALP values at 12 months of treatment compared to placebo and to evaluate the effect of seladelpar on pruritus at 6 months of treatment compared to placebo in subjects with baseline moderate to severe pruritus.
    Other secondary objectives of the study are to evaluate the effect of seladelpar on other measures of cholestasis, metabolic markers, and PBC prognostic criteria; to evaluate the effect of seladelpar on quality of life (QoL); and to evaluate the effect of seladelpar on PBC-associated clinical outcomes.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria to be eligible for study participation:
    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 based on any two of the following criteria:
    a. History of ALP above 1.0× ULN for at least 6 months
    b. Positive AMA titer (>1:40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay [ELISA]) or positive PBC-specific antinuclear antibodies (ANAs)
    c. Documented liver biopsy results consistent with PBC
    4. UDCA for the past 12 months (stable dose for >3 months prior to screening) OR intolerant to UDCA (last dose of UDCA >3 months prior to screening)
    5. Laboratory parameters measured by the Central Laboratory at screening:
    a. ALP ≥1.67× ULN
    b. Aspartate aminotransferase (AST) ≤3× ULN
    c. ALT ≤3× ULN
    d. Total bilirubin ≤2× ULN
    e. Estimated glomerular filtration rate (eGFR) >45 mL/min/1.73 m2 (calculated by the Modification of Diet in Renal Disease study equation)
    f. International normalized ratio (INR) below 1.1× ULN
    For subjects on anticoagulation therapy, INR must be maintained in the range required for prophylaxis for their specific disease.
    g. Platelet count ≥100×103/μL
    NOTE: PT, INR, and platelets can be performed locally at the Screening Visit, if deemed necessary by the investigator after consultation with the
    medical monitor, in cases where centrally read samples are deemed invalid
    6. Females of reproductive potential (refer to Section 8.1.1) must use at least 1 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
    E.4Principal 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 (e.g., cancer) or confound its results (e.g., Paget’s disease, any active infection)
    3. Advanced PBC as defined by the Rotterdam criteria (albumin below the lower limit of normal AND total bilirubin above 1.0× ULN)
    4. Presence of clinically important hepatic decompensation, including the following:
    a. History of liver transplantation, current placement on liver transplantation list, or current Model for End-Stage Liver Disease (MELD) score ≥12. For subjects on anticoagulation medication, evaluation of the baseline INR, in concert with their current dose adjustments of their anticoagulant medication, will be taken into account when calculating the MELD score. This will be done in consultation with the medical monitor.
    b. Complications of portal hypertension, including known esophageal varices, history of variceal bleeds or related interventions (e.g., transjugular intrahepatic portosystemic shunt placement), ascites, and hepatic encephalopathy
    c. Cirrhosis with complications, including history or presence of spontaneous bacterial peritonitis, hepatocellular carcinoma, or hepatorenal syndrome
    5. Other chronic liver diseases:
    a. Current features of autoimmune hepatitis as determined by the investigator based on immunoserology, liver biochemistry, or historic confirmed liver histology
    b. Primary sclerosing cholangitis determined by the 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. History of biopsy confirmed NASH
    f. History or evidence of Gilbert’s syndrome with elevated total bilirubin
    g. History or evidence of hemochromatosis
    h. Hepatitis B, defined as the presence of hepatitis B surface antigen (HBsAg)
    i. Hepatitis C, defined as the presence of hepatitis C virus (HCV) ribonucleic acid (RNA)
    j. History, evidence, or high suspicion of hepatobiliary malignancy based on imaging, screening laboratory values, and/or clinical symptoms
    6. Known history of human immunodeficiency virus (HIV) or positive antibody test at screening
    7. Clinically important alcohol consumption, defined as more than 2 drink units per day (equivalent to 20 g) in women and 3 drink units per day (equivalent to 30 g) in men, or inability to quantify alcohol intake reliably
    8. History of malignancy diagnosed or treated, actively or within 2 years, or ongoing evaluation for malignancy; localized treatment of squamous or noninvasive basal cell skin cancers and cervical carcinoma in situ is allowed if appropriately treated prior to screening.
    9. Treatment with OCA, and fibrates (e.g., bezafibrate, fenofibrate, elafibranor, lanifibranor, pemafibrate, saroglitizar) 6 weeks prior to screening
    10. Treatment with colchicine, methotrexate, azathioprine, or long-term systemic corticosteroids (>2 weeks) during 2 months prior to screening. See Section 7 for additional medications that may be excluded.
    11. Treatment with anti-pruritic drugs (e.g., cholestyramine, naltrexone, rifampicin, sertraline, or any experimental approach) must be on a stable dose within 1 month prior to screening
    12. Treatment with any other investigational therapy or device within 30 days or within 5 half-lives, whichever is longer, prior to screening
    13. For females, pregnancy or breastfeeding
    14. 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.
    15. Immunosuppressant therapies (e.g., cyclosporine, tacrolimus, anti- TNF or other immunosuppressive biologics).
    16. 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.
    17. Active COVID-19 infection during Screening.
    E.5 End points
    E.5.1Primary end point(s)
    1. Proportion of subjects who are considered responders at 12 months based on the following composite endpoint of ALP and total bilirubin at 12 months requiring
    a. ALP <1.67× ULN
    b. ≥15% decrease in ALP
    c. Total bilirubin ≤1.0× ULN
    2. Assessment of TEAEs (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE] Version 5.0), biochemistry, and hematology
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. 12 months
    2. Throughout the study
    E.5.2Secondary end point(s)
    1. Proportion of subjects with ALP ≤1.0× ULN at 12 months (ie, normalization)
    2. Change from baseline in weekly averaged pruritus NRS in subjects with baseline NRS ≥4 at 6 months
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. 12 months
    2. 6 months
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA96
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Canada
    Chile
    Israel
    Korea, Republic of
    Mexico
    New Zealand
    United States
    France
    Poland
    Netherlands
    Romania
    Spain
    Switzerland
    Czechia
    Germany
    Greece
    Italy
    Belgium
    Hungary
    Russian Federation
    Turkey
    Ukraine
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS is the Post-Treatment Safety Follow-up visit that will occur 1 month (4 weeks±7 days) after the last dose of the study drug.
    At the end of the visit, subjects will be invited to participate in the long-term study (Study CB8025-31731-RE).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 125
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 25
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 180
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who will successfully complete this study will be offered open-label treatment in a long-term study (Study CB8025-31731-RE); subjects receiving placebo will be switched to the active treatment with seladelpar.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-03-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-08-11
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