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    Summary
    EudraCT Number:2014-004856-68
    Sponsor's Protocol Code Number:CB8025-21427
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-05-19
    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 ConcernedFrance - ANSM
    A.2EudraCT number2014-004856-68
    A.3Full title of the trial
    A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH)
    Étude de phase II de titrage de dose en ouvert, d’une durée de 12 semaines, visant à évaluer les effets de MBX-8025 chez des patients atteints d’hypercholestérolémie homozygote familiale (HHF).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH)
    Étude d'une durée de 12 semaines, en ouvert, tous les patients recevront d'abord un placebo pendant 2 semaines et ensuite un titrage de dose du médicament actif : chaque patient recevra d'abord 50 mg, après évaluation il sera possible d'augmenter la dose à 100 mg et après une autre évaluation, à 200 mg du traitement à l'étude. Étude de phase II, visant à évaluer les effets de MBX-8025 chez des patients atteints d'hypercholestérolémie homozygote familiale (HHF)
    A.4.1Sponsor's protocol code numberCB8025-21427
    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 pointPol Boudes
    B.5.3 Address:
    B.5.3.1Street Address7999 Gateway Blvd, suite 130
    B.5.3.2Town/ cityNewark CA
    B.5.3.3Post code94560
    B.5.3.4CountryUnited States
    B.5.4Telephone number0015102938815
    B.5.5Fax number0015102936853
    B.5.6E-mailpboudes@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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameMBX-8025
    D.3.2Product code MBX-8025
    D.3.4Pharmaceutical form Capsule
    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 INNMBX-8025
    D.3.9.1CAS number 928821-40-3
    D.3.9.2Current sponsor codeMBX-8025
    D.3.9.4EV Substance CodeSUB170472
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number50 to 100
    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
    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
    Homozygous Familial Hypercholesterolemia
    Hypercholestérolémie homozygote familiale (HHF)
    E.1.1.1Medical condition in easily understood language
    High cholesterol due to genetic disorder
    Cholestérol élevé causé par un trouble génétique
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    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
    Primary:
    To evaluate the effect of MBX-8025 on Low Density Lipoprotein Cholesterol (LDL-C).
    Primaire :
    Évaluer l’effet de MBX-8025 sur le cholestérol LDL-C (lipoprotéine de basse densité).
    E.2.2Secondary objectives of the trial
    To evaluate the effects of MBX-8025 on other lipid parameters
    To evaluate the safety and tolerability of MBX-8025 in patients with HoFH.
    To evaluate steady-state trough plasma levels of MBX-8025 and its metabolites, M1, M2 and M3
    Exploratory:
    To evaluate the effects of MBX-8025 on proprotein convertase subtilisin/kexin type 9 (PCSK9) and high sensitivity C reactive protein (hs-CRP).
    Évaluer l’effet de MBX-8025 sur d'autres paramètres lipidiques
    Évaluer l'innocuité et la tolérance de MBX-8025 chez des patients atteints de HHF.
    Évaluer les concentrations plasmatiques minimales à l’état d’équilibre de MBX-8025 et de ses métabolites M1, M2 et M3
    Exploratoire :
    Évaluer les effets de MBX-8025 sur la proprotéine convertase subtilisine / kexine type 9 (PCSK9) et la protéine C-réactive ultrasensible (hs-CRP).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusions:
    1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
    2. Male or female with HoFH confirmed by genotype (two mutants alleles at the LDL-Receptor gene locus).
    3. 18 years of age or older.
    4. Existing lipid lowering therapies (statins, cholesterol absorption inhibitors, bile acid sequestrants, nicotinic acid and their combinations, LDL-C apheresis) on a stable regimen for at least four weeks before screening visit.
    5. Stable lipid lowering diet compatible with a Step I diet of the AHA.
    6. Fasting LDL-C ≥ 4.8 mmol/L (≥ 185.6 mg/dL) during screening.
    7. 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.
    1. Patient ayant signé un formulaire de consentement éclairé (daté et signé) et tout autre autorisation requise par les lois en vigueur et capable de respecter toutes les obligations de l’étude.

    2. Être un homme ou femme atteint(e) de HHF confirmée par génotype (deux allèles mutants au niveau du locus du gène du récepteur LDL)

    3. Avoir 18 ans ou plus

    4. Être sous traitement hypolipémiant stable (statines, inhibiteurs de l'absorption du cholestérol, chélateurs des acides biliaires, acide nicotinique et ses dérivés, aphérèse du LDL-C) depuis au moins quatre semaines avant la Visite de sélection.

    5. Suivre un régime hypolipémiant stable compatible avec un régime d'étape I de l'American Heart Association (AHA).

    6. Présenter un taux de LDL-C à jeun ≥ 4,8 mmol/l (≥ 185,6 mg/dl) lors de la sélection.

    7. Pour les femmes en âge de procréer, utiliser au moins une méthode contraceptive de barrière ainsi qu'un deuxième moyen de contraception efficace pendant l'étude et pendant au moins deux semaines après la dernière dose. Pour les hommes, utiliser une méthode contraceptive appropriée (i.e. préservatifs) pour que leurs partenaires féminines en âge de procréer ne tombent pas enceinte pendant l’étude et au moins 2 semaines après la dernière dose.
    E.4Principal exclusion criteria
    Exclusions:
    1. Treatment with lomitapide or mipomersen within two months of screening.
    2. Heart Failure (HF) with New York Heart Association (NYHA) class III and class IV or a left ventricular ejection fraction (LVEF) of less than 30%.
    3. Uncontrolled cardiac arrhythmia during the past three months of screening.
    4. Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass graft or stroke during the past three months prior to Screening Visit.
    5. Planned cardiac surgery, or planned revascularization, in the next four months.
    6. Uncontrolled hypertension.
    7. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN).
    8. Unexplained creatine kinase (CK) ≥ 5 times the ULN.
    9. For females, pregnancy or breast-feeding.
    10. Any other condition(s) that would compromise the safety of the patient or compromise the quality of the clinical study as judged by the Investigator and/or Medical Monitor
    1. Avoir reçu un traitement par lomitapide ou mipomersen dans les deux mois précédant la sélection.

    2. Présenter une insuffisance cardiaque (IC) de classe III ou de classe IV de la New York Heart Association (NYHA) ou une fraction d'éjection du ventricule gauche (FEVG) inférieure à 30 %.

    3. Avoir présenté une arythmie cardiaque incontrôlée au cours des trois mois précédant la sélection.

    4. Avoir subi un infarctus du myocarde, un angor instable, une intervention coronarienne percutanée, un pontage aorto-coronarien ou un AVC au cours des trois mois précédant la Visite de sélection.

    5. Avoir une intervention de chirurgie cardiaque ou de revascularisation prévue au cours des quatre prochains mois.

    6. Souffrir d'hypertension non contrôlée.

    7. Présenter un taux d'aspartate aminotransférase (ASAT) ou d'alanine aminotransférase (ALAT) ≥ 3 x la limite supérieure de la normale (LSN).

    8. Présenter un taux inexpliqué de créatine kinase (CK) ≥ 5 fois la LSN.

    9. Pour les femmes, être enceinte ou allaiter.

    10. Toute(s) autre(s) condition(s) susceptible(s) de compromettre la sécurité du patient ou la qualité de l'étude clinique selon le jugement de l'investigateur et/ou du moniteur médical
    E.5 End points
    E.5.1Primary end point(s)
    The effect of MBX-8025 on Serum LDL-C.
    Les effets du MBX-8025 sur le LDL-C sérique.
    E.5.1.1Timepoint(s) of evaluation of this end point
    All visits after first drug administration: visit 4-10
    Toutes les visites après la première administration du traitement : visite 4-10
    E.5.2Secondary end point(s)
    The effect of MBX-8025 on other lipid parameters, safety and tolerability and PK of MBX-8025 and it's metabolites.
    Les effet de MBX-8025 sur d'autres paramètres lipidiques.
    L'innocuité, la tolérance et la PK de MBX-8025 et de ses métabolites.
    E.5.2.1Timepoint(s) of evaluation of this end point
    All visits after first drug administration: visit 4-10
    Toutes les visites après la première administration du traitement : visite 4-10
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    DVDP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months6
    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: 7
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 8
    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)
    None
    Aucun
    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 Decision2015-03-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-03-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-02-15
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