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    Summary
    EudraCT Number:2011-003998-28
    Sponsor's Protocol Code Number:CER-001-CLIN-003
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-11-14
    Trial 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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2011-003998-28
    A.3Full title of the trial
    Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia
    A.3.2Name or abbreviated title of the trial where available
    MODE
    A.4.1Sponsor's protocol code numberCER-001-CLIN-003
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01412034
    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 SponsorCerenis Therapeutics
    B.1.3.4CountryFrance
    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 supportCerenis Therapeutics
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCerenis Therapeutics
    B.5.2Functional name of contact pointChristopher Prue
    B.5.3 Address:
    B.5.3.1Street AddressBP87519-265 rue de la Decouverte
    B.5.3.2Town/ cityLabege
    B.5.3.3Post code31675
    B.5.3.4CountryFrance
    B.5.4Telephone number+33(0)674 91 55 36
    B.5.5Fax number+33(0)562 19 04 17
    B.5.6E-mailprue@cerenis.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 nameRecombinant Human Apolipoprotein A-I/Phospholipids Complex
    D.3.2Product code CER-001
    D.3.4Pharmaceutical form Sterile concentrate
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeCER-001
    D.3.9.3Other descriptive namerecombinant human apolipoprotein A-I
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8.0
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.3Other descriptive namesphingomyelin
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20.95
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.3Other descriptive name1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.65
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    homozygous familial hypercholesterolemia
    E.1.1.1Medical condition in easily understood language
    genetic defect for high cholesterol in blood
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level LLT
    E.1.2Classification code 10020604
    E.1.2Term Hypercholesterolemia
    E.1.2System Organ Class 100000004861
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of the base treatment phase is to assess the impact of 12 intravenous infusions of 8 mg/kg CER-001 given at 2-week intervals (i.e., every 10 to 18 days) on descending thoracic aorta and carotid wall area and volume using 3T magnetic resonance imaging (3TMRI), when administered to patients with genetically-confirmed homozygous familial hypercholesterolemia (HoFH). The objective of the extension phase is to assess the additional benefit as well as the safety of continued use of CER-001 for a one year period (24 total infusions).
    E.2.2Secondary objectives of the trial
    Base treatment phase:
    •Assess 6M efficacy data to determine continuation of ext.phase
    •% change in carotid vessel wall vol.
    •Abs. change carotid vessel wall vol.
    •%change the mean of the carotid normalized wall index
    •%change maximum vessel wall thickness
    •%change mean vessel wall thickness
    •%change thoracic aorta vessel wall vol.measured by 3TMRI
    •%change thoracic aorta mean vessel wall area
    •Abs.change thoracic aorta vessel wall vol.
    •%change carotid lipid core vol.
    •Abs.change carotid lipid core vol.
    •%change carotid calcified vessel wall vol.
    •Abs. change carotid calcified vessel wall vol.
    •%change carotid intra-plaque hemorrhage vol.
    •Abs. change carotid intra-plaque hemorrhage vol.
    Ext. phase:
    •%change baseline to 12M carotid mean vessel wall area measured by 3TMRI
    •%change 6M to12M carotid mean vessel wall area measured by 3TMRI
    •Overall %change baseline to 12M carotid mean vessel wall area measured by 3TMRI
    •Continued safety monitoring --> study duration
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Male or female subjects 12 year or older with homozygous FH (confirmed by genetic testing)
    Subject or subject guardian must sign informed consent (pediatric subject must sign assent if applciable)
    Subject must be willing to participate and comply with all protocol requirements.

    Subject must be enrolled in MODE before 31Dec2012 to be eligible to participate in safety extension phase
    E.4Principal exclusion criteria
    Subjects weighing more than 100 kg at screening.
    Subjects with significant health problems in the recent past including blood disorders, cancer or digestive problems.
    Females who are pregnant, breastfeeding, or plan to become pregnant during the study.
    Subject has known major hematologic, reanl, hepatic, metabolic, GI or endocrine dysfunction.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint in this study is the percent change from baseline to follow-up in carotid mean vessel wall area as measured by 3TMRI.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and 1-3 weeks after last infusion:
    6m (1-3 weeks after infusion 12)
    12m (1-3 weeks after infusion 24)
    E.5.2Secondary end point(s)
    Secondary outcome measurements for the base treatment phase will include:
    • Assessment of six month efficacy data for determination of continuation of extension
    phase
    • Percent change in carotid vessel wall volume
    • Absolute change in carotid vessel wall volume
    • Percent change in the mean of the carotid normalized wall index
    • Percent change in maximum vessel wall thickness
    • Percent change in mean vessel wall thickness
    • Percent change in thoracic aorta vessel wall volume as measured by 3TMRI
    • Percent change in thoracic aorta mean vessel wall area
    • Absolute change in thoracic aorta vessel wall volume
    • Percent change in carotid lipid core volume
    • Absolute change in carotid lipid core volume
    • Percentage change in carotid calcified vessel wall volume
    • Absolute change in carotid calcified vessel wall volume
    • Percent change in carotid intra-plaque hemorrhage volume
    • Absolute change in carotid intra-plaque hemorrhage volume

    Secondary efficacy neasurements for the extension phase will include:
    • Percent change from baseline to twelve months in carotid mean vessel wall area as
    measured by 3TMRI
    • Percent change from six months to twelve months in carotid mean vessel wall area as
    measured by 3TMRI
    • Overall percent change from baseline to twelve months carotid mean vessel wall area
    as measured by 3TMRI
    • Continued safety monitoring throughout the study duration
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline and 1-3 weeks after last infusion:
    6m (1-3 weeks after infusion 12)
    12m (1-3 weeks after infusion 24)
    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 No
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Canada
    France
    Italy
    Netherlands
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    Last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    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 Yes
    F.1.1Number of subjects for this age range: 15
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 0
    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 state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 30
    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)
    No immediate plans are outlined in the protocol. It is possible that a follow-up safety extension could be started for these patients.
    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 Decision2011-11-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-07-08
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