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    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2011-003998-28
    Sponsor's Protocol Code Number:CER-001-CLIN-003
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-10-19
    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 ConcernedUK - MHRA
    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 Address265 rue de la Decouverte, Bat. A
    B.5.3.2Town/ cityLabege
    B.5.3.3Post code31670
    B.5.3.4CountryFrance
    B.5.4Telephone number+33 (0)6 74 91 55 36
    B.5.5Fax number+33 (0)5 62 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.3.8INN - Proposed INNrecombinant human apolipoprotein a1
    D.3.9.2Current sponsor codeCER-001
    D.3.9.3Other descriptive namerecombinant human apolipoprotein A-I
    D.3.9.4EV Substance CodeAS1
    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.3.8INN - Proposed INNsphingomyelin
    D.3.9.3Other descriptive namesphingomyelin
    D.3.9.4EV Substance CodeAS2
    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.3.8INN - Proposed INNDPPG
    D.3.9.3Other descriptive name1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)
    D.3.9.4EV Substance CodeAS3
    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 15.0
    E.1.2Level LLT
    E.1.2Classification code 10020604
    E.1.2Term Hypercholesterolemia
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the impact of 12 intravnous infusions of 8 mg/kg CER-001 given at 2-week intervals on descending thoracic aorta and carotid plaque volume, determined from vessel wall volume of diseased arterial segments using 3T magnetic resonance imaging (3TMRI).
    E.2.2Secondary objectives of the trial
    Absolute change from baseline in carotid plaque volume. Absolute change in baseline in descending thoracid aorta plaque volume. Percent change from baseline in carotid plaque lipid rich necrotic core volume, carotid plauqe calcium volume and coratid intraplaque hemorrhage.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject or Subject guardian must sign the informed consent, and subject must sign assent if applicable, as approved by the EC prior to performance of any screening procedures
    2. Male or female subject 12 years or older
    3.Females of childbearing potential that agree and commit to use an acceptable form of birth control for the entire study. Acceptable forms of birth control for this study are defined as a barrier method plus hormonal therapy (implants, injections, oral contraceptives and IUDs) or abstinence.
    4. Subject diagnosis of Homozygous FH defined as:
    • Genetic testing on record classifying the subject as Homozygous FH
    -or-
    •Genetic testing on record classifying the subject as Compound Heterozygous FH
    with accompanying phenotypical criteria of:
    • a history of LDL-C ≥220 mg/dl (5.69 mmol/L) while receiving maximally tolerated lipid-lowering therapy with <15% response
    -and-
    •LDLC above the 90th percentile in ≥2 firstdegree relatives
    -and-
    • the presence of tendinous xanthomas and/or manifestations of premature
    coronary heart disease or corneal arcus.
    5. Subject must be willing to participate in the study and comply with all protocol
    requirements, including willingness to:
    • Undergo two baseline contrastenhanced 3TMRI evaluations separated by about 510 days.
    • Return to the clinic every two weeks for a total of twelve IV infusions of study drug.
    • Return to the clinic at the end of the study for a followup contrastenhanced 3TMRI procedure.
    E.4Principal exclusion criteria
    1. Subjects weighing more than 100 kg at Screening.
    2. Subjects with significant health problems in the recent past including blood disorders, cancer, or digestive problems but not cardiovascular disease as manifested in inclusion criteria #3 above.
    3. Females who are prenant, breastfeeding, or plan to become pregnant during the study.
    4. Subject has known major hematologic, renal [serum creatinine > 2.0 mg/dL (180 μmol/L)], hepatic (liver enzymes greater than twice the upper limits of normal for the performing laboratory), metabolic, gastrointestinal or endocrine dysfunction in the judgment of the Investigator
    5. Any clinically significant medical condition or presence of any laboratory abnormality performed prior to randomization that is considered by the investigator to be clinically important and could interfere with the conduct of the study.
    6. Subject is likely to be unreliable as a study participant based on then Investigator's (or designee’s) knowledge of the subject (e.g., alcohol or other drug abuse, inability or unwillingness to adhere to the protocol, or psychosis).
    7. Subject has a contraindication to MRI scanning such as imbedded metal (e.g., schrapnel), implanted metal objects (e.g., pacemaker), claustrophobia, allergy to gadolinium chelate contrast or severe renal insufficiency (e.g., GFR < 30mL/min/1.73m2) that would preclude the use of contrastenhanced 3TMRI.
    8. Subject has participated in any investigational drug study within 30 days prior to randomization, or expects to participate in any other investigational drug study during his/her planned participation in this study. The last dose of any investigational product must have been taken at least 30 days prior to the first dose of CER001.
    9. Subject has previously participated in this study or another study involving
    CER001.
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline to follow-up in carotid plaque volume measured as arterial wall volume of diseased portions of the arterial wall using 3TMRI.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and 1-3 weeks after last infusion.
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints will include:
    Absolute change from baseline to followup in carotid plaque volume.
    Percent change and absolute change from baseline to followup in descending
    thoracic aorta plaque volume.
    Percent change and absolute change from baseline to followup in total descending
    thoracic aorta plus carotid plaque volume.
    Percent change from baseline in carotid plaque lipid –rich necrotic core volume.
    Percent change from baseline in carotid plaque calcium volume.
    Percent change from baseline in carotid intraplaque hemorrhage (IPH).
    Most severe % stenosis of right and of left carotid arteries by magnetic resonance angiography (MRA) or by noncontrast MRI luminal area reduction if MRA is not available.
    Minimum thickness of carotid fibrous cap in carotids with a stenosis >50%.

    The safety endpoints in this study include:
    Incidence and severity of AEs from routine monitoring;
    Incidence of abnormalities and changes from baseline in clinical laboratory parameters from testing of blood and urine, including biomarkers for immunogenicity;
    Incidence of adverse changes from baseline vital sign values; and
    Incidence of adverse changes from baseline physical examinations.
    Minimum thickness of carotid fibrous cap in carotids with a stenosis >50%.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy Endpoints: Baseline and 1-3 weeks after last infusion.
    Safety Endpoints: From Visit 3 (first drug infusion) through to Visit 15, 1-3 weeks after last drug infusion.
    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 No
    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.8.2.4Number of treatment arms in the trial1
    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 EEA7
    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
    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 years0
    E.8.9.1In the Member State concerned months9
    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 months1
    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.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    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) No
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    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. Patients otherwise will receive their usual standard of care.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-12-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-07-08
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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