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    EudraCT Number:2017-002158-35
    Sponsor's Protocol Code Number:IVA_01_ODI_HMPS_17_002
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-08-16
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2017-002158-35
    A.3Full title of the trial
    A phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to investigate the fate of odiparcil since its administration up to the point is completely eliminated, its safety and efficacy in patients 16 years and above with mucopolysaccharidosis (MPS) type VI
    A.4.1Sponsor's protocol code numberIVA_01_ODI_HMPS_17_002
    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 SponsorInventiva S.A.
    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 supportInventiva S.A.
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInventiva S.A
    B.5.2Functional name of contact pointVéronique BERGER
    B.5.3 Address:
    B.5.3.1Street Address50 rue de Dijon
    B.5.3.2Town/ cityDAIX
    B.5.3.3Post code21121
    B.5.4Telephone number+33380 447 697
    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 nameOdiparcil
    D.3.2Product code IVA 336
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNOdiparcil
    D.3.9.1CAS number 137215-12-4
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 placeboFilm-coated tablet
    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
    Mucopolysaccharidosis (MPS) type VI.
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis (MPS) type VI.
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10056892
    E.1.2Term Mucopolysaccharidosis VI
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 the study is to assess the safety and efficacy of two doses of odiparcil in MPS VI patients and to provide evidence to enable the selection of the relevant dose of odiparcil for phase III study.
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to characterize the dose response, PK and PD of odiparcil.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Optional pharmacogenomic sub-study
    E.3Principal inclusion criteria
    1. Male or female gender.
    2. Age ≥18 years.
    3. Diagnosis of MPS VI.
    4. Urine GAG above upper limit of normal (ULN) based on historical data.
    5. Willing and able to provide written, dated, signed informed consent, after the nature of the study has been explained, and prior to any research-related procedures or study assessment.
    6. Able to comply with all study procedures.
    7. Women with childbearing potential (i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy) must agree to use a highly effective method of birth control during the study and at least 4 weeks after last administration.The following can be considered to be examples of highly effective methods of contraception preferably with low user dependency:
    - combined (estrogen and progestogen containing) hormonal contraception associated wiht inhibition of ovulation (oral, intravanginal or transdermal)
    - progestogen-only hormonal contraceptioni associated with inhibition of ovulation (oral, injectable, implantable) 1
    - Intrauternine device (IUD) 1
    - Intrauterine hormone-releasing system (IUS) 1
    - Bilateral tubal occulusion 1
    - Vasectomised partner 1
    - Sexual abstinence
    These methods of contraception must be supplemented with a barrier method (preferably male condom).

    1 Contraception methods that are considered to have low user dependency.

    Inclusion criteria for Enzyme Replacement Therapy (ERT) receiving group
    1. Patients with MPS Type VI receiving enzyme replacement therapy (Naglazyme) for at least 6 months on the licensed dosage or as per local guidelines.

    Inclusion criteria for not ERT receiving group:
    Patients with MPS Type VI not receiving enzyme replacement therapy for the following reasons:
    1. Patients not treated with ERT (treatment naive) or previously treated with ERT but having discontinued for more than 3 months before inclusion, either due to medical decision or personal choice (e.g. for quality of life preferences). Under no circumstances patients will be taken of ERT for the only purpose of participating to the study. Reason for not receiving ERT should be specified by the invetigator.
    2. Patients allergic to ERT therapy
    3. Patients that have had a previous hematopoietic stem cell transplant (HSCT)

    E.4Principal exclusion criteria
    Exclusion criteria for the entire cohort
    1. Use of any investigational product or investigational medical device within 30 days prior to screening. This will include product bought over the counter specifically compounds like genistein and pentosane polysulphate which may not be considered as investigational products by patients and some health care professionals.
    2. Concurrent disease or condition that would interfere with study participation or pose a safety concern for example patient with: severe cardiac insufficiency as define NYHA class > II, and severe restrictive chronic respiratory insufficiency as reflected by serum [HCO3-] ≥28 mEq/L.
    3. Subjects who had surgery within 3 months before study starts, or for whom surgery is planned during study period.
    4. Patient with spinal cord compression requiring surgical intervention.
    5. Subjects with the following liver test abnomalies: any ALT, AST > 3 x ULN or bilirubin >1.5 x ULN (except if Gilbert syndrome) at screening visit.
    6. Evidence of an immunosuppressive state, including known HIV infection, agammaglubilinemias, T-Cell deficiencies.
    7. Subjects with history of chronic infections, including but not limited to subjects with history of viral hepatitis C, or B, with recent history of serious or life-threatening infection or any current signs or symptoms that may indicate infection at visit V-1 of study as per investigators clinical judgement.
    8. History of malignant cancer except of cervical carcinoma in situ, basal cell carcinoma, dermatological squamous cell carcinoma.
    9. Subjects with significant haematologic abnormalities, such as hemoglobin <8 g/dL, or WBC<2000 /mm3 or absolute neutrophil count <1300 /mm3, or platelet <30.000 /mm3.
    Subjects with other haematological abnormalities not corresponding to the above criteria should not be included in preliminary safety assessment.
    10. International Normalized Ratio (INR), activated partial thromboplastin time (aPTT) or thrombin time (TT) values above the central laboratory reference range at screening considered as clinically significant by the investigator. For patients on anticoagulants, they should be within their target effect on INR and be stable.
    11. Any history of bleeding diathesis.
    12. Patient with coexistence of corneal pathologies other than corneal clouding (e.g. exposure keratopathy)
    13. An unwillingness on the part of male patients to abstain from sexual intercourse with pregnant or lactating women; or an unwillingness to use highly effective form of birth control if engaging in sexual intercourse with a woman who could become pregnant from the time of the first dose of study medication until completion of follow-up procedures.
    14. An unwillingness on the part of female patients to use highly effective form of birth control if engaging in sexual intercourse and to have a monthly pregnancy test during treatment and until completion of follow-up procedures.
    15. Pregnant or lactating women.
    16. Have a known hypersensitivity to any of the ingredients or excipients of the IMP including: Microcrystalline Cellulose, Povidone, Sodium starch glycolate (type A), Magnesium stearate, Opadry™ II 85F18422

    Exclusion criteria for ERT treated group:
    1. Previous hematopoietic stem cell transplant (HSCT)
    E.5 End points
    E.5.1Primary end point(s)
    1. Preliminary safety assessment
    The goal of preliminary safety assessment is to focus on clinical tolerance and lab safety such as coagulation, liver enzymes and cristalluria.
    2. Core study
    a. Safety outcomes:
    a1. Safety outcomes will be evaluated on the following safety variables: incidence of AEs/SAEs, patient withdrawals from study due to AEs/SAEs, change from baseline in laboratory safety test, change from baseline in vital signs parameters.
    a2.1 Twelve-lead-ECG
    a2.2 Bone biomarkers (osteocalcin, beta-crosslaps, bone-specific alkaline phosphatase (BSAP), N-terminal propeptide of type 1 collagen (P1NP)).

    b. Efficacy outcomes:
    b1. Mobility: 6-minute walk test, 9-hole PEG test, range of motion of the shoulder
    b2. Pain assessment: Brief Pain Inventory BPI
    b3. Respiratory function: FEV1, FVC, MVV
    b4. Cardiac and vascular tests: echochardiogram and carotid intima media thickness
    b5. Audiology assessments: pure tone audiometry and whisper voice test
    b6. Ophthalmology assessments: corneal opacification, level of retinopathy and optic nerve involvement, intra-ocular pressure, and visual acuity
    b7. Questionnaires: EQ-5D-5L, Zarit caregiver burden, Fatigue Severity Scale

    c. Pharmacokinetic endpoints:
    c1. Odiparcil concentration in plasma and its metabolites.
    c2. Pre-dose samples will be collected to measure the odiparcil concentration remaining
    c3. Metabolite identification

    d. Pharmacodynamic endpoints:
    d1. GAG concentrations in urine and leukocytes isolated from peripheral blood
    d2. GAG content in skin biopsies
    d3. Anti-thrombin IIa activity in plasma
    d4. Thrombin generation assay (TGA)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Preliminary safety assessment
    V-1, V0, Va (D1), Vb (D7), Vc (D14)
    2. Core study
    a. Safety:
    a1. V-1, V0, V1, V2, V4, V5, V6, V7 and V8
    a2.1 V-1, V0, V2, V4, V7 and V8
    a2.2 V0 and V7

    b. Efficacy outcomes:
    b1. V-1, V0, V2, V4, V7 and V8
    b2. V0, V7
    b3. V-1, V0 and V7
    b4. V0 and V7
    b5. V0 and V7
    b6. V0 and V7
    b7. V-1, V0, V4 and V7

    c. Pharmacokinetic endpoints:
    c1. V2 (0, 0.5, 1, 2, 3, 4, 8, 12 h post dose)
    c2. V4 and V7
    c3. V2

    d. Pharmacodynamic endpoints
    d1. V-1, V0, V2, V4, V7 and V8
    d2. V6 and V7
    d3. V-1, V0, V2, V4, V7 and V8
    d4. V-1, V0, V1, V2, V3, V4, V5, V6, V7 and V8
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    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 Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    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. 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 Yes
    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 Yes
    E. trial design description
    Preliminary Safety assessment open-label. Core study 1st cohort double blind 2nd cohort open label.
    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 trial4
    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 EEA4
    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
    The end of the study is defined as completion of the last follow-up visit by the last patient
    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 months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    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: 10
    F.1.3Elderly (>=65 years) No
    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 20
    F.4.2.2In the whole clinical trial 20
    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)
    Given this is the first trial of odiparcil in MPS VI patients and given that the goal of the trial is to assess safety, it is difficult at this stage to commit to a compassionate protocol without having the results of the trial or at least early indications of the safety/efficacy of the drug.
    The sponsor commits to discuss with investigators, DSMB members, patient associations and regulatory authorities to provide as early as possible during the study a compassionate study protocol.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Society for Mucopolysaccharide Diseases
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-12-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-03
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-10-22
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