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    Summary
    EudraCT Number:2012-000856-33
    Sponsor's Protocol Code Number:2012-40
    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:2014-05-15
    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 number2012-000856-33
    A.3Full title of the trial
    A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase cDNA in children with Sanfilippo type B syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Intracerebral Gene therapy in children with Sanfilippo type B syndrome
    A.4.1Sponsor's protocol code number2012-40
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN19853672
    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 SponsorInstitut Pasteur
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAssociation Française contre les Myopathies
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportInstitut Pasteur
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportVaincre les Maladies Lysosomales
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportAstellas
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInstitut Pasteur
    B.5.2Functional name of contact pointPIRC
    B.5.3 Address:
    B.5.3.1Street Address25-28 rue du Dr Roux
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75015
    B.5.3.4CountryFrance
    B.5.4Telephone number+33144389101
    B.5.5Fax number+33140613977
    B.5.6E-mailecsanfilippo@pasteur.fr
    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/3/11/917
    D.3 Description of the IMP
    D.3.1Product namerAAV2/5-hNaGlu
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntracerebral use
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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
    Mucopolysaccharidosis III B
    E.1.1.1Medical condition in easily understood language
    sanfilippo B syndrome
    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 17.0
    E.1.2Level PT
    E.1.2Classification code 10056890
    E.1.2Term Mucopolysaccharidosis III
    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
    to evaluate the clinical, radiological, biological tolerance associated to the proposed treatment
    E.2.2Secondary objectives of the trial
    To collect samples and data to define exploratory tests that could become evaluation criteria and further clinical efficacy studies (Brain MRI; neurological tests and biological markers)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Age: 18 months up to the 5th birthday;
    Onset of clinical manifestations related to MPSIIIB;
    NaGlu activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls;
    Patient affiliated to, or covered by a French social security regimen or European patients with European Health Insurance Card
    Family understanding the procedure and the informed consent;
    Signed informed consent by both parents or legal representative
    Vital laboratory parameters within normal range
    E.4Principal exclusion criteria
    Presence of brain atrophy on pre-inclusion MRI judged on a cortico-dural distance of more than 0.6 cm
    Any condition that would contraindicate general anaesthesia;
    Any other permanent medical condition not related to MPSIIIB that could contraindicate the study participation;
    No independent walking (Ability to walk without help);
    Any medication aiming at modifying the natural course of MPSIIIB given during the 6 months before vector injection (sleep and mood regulators are accepted)
    Any condition that would contraindicate treatment with Tacrolimus, Cellcept® and Solupred®/Solumedrol®.
    E.5 End points
    E.5.1Primary end point(s)
    Occurrence, intensity and relationship of all TEAEs. It will be presented overall by Prefered Term and System Organ Class (SOC).
    Occurrence, intensity and relationship of TEAEs per periods: 15 days post-surgery and during the 1 year follow-up in order to discriminate short- and mid-term safety.
    Occurrence and relationship of all TESAEs occurring during the study period.
    Occurrence, intensity and relationship of clinically significant abnormal haematology or biochemistry values (grade 3 or 4) occurring from immunosuppressant regimen start to the end of the study participation.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1year
    E.5.2Secondary end point(s)
    Endpoints related to the secondary objective of the protocol will show the accuracy of the data and samples collection and the ability of the children to be evaluated by the different neuropsychological tests that have been chosen.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1year
    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 No
    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) Yes
    E.7.1.1First administration to humans Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 No
    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
    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
    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: 4
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 4
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 4
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    patients are under 5 years old at inclusion. Sanfilippo type B disease induces severe mental deficiency when growing-up.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    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)
    An extension study will be proposed for the long term safety evaluation of the proposed treatment. Immunosuppressant treatment under tacrolimus will be extended. The initial duration of the extension phase will be of one year and will be repeated each year. Parents will sign a new informed consent before each extension phase.
    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 Decision2013-09-09
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-11-27
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