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    Summary
    EudraCT Number:2013-001479-18
    Sponsor's Protocol Code Number:GENiSIS2013
    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:2014-05-01
    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 number2013-001479-18
    A.3Full title of the trial
    A Phase III, Double Blinded, Randomised, Placebo Controlled Clinical Trial of High Dose Oral Genistein Aglycone in Patients with Sanfilippo Syndrome (Mucopolysaccharidosis III)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    High Dose Genistein in Sanfilippo Syndrome
    A.3.2Name or abbreviated title of the trial where available
    High Dose Genistein in Sanfilippo Syndrome
    A.4.1Sponsor's protocol code numberGENiSIS2013
    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 SponsorManchester University NHS Foundation Trust
    B.1.3.4CountryUnited Kingdom
    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 supportDSM Nutritionals
    B.4.2CountrySwitzerland
    B.4.1Name of organisation providing supportSociety for Mucopolysaccharide Diseases
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationManchester University NHS Foundation Trust
    B.5.2Functional name of contact pointLynne Webster
    B.5.3 Address:
    B.5.3.1Street AddressResearch Office, Manchester Royal Infirmary, Oxford Road
    B.5.3.2Town/ cityManchester
    B.5.3.3Post codeM13 9WL
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44 161 2764125
    B.5.6E-maillynne.webster@mft.nhs.uk
    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 nameGenistein aglycone
    D.3.2Product code 500 0500 0
    D.3.4Pharmaceutical form Oral powder
    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 INNGenistein aglycone
    D.3.9.1CAS number 446-72-0
    D.3.9.3Other descriptive nameGENISTEIN
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number98
    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 placeboOral powder
    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
    Sanfilippo syndrome (Mucopolysaccharidosis III)
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis III is a disorder marked by severe neurological symptoms and is caused by deficiencies of specific lysosomal enzymes
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10056918
    E.1.2Term Sanfilippo's syndrome
    E.1.2System Organ Class 100000004850
    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 this trial is to evaluate oral genistein aglycone therapy in patients between 2 and 15 years old with MPS III A, B or C
    E.2.2Secondary objectives of the trial
    Following 52 weeks of treatment are there changes in urinary GAG excretion; urine and plasma heparan sulphate; neuropsychological measures; actigraphy.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Written informed consent of a legally authorised guardian(s);
    • Aged between 2 and 15 years of age (inclusive), at the time of informed consent;
    • The patient must be able to walk unaided;
    • The patient must have confirmed MPS III A ,B or C with: a fibroblast or leukocyte HeparanNsulphatase (HNS) or Nalphaacetylglucosaminidase (NAGLU) or HeparanalphaglucosaminideNAcetyltransferase (HGSNAT) activity level
    of less than 10% of the lower limit of the normal range, or below the detection range of the measuring laboratory or have HNS or NAGLU;
    AND/OR
    • HGSNAT mutations known to be pathogenic;
    •A clinical diagnosis of MPS III.
    E.4Principal exclusion criteria
    • The patient has undergone bone marrow transplantation.
    • The patient is unable to participate in study activities in the opinion of the investigator.
    • The patient has a clinically significant organic disease (with the exception of symptoms relating to MPS III A or B or C) including: cardiovascular, hepatic, pulmonary, neurologic, or renal disease, other serious intercurrent illness, or
    extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival.
    • Patients that have had a change in psychotropic medication within one month of randomisation.
    • The patient receives any investigational medicinal product within 90 days prior to trial enrolment.
    • Patients should not have received genistein in any form for 30 days prior to enrolling into the study.
    • The patient has known hypersensitivity or adverse reactions to genistein.
    • A positive pregnancy test in a patient of childbearing potential.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint will be CSF heparan sulphate at 52 weeks, adjusted for baseline, in patients allocated to drug compared with patients allocated to placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    CSF samples will be collected at weeks 0, 52 and analysed. They will also be collected and analysed following the 52 week open label phase.
    E.5.2Secondary end point(s)
    Urinary GAG excretion at 52 weeks.
    Urinary and plasma heparan sulphate at 52 weeks.
    Neuropsychological measures, including the Vineland adaptive behaviour
    Questionnaire; Connors questionnaire; Bayley scale III; and MPS III symptom checklist at 52 weeks.
    Actigraphy at 52 weeks.
    E.5.2.1Timepoint(s) of evaluation of this end point
    These endpoints will be evaluated at 0 and 52 weeks, and following the 52 week open label phase
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    There is a 52 week open label period following the double blind component
    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 trial2
    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 years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 24
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 16
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    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 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
    Subjects are minors. Mucopolysaccharidosis III is a disorder marked by severe neurological symptoms and adolescents or children with this disorder would not be capable of providing informed consent or assent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state24
    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)
    If the trial is successful a submission will be made to the regulatory authorities for a marketing authorisation. The aim
    would be to submit this following the placebo controlled phase rather than open label. Supply of the drug cannot be
    guaranteed, but the aim would be to keep any gap in treatment to a minimum.
    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 Decision2014-06-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-04-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-06-30
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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