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    Summary
    EudraCT Number:2012-000814-13
    Sponsor's Protocol Code Number:ML7981
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-02-17
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2012-000814-13
    A.3Full title of the trial
    Glibenclamide treatment in hypotonia-cystinuria syndrome
    Glibenclamide ter behandeling van hypotonie-cystinurie syndroom
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Glibenclamide treatment in hypotonia-cystinuria syndrome
    Glibenclamide ter behandeling van hypotonie-cystinurie syndroom
    A.3.2Name or abbreviated title of the trial where available
    Glibenclamide treatment in hypotonia-cystinuria syndrome
    Glibenclamide ter behandeling van hypotonie-cystinurie syndroom
    A.4.1Sponsor's protocol code numberML7981
    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 SponsorUniversity Hospital Leuven
    B.1.3.4CountryBelgium
    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 supportUniversity Hospital Leuven
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Hospital Leuven
    B.5.2Functional name of contact pointHCS trial
    B.5.3 Address:
    B.5.3.1Street AddressHerestraat 49
    B.5.3.2Town/ cityLeuven
    B.5.3.3Post code3000
    B.5.3.4CountryBelgium
    B.5.4Telephone number003216343820
    B.5.5Fax number003216343820
    B.5.6E-mailluc.regal@uzleuven.be
    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 Yes
    D.2.1.1.1Trade name Daonil
    D.2.1.1.2Name of the Marketing Authorisation holderAventis Pharma SA
    D.2.1.2Country which granted the Marketing AuthorisationBelgium
    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.3.4Pharmaceutical form Capsule
    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 INNGLIBENCLAMIDE
    D.3.9.1CAS number 10238-21-8
    D.3.9.4EV Substance CodeSUB07916MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.0125 to 0.2
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hypotonia-cystinuria syndrome/PREPL deficiency
    Hypotonie-cystinurie syndroom/PREPL deficiëntie
    E.1.1.1Medical condition in easily understood language
    Hypotonia-cystinuria syndrome/PREPL deficiency
    Hypotonie-cystinurie syndroom/PREPL deficiëntie
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluation of the effect of glibenclamide on hypotonia in hypotonia-cystinuria syndrome/PREPL deficiency. Evaluation of the effect glibenclamide on growth hormone secretion in hypotonia-cystinuria syndrome/PREPL deficiency.
    Evaluatie van het effect van glibenclamide op hypotonie bij hypotonie-cystinurie syndroom/PREPL deficiëntie. Evaluatie van het effect van glibenclamide op groeihormoon secretie bij hypotonie-cystinurie syndroom/PREPL deficiëntie.
    E.2.2Secondary objectives of the trial
    no
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Diagnosis of hypotonia-cystinuria syndrome/PREPL deficiency, confirmed by molecular genetic analysis.
    Age <16y.
    Written informed consent.
    Diagnose van hypotonie-cystinurie syndroom/PREPL deficiëntie, genetisch bevestigd.
    Leeftijd onder 16 jaar
    Geschreven geïnformeerde instemming.
    E.4Principal exclusion criteria
    Intolerance for glibenclamide.
    History of hyperinsulinism or other contraindications for glibenclamide.
    Intolerantie voor glibenclamide
    Voorgeschiedenis van hyperinsulinisme of andere contra-indicaties voor glibenclamide
    E.5 End points
    E.5.1Primary end point(s)
    Evolution of hypotonia (6min walk test, maximal inspiratory and exspiratory pressure, manual newtonmetry, MG-ADL, MG composite, AIMS score) after start of glibenclamide. If improvement is seen: evolution 2days after stop of optimal dose of glibenclamide.
    Evolution of glycemia during 24h, IGF1 after 24h, IGFBP3 after 24h treatment, GH first 4hours, insulin first 4 hours
    Evolutie van hypotonie (6min walk test, maximale inspiratoire en exspiratoire druk, manuele newtonmetrie, MG-ADL, MG composite, AIMS score) na start van glibenclamide. Indien verbetering gezien wordt: evolutie 2 d na stop van optimale dosis van glibenclamide.
    Evolutie van glycemie gedurende 24u, IGF1 na 1d, IGFBP na 1d, groeihormoon de eerste 4u, insuline de eerste 4u.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Hypotonia evaluation: 4 hours after intake of glibenclamide
    glycemia: every 30 min during first 4h, afterwards every 1h
    GH and insulin every 30 min (4h)
    Hypotonie evaluatie: 4u na inname van glibenclamide
    Glycemie:om de 30 min gedurende 4u, daarna elk uur tot 24u na inname. Groeihormoon en insuline elk half uur gedurende 4 uur.
    E.5.2Secondary end point(s)
    no
    E.5.2.1Timepoint(s) of evaluation of this end point
    no
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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.8.3 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 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
    If no effect on hypotonia/growth hormone is seen in the first 3 patients with a dose of glibenclamide low enough not to cause hypoglycemia.
    Otherwise: last visit of last subject undergoing the trial
    Indien geen effect op hypotonie/groeihormoon gezien wordt bij de eerste drie patiënten bij de laagste dosis glibenclamide die geen hypoglycemie veroorzaakt.
    Anders: laatste visite van laatste patiënt in de 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 months6
    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: 8
    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: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    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 treatment is effective on hypotonia and/or growth hormone deficiency and well tolerated (no hypoglycemia), it will be continued. A capillary glucometer and guidelines for action in case of hypoglycemia will be provided in that case.
    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 Decision2012-03-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-04-20
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