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    Summary
    EudraCT Number:2018-004191-35
    Sponsor's Protocol Code Number:GLYCO-1B
    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:2019-03-05
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2018-004191-35
    A.3Full title of the trial
    Evaluation of the safety and efficacy of administration of Empagliflozin in a new treatment for neutropenia in patients with Glycogen Storage Disease type 1b (GSD1b) and G6PC3 deficiency.
    Évaluation de la sécurité et de l'efficacité de l'administration d'Empagliflozine dans un nouveau traitement pour la neutropénie des patients atteints d’une Glycogénose de type 1b (GSD1b) et d’une déficience en G6PC3.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Use of an antidiabetic drug (Empagliflozin) to lower the blood level of 1,5-anhydroglucitol in patients deficient in the glucose-6-phosphate transporter (GSD1b) and the phosphatase G6PC3, both of the endoplasmic reticulum, to treat their recurrent infections by normalizing their blood neutrophil counts. Neutrophils are the most abundant white blood cells in our blood that are essential to help fighting infections. 1,5-anhydroglucitol is a sugar derivative with no known function.
    Evaluation de l’usage d’un antidiabétique (Empagliflozin) pour baisser le niveau de 1,5-anhydroglucitol du sang des patients déficients dans le transporteur du glucose-6-phosphate du réticulum endoplasmique (GSD1b) et de la phosphatase G6PC3 pour traiter leurs infections récurrentes en normalisant le nombre de leurs neutrophiles. Les neutrophiles sont des globules blancs permettant de luter contre les infections. Le 1,5-anhydroglucitol est un polyol sans fonction connue.
    A.3.2Name or abbreviated title of the trial where available
    GLYCO-1B
    A.4.1Sponsor's protocol code numberGLYCO-1B
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCliniques universitaires Saint-Luc
    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 supportCliniques universitaires Saint-Luc
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCliniques universitaires Saint-Luc
    B.5.2Functional name of contact pointgastroentérologie-hépatologie pedia
    B.5.3 Address:
    B.5.3.1Street AddressAvenue Hippocrate, 10
    B.5.3.2Town/ cityBruxelles
    B.5.3.3Post code1200
    B.5.3.4CountryBelgium
    B.5.4Telephone number3227641933
    B.5.6E-mailpcic-saintluc@uclouvain.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 Jardiance
    D.2.1.1.2Name of the Marketing Authorisation holderBoehringer Ingelheim International GmbH
    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.3.1Product nameEmpagliflozin
    D.3.2Product code A10BK03
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.11 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
    (1) Severe Congenital Neutropenia type 4 (SNC4) due to a deficiency in G6PC3, a phosphatase of the endoplasmic also known a Ubiquitous glucose-6-phosphatase and (2) the neutropenia in Glycogen Storage Disease type 1b due to a deficiency in the glucose-6-phosphate transporter (G6PT / SLC37A4) of the endoplasmic reticulum.
    (1) Neutropénie Sévère Congénitale de type 4 due à la déficience en G6PC3, une phosphatase du réticulum endoplasmique aussi connu comme la glucose-6-phosphatase ubiquiste et (2) la neutropénie dans la Glycogénose de type 1b due a une déficience dans le transporteur de glucose-6-phosphate (G6PT / SLC37A4) du réticulum endoplasmique.
    E.1.1.1Medical condition in easily understood language
    This study treats the neutropenia of patients deficient in glucose-6-phosphate transporter (GSD1b) and those that are deficient in G6PC3 and thus aims at reducing their number of infectious episodes.
    Cette étude traite la neutropénie des patients déficients en transporteur glucose-6-phosphate (GSD1b) et ceux déficients en G6PC3 et est centrée sur la baisse du nombre de leurs épisodes infectieux.
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Ubiquitous glucose-6-phosphatase deficiency (G6PC3) and glucose-6-phosphate transporter deficiency (G6PT / SLC37A4) both cause neutropenia. The purpose of our trial is to test the effect of the effectiveness of Empagliflozin on the urinary excretion and elimination of 1,5-anhydroglucitol from the blood in patients with G6PT deficiency (GSD Ib) and those who are deficient in G6PC3. This should allow patients to significantly lower the level of the toxic compound 1,5-anhydroglucitol-6-phosphate, that we found to accumulate in their neutrophils, and thereby treat their neutropenia. Empagliflozin (marketed in Belgium as Jardiance®) belongs to the class of drugs known as oral hypoglycemic agents. This project is the outcome of the work from researchers and doctors looking after these patients to translate the results from fundamental research, to be soon published in PNAS (Veiga-da-Cunha et al.), into a clinical trial that will very likely benefit the patients.
    Le déficit en glucose-6-phosphatase (G6PC3) et en transporteur de glucose-6-phosphate (G6PT / SLC37A4) du reticulum endoplasmique entraînent tous deux une neutropénie. Le but de notre essai clinique est de tester l'effet de l'efficacité de l'Empagliflozine sur l'excrétion urinaire et l'élimination du 1,5-anhydroglucitol du sang chez des patients présentant un déficit en G6PT (GSD Ib) ou en G6PC3. Cela devrait permettre aux patients de réduire considérablement le niveau du composé toxique, le 1,5-anhydroglucitol-6-phosphate, que nous avons constaté s’accumule dans leurs neutrophiles, et ainsi guérir leur neutropénie. L'empagliflozine (Jardiance®) appartient à la classe des médicaments appelés agents hypoglycémiants oraux. Ce projet est l'aboutissement de travaux de chercheurs et de médecins s'occupant de ces patients, à paraître dans PNAS (Veiga-da-Cunha et al.), le but est de les traduire en un essai clinique qui bénéficiera très probablement à ces patients.
    E.2.2Secondary objectives of the trial
    Not applicable
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Glycogenosis type 1b confirmed by biochemical analyzes and / or genetic analysis.
    - Alternatively, G6PC3 deficiency confirmed by genetic analysis
    - Informed consent signed by the recipient and / or parents / assigns.
    - Information and agreement of the referring medical team.
    -Critères d’inclusion
    - Glycogénose type 1b confirmée par des analyses biochimiques et/ou analyse génétique.
    - Alternativement, déficience en G6PC3 confirmée par analyse génétique
    - Consentement informé signé par le receveur et/ ou les parents / ayants droits.
    - Information et accord de l’équipe médicale référente.
    E.4Principal exclusion criteria
    - Presence of advanced fibrosis (Metavir F4) or cirrhosis.
    - Impossibility of long-term and / or non-compliance monitoring.
    - Other medical problems which, in the opinion of the physicians in charge of the patient, would constitute a contraindication to the procedure.
    - Présence d’une fibrose avancée (Metavir F4) ou de cirrhose.
    - Impossibilité d’un suivi à long terme et/ou non compliance.
    - Autres problèmes médicaux qui, selon l’avis des médecins en charge du patient, constitueraient une contre-indication à la procédure.
    E.5 End points
    E.5.1Primary end point(s)
    -Safety: absence of hypoglycaemia due to gliflozin treatment.
    -Efficacy: Increased neutrophil count
    -Sécurité : absence d’hypoglycémies dues au traitement par les gliflozines.
    -Efficacité : Augmentation du taux de neutrophiles
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 2, day 3, day 5, day 7, day 14, day 21, day 28, day 35, day 42, day 49, day 56
    Jour 2, jour 3, jour 5, jour 7, jour 14, jour 21, jour 28, jour 35, jour 42, jour 49, jour 56
    E.5.2Secondary end point(s)
    -Efficacy:
    -Decrease in the number of infections
    -Decrease in the number of episodes of oral aphtosis (stomatitis)
    -Decrease of blood 1,5-anhydroglucitol
    -Decrease in the level of 1,5-anhydroglucitol-6-phosphate in neutrophils
    -Increased excretion of urinary 1,5-anhydroglucitol
    -Improved neutrophilic function
    -Improved platelet function
    -Efficacité :
    -Diminution du nombre d’infections
    -Diminution du nombre d’épisodes d’aphtose buccale (stomatite)
    -Diminution du taux de 1,5-anhydroglucitol sanguin
    -Diminution du taux de 1,5-anhydroglucitol-6-phosphate dans les neutrophiles
    -Augmentation de l’excrétion de 1,5-anhydroglucitol urinaire
    -Amélioration de la fonction neutrophilique
    -Amélioration de la fonction plaquettaire
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 2, day 3, day 5, day 7, day 14, day 21, day 28, day 35, day 42, day 49, day 56
    Jour 2, jour 3, jour 5, jour 7, jour 14, jour 21, jour 28, jour 35, jour 42, jour 49, jour 56
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Our study concerns the repurposing of a drug already used for another indication (phase 4 equivalent)
    Notre étude concerne le « repurposing » d’un médicament déjà utilisé pour une autre indication (équivalent phase 4)
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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) 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 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
    LVLS
    dernier sujet dernière visite
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    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: 5
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    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 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 state5
    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)
    None
    Aucun
    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 Decision2019-04-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-12
    P. End of Trial
    P.End of Trial StatusCompleted
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