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    Summary
    EudraCT Number:2021-001351-13
    Sponsor's Protocol Code Number:APHP201133
    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:2021-09-09
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-001351-13
    A.3Full title of the trial
    Interest of famotidine in reducing endothelial expression of P-selectin in children with sickle cell disease: pilot study, single-center, prospective, non-comparative
    Intérêt de la famotidine pour diminuer l’expression endothéliale de P-sélectine chez les enfants drépanocytaires : étude pilote, monocentrique, prospective, non comparative
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Interest of famotidine in reducing endothelial expression of P-selectin in children with sickle cell disease: pilot study, single-center, prospective, non-comparative
    Intérêt de la famotidine pour diminuer l’expression endothéliale de P-sélectine chez les enfants drépanocytaires : étude pilote, monocentrique, prospective, non comparative
    A.3.2Name or abbreviated title of the trial where available
    FAMODREP
    FAMODREP
    A.4.1Sponsor's protocol code numberAPHP201133
    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 SponsorAssistance Publique des Hôpitaux de Paris(AP-HP)
    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 supportDGOS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAssistance Publique des Hôpitaux de Paris(AP-HP)
    B.5.2Functional name of contact pointDRCI, Hôpital St Louis,
    B.5.3 Address:
    B.5.3.1Street Address1 avenue Claude Vellefaux
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.4Telephone number33144 84 17 12
    B.5.5Fax number33144 84 17 01
    B.5.6E-mailalexandra.bruneau@aphp.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 Yes
    D.2.1.1.1Trade name FAMOTIDINE 40mg/5mL, powder for oral suspension
    D.2.1.1.2Name of the Marketing Authorisation holderLupin Pharmaceuticals , Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameFAMOTIDINE
    D.3.2Product code NDC 68180-150-01
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNFAMOTIDINE
    D.3.9.3Other descriptive nameFAMOTIDINE
    D.3.9.4EV Substance CodeSUB07503MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8
    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
    major sickle cell syndrome
    syndrome drépanocytaire majeur
    E.1.1.1Medical condition in easily understood language
    major sickle cell syndrome
    syndrome drépanocytaire majeur
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the effect of famotidine on P-selectin expression after 29 days of treatment.
    Evaluer l’effet de la famotidine sur l’expression de P-sélectine après 29 jours de traitement.
    E.2.2Secondary objectives of the trial
    - To assess the effect of famotidine on the expression of other endothelial activation markers after 29 days of treatment;
    - To assess the effect of famotidine on the biomarkers of hemolysis and inflammation after 29 days of treatment;
    - To assess the adverse effects of famotidine in a pediatric population suffering from sickle cell disease
    - Evaluer l’effet de la famotidine sur l’expression des autres marqueurs d’activation endothéliale après 29 jours de traitement ;
    - Evaluer l’effet de la famotidine sur les marqueurs biologiques d’hémolyse et d’inflammation après 29 jours de traitement ;
    - Evaluer les effets indésirables de la famotidine dans une population pédiatrique atteinte de drépanocytose
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - child or adolescent aged from 1 year to at most 17 years and 10 months, followed at the Necker-Enfants Malades Hospital for a major sickle cell syndrome SS or Sβ0;
    - having at least one vaso-occlusive crisis in the year prior to inclusion;
    - signed informed consent of the 2 parents or legal representative (s), and, oral and if possible signed consent of the child of expressive age or the adolescent;
    - beneficiary of social security coverage or entitled (excluding SMA)
    - enfant ou adolescent âgé de 1 an à au plus 17 ans et 10 mois, suivi à l’hôpital Necker-Enfants malades pour un syndrome drépanocytaire majeur SS ou Sβ0 ;
    - ayant au moins une crise vaso-occlusive au cours de l’année précédant l’inclusion ;
    - consentement éclairé signé des 2 parents ou représentant(s) légal(aux), et, consentement oral et si possible signé de l’enfant en âge de s’exprimer ou de l'adolescent ;
    - bénéficiaire d’une couverture sociale ou ayant droit (hors AME)
    E.4Principal exclusion criteria
    - treatment with crizanlizumab (anti-P-selectin antibody);
    - treatment with atazanavir / ritonavir in combination with tenofovir;
    - known hypersensitivity to famotidine or to other histamine type 2 (H2) receptor antagonists;
    - cardiovascular history such as: arrhythmia, AVB (atrioventricular block), QT prolongation;
    - Renal failure characterized by creatinine clearance <60 mL / min;
    - hepatic cytolysis (ALAT ≥ 3N)
    - neutropenia (<1 G / L), thrombocytopenia (<80 G / L), reticulopenia (<80 G / L);
    - predictable poor adherence to treatment;
    - participation in another interventional research involving the human person.
    - bone marrow transplant or gene therapy project within one month of inclusion

    Within 3 months prior to inclusion:
    - erythrocyte transfusion;
    - introduction of hydroxyurea or modification of the doses of hydroxyurea;
    - introduction of L-glutamine or modification of the doses of L-glutamine;
    - introduction of voxelotor or modification of voxelotor doses;
    - taking oral or IV corticosteroids or any other immunomodulatory treatment;
    - taking an antihistamine treatment.

    In the month preceding inclusion:
    - occurrence of a vaso-occlusive crisis, acute chest syndrome or any vaso-occlusive phenomenon (acute splenic sequestration, priapism, stroke, occlusion of the central retinal artery, papillary necrosis);
    - occurrence of fever (≥38 ° C) or any infectious episode, febrile or not, suspected or confirmed, of a viral, bacterial, fungal or parasitic nature;
    - occurrence of an acute hemolytic episode (increase in jaundice and pallor, decrease in hemoglobin ≥ 1g / dL compared to baseline hemoglobin, increase in LDH and / or AST and / or free bilirubin deemed significant by the child's referring physician).
    - traitement par crizanlizumab (anticorps anti-P-sélectine) ;
    - traitement par atazanavir/ritonavir en association avec le ténofovir ;
    - hypersensibilité connue à la famotidine ou à d'autres antagonistes des récepteurs histaminiques de type 2 (H2) ;
    - antécédent cardiovasculaire à type de : arythmie, BAV (bloc auriculoventriculaire), allongement du QT ;
    - Insuffisance rénale caractérisée par une clairance de la créatinine < 60 mL/min ;
    - cytolyse hépatique (ALAT ≥ 3N)
    - neutropénie (< 1 G/L), thrombopénie (< 80 G/L), réticulopénie (< 80 G/L) ;
    - mauvaise adhésion au traitement prévisible ;
    - participation à une autre recherche interventionnelle impliquant la personne humaine.
    - projet de greffe de moelle ou de thérapie génique dans le mois suivant l’inclusion

    Dans les 3 mois précédant l’inclusion :
    - transfusion érythrocytaire ;
    - introduction d’hydroxyurée ou modification des doses d’hydroxyurée ;
    - introduction de L-glutamine ou modification des doses de L-glutamine ;
    - introduction de voxelotor ou modification des doses de voxelotor ;
    - prise de corticoïdes oraux ou IV ou de tout autre traitement immunomodulateur ;
    - prise d’un traitement antihistaminique.

    Dans le mois précédant l’inclusion :
    - survenue d’une crise vaso-occlusive, d’un syndrome thoracique aigu ou de tout phénomène vaso-occlusif (séquestration splénique aigue, priapisme, accident vasculaire cérébral, occlusion de l’artère centrale de la rétine, nécrose papillaire) ;
    - survenue d’une fièvre (≥38°C) ou de tout épisode infectieux, fébrile ou non, suspecté ou confirmé, de nature virale, bactérienne, fongique ou parasitaire ;
    - survenue d’un épisode hémolytique aigu (majoration de l’ictère et de la pâleur, diminution de l’hémoglobine de ≥ 1g/dL par rapport à l’hémoglobine de base, augmentation des LDH et/ou des ASAT et/ou de la bilirubine libre jugée significative par le médecin référent de l’enfant).

    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint will be the difference in the plasma concentration of soluble P-selectin measured by ELISA technique (Human P-selectin / CD62P Quantikine ELISA kit, R&D) before and after 29 days of treatment with famotidine.
    Le critère d’évaluation principal sera la différence de concentration plasmatique de P-sélectine soluble mesurée par technique ELISA (Human P-selectin/CD62P Quantikine ELISA kit, R&D) avant et après 29 jours de traitement par famotidine.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Follow-up visit / end of treatment: D29
    Visite de suivi / fin de traitement : J29
    E.5.2Secondary end point(s)
    The secondary endpoints will be:
    - the differences in plasma concentration of soluble adhesion molecules E-selectin, VCAM-1, and ICAM-1 measured by ELISA technique (Human E-selectin / CD62E, Human sVCAM-1 / CD106 and Human ICAM-1 / CD54 Quantikine ELISA kits, R&D) before and after 29 days of treatment with famotidine.
    - The differences in blood values ​​of hemoglobin, reticulocytes, ASAT, free bilirubin, LDH, and CRP (measured in the hematology / hemostasis and biochemistry laboratories of the Necker-Enfants Malades hospital) before and after 29 days of treatment by famotidine.
    - Occurrence of serious or non-serious adverse event (s)
    Les critères d’évaluation secondaires seront :
    - les différences de concentration plasmatique des molécules d’adhérence solubles E-sélectine, VCAM-1, et ICAM-1 mesurées par technique ELISA (Human E-selectin/CD62E, Human sVCAM-1/CD106 et Human ICAM-1/CD54 Quantikine ELISA kits, R&D) avant et après 29 jours de traitement par famotidine.
    - Les différences de valeurs sanguines d’hémoglobine, réticulocytes, ASAT, bilirubine libre, LDH, et CRP (mesurées dans les laboratoires d’hématologie/hémostase et de biochimie de l’hôpital Necker-Enfants malades) avant et après 29 jours de traitement par famotidine.
    - Survenue d’événement(s) indésirable(s) grave(s) ou non grave(s)
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Inclusion visit: D0
    - Follow-up visit / end of treatment: D29
    - Phone call / end of research: D36
    - Visite d'inclusion: J0
    - Visite de suivi / fin du traitement : J29
    - Appel téléphonique/ fin de recherche : J36
    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) 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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months13
    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: 30
    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: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    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 state30
    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
    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 Decision2021-11-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-20
    P. End of Trial
    P.End of Trial StatusCompleted
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