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    The EU Clinical Trials Register currently displays   43865   clinical trials with a EudraCT protocol, of which   7286   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2018-004140-28
    Sponsor's Protocol Code Number:D20180338
    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:2018-11-29
    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 number2018-004140-28
    A.3Full title of the trial
    NA
    « Administration de méthionine par voie orale ou entérale chez les patients atteints de protéinose alvéolaire primitive par mutations du gène MARS»
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA
    A.3.2Name or abbreviated title of the trial where available
    MetPAP
    A.4.1Sponsor's protocol code numberD20180338
    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-HOPITAUX 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 supportAP-HP
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASSISTANCE PUBLIQUE-HOPITAUX DE PARIS (AP-HP)
    B.5.2Functional name of contact pointDRCI Hôpital Saint 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.5.6E-mailsylvie.prieur@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 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 nameL-méthionine
    D.3.2Product code préparation magistrale
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPNasogastric use (Noncurrent)
    Gastric use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNL-METHIONINE
    D.3.9.1CAS number 63-68-3
    D.3.9.4EV Substance CodeSUB21984
    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
    NA
    Patients mineurs âgés de moins de 10 ans atteints de protéinose alvéolaire par double mutation Ala393Thr et Ser567Leu du gène MARS génétiquement prouvée.
    E.1.1.1Medical condition in easily understood language
    NA
    NA
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    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
    NA
    Evaluer la tolérance d’une supplémentation quotidienne prolongée en méthionine chez les patients atteints de protéinose alvéolaire primitive par mutations du gène MARS.
    E.2.2Secondary objectives of the trial
    NA
    Evaluer l’efficacité de 2 mois de supplémentation quotidienne en Méthionine sur :
    - l’état respiratoire clinique
    - l’aspect des lésions pulmonaires à la tomodensitométrie thoracique
    - la production de matériel lipo-protéinacé au LBA
    - l’état nutritionnel
    - l’atteinte hépatique
    - l’inflammation systémique
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    NA
    - Patient mineur âgé de moins de 10 ans atteint de protéinose alvéolaire par double mutation Ala393Thr et Ser567Leu du gène MARS génétiquement prouvée.
    - Patient nécessitant une hospitalisation prolongée à Necker pour programme de lavages broncho-alvéolaires (LBA) thérapeutiques dans le cadre du soin
    - Patient pour lequel la méthionine peut être administrée par voie orale ou par sonde entérale (sonde nasogastrique ou de gastrostomie)
    - Consentement signé par les titulaires de l’autorité parentale
    E.4Principal exclusion criteria
    NA
    - Patient atteint de protéinose alvéolaire par d’autres mutations du gène MARS
    - Patient atteint de protéinose alvéolaire secondaire à une autre étiologie ou sans cause identifiée
    - Refus de participation à l’étude
    - Hypertension artérielle nécessitant un traitement médicamenteux
    - Insuffisance cardiaque
    - Hypersensibilité connue à l’une des substances utilisées ou potentiellement utilisées pour l’étude : méthionine, vitamines B6, B12, B9 et C
    - Hyperméthioninémie préalable (méthioninémie > +2 DS de la normale pour l’âge) quelle qu’en soit la cause
    E.5 End points
    E.5.1Primary end point(s)
    NA
    La tolérance est appréciée par l’absence de survenue d’évènement indésirable entre J1 et M2,5.
    E.5.1.1Timepoint(s) of evaluation of this end point
    NA
    M2 = à 2 mois
    E.5.2Secondary end point(s)
    NA
    - Etat respiratoire : fréquence respiratoire, besoin en O2, signes de lutte respiratoires
    - Lésions pulmonaires : aspect des lésions élémentaires à la TDM thoracique, chacune scorée de 1 à 4
    - Matériel lipo-protéinacé : examen du liquide à chaque LBA
    - Etat nutritionnel : poids et rapport périmètre brachial / périmètre crânien
    - Atteinte hépatique : (i) Clinique : hépatomégalie, signes d’insuffisance hépatocellulaire, signes d’hypertension portale (ii) Biologique : cholestase et cytolyse hépatique, fonctions de synthèse et (iii) Echographique : taille du foie et de la rate, stéatose, parenchyme hépatique, circulation veineuse collatérale
    - Inflammation systémique : CRP, VS, IgG, anémie inflammatoire
    E.5.2.1Timepoint(s) of evaluation of this end point
    NA
    L’efficacité est évaluée selon les critères suivants :
    Etat Respiratoire : à l’inclusion, J15, J30, J45, J60, J75
    Lésions pulmonaires : aspect des lésions suivantes à la tomodensitométrie thoracique à l’inclusion et J60.
    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) 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 Yes
    E.8.1.7.1Other trial design description
    Non comparatif
    NA
    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 No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    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
    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 months2
    E.8.9.1In the Member State concerned days15
    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: 3
    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: 3
    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 No
    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 state3
    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)
    NA
    NA
    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 Decision2019-01-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-05
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-06-02
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