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    Summary
    EudraCT Number:2013-002702-30
    Sponsor's Protocol Code Number:ALXN1101-MCD-202
    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:2016-05-19
    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 ConcernedFrance - ANSM
    A.2EudraCT number2013-002702-30
    A.3Full title of the trial
    A PHASE 2/3, MULTICENTER, MULTINATIONAL, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALXN1101 IN NEONATES WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A
    Etude en ouvert de phase II/III, multicentrique et internationale, visant à évaluer l'efficacité et la sécurité d'emploi de l'ALXN1101 chez des nouveau-nés présentant un déficit en cofacteur molybdène (MoCoD) de type A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    STUDY OF ALXN1101 IN NEWBORNS WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A
    Etude de l'ALXN1101 chez des nouveau-nés présentant un deficit en cofacteur molybdène (MoCoD) de type A
    A.4.1Sponsor's protocol code numberALXN1101-MCD-202
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02629393
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/071/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAlexion Pharma GmbH
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAlexion Pharma GmbH
    B.4.2CountrySwitzerland
    B.4.1Name of organisation providing supportAlexion Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationALEXION EUROPE SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address1-15 Avenue Edouard Belin
    B.5.3.2Town/ cityRueil-Malmaison
    B.5.3.3Post code92500
    B.5.3.4CountryFrance
    B.5.4Telephone number+3314710 0606
    B.5.5Fax number+3314710 0611
    B.5.6E-mailclinicaltrials.eu@alxn.com
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/777
    D.3 Description of the IMP
    D.3.1Product nameCyclic Pyranopterin Monophosphate Monohydrobromide Dihydrate
    D.3.2Product code ALXN1101
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCyclic pyranopterin monophosphate monohydrobromide dihydrate
    D.3.9.2Current sponsor codeALXN1101
    D.3.9.3Other descriptive nameALXN1101
    D.3.9.4EV Substance CodeSUB125948
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.5
    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
    Molybdenum CoFactor Deficiency (MoCD) Type A
    Déficit en molybdène (MoCoD) de type A
    E.1.1.1Medical condition in easily understood language
    Molybdenum CoFactor Deficiency
    Déficit en molybdène
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10069687
    E.1.2Term Molybdenum cofactor deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and efficacy of ALXN1101 in neonate patients with MoCD Type A
    Evaluer l'efficacité et la sécurité d'emploi de l'ALXN1101 chez les nouveau-nés atteints de MoCoD de type A
    E.2.2Secondary objectives of the trial
    - To evaluate the effect of ALXN1101 on acquisition of developmental milestones
    - To evaluate the effect of ALXN1101 on pediatric measures of functional ability and activities of daily living
    - To characterize the pharmacokinetics (PK) of ALXN1101
    - Evaluer l'effet de l'ALXN1101 sur l'acquisition des étapes principales du développement
    - Evaluer l'effet de l'ALXN1101 sur les mesures pédiatriques des capacités fonctionnelles et des activités de la vie quotidienne
    - Caractériser la pharmacocinétique (PK) de l'ALXN1101
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female neonatal patients (1 to 28 days of age, [inclusive] at the time of ALXN1101 administration, with day 1 of age corresponding to the day of birth)
    - Diagnosis of MoCD Type A, based on:
     Prenatal genetic diagnosis, or
     Onset of clinical and/or laboratory signs and symptoms consistent with MoCD Type A within the first 28 days after birth
    - Parent or legal guardian must have signed the ICF prior to any study procedures being performed
    - Nouveau-né de sexe masculin ou féminin (âgé de 1 à 28 jours [inclus] au moment de l’administration de l’ALXN1101, le Jour 1 correspondant à son jour de naissance)
    - Diagnostic de MoCoD de type A reposant sur :
    Un diagnostic génétique prénatal ou
    L’apparition de signes et symptômes cliniques et/ou des données de laboratoire correspondant à un MoCoD de type A dans les 28 jours suivant la naissance
    - Le parent ou tuteur légal doit avoir signé le formulaire de consentement avant l’exécution de toute procédure de l’étude.
    E.4Principal exclusion criteria
    - Diagnosis other than MoCD Type A (may be determined after the initiation of study drug)
    - Condition that is considered by the treating physician to be a contraindication to therapy, including evidence of abnormalities on brain imaging not attributable to MoCD, or that might otherwise interfere with the patient’s participation in the study, pose any additional risk for the patient, or confound patient assessments
    - Antenatal and/or postnatal brain imaging prior to initiation of treatment with ALXN1101 that indicates cortical or subcortical cystic encephalomalacia, clinically significant intracranial hemorrhage, or other abnormalities on brain imaging determined by the treating physician to be clinically significant
    - Modified Gasgow Coma Scale (mGCS) for Infants and Children score of less than 7 for more than 24 hours (This criterion does not apply to children less than 1 day of age)
    - Un diagnostic autre qu’un MoCoD de type A (qui peut être déterminé après l’initiation du produit expérimental)
    - Une condition qui de l’avis du médecin traitant est une contre-indication au traitement, notamment les signes sur les images cérébrales d’anomalies non attribuables au MoCoD de type A, ou susceptibles sinon de perturber la participation des patients à l’étude, de représenter un risque supplémentaire pour le patient ou de fausser ses évaluations
    - Images cérébrales anténatales et/ou postnatales avant l’initiation du traitement par ALXN1101 indiquant une encéphalomalacie kystique corticale ou sous-corticale, hémorragie intracrânienne cliniquement significative ou autres anomalies à l’imagerie cérébrale que le médecin traitant aura déterminées comme étant cliniquement significatives
    - Un score de Glasgow modifié [mGCS (modified Glasgow Coma Scale)] des nourrissons et des enfants inférieur à 7 pendant plus de 24 heures (ce critère ne s’applique pas aux enfants âgés de moins d’un jour)
    E.5 End points
    E.5.1Primary end point(s)
    Response, defined as patients alive and able to sit upright independently for at least 30 seconds
    Réponse, définie par des patients toujours en vie et en mesure de rester assis bien droit sans assistance pendant au moins 30 secondes
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 12
    Mois 12
    E.5.2Secondary end point(s)
    - Bayley Scales of Infant Development®- Third Edition (Bayley - III®) Cognitive and Motor Scales as measured
    - Functional ability and activities of daily living, measured by the Pediatric Evaluation of Disability Inventory (PEDI)
    - PK parameters
    - Safety
    - Test de Bayley (Bayley Scales of Infant Development® – Troisième édition [Bayley – III]) Évaluations sur le développement cognitif et moteur mesurées
    - Capacité fonctionnelle et activités de la vie quotidienne, mesurées par l’évaluation pédiatrique de l’inventaire d’invalidité (Pediatric Evaluation of Disability Inventory - PEDI)
    - parameter pharmacocinetique
    - Sécurité
    E.5.2.1Timepoint(s) of evaluation of this end point
    through Month 12
    jusqu'au mois 12
    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 Yes
    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) Yes
    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 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 Yes
    E.8.5.1Number of sites anticipated in the EEA12
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Germany
    Israel
    Italy
    Malaysia
    Netherlands
    Saudi Arabia
    Spain
    Taiwan
    Tunisia
    Turkey
    United Kingdom
    United States
    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 years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial 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: 10
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 10
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    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 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
    Newborns to be enrolled. The parent or legal guardian will provide informed consent
    Des nouveau-nés seront inclus dans l'étude. Le parent ou le tuteur legal donnera son consentement
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 10
    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)
    After the 12 month evaluation point, the subject will enter in the 2 years extension treatment period
    Suite aux critères d'évaluations au douxième mois, le sujet entrera dans une période d'extension de 2ans
    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 Decision2016-05-17
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-09-13
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