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    Summary
    EudraCT Number:2013-002702-30
    Sponsor's Protocol Code Number:ALXN1101-MCD-202
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2016-04-20
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2013-002702-30
    A.3Full title of the trial
    A PHASE 2/3, MULTICENTER, MULTICENTER, MULTINATIONAL, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALX1101 IN NEONATES WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A
    Estudio abierto, multicéntrico y multinacional, de fase II/III, para evaluar la eficacia y la seguridad de ALXN1101 en recién nacidos con déficit del cofactor molibdeno (DCMo) de tipo 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
    A.4.1Sponsor's protocol code numberALXN1101-MCD-202
    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 del cofactor molibdeno (DCMo) de tipo A
    E.1.1.1Medical condition in easily understood language
    Molybdenum CoFactor Deficiency
    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
    Evaluar la eficacia y la seguridad de ALXN1101 en pacientes neonatos con DCMo de tipo 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
    -Evaluar el efecto de ALXN1101 en la adquisición de hitos en el desarrollo
    -Evaluar el efecto de ALXN1101 en medidas pediátricas de la capacidad funcional y de las actividades de la vida cotidiana
    -Caracterizar la farmacocinética (FC) de ALXN1101
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female neonatal patient (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 informed consent form (ICF) prior to any study procedures being performed
    -Pacientes neonatos de ambos sexos (de 1 a 28 días de edad [inclusive] en el momento de la administración de ALXN1101, donde el día 1 se corresponde con la fecha de nacimiento).
    -Diagnóstico de DCMo de tipo A, en base a
    o Diagnósticos genéticos prenatales, o
    o Aparición de signos y síntomas clínicos y/o analíticos coherentes con el DCMo de tipo A
    en los primeros 28 días tras el nacimiento
    - El progenitor o tutor legal debe haber firmado el formulario de consentimiento informado (FCI) antes de llevar a cabo cualquier procedimiento del estudio
    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 Type A, 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 Glasgow 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)
    -Diagnóstico diferente a DCMo de tipo A (puede determinarse después del inicio del fármaco del estudio).
    -Trastorno que el médico responsable considere como una contraindicación al tratamiento, incluidas evidencias de anomalías en pruebas por imagen del cerebro no atribuibles al DCMo de tipo A, o que pueda interferir con la participación del paciente en el estudio, conllevar un riesgo adicional para el paciente o confundir sus evaluaciones.
    -Pruebas por imagen del cerebro prenatales y/o posnatales previas al inicio del tratamiento con ALXN1101 que indican encefalomalacia quística cortical o subcortical, hemorragia intracraneal clínicamente significativa u otras anomalías en las pruebas por imagen del cerebro determinadas por el médico responsable del tratamiento como clínicamente significativas.
    -Escala de coma de Glasgow modificada (modified Glasgow Coma Scale, mGCS) para lactantes y niños con una puntuación inferior a 7 durante más de 24 horas (este criterio no se aplica a niños menores de 1 día de edad).
    E.5 End points
    E.5.1Primary end point(s)
    Response, defined as patients being alive and able to sit upright independently for at least 30 seconds
    Respuesta, definida como un paciente que ha sobrevivido y que puede sentarse erguido de forma autónoma durante al menos 30 segundos en el mes 12
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 12
    12 meses
    E.5.2Secondary end point(s)
    - Bayley Scales of Infant Development® ? Third Edition (Bayley ? III®) Cognitive and Motor Scales as measured through Month 12
    - Functional ability and activities of daily living, measured by the Pediatric Evaluation of Disability Inventory (PEDI) through Month 12
    -Escalas de Bayley del desarrollo infantil®, tercera edición (Bayley – III) Escalas de la función cognitiva y motora según las mediciones hasta el mes 12
    -Capacidad funcional y actividades de la vida cotidiana, medidas por la Evaluación pediátrica del inventario de discapacidad (Pediatric Evaluation of Disability Inventory, PEDI) hasta el mes 12
    E.5.2.1Timepoint(s) of evaluation of this end point
    Month 12
    12 meses
    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
    France
    Germany
    Israel
    Italy
    Malaysia
    Netherlands
    Saudi Arabia
    Taiwan
    Tunisia
    Turkey
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    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
    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 Decision2016-05-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-26
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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