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    Summary
    EudraCT Number:2006-000839-10
    Sponsor's Protocol Code Number:PKU-008
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2006-07-13
    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 number2006-000839-10
    A.3Full title of the trial
    A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

    Estudio de extensión fase 3b, multicéntrico y abierto de PhenoptinTM en sujetos fenilcetonúricos que participaron en los estudios PKU-004 o PKU-006
    A.4.1Sponsor's protocol code numberPKU-008
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBioMarin Pharmaceutical Inc.
    B.1.3.4CountryUnited States
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 numberEMEA/OD/077/03
    D.3 Description of the IMP
    D.3.1Product namePhenoptin
    D.3.2Product code T1401
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNSapropterin
    D.3.9.1CAS number 69056-38-8
    D.3.9.2Current sponsor codeT1401
    D.3.9.3Other descriptive namesapropterin dihydrochloride
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number100
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    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 Yes
    D.3.11.13.1Other medicinal product typesynthetic drug product
    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
    Fenilcetonuria (PKU)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 8.1
    E.1.2Level LLT
    E.1.2Classification code 10034872
    E.1.2Term Phenylketonuria
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Este estudio tiene como objetivo valorar la seguridad del tratamiento a largo plazo con Phenoptin en sujetos con fenilcetonuria (PKU) que participaron en los estudios clínicos fase 3 sobre Phenoptin.
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study Information not present in EudraCT
    E.3Principal inclusion criteria
    • Hayan participado en el estudio PKU-004 o PKU-006.
    • Estén dispuestas y sean capaces de proporcionar consentimiento informado por escrito y firmado o, en el caso de sujetos menores de 18 años de edad, proporcionar un asentimiento por escrito (si se requiere) y un consentimiento informado firmado por uno de los padres o tutor legal, una vez que les sea explicada la naturaleza del estudio y antes de que se efectúe cualquier procedimiento relacionado con la investigación.
    • Presenten una prueba urinaria de embarazo negativa el día de la valoración de selección (pacientes femeninas en edad fértil).
    • Estén dispuestas a cumplir con los procedimientos del estudio y sean capaces de hacerlo.
    E.4Principal exclusion criteria
    • No hayan respondido a tratamientos anteriores con Phenoptin durante su participación en PKU-004 o PKU-006.
    • Sean consideradas como poco fiables o no disponibles para participar en el estudio; o, en el caso de menores de 18 años, tengan padres o tutores legales que sean considerados como poco fiables o no disponibles.
    • Hayan suspendido prematuramente su participación en PKU-004 o PKU-006, salvo en el caso de sujetos del estudio PKU-004 que pasaron al estudio PKU-008 en la semana 22, sujetos del estudio PKU-006 que pasaron al estudio PKU-008 en la semana 10, o sujetos del estudio PKU-006 que suspendieron su participación porque presentaban altas concentraciones de Phe después de los aumentos de Phe alimentaria.
    • Hayan recibido algún producto experimental aparte de Phenoptin los 30 días anteriores a la valoración de selección, o tengan previsto requerir algún tratamiento experimental antes de que finalicen todas las valoraciones planificadas para el estudio.
    • Hayan presentado una prueba urinaria de embarazo positiva en el momento de la selección (sólo en el caso de pacientes femeninas no estériles en edad fértil), se conozca que están embarazadas o amamantando, o estén planificando su embarazo (pacientes femeninas) o el de su pareja (pacientes masculinos).
    • Las pacientes femeninas en edad fértil deben practicar un método anticonceptivo eficaz, según lo determine el IP, y estar dispuestas a seguir utilizando medidas aceptables de control de la natalidad
    • Sufran una enfermedad o afección simultánea que interferiría en la participación o la seguridad del estudio (por ej., trastornos convulsivos, asma dependiente de esteroides orales u otra afección que requiera la administración de corticosteroides orales o parenterales, o diabetes insulinodependiente).
    • Se vean afectadas por cualquier circunstancia que, en la opinión del IP, las expongan a un alto riesgo si cumplen con el tratamiento y/o finalizan el estudio.
    • Tengan un valor de ALT > 2 veces mayor que el límite superior de normalidad (es decir, de grado 1 o superior según los criterios de toxicidad de la Organización Mundial de la Salud; apéndice 2) en la visita de selección.
    • Sufran una patología neuropsiquiátrica grave (por ej., depresión importante) que no esté bajo tratamiento médico actual.
    • Tengan antecedentes de trasplante de órganos.
    • Necesiten tratamiento concomitante con cualquier medicamento que haya demostrado inhibir la síntesis de folato (por ej., metotrexato).
    • Estén tomando levodopa.
    Tengan un diagnóstico clínico de deficiencia primaria de BH4.
    E.5 End points
    E.5.1Primary end point(s)
    Este estudio tiene como objetivo valorar la seguridad del tratamiento a largo plazo con Phenoptin en sujetos con fenilcetonuria (PKU) que participaron en los estudios clínicos fase 3 sobre Phenoptin.
    Se determinará la seguridad haciendo un seguimiento de las concentraciones sanguíneas de Phe y tabulando la incidencia y frecuencia de todos los AA y AAG que se presenten durante el estudio. Se valorará la seguridad en relación con la frecuencia y el tipo de AA, cambios clínicamente significativos en las constantes vitales o resultados de los reconocimientos médicos, y cambios clínicamente significativos en los resultados de los análisis de laboratorio (bioquímica, hematología, concentración sanguínea de Phe y análisis de orina).
    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 No
    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) 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 No
    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.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    Última visita del último paciente que este llevando a cabo el estudio.
    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 days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    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 Yes
    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
    En el caso de sujetos<18 años, estos otorgarán su consentimiento por escrito (si es requerido) y siempre tendrán que otorgar consentimiento escrito los padres o representantes legales del paciente.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 52
    F.4.2.2In the whole clinical trial 128
    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 Decision2006-10-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2006-08-11
    P. End of Trial
    P.End of Trial StatusOngoing
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