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    Summary
    EudraCT Number:2011-004104-38
    Sponsor's Protocol Code Number:ARPA-IGF1-2011
    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:2012-01-19
    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 number2011-004104-38
    A.3Full title of the trial
    ESTUDIO ALEATORIZADO, DOBLE CIEGO, CONTROLADO CON PLACEBO, DE EFICACIA, TOLERANCIA Y SEGURIDAD DEL TRATAMIENTO CON EL FACTOR TRÓFICO IGF-I EN LA ATAXIA DE FRIEDREICH
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    ESTUDIO ALEATORIZADO, DOBLE CIEGO, CONTROLADO CON PLACEBO, DE EFICACIA, TOLERANCIA Y SEGURIDAD DEL TRATAMIENTO CON EL FACTOR TRÓFICO IGF-I EN LA ATAXIA DE FRIEDREICH
    A.4.1Sponsor's protocol code numberARPA-IGF1-2011
    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 SponsorFIBHULP
    B.1.3.4CountrySpain
    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 supportMinisterio de Sanidad, Política Social e Igualdad
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFIBHULP
    B.5.2Functional name of contact pointFIBHULP
    B.5.3 Address:
    B.5.3.1Street AddressPaseo de la Castellana, 261
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28046
    B.5.3.4CountrySpain
    B.5.4Telephone number34917277576
    B.5.5Fax number34912071061
    B.5.6E-mailfundacion.hulp@salud.madrid.org
    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 INCRELEX 10 mg/ml solución inyectable
    D.2.1.1.2Name of the Marketing Authorisation holderIpsen Pharma
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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/3/06/373
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMECASERMIN
    D.3.9.1CAS number 68562-41-4
    D.3.9.4EV Substance CodeSUB08672MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.05
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pacientes con ataxia de Friedreich
    E.1.1.1Medical condition in easily understood language
    Pacientes con ataxia de Friedreich
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10017374
    E.1.2Term Friedreich's ataxia
    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
    Objetivos principales:
    • Determinar la eficacia de IGF-1 en pacientes con ataxia de Friedreich de grado leve a moderado, medida mediante la escala SARA.
    • Determinar los cambios en los niveles en sangre de IGF-1 y los niveles de frataxina en pacientes con ataxia de Friedreich de grado leve a moderado, antes y después de iniciado el tratamiento.
    E.2.2Secondary objectives of the trial
    Objetivos secundarios:
    • Ecocardiograma.
    • Valoración neuro-oftalmológica.
    • Potenciales evocados visuales.
    • Examen otoneurológico.
    • RM cerebral y cervical.
    • Determinar los cambios en los niveles en sangre de IGF-1 en pacientes con ataxia de Friedreich de grado leve a moderado, antes y después de iniciado el tratamiento.
    Objetivos de seguridad y tolerancia:
    • Hemograma
    • Bioquímica General
    • Marcadores tumorales
    • ECG
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Criterios de inclusión:
    - Edad igual o mayor de 14 años y menor de 85 años.
    - Pacientes con ataxia de Friedreich con puntuaciones entre 8 y 32 puntos en la escala SARA y una puntuación igual o menor de 7 puntos en el item 1 de la escala SARA.
    - Pacientes con y sin miocardiopatía.
    - Pueden ser incluidos pacientes en tratamiento estable con idebenona y/o riboflavina y/o deferiprona o darbepoetina durante al menos 1 año.
    - Haber dado su consentimiento informado.
    E.4Principal exclusion criteria
    CUALQUIERA de los siguientes criterios excluirá a un paciente de este estudio:
    - Edad menor de 14 ó mayor de 85 años.
    - Puntuación en la escala SARA <8 y >32 puntos.
    - Fuerza muscular alterada a nivel proximal de las extremidades inferiores en un grado 4 puntos en la Escala Neurológica Gradual.
    - Función sensitiva (modalidades primarias + estereognosia) alterada en un grado de 9 en la Escala Neurológica Gradual.
    - Enfermedad neurológica significativa que pueda afectar la cognición: demencias, síndrome de Down.
    - Presencia actual de trastorno o síntoma (por ejemplo, alucinaciones) psiquiátrico mayor clínicamente significativo, de acuerdo con los criterios del Diagnostic and Statistical Manual of Mental Disorders, Cuarta Edición (DSM-IV), que pueda afectar a la capacidad del paciente para completar el estudio.
    - Enfermedad sistémica o síntoma (por ejemplo, enfermedad cardiovascular, enfermedad renal con creatinina > 2,0 mg/dL) actual clínicamente significativa que pueda deteriorar o afectar la seguridad del paciente o su capacidad de cooperar con los exámenes durante el estudio.
    - Historia de ictus clínicamente evidente.
    - Infarto de miocardio dentro de los 2 últimos años.
    - Historia de neoplasia, incluyendo melanoma, dentro de los últimos 5 años, con la excepción del carcinoma cutáneo de células basales no metastásico y el carcinoma in-situ de cérvix.
    - Otra anomalía clínicamente significativa en el examen físico, neurológico o de laboratorio o en el electrocardiograma (ECG) que, en opinión del Investigador, descarte al paciente del estudio.
    - Encontrarse gestante o amamantando.
    - Historia de dependencia del alcohol o de drogas dentro de los últimos 2 años.
    - Exposición a tóxicos industriales y ambientales: mercurio, tolueno, plomo, monóxido de carbono, bromuro y talio.
    - Uso de ciertos medicamentos: fenitoína, litio, citarabina.
    - Síndrome carencial: vitaminas E, B12 y B1.
    - Historia o presencia de cualquier enfermedad autoinmune o de trastornos de deficiencia inmune significativa.
    - Afecciones inflamatorias: Enfermedad celiaca, enfermedad de Behçet, lupus eritematoso diseminado, enfermedad de Wegener, anticuerpos antiglutamato decarboxilasa (anti-GAD), y anticuerpos antirreceptor del glutamato, y anticuerpos antigliadina.
    - Sospecha de afección paraneoplásica (atrofias cerebelosas paraneoplásicas asociadas o no a anticuerpos antineuronales).
    - Afectación infecciosa y postinfecciosa recientes por los agentes habituales (virus, bacterias, parásitos).
    - Presencia de hipotiroidismo no compensado, atrofia cruzada, hemosiderosis marginal, síndrome de la persona rígida y degeneración hepatocerebral y antecedente de hipertermia maligna.
    - Haber usado un fármaco en ensayo clínico dentro de los 30 días previos a la visita basal.
    - Mujeres fértiles, salvo que estén utilizando un método anticonceptivo de probada eficacia.
    - Pacientes en los que no se puede obtener una RM cerebral y medular
    E.5 End points
    E.5.1Primary end point(s)
    Evaluación neurológica (eficacia). Escala SARA (Scale for the Assessment and Rating of Ataxia).
    Otras pruebas son: 9HPT, Click test y Writing test
    E.5.1.1Timepoint(s) of evaluation of this end point
    Mes 12
    E.5.2Secondary end point(s)
    Análisis de la expresión de frataxina, basal y trimestralmente
    Niveles de IGF-1 en sangre basal.
    Variables de seguridad: Exploración física completa en todas las visitas, constantes vitales, altura peso y determinaciones analíticas en cada visita.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Frataxina, trimestralmente, IGF-1 en sangre basal y variables de seguridad en cada visita. Hasta los 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 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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
    El final del estudio se produce en la visita del mes 12 salvo aquellos pacientes que tengan que retirarse prematuramente, en cuyo caso, deberán completar la visita de retirada prematura en los 7 días siguientes a la administración de la última dosis.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned 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: 5
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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)
    Dadas las características de estos pacientes y las escasas alternativas terapéuticas existentes se mantendría tratamiento por uso compasivo
    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 Decision2012-02-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-07-17
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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