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    Summary
    EudraCT Number:2018-000338-36
    Sponsor's Protocol Code Number:IEDAT-03-2018
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-04-05
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2018-000338-36
    A.3Full title of the trial
    Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
    Tratamiento de extensión a largo plazo y sin enmascaramiento usando fosfato sódico de dexametasona intraeritrocitaria en pacientes con ataxia telangiectasia que participaron en el estudio IEDAT-02-2015
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Not Applicable
    No aplicable
    A.3.2Name or abbreviated title of the trial where available
    OLE-IEDAT
    OLE-IEDAT
    A.4.1Sponsor's protocol code numberIEDAT-03-2018
    A.5.4Other Identifiers
    Name:US INDNumber:115929
    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 SponsorEryDel S.p.A.
    B.1.3.4CountryItaly
    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 supportEryDel S.p.A.
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDr. Guenter Janhofer, MD at EryDel S.p.A.
    B.5.2Functional name of contact pointCHIEF MEDICAL OFFICER
    B.5.3 Address:
    B.5.3.1Street AddressVia A. Meucci, 3
    B.5.3.2Town/ cityBresso (MI)
    B.5.3.3Post code20091
    B.5.3.4CountryItaly
    B.5.4Telephone number+391 484 350 0708
    B.5.6E-mailGuenter.Janhofer@erydel.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 numberEMA/COMP/306983/2013
    D.3 Description of the IMP
    D.3.1Product nameDexamethasone sodium phosphate
    D.3.4Pharmaceutical form Solution for blood fraction modification
    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 INNDexamethasone sodium phosphate
    D.3.9.1CAS number 2392-39-4
    D.3.9.2Current sponsor codeDSP
    D.3.9.3Other descriptive nameDEXAMETHASONE SODIUM PHOSPHATE PH. EUR.
    D.3.9.4EV Substance CodeSUB174329
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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 Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeThe IMP refers to dexamethasone sodium phosphate (DSP) for encapsulation into human autologous erythrocytes. DSP is formulated at 25 mg/mL in WFI to produce the drug product, DSP Solution.
    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
    Patient with neurological symptoms of Ataxia Telangiectasia
    Pacientes con síntomas neurológicos de ataxia telangiectasia
    E.1.1.1Medical condition in easily understood language
    Patient with neurological symptoms of Ataxia Telangiectasia
    Pacientes con síntomas neurológicos de ataxia telangiectasia
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10003594
    E.1.2Term Ataxia telangiectasia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.
    •To evaluate the long term effect of EDS-EP in treating CNS symptoms as measured by the “Modified” International Cooperative Ataxia Rating Scale (mICARS), and Clinical Global Impression of severity and change (CGI-S/C)
    •Seguir y evaluar la seguridad a largo plazo y la tolerabilidad del EDS-EP en pacientes con AT.
    •Evaluar el efecto a largo plazo del EDS-EP en el tratamiento de los síntomas del SNC medidos con la escala cooperativa internacional para la cuantificación de la ataxia “modificada” (ICARSm) y la impresión global clínica de la gravedad y el cambio (CGI-S/C).
    E.2.2Secondary objectives of the trial
    To evaluate the long-term effect of EDS-EP on health related Quality of Life (QoL; EQ-5D-5L scale).
    •Evaluar el efecto a largo plazo del EDS-EP en la calidad de vida (Quality of Life, QoL) relacionada con la salud mediante la escala EQ-5D-5L.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Patient completed the double-blind period in the IEDAT-02-2015 trial and must have completed the final (Visit 15/Month 12) efficacy assessments of IEDAT-02-2015.
    2.Patient tolerated the study medication, without any evidence of steroid adverse events, or treatment-related severe/ serious adverse events.
    3.Body weight > 15 kg.
    4.The patient and his/her parent/caregiver (if below the age of consent), or a legal representative, has provided written informed consent to participate. If consent is provided solely by the caregiver in accordance with local regulations, the patient must provide assent to participate in the study.
    5.Patient benefitted from treatment with the study medication, or did not worsen significantly, as determined by the Principal Investigator (PI) according to the procedures described below.

    Procedure for selecting patients for further treatment in IEDAT-03-2018
    •The Principal Investigator will ask all patients who meet the above requirements, and determine their interest in continuing to receive treatment with the study medication in a new protocol. This information will be submitted to the CRO, where an individual will access the study randomization and determine that patient shows evidence of disease stabilization or improvement, as determined by the ratings of the CGI-C.
    The CRO will provide the decision on the patient’s eligibility to the PI, within 2 weeks of receipt of the request, without indicating the previous treatment, or the criteria used to determine eligibility.
    1.El paciente completó el período de doble enmascaramiento en el ensayo IEDAT-02-2015 y tiene que haber completado las evaluaciones finales (Visita 15 / Mes 12) de eficacia del IEDAT-02-2015.
    2.El paciente toleró el fármaco investigado sin ningún indicio de acontecimientos adversos relacionados con esteroides ni acontecimientos adversos intensos o graves relacionados con el tratamiento.
    3.Peso corporal > 15 kg
    4.El paciente y su padre/madre/cuidador (si es menor de edad para otorgar el consentimiento), o un representante legal, han proporcionado el consentimiento informado por escrito para participar. Si solamente el cuidador proporciona el consentimiento de conformidad con las regulaciones locales, el paciente debe proporcionar el asentimiento para participar en el estudio.
    5.El paciente mejoró al ser tratado con el fármaco investigado o no empeoró significativamente, según determinó el investigador principal (IP) siguiendo los procedimientos descritos a continuación.

    Procedimiento para seleccionar pacientes para el tratamiento adicional en IEDAT-03-2018
    •El investigador principal preguntará a los pacientes que cumplan los requisitos anteriores si les interesa continuar tratándose con el fármaco investigado en un nuevo protocolo. Esta información se enviará a la OIC, donde una persona con acceso a la aleatorización del estudio determinará si el paciente presenta indicios de estabilización de la enfermedad o mejoría, según las clasificaciones de la CGI-C.
    La OIC comunicará al IP la decisión acerca de la elegibilidad del paciente en el plazo de 2 semanas tras la recepción de la petición, sin indicar cuál era el tratamiento previo ni los criterios usados para determinar la elegibilidad.
    E.4Principal exclusion criteria
    1.Females that are
    a.pregnant, or are breast-feeding (for EU countries only);
    b.of childbearing potential, pregnant, or are breast-feeding (for US and Rest of World countries).
    Females of childbearing potential using adequate birth control, as determined by their Health Care Provider, will be eligible.
    2.A disability that may prevent the patient from completing all study requirements.
    3.Current participation in a clinical study with another investigational drug.
    Medical History and Current Status
    4.CD4+ lymphocytes count <400/mm3 (for patients 6 years of age) or <200/mm3 (for patients >6 years).
    5.Current neoplastic disease.
    6.Severe impairment of the immunological system.
    7.Severe or unstable pulmonary disease.
    8.Uncontrolled diabetes.
    Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible.
    9.Any other severe, unstable, or serious disease or condition that in the Investigator’s opinion would put the patient at risk for imminent life-threatening morbidity, need for hospitalization, or mortality.
    10.Eligibility of patients with abnormal laboratory test values will be determined by the Investigator.
    11.Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or thalassemia.
    12.Moderate or severe renal and/or hepatic impairment.
    13.Patients who experienced moderate/ severe steroid side effects, or moderate/ severe adverse events associated with the study medication administered in the IEDAT-02 study.
    Prior/Concomitant Medication
    14.Requires treatment with an oral or parenteral steroid. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted.
    15.Requires any other concomitant medication prohibited by the protocol.
    16.Use of any drug that is a strong inducer/inhibitor of CYP3A4.
    1.Mujeres que estén
    a.embarazadas o en período de lactancia (solo para los países de la UE);
    b.en edad fértil, embarazadas o en período de lactancia (para EE. UU. y el resto de países).
    Las mujeres en edad fértil que usen un método anticonceptivo adecuado, según lo determinado por el profesional sanitario que las atienda, serán elegibles.
    2.Una discapacidad que pueda impedir que el paciente complete todos los requisitos del estudio.
    3.Participación actual en otro estudio clínico con otro fármaco en investigación.
    Antecedentes médicos y condición actual
    4.Recuento de linfocitos CD4+ <400/mm3 (para pacientes de 6 años de edad) o <200/mm3 (para pacientes >6 años).
    5.Padece actualmente una enfermedad neoplásica.
    6.Afectación grave del sistema inmunitario.
    7.Enfermedad pulmonar grave o inestable.
    8.Diabetes no controlada.
    Los pacientes con diabetes estabilizada (es decir, con ausencia de episodios hipoglucémicos o hiperglucémicos en los últimos 3 meses) serán elegibles.
    9.Cualquier otra enfermedad o afección grave, inestable o seria que, a criterio del investigador, pueda poner al paciente en riesgo de presentar una morbilidad inminente que represente un riesgo para la vida, de necesitar hospitalización o de mortalidad.
    10.La elegibilidad de los pacientes con valores anómalos en las pruebas analíticas estará determinada por el investigador.
    11.Hemoglobinopatías confirmadas, p. ej., hemoglobinopatía C, anemia drepanocítica o talasemia.
    12.Insuficiencia renal y/o hepática moderada o grave.
    13.Pacientes que presentaron efectos secundarios moderados o intensos relacionados con los esteroides, o acontecimientos adversos moderados o intensos asociados con el fármaco investigado y administrado en el estudio IEDAT-02.
    Medicamentos previos/concomitantes
    14.Requiere tratamiento con un esteroide por vía oral o parenteral. Se permitirá el tratamiento con esteroides inhalados o intranasales para el asma o alergias, así como también el uso de esteroides tópicos.
    15.Requiere cualquier otro medicamento concomitante prohibido por el protocolo.
    16.Uso de cualquier fármaco que sea un fuerte inductor o inhibidor del CYP3A4.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint in the AT study will be the mean change from baseline to endpoint (Month 6 or early discontinuation) in the total score on the ‘Modified’ International Cooperative Ataxia Rating Scale (ICARS).
    La variable principal de eficacia en el estudio AT será el cambio medio desde el inicio hasta el punto final (mes 6 o interrupción temprana) en la puntuación total en la escala cooperativa internacional para la cuantificación de la ataxia “modificada” (ICARSm)
    E.5.1.1Timepoint(s) of evaluation of this end point
    To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients.
    Seguir y evaluar la seguridad a largo plazo y la tolerabilidad del EDS-EP en pacientes con AT.
    E.5.2Secondary end point(s)
    To evaluate the long-term effect of EDS-EP on health related Quality of Life (QoL; EQ-5D-5L scale).
    Evaluar el efecto a largo plazo del EDS-EP en la calidad de vida ( QoL; escala EQ-5D-5L)
    E.5.2.1Timepoint(s) of evaluation of this end point
    A patient/caregiver-rated assessment of quality of life (QoL) will be performed using the EQ-5D-5L scale. The EQ-5D is a standardized instrument for assessing health-related QoL, which provides a simple descriptive profile and a single index value for health status in a variety of health conditions
    Se realizará una evaluación de calidad de vida (CdV) calificada por el paciente / cuidador utilizando la escala EQ-5D-5L. El EQ-5D es un instrumento estandarizado para evaluar la CdV relacionada con la salud, que proporciona un perfil descriptivo simple y un valor de índice único para el estado de salud en una variedad de condiciones de salud.
    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 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 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.8.3 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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 Yes
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Germany
    India
    Israel
    Italy
    Norway
    Poland
    Spain
    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
    LPLV

    The open-label treatment period is planned for 12 months, but may be extended further and continue until patients eventually withdraw consent or the Investigator decides to discontinue treatment based on a risk/ benefit assessment
    LPLV
    El período de tratamiento abierto está planificado para 12 meses, pero puede extenderse más y continuar hasta que los pacientes finalmente retiren el consentimiento o el Investigador decida suspender el tratamiento según una evaluación de riesgo / beneficio
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 15
    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: 5
    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: 5
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 19
    F.4.2.2In the whole clinical trial 61
    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)
    Standard Care
    Práctica Clínica Habitual
    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 Decision2018-05-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-24
    P. End of Trial
    P.End of Trial StatusCompleted
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