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    EudraCT Number:2017-000397-10
    Sponsor's Protocol Code Number:DSC/14/2357/51
    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:2019-04-11
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2017-000397-10
    A.3Full title of the trial
    Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies
    Estudio abierto, de seguridad a largo plazo, de tolerabilidad y de eficacia de GIVINOSTAT en todos los pacientes con DMD que hayan sido tratados previamente en uno de los estudios de GIVINOSTAT.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past
    Estudio en el que todos los pacientes toman el mismo fármaco de investigación con el objetivo de evaluar la seguridad a largo plazo, la tolerabilidad y la eficacia de GIVINOSTAT en todos los pacientes afectados con Distrofia Muscular de Duchenne que ya han sido tratados en uno de los estudios de GIVINOSTAT en pasado
    A.3.2Name or abbreviated title of the trial where available
    Givinostat DMD study long term
    Givinostat DMD estudio a largo plazo
    A.4.1Sponsor's protocol code numberDSC/14/2357/51
    A.5.4Other Identifiers
    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 SponsorITALFARMACO S.p.A.
    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 supportITALFARMACO S.p.A.
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationITALFARMACO S.p.A.
    B.5.2Functional name of contact pointClinical Research and Development
    B.5.3 Address:
    B.5.3.1Street AddressVia dei Lavoratori, 54
    B.5.3.2Town/ cityCinisello Balsamo (MI)
    B.5.3.3Post code20092
    B.5.4Telephone number+390264431
    B.5.5Fax number+390264433554
    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/3/12/1009
    D.3 Description of the IMP
    D.3.1Product nameGivinostat (hydrochloride monohydrate)
    D.3.2Product code ITF2357
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation Yes
    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 INNGivinostat (hydrochloride monohydrate)
    D.3.9.1CAS number 732302-99-7
    D.3.9.2Current sponsor codeITF2357
    D.3.9.3Other descriptive nameGIVINOSTAT
    D.3.9.4EV Substance CodeSUB30834
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Duchenne Muscular Dystrophy (DMD)
    Distrofia muscular de Duchenne (DMD)
    E.1.1.1Medical condition in easily understood language
    A neuromuscular disease characterized by rapidly progressive muscle weakness and wasting
    Una enfermedad neuromuscular caracterizada por debilidad muscular rápidamente progresiva y degenerativa.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    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 assess the long term safety and tolerability of GIVINOSTAT in patients with DMD following core protocols program
    Evaluar la seguridad y tolerabilidad a largo plazo de GIVINOSTAT en pacientes con DMD siguiendo el programa de protocolos básicos.
    E.2.2Secondary objectives of the trial
    To evaluate the effects of long-term administration of GIVINOSTAT on muscular function and strength;
    To evaluate the effects of long-term administration of GIVINOSTAT on respiratory function;
    To evaluate the impact on daily activities and quality of life following long-term administration of GIVINOSTAT
    -Evaluar los efectos de la administración a largo plazo de GIVINOSTAT en la función y la fuerza musculares.

    -Evaluar los efectos de la administración a largo plazo de GIVINOSTAT en la función respiratoria.

    -Evaluar el impacto en las actividades diarias y la calidad de vida después de la administración a largo plazo de GIVINOSTAT.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all the following inclusion criteria:
    1. Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit;
    2. Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations);
    3. Subjects must be willing to use adequate contraception:
    Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following:
    oTrue abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.
    oCondom with spermicide and the female partner must use an acceptable method of contraception, such as an oral, transdermal, injectable or implanted steroid-based contraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such as for example cervical cap with spermicide jelly.
    Los sujetos deben cumplir todos los siguientes criterios de inclusión:

    1. Haber participado en alguno de los estudios anteriores con GIVINOSTAT para la DMD y haber asistido a la visita de final del estudio.

    2. Ser capaces de dar su asentimiento y/o consentimiento informado por escrito firmado por el sujeto y/o un progenitor/tutor legal (de acuerdo con las normativas locales).

    3. Los sujetos deben estar dispuestos a usar anticonceptivos adecuados. Los métodos anticonceptivos deben usarse desde el estudio anterior de GIVINOSTAT hasta 3 meses después de la última dosis del fármaco de estudio e incluyen los siguientes:
    *Abstinencia real (ausencia de relaciones sexuales) cuando coincida con el estilo de vida preferido y habitual del sujeto. La abstinencia periódica (p. ej., métodos del calendario, de la ovulación, sintotérmicos, postovulación) y la marcha atrás no son métodos anticonceptivos aceptables.

    *El preservativo con espermicida y la pareja femenina debe usar un método anticonceptivo aceptable, como un anticonceptivo con esteroides oral, transdérmico, inyectable o implantado; un diafragma; o un método anticonceptivo de barrera junto con gelatina espermicida como, por ejemplo, un capuchón cervical con gelatina espermicida.
    E.4Principal exclusion criteria
    Subjects presenting with any of the following criteria will not be included in the study:
    1. Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;
    2. Use of any current investigational drug other than Givinostat;
    3. Have presence of other clinically significant disease, which, in the Investigator’s opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results;
    4. Have a diagnosis of other neurological diseases or presence of relevant somatic disorders that are not related to DMD;
    5. Have platelets count, White Blood Cell and Hemoglobin at screening < Lower Limit of Normal (LLN) (for abnormal screening laboratory test results (<LLN), the platelets count, White Blood Cell and Hemoglobin will be repeated once; if the repeat test result is still <LLN, then exclusionary);
    6. Have heart failure (New York Heart Association Class III or IV)
    7. Have a current liver disease or impairment, including but not limited to an elevated total bilirubin(i.e. > 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's;
    8. Have a baseline QTcF >450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome);
    9. Have a psychiatric illness/social situations rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures.
    10. Have any hypersensitivity to the components of study medication;
    11. Have a sorbitol intolerance or sorbitol malabsorption, or have the hereditary form of fructose intolerance.
    No se incluirá en el estudio a los sujetos que presenten alguno de los siguientes criterios:

    1. Se permitirá el uso de cualquier tratamiento farmacológico, aparte de los corticoesteroides, que pudiera haber tenido efectos en la fuerza o función musculares en los 3 meses anteriores a la inscripción en este estudio (p. ej., hormona del crecimiento); el uso de vitamina D, calcio y cualquier otro suplemento.
    2. Uso de cualquier fármaco actualmente en fase de investigación que no sea givinostat.
    3. Padecer otra enfermedad clínicamente significativa, que, en opinión del investigador, podría afectar negativamente a la seguridad del sujeto, haciendo poco probable la finalización del ciclo de tratamiento o del seguimiento, o que podría perjudicar la evaluación de los resultados del estudio.
    4. Tener un diagnóstico de otras enfermedades neurológicas o padecer trastornos somáticos relevantes que no estén relacionados con la DMD.
    5. Tener un recuento de plaquetas, glóbulos blancos y hemoglobina en la selección < límite inferior de la normalidad (LIN)* [para resultados anómalos de pruebas de laboratorio de selección (<LIN), el recuento de plaquetas, glóbulos blancos y hemoglobina se repetirá una vez; si el resultado de la prueba de repetición sigue siendo <LIN, será excluyente].
    6. Padecer insuficiencia cardíaca (clases III o IV de la New York Heart Association).
    7. Tener una enfermedad o insuficiencia hepática actual, entre otras, bilirrubina* total elevada (es decir, >1,5 x LSN) a menos que sea secundaria a la enfermedad de Gilbert o tenga un patrón congruente con esta.
    8. Tener un valor inicial de QTcF >450 mseg (como media de 3 lecturas consecutivas con 5 minutos de diferencia) o antecedentes de factores de riesgo adicionales de taquicardia ventricular helicoidal (p. ej., insuficiencia cardíaca, hipocaliemia o antecedentes familiares de síndrome de QT largo).
    9. Padecer una enfermedad psiquiátrica o encontrarse en situaciones sociales que hagan que el posible sujeto sea incapaz de entender y cumplir las pruebas de función muscular y/o los procedimientos del protocolo del estudio.
    10. Sufrir hipersensibilidad a los componentes del medicamento del estudio.
    11. Tener intolerancia al sorbitol o mala absorción del sorbitol, o padecer un tipo hereditario de intolerancia a la fructosa.
    E.5 End points
    E.5.1Primary end point(s)
    Type, incidence, and severity of treatment related/not related AEs and SAEs
    Tipo, incidencia e intensidad de los AA y AAG relacionados y no relacionados con el tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline through week 48 and then yearly till the end of the study
    Desde el inicio hasta la semana 48 y luego anualmente hasta el final del estudio
    E.5.2Secondary end point(s)
    For ambulant patients:
    • Change from baseline in physical function as measured by 6MWT, NSAA, Time function tests (e.g. time to rise from floor, time to climb 4-stairs, time to 10m walk)(week 48 and then yearly till the end of the study)
    • Change from baseline in muscle strength (e.g. knee extension and elbow flexion) as measured by HHM (week 48 and then yearly till the end of the study)
    • Time to major disease milestones (e.g. time to loss of ambulation).
    For non-ambulant patients:
    • Change in physical function from baseline in the Egen Klassifikation (EK) score (week 48 and then yearly till the end of the study)
    • Change in patient and/or parent/caregiver reports of activities of daily living as measured by Barthel Index (week 48 and then yearly till the end of the study
    • Change in upper limbs muscle strength (week 48 and then yearly till the end of the study) evaluated by handheld myometry (HHM)
    For all patients:
    • Change from baseline in physical function as measured by the Performance of Upper Limb (PUL) and MFM (week 48 and then yearly till the end of the study)
    • Change from baseline in respiratory function (week 48 and then yearly till the end of the study) (e.g. FVC, FEV1, PEF)
    • Change in patient and/or parent/caregiver reports of quality of life as measured by PedsQL (week 48 and then yearly till the end of the study),
    • Time to major disease milestones (e.g. time to respiratory support needed during the day, time to scoliosis surgery, time to death).
    Para pacientes ambulantes:

    • Cambio desde el inicio en la función física medida por la PM6M, NSAA, pruebas de tiempo de funciones (p. ej., tiempo para levantarse del suelo, tiempo para subir 4 escaleras, tiempo para caminar 10 m) (semana 48 y luego anualmente hasta el final del estudio).
    • Cambio desde el inicio en la fuerza muscular (p. ej., extensión de la rodilla y flexión del codo) medido por MM (semana 48 y luego anualmente hasta el final del estudio).
    • Tiempo hasta los hitos principales de la enfermedad (p. ej., tiempo hasta la pérdida de la deambulación).

    Para pacientes no ambulantes:

    • Cambio en la función física desde el inicio en la puntuación de la Egen Klassifikation (EK) (semana 48 y luego anualmente hasta el final del estudio).
    • Cambio en los informes de los pacientes y/o padres/cuidadores sobre las actividades de la vida diaria medidas por el Índice de Barthel (semana 48 y luego anualmente hasta el final del estudio).
    • Cambio en la fuerza muscular de las extremidades superiores (semana 48 y luego anualmente hasta el final del estudio) evaluada por miometría manual (MM).

    Para todos los pacientes:

    • Cambio desde el inicio en la función física medida por el funcionamiento de las extremidades superiores (FES) y MFM (semana 48 y luego anualmente hasta el final del estudio).
    • Cambio desde el inicio en la función respiratoria (semana 48 y luego anualmente hasta el final del estudio) (p. ej., FVC, FEV1, PEF).
    • Cambio en los informes de los pacientes y/o padres/cuidadores sobre la calidad de vida medida por el PedsQL (semana 48 y luego anualmente hasta el final del estudio).
    • Tiempo hasta los hitos principales de la enfermedad (p. ej., tiempo hasta el apoyo respiratorio necesario durante el día, tiempo hasta la cirugía de escoliosis, tiempo hasta la muerte).
    Además de los puntos temporales indicados, el análisis estadístico se realizará cada año de tratamiento hasta que se conceda la autorización de comercialización.
    E.5.2.1Timepoint(s) of evaluation of this end point
    timepoint given in endpoints description
    puntos de evaluación dado en la descripción de puntos finales
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    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. 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.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    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
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months5
    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: 175
    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. of subjects for this age range: 138
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 37
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female No
    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. of subjects incapable of giving consent
    Subjects who could be not able to give informed written assent/consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state48
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 153
    F.4.2.2In the whole clinical trial 185
    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)
    The treatment may continue up to the Marketing Authorization of GIVINOSTAT or different decision taken by the Sponsor and /or the Competent Authorities, based on safety and/or efficacy reasons, for all the DMD population
    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 Decision2019-05-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-29
    P. End of Trial
    P.End of Trial StatusOngoing
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