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    Summary
    EudraCT Number:2010-018454-13
    Sponsor's Protocol Code Number:SMB-NAL-III-09-1
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2010-05-31
    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 number2010-018454-13
    A.3Full title of the trial
    A Phase III, Multicentre, Randomised, Placebo-Controlled, Double Blind Study of the Incidence of Recurring Pulmonary Exacerbations in Cystic Fibrosis Patients using Two Different Doses of Inhaled Nacystelyn®

    Estudio de fase III multicéntrico, doble ciego, aleatorizado y controlado con placebo de la incidencia de recurrencia de las exacerbaciones pulmonares en pacientes con fibrosis quística utilizando dos dosis diferentes de Nacystelyn® inhalado
    A.4.1Sponsor's protocol code numberSMB-NAL-III-09-1
    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 SponsorLaboratoires SMB S.A.
    B.1.3.4CountryBelgium
    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 numberEU/3/01/026
    D.3 Description of the IMP
    D.3.1Product nameNacystelyn®
    D.3.2Product code NAL
    D.3.4Pharmaceutical form Inhalation powder, hard capsule
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNL-Lysine- N-acetylcysteinate
    D.3.9.2Current sponsor codeNAL
    D.3.9.3Other descriptive nameNacystelyn®
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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) 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 No
    D.3.11.8Extractive medicinal product No
    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 No
    D.IMP: 2
    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/3/01/026
    D.3 Description of the IMP
    D.3.1Product nameNacystelyn®
    D.3.2Product code NAL
    D.3.4Pharmaceutical form Inhalation powder, hard capsule
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNL-Lysine- N-acetylcysteinate
    D.3.9.2Current sponsor codeNAL
    D.3.9.3Other descriptive nameNacystelyn®
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 No
    D.3.11.8Extractive medicinal product No
    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 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 placeboInhalation powder, hard capsule
    D.8.4Route of administration of the placeboInhalation use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic fibrosis

    Fibrosis quistica
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.1
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Determinar la seguridad y la eficacia a largo plazo de Nacystelyn® inhalado, en presencia o ausencia de rhDNasa, en pacientes con fibrosis quística, reflejadas por un cambio en el tiempo hasta la primera exacerbación pulmonar (EP) por un grupo de tratamiento durante el periodo de estudio.

    To determine the long-term safety and efficacy of inhaled Nacystelyn®, in the presence or absence of rhDNase, in patients with CF as reflected in a change per treatment group in the time to first pulmonary exacerbation (PE) during the study period.
    E.2.2Secondary objectives of the trial
    Determinar los efectos de 52 semanas de Nacystelyn® inhalado, en presencia o ausencia de rhDNasa, sobre la función pulmonar, el número de pacientes que experimenten una exacerbación pulmonar, el número total de exacerbaciones pulmonares experimentadas, el número de pacientes tratados con antibióticos intravenosos por una exacerbación pulmonar, el número de exacerbaciones pulmonares tratadas con antibióticos intravenosos, la duración total del uso de antibióticos para el tratamiento de las exacerbaciones pulmonares, la duración total de las hospitalizaciones por exacerbación pulmonar y la calidad de vida (CdV).

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Tienen un diagnóstico confirmado de fibrosis quística basado en lo siguiente: cloro en sudor > 40 mEq/l y/o genotipo con dos mutaciones identificables compatibles con la fibrosis quística y una o más características clínicas compatibles con la fibrosis quística;
    2. Tienen ≥ 12 años de edad;
    3. Tienen un volumen espiratorio forzado en un segundo (FEV1) > 40,0% del previsto;
    4. Han experimentado como mínimo una exacerbación pulmonar tratada en los 12 meses previos a la selección;
    5. Han estado en tratamiento estable con rhDNasa (2,5 mg una vez al día con nebulizador) durante tres meses antes de la selección (sólo para los pacientes que tomen rhDNasa durante el estudio);
    6. Tienen >80% y <120% cumplimiento durante el periodo de run-in (prealeatorización);
    7. Han firmado un hoja de consentimiento informada por escrito (o de asentimiento proporcionado por los padres o tutores legales) y están dispuestos a, y son capaces de, cumplir todos los procedimientos del estudio
    E.4Principal exclusion criteria
    1. El paciente ha experimentado una exacerbación pulmonar tratada, resuelta o no resuelta, en el mes anterior a ser seleccionado;
    2. El paciente ha sido hospitalizado o ha experimentado cambios en los antibióticos, antiinflamatorios, corticosteroides o broncodilatadores de acción prolongada en las dos semanas previas a la aleatorización
    3. El paciente ha estado tomando algún mucolítico, excepto la rhDNasa, en las dos semanas previas a la aleatorización;
    4. El paciente tiene antecedentes de hemoptisis (> 30 cc) en los tres meses previos a la aleatorización
    5. El paciente ha participado en otro estudio clínico en el mes previo al aleatorización;
    6. El paciente es fumador de > 10 cigarrillos/día (o equivalente) o tiene antecedentes de tabaquismo de >10 paquetes año;
    7. El paciente tiene antecedentes de drogadicción, alcoholismo o abuso de medicamentos en los 12 meses previos;
    8. La paciente está embarazada o en periodo de lactancia;
    9. La paciente es fértil y ella o su pareja no están utilizando una forma aceptable de anticoncepción (que se define como el uso de un dispositivo intrauterino [DIU], un método de barrera con espermicida, preservativos, implantes subdérmicos o anticonceptivos orales);
    10. El paciente tiene antecedentes de neoplasias malignas en los cinco años previos;
    11. El paciente tiene antecedentes de trasplante de pulmón, se ha planificado que reciba un trasplante de pulmón durante el periodo del estudio o está actualmente en lista para trasplante;
    12. Se sabe que el paciente tiene, o está en situación de riesgo de contraer, el virus de la inmunodeficiencia humana (VIH), de la hepatitis B, de la hepatitis C o la tuberculosis;
    13. Pacientes con hipersensibilidad o intolerancia conocidas a nacystelyn, acetilcisteína, lisina o lactosa;
    14. El paciente tiene una dolencia o anomalía de los resultados basales de los análisis clínicos que, en opinión del investigador principal, afectarían a la seguridad del paciente.
    E.5 End points
    E.5.1Primary end point(s)

    • Tiempo hasta la primera exacerbación pulmonar mientras dure el estudio en una población de intención de tratar (IdT)

    Time to first PE within the study duration on an ITT population.
    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 Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    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.8.3 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 concerned11
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA82
    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
    Ultimo paciente, ultima visita

    Last patient last visit
    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 months2
    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 months2
    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.1.1.1In 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.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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
    Los padres de los adolescentes participantes en el estudio podran firmar el consentimiento para su participacion en el estudio, asi como los propios adolescentes.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state66
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 450
    F.4.2.2In the whole clinical trial 552
    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 Decision2010-09-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-07-30
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2010-10-15
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