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    Summary
    EudraCT Number:2015-003170-33
    Sponsor's Protocol Code Number:INS-312
    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:2016-01-13
    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 number2015-003170-33
    A.3Full title of the trial
    An Open-Label Safety Extension Study to a Multicenter Study of Liposomal Amikacin for Inhalation (LAI) in Adult Patients with Nontuberculous Mycobacterial (NTM) Lung Infections caused by Mycobacterium avium complex (MAC) that are refractory to treatment
    Estudio abierto de seguridad de extension del estudio multicéntrico de la amicacina liposomal para inhalación (LAI) en pacientes adultos con infecciones pulmonares por micobacterias no tuberculosas (MNT) causadas por el complejo Mycobacterium avium (CMA) que son resistentes al tratamiento
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension study of Liposomal Amikacin in Adults with Nontuberculous Mycobacteria (INS-312)
    Estudio de extension de Amicacina Liposomal en Adultos con Micobacterias Notuberculosas (INS-312)
    A.4.1Sponsor's protocol code numberINS-312
    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 SponsorInsmed 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 supportInsmed Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInsmed Inc.
    B.5.2Functional name of contact pointGina Eagle
    B.5.3 Address:
    B.5.3.1Street Address10 Finderne Avenue, Building 10
    B.5.3.2Town/ cityBridgewater
    B.5.3.3Post codeNJ 08807-3365
    B.5.3.4CountryUnited States
    B.5.4Telephone number+19089474388
    B.5.5Fax number+19085264047
    B.5.6E-mailgina.eagle@insmed.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 numberEU/3/14/1259
    D.3 Description of the IMP
    D.3.1Product nameLiposomal Amikacin for Inhalation (LAI)
    D.3.4Pharmaceutical form Nebuliser suspension
    D.3.4.1Specific paediatric formulation No
    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 INNAMIKACIN SULFATE
    D.3.9.1CAS number 39831-55-5
    D.3.9.3Other descriptive nameAmikacin Sulfate
    D.3.9.4EV Substance CodeSUB00444MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number70
    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 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
    Nontuberculous Mycobacterial (NTM) Lung Infections caused by Mycobacterium avium complex (MAC) that are refractory to treatment
    Infecciones pulmonares por micobacterias no tuberculosis causadas por Mycobacterium avium complex (MAC) que son resistentes al tratamiento
    E.1.1.1Medical condition in easily understood language
    Nontuberculous Mycobacterial (NTM) Lung Infections caused by Mycobacterium avium complex (MAC) that are refractory to treatment
    Infecciones pulmonares por micobacterias no tuberculosis causadas por Mycobacterium avium complex (MAC) que son resistentes al tratamiento
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level PT
    E.1.2Classification code 10058806
    E.1.2Term Mycobacterium avium complex infection
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate long term safety and tolerability of LAI (590 mg) administered once daily (QD) for up to 12 months in subjects who were refractory to standard multi-drug treatment and failed to convert in Study INS-212
    Evaluar la seguridad y tolerabilidad a largo plazo de LAI (590 mg) administrada 1/d durante un máximo de 12 meses en sujetos que presentaron resistencia a la politerapia estándar y no lograron la conversión en el estudio INS-212
    E.2.2Secondary objectives of the trial
    Secondary Objectives
    1. To evaluate the number of subjects achieving culture conversion (3 consecutive negative sputum cultures) by Month 6
    2. To evaluate the number of subjects achieving culture conversion by Month 12/EOT (end of treatment)
    3. To evaluate the time to culture conversion
    4. To evaluate the change in the six-minute walk test (6MWT) distance at Month 6 and Month 12/EOT

    Exploratory Objectives
    1. To assess the proportion of subjects achieving culture conversion by Month 6 and 12/EOT
    2. To assess subject-reported symptoms of NTM and change from Baseline in quality of life scores on the St George?s Respiratory Questionnaire (SGRQ) and quality of life scores on the SGRQ ? Part 2 (Activities of Daily Living) at Month 6 and Month 12/EOT
    3. To assess the change from Baseline in the EQ-5D-3L questionnaire subject-reported health outcomes at Month 6 and Month 12/EOT
    Objetivos secundarios
    1.Evaluar el nº de sujetos que logran la conversión de los cultivos (3 cultivos de esputo negativos consecutivos) para el mes 6
    2.Evaluar el nº de sujetos que logran la conversión de los cultivos para el mes 12/FdT
    3.Evaluar el tiempo hasta la conversión de los cultivos.
    4.Evaluar el cambio en la distancia recorrida en la prueba de la marcha de 6 minutos en el mes 6 y mes 12/FdT
    Objetivos exploratorios
    1.Evaluar la proporción de sujetos que logran la conversión de los cultivos para el mes 6 y el mes 12/FdT
    2.Evaluar los síntomas de MNT referidos por el paciente y el cambio respecto al inicio en las puntuaciones de calidad de vida en el Cuestionario respiratorio de St. George y las puntuaciones de calidad de vida en el SGRQ, parte 2 en el mes 6 y mes 12/FdT
    3.Evaluar el cambio respecto al inicio en los resultados de salud percibido por el paciente del cuestionario EQ-5D-3L en el mes 6 y el mes 12/FdT
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects are eligible to participate in the study if they meet all the following inclusion criteria:
    1. have successfully completed the Month 6 and End of Treatment(EoT) visits in INS-212
    2. have not achieved the INS-212 protocol definition of culture conversion (3 consecutive negative sputum cultures) by Month 6 in INS-212
    OR
    have experienced a relapse or recurrence (agar positive or more than 2 consecutive broth positive results after culture conversion has occurred) by Month 6 in INS-212
    3. have demonstrated compliance with study medication in INS-212
    4. willing to adhere to multi-drug treatment regimen during the course of the study
    5. female of childbearing potential agrees to practice an acceptable method of birth control (e.g., abstinence, hormonal or barrier methods, partner sterilization, or intrauterine device (IUD))
    6. provide written informed consent before performing any study related procedure
    7. willing to have specimens stored
    8. able to comply with study medication use, study visits, and study procedures as determined by the investigator
    Los sujetos son aptos para participar en el estudio si cumplen todos los criterios de inclusión siguientes:
    1.Haber completado satisfactoriamente las visitas del mes 6 y FdT en INS-212
    2.No haber logrado la conversión de los cultivos según la definición del protocolo INS-212 (3 cultivos de esputo negativos consecutivos) para el mes 6 en INS-212
    O
    haber sufrido una recidiva o recaída (agar positivo o más de 2 resultados positivos consecutivos en medio después de que se hubiera producido la conversión de los cultivos) para el mes 6 en INS-212
    3.Haber demostrado cumplimiento con la medicación del estudio en INS-212
    4.Estar dispuesto a cumplir una politerapia durante el transcurso del estudio
    5.Si es mujer en edad fértil, acceder a utilizar un método anticonceptivo aceptable (p. ej., abstinencia, métodos hormonales o de barrera, esterilización de la pareja o dispositivo intrauterino [DIU])
    6.Dar su consentimiento informado por escrito antes de la realización de los procedimientos del estudio
    7.Estar dispuesto al almacenamiento de muestras
    8.Ser capaz de cumplir el uso de la medicación del estudio, las visitas y los procedimientos del estudio que determine el investigador
    E.4Principal exclusion criteria
    Subjects are not eligible to participate in the study if they meet any of the following criteria:
    1. achieved culture conversion without relapse or recurrence in the INS-212 study by Month 6
    2. early discontinuation (prior to Month 6 study visit) from INS-212
    3. met any of the exclusion criteria of the INS-212 study, with the exception of ?unable to perform the 6 Minute Walk Test (6MWT)? or ?receiving continuous oxygen therapy? (if these occurred during the INS-212 study)
    4. met any of the discontinuation criteria of the INS-212 study
    5. positive pregnancy test or lactation. All women of child bearing potential will be tested. Women not of childbearing potential are defined as postmenopausal (i.e., amenorrheic for at least 1 year), or surgically or naturally sterile.
    6. significant (as determined by the investigator) hearing loss, vestibular dysfunction, or neuromuscular weakness where the potential risk of aminoglycoside toxicity outweighs the potential benefit
    7. aspartate aminotransferase or alanine aminotransferase ? 3 times the upper limit of normal (ULN) and/or total bilirubin ? 2 times the upper limit of normal (ULN) at their EOT study visit in INS-212
    8. absolute neutrophil count ?500/?L at their EOT study visit in INS-212
    9. serum creatinine >2 times ULN at their EOT study visit in INS-212
    10. current alcohol, medication or illicit drug abuse
    11. any condition that, in the opinion of the Investigator, interferes with ability to safely complete the study or adhere to study requirements.
    Los sujetos no son aptos para participar en el estudio si cumplen cualquiera de los siguientes criterios:
    1.Haber logrado la conversión de los cultivos sin recidiva o recaída en el estudio INS-212 para el mes 6
    2.Interrupción anticipada (antes de la visita del mes 6 del estudio) de INS-212
    3.Cumplir cualquiera de los criterios de exclusión del estudio INS-212, a excepción de ?incapaz de realizar la PM6M? o ?reciben terapia de oxígeno continua? (si estos tuvieron lugar durante el estudio INS-212)
    4.Cumplir cualquiera de los criterios de interrupción del estudio INS-212
    5.Prueba de embarazo positiva o lactancia. Todas las mujeres en edad fértil se someterán a la prueba. Las mujeres que no están en edad fértil son las posmenopáusicas (esto es, amenorreicas durante al menos 1 año) o las estériles quirúrgicamente o de manera natural.
    6.Pérdida auditiva importante (según lo determine el investigador), disfunción vestibular o debilidad neuromuscular en que el posible riesgo de toxicidad de los aminoglucósidos supere el posible beneficio
    7.Aspartato aminotransferasa o alanina aminotransferasa ? 3 veces el límite superior de la normalidad (LSN) o bilirrubina total ? 2 veces el LSN en su visita de FdT del estudio INS-212
    8.Cifra absoluta de neutrófilos ? 500/?l en su visita de FdT del estudio INS-212
    9.Creatinina sérica > 2 veces el LSN en su visita de FdT del estudio INS-212
    10.Alcoholismo, abuso de medicamentos o drogodependencia en la actualidad
    11.Cualquier afección que, en opinión del investigador, interfiera en la capacidad de realizar el estudio con seguridad o cumplir con sus requisitos.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the frequency of treatment-emergent adverse events (TEAEs), TEAEs leading to withdrawal from study, treatment-emergent serious adverse events (SAEs), AEs of special interest, clinically significant abnormal laboratory test results, and vital signs measurements.
    El criterio de evaluación principal es la frecuencia de acontecimientos adversos surgidos durante el tratamiento (AAST), AAST que provocan la retirada del estudio, acontecimientos adversos graves (AAG) surgidos durante el tratamiento, AA de especial interés, resultados anormales de las pruebas de laboratorio clínicamente significativos y evaluación de los signos vitales.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline until end of study
    Desde el inicio hasta el final del estudio
    E.5.2Secondary end point(s)
    1. Proportion of subjects achieving culture conversion (3 consecutive negative sputum cultures without relapse or recurrence) by Month 6
    2. Proportion of subjects achieving culture conversion by Month 12/EOT
    3. Time to culture conversion. The date of conversion is defined by the date of the first of at least 3 consecutive monthly culture specimens that are MAC negative.
    4. The mean change from Baseline in 6MWT distance at Month 6 and Month 12/EOT
    EXPLORATORY ENDPOINTS - EFFICACY
    1. Proportion of subjects achieving culture conversion by Month 6 and 12/EOT
    2. The mean change from Baseline at Month 6 and Month 12/EOT in the overall SGRQ and SGRQ ? Part 2 (Activities of Daily Living)
    3. The mean change from Baseline at Month 6 and Month 12/EOT in the EQ 5D-3L
    1.Proporción de sujetos que logran la conversión de los cultivos (3 cultivos de esputo negativos consecutivos sin recidiva o recaída) para el mes 6
    2.Proporción de sujetos que logran la conversión de los cultivos para el mes 12/FdT
    3.Tiempo hasta la conversión de los cultivos. La fecha de la conversión se define por la fecha de la primera de, al menos, 3 muestras consecutivas de cultivo mensual que son negativas para CMA.
    4.Cambio medio respecto al inicio en la distancia recorrida en la PM6M en el mes 6 y mes 12/FdT
    CRITERIOS DE EVALUACIÓN SECUNDARIOS-EFICACIA
    1.Proporción de sujetos que logran la conversión de los cultivos para el mes 6 y mes 12/FdT
    2.Cambio medio respecto al inicio en el mes 6 y mes 12/FdT en el SGRQ global y SGRQ, parte 2 (actividades de la vida diaria)
    3.Cambio medio respecto al inicio en el mes 6 y mes 12/FdT en el EQ 5D-3L
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline until end of study
    Desde el inicio hasta el final del estudio
    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 Yes
    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 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) Yes
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA40
    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
    Australia
    Austria
    Canada
    France
    Germany
    Israel
    Italy
    Japan
    Netherlands
    New Zealand
    Poland
    Spain
    Taiwan
    Thailand
    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
    LVLS
    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 months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months10
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 134
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 66
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 44
    F.4.2.2In the whole clinical trial 200
    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)
    All patients who end their participation in the study will continue to be monitored by their respective physicians, using standard of care
    No special procedures or extra medical care is needed after the trial that would not be otherwise used in patients who have not participated in the trial.
    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 Decision2016-03-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-03-03
    P. End of Trial
    P.End of Trial StatusCompleted
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