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    Summary
    EudraCT Number:2015-003170-33
    Sponsor's Protocol Code Number:INS-312
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-11-04
    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 ConcernedItaly - Italian Medicines Agency
    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
    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
    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)
    Studio di estensione di Amikacina Liposomiale in pazienti adulti con infezioni polmonari da micobatteri non tubercolari causate da Micobatterio Avium Complex refrattarie al trattamento
    A.3.2Name or abbreviated title of the trial where available
    Extension study of Liposomal Amikacin in Adults with Nontuberculous Mycobacteria (INS-312)
    Studio di estensione con Amicacina liposomiale in pazienti adulti con MNT causate da MAC
    A.4.1Sponsor's protocol code numberINS-312
    A.5.4Other Identifiers
    Name:INS-312Number:INS-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 INCORPORATED
    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 Inc
    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 number0019089474388
    B.5.5Fax number0019085264047
    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.3.4Pharmaceutical 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 INNAMICACINA SOLFATO
    D.3.9.1CAS number 39831-55-5
    D.3.9.2Current sponsor codeND
    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
    Infezioni polmonari da micobatteri non tubercolari (MNT) causate da Mycobacterium avium complex (MAC), refrattarie al trattamento
    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
    Infezioni polmonari da micobatteri non tubercolari (MNT) causate da Mycobacterium avium complex (MAC), refrattarie al trattamento
    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 20.1
    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
    Valutare la sicurezza e la tollerabilit¿ a lungo termine di LAI (590 mg) somministrato una volta al giorno (QD) per un massimo di 12 mesi in soggetti refrattari al trattamento multifarmaco standard che non hanno ottenuto una conversione dell¿esame colturale nello studio 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
    1.Valutare il numero di soggetti che ottengono una conversione dell¿esame colturale (3 colture dell¿espettorato negative consecutive) al Mese 6
    2.Valutare il numero di soggetti che ottengono una conversione dell¿esame colturale al Mese 12/Fine trattamento (EOT)
    3.Valutare il tempo alla conversione dell¿esame colturale
    4.Valutare la variazione della distanza percorsa al test del cammino dei sei minuti (6MWT) al Mese 6 e al Mese 12/EOT

    Obiettivi esploratori
    1.Valutare la proporzione di soggetti che ottengono una conversione dell¿esame colturale al Mese 6 e 12/EOT
    2.Valutare i sintomi riferiti dal soggetto di infezione da MNT e la variazione dal Basale nei punteggi di qualit¿ della vita al questionario respiratorio di St. George (SGRQ) e nei punteggi di qualit¿ della vita al SGRQ - Parte 2 (Attivit¿ della vita quotidiana) al Mese 6 e al Mese 12/EOT
    3.Valutare la variazione dal Basale degli esiti sulla salute riferiti dal soggetto nel questionario europeo di qualit¿ della vita a 5 dime
    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
    I soggetti sono eleggibili per la partecipazione allo studio se soddisfano tutti i seguenti criteri di inclusione:
    1.hanno completato con successo le visite del Mese 6 e EOT in INS-212
    2.non hanno soddisfatto la definizione secondo il protocollo dell’INS-212 di conversione dell’esame colturale (3 colture dell’espettorato negative consecutive) al Mese 6 in INS-212
    O
    hanno manifestato una recidiva o ricorrenza (risultato positivo in agar o più di 2 risultati positivi consecutivi in brodo dopo raggiungimento della conversione colturale) al Mese 6 in INS-212
    3.hanno dimostrato di aderire al medicinale in studio in INS-212
    4.sono disponibili ad aderire a un regime di trattamento multifarmaco nel corso dello studio
    5.le donne potenzialmente fertili acconsentono ad adottare un metodo di contraccezione accettabile (ad esempio, astinenza, metodi ormonali o barriera, sterilizzazione del partner o dispositivo intrauterino [IUD])
    6.consenso informato scritto fornito prima dell’esecuzione di qualsiasi procedura correlata allo studio
    7.consenso alla conservazione dei propri campioni
    8.capacità di aderire all’uso del medicinale in studio, alle visite dello studio e alle procedure dello studio, secondo quanto stabilito dallo sperimentatore
    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/µL 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.
    I soggetti non sono eleggibili a partecipare allo studio se soddisfano uno qualsiasi dei seguenti criteri:
    1.hanno ottenuto una conversione dell’esame colturale senza recidiva o ricorrenza nello studio INS-212 al Mese 6
    2.interruzione anticipata (prima della visita dello studio del Mese 6) dello studio INS-212
    3.hanno soddisfatto uno qualsiasi dei criteri di esclusione dello studio INS-212, ad eccezione di ‘impossibilità a effettuare il 6MWT’ o ‘sta ricevendo un’ossigenoterapia continuativa’ (se questi si sono manifestati durante lo studio INS-212)
    4.hanno soddisfatto uno qualsiasi dei criteri di interruzione dello studio INS-212
    5.test di gravidanza positivo o paziente in fase di allattamento. Tutte le donne potenzialmente fertili saranno sottoposte a test di gravidanza. Sono definite non potenzialmente fertili le donne in età postmenopausale (ovvero, con amenorrea da almeno 1 anno), oppure chirurgicamente o naturalmente sterili.
    6.perdita dell’udito significativa (come stabilito dallo sperimentatore), disfunzione vestibolare o debolezza neuromuscolare, tali per cui il potenziale rischio di tossicità da aminoglicosidi ecceda il beneficio potenziale
    7.livelli di aspartato aminotransferasi o alanina aminotransferasi = 3 volte il limite superiore della normalità (ULN) e/o bilirubina totale = 2 volte il limite superiore della normalità (ULN) alla visita dello studio EOT in INS-212
    8.conta assoluta dei neutrofili =500/µL alla visita dello studio EOT in INS-212
    9.creatinina sierica >2 volte l’ULN alla visita dello studio EOT in INS-212
    10.attuale abuso di alcol, medicinali o sostanze illecite
    11.qualsiasi condizione che, a giudizio dello sperimentatore, interferisca con la capacità di portare a termine lo studio in modo sicuro o di aderire ai requisiti dello studio
    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.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline until end of study
    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
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline until end of study
    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 concerned8
    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 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
    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
    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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    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.
    Tutti i pazienti che hanno terminato lo studio continueranno ad essere seguito dal loro medico, e seguiranno la terapia standard. Nessuna procedura speciale o al di fuori dalla pratica clinica sar¿ necessaria dopo lo studio che non sarebbe utilizzata in pazienti che non hanno partecipato allo studio.
    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 Decision2016-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-17
    P. End of Trial
    P.End of Trial StatusCompleted
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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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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