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    Summary
    EudraCT Number:2016-002749-42
    Sponsor's Protocol Code Number:GS-US-205-1850
    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:2017-08-08
    Trial 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 number2016-002749-42
    A.3Full title of the trial
    Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization
    Ensayo aleatorizado y doble ciego de fase IIIb para evaluar la seguridad y la eficacia de 2 regímenes de tratamiento con Aztreonam 75 mg polvo y disolvente para solución para inhalación por nebulizador / Aztreonam solución para inhalación (AZLI) en pacientes pediátricos con fibrosis quística (FQ) e infección/colonización de las vías respiratorias por Pseudomonas aeruginosa (PA) de nueva aparición
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteria
    Estudio con aztreonam para inhalación en niños con fibrosis quística e infección nueva de las vías respiratorias por la bacteria Pseudomonas aeruginosa.
    A.3.2Name or abbreviated title of the trial where available
    ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2)
    A.4.1Sponsor's protocol code numberGS-US-205-1850
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/064/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGilead Sciences, 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 supportGilead Sciences, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGilead Sciences International Ltd.
    B.5.2Functional name of contact pointClinical Trials Mailbox
    B.5.3 Address:
    B.5.3.1Street AddressFlowers Building, Granta Park
    B.5.3.2Town/ cityAbington, Cambridge
    B.5.3.3Post codeCB21 6GT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+34913 78 98 30
    B.5.5Fax number+34913 78 98 41
    B.5.6E-mailRegulatory.Spain@gilead.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 Yes
    D.2.1.1.1Trade name Cayston Aztreonam 75 mg powder and solvent for nebuliser solution
    D.2.1.1.2Name of the Marketing Authorisation holderGilead Sciences International Ltd.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/04/204
    D.3 Description of the IMP
    D.3.2Product code AZLI
    D.3.4Pharmaceutical form Powder and solvent for nebuliser solution
    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 INNAZTREONAM
    D.3.9.2Current sponsor codeAZLI
    D.3.9.4EV Substance CodeSUB05664MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder and solvent for nebuliser solution
    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 and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
    Fibrosis quística y cultivo positivo de Pseudomonas aeruginosa de nueva aparición en la vias respiratorias bajas.
    E.1.1.1Medical condition in easily understood language
    Cystic fibrosis and a newly acquired lung infection with a bacterium called Pseudomonas aeruginosa
    Fibrosis quística e infección pulmonar de nueva aparición por bacteria Pseudomonas aeruginosa
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10068288
    E.1.2Term Cystic fibrosis pulmonary exacerbation
    E.1.2System Organ Class 100000113915
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the safety and efficacy of a 14-day course vs a 28-day course of AZLI 75 mg three times a day (TID) in subjects with new onset PA respiratory tract colonization/infection as determined by PA eradication over a 28-day post-treatment follow-up period.
    El objetivo principal de este estudio es evaluar la seguridad y la eficacia de un ciclo de 14 días frente a un ciclo de 28 días de AZLI 75 mg administrado tres veces al día (3 v/d) en pacientes con colonización/infección de las vías respiratorias por PA de nueva aparición según lo establecido para la erradicación de PA durante un período de seguimiento de 28 días después del tratamiento.
    E.2.2Secondary objectives of the trial
    - To evaluate the time from primary eradication to PA recurrence over a 108-week
    post-treatment follow-up period.
    - To compare the efficacy of AZLI 75 mg TID for 14 days vs historical pooled tobramycin nebulizer solution (TNS) twice daily (BID) for 28 days as determined by PA eradication over a 28-day post-treatment follow-up period
    - To evaluate the time to PA recurrence for a sub-group of subjects matching the population in the TNS ELITE Study over a 108-week post-treatment follow-up period
    - Evaluar el tiempo desde la erradicación primaria hasta la recaída de PA durante un período de seguimiento de 180 semanas después del tratamiento.
    - Comparar la eficacia de AZLI 75 mg 3 v/d durante 14 días con los datos históricos combinados de trobramicina solución para inhalación por nebulizador (TSN) administrada dos veces al día (2 v/d) durante 28 días, según lo establecido para la erradicación de PA durante un período de seguimiento de 28 días después del tratamiento.
    - Evaluar el tiempo hasta la recaída de PA en un subgrupo de pacientes idéntico a la población del estudio TSN ELITE {Ratjen et al 2009} durante un período de seguimiento de 180 semanas después del tratamiento
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Male or female aged 3 months to less than 18 years at screening
    2) Diagnosis of CF as determined by the 2008 CF Consensus Conference criteria
    3) Documented new onset of positive respiratory tract culture for PA within 30 days of Screening defined as either first lifetime documented PA-positive culture, or PA recovered after at least a 2-year history of PA-negative respiratory cultures (at least 2 cultures per year)
    4) FEV1 ≥ 80% predicted
    5) Clinically stable with no evidence of acute significant respiratory symptoms that would require administration of IV antipseudomonal antibiotics, oxygen supplementation, or hospitalization
    6) A negative serum pregnancy test is required for female subjects of child bearing potential who have a positive urine pregnancy test at screening
    7) Male subjects and female subjects of childbearing potential who engage in heterosexual intercourse must agree to use protocol specified method(s) of contraception
    8) Lactating females must agree to discontinue nursing before administration of study drug
    9) Subjects and/or parent/guardian must be able to give written informed consent prior to study related procedures
    1) Niños o adolescentes de ambos sexos de entre 3 meses y menos de 18 años de edad.
    2) Diagnóstico de FQ según los criterios de la Conferencia de Consenso sobre la FQ
    3) Cultivo de las vías respiratorias con un resultado positivo documentado para PA de nueva aparición en los 30 días siguientes a la selección, definido como el primer cultivo positivo para PA documentado en la vida del paciente o reaparición de PA después de al menos 2 años con cultivos respiratorios negativos para PA (un mínimo de 2 cultivos al año).
    4) FEV1 ≥ 80% del valor previsto
    5) Estable clínicamente sin presencia de síntomas respiratorios agudos importantes que justifiquen la administración de antibióticos IV contra Pseudomonas; oxigenoterapia u hospitalización.
    6) Se requiere una prueba de embarazo en suero negativa para las mujeres con potencial de tener hijos que tengan una prueba de embarazo de orina positiva en el screening.
    7) Los sujetos masculinos y sujetos femeninos con potencial reproductivo que mantengan relaciones sexuales heterosexuales deben estar conformes con el uso de métodosde anticoncepción especificados en el protocolo.
    8) Las mujeres lactantes deben estar conformes con suspender la lactancia antes de la administración del fármaco del estudio.
    9) Los sujetos y / o padres / tutores deben ser capaces de dar consentimiento informado por escrito antes de los procedimientos relacionados con el estudio
    E.4Principal exclusion criteria
    1) Use of IV or inhaled antipseudomonal antibiotics within 2 years of Screening
    2) Use of oral antipseudomonal antibiotics for a respiratory event within 30 days of study entry (Screening visit)
    3) History of intolerance to inhaled short acting β2 agonists
    4) History of lung transplantation
    5) Administration of any investigational drug or device within 28 days prior to Screening
    6) Concurrent use of oral corticosteroids in doses exceeding the equivalent of 10 mg prednisone per day or 20 mg prednisone every other day
    7) Current requirement for daily continuous oxygen supplementation or requirement of more than 2 L/minute at night
    8) Hospitalization for a respiratory event within 30 days prior to Screening
    9) Changes in antimicrobial, bronchodilator, corticosteroid, dornase alfa, or hypertonic saline medications within 7 days prior to Screening
    10) Significant changes (per investigators discretion) in physiotherapy technique or schedule within 7 days prior to Screening
    11) Abnormal hepatic or renal function results at most recent test within the previous 12 months, defined as
    - AST or ALT >5 times upper limit of normal (ULN), or
    - Serum creatinine > 2 times ULN for age
    12) Presence of a condition or abnormality that would, in the opinion of the Investigator, compromise the subject’s safety or the quality of the study data
    13) Known hypersensitivity to aztreonam, its metabolites, or formulation excipients present in AZLI
    14) Respiratory cultures performed within 2 years prior to Screening that are positive for ANY Burkholderia spp. or NTM
    1) Uso de antibióticos contra Pseudomonas por vía IV o inhalados en los 2 años previos a la selección.
    2) Uso de antibióticos contra Pseudomonas por vía oral para un acontecimiento respiratorio ocurrido en los 30 días previos al inicio del estudio (visita de selección).
    3) Antecedentes de intolerancia a agonistas β2 de acción corta inhalados.
    4) Antecedentes de trasplante de pulmón
    5) Administración de cualquier fármaco o dispositivo de investigación dentro de los 28 días anteriores a la visita de seleccion.
    6) Uso simultáneo de corticosteroides orales en dosis superiores al equivalente de 10 mg de prednisona al día o 20 mg de prednisona cada dos días
    7) Necesidad actual de oxigenoterapia diaria continua o necesidad de más de 2 l/minuto por la noche
    8) Hospitalización por un acontecimiento respiratorio ocurrido en los 30 días previos a la selección
    9) Cambios en la medicación con antimicrobianos, broncodilatadores, corticosteroides, dornasa alfa o solución salina hipertónica en los 7 días previos a la selección.
    10) Cambios significativos (según el criterio del investigador) en la técnica o el calendario de fisioterapia en los 7 días previos a la selección.
    11) Resultados anormales de la función renal o hepática en el análisis más reciente realizado en los últimos 12 meses, definidos como:
    - AST o ALT > 5 veces el límite superior de la normalidad (LSN), o
    - Creatinina sérica > 2 veces el LSN para la edad.
    12) Presencia de un trastorno o alteración que, en opinión del investigador, comprometería la seguridad del paciente o la calidad de los datos del estudio.
    13) Hipersensibilidad conocida a aztreonam, sus metabolitos o los excipientes de la formulación en AZLI.
    14) Resultados positivos para CUALQUIER especie de Burkholderia o para micobacterias no tuberculosas (MNT) en cultivos respiratorios realizados en los 24 meses previos a la selección.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is:
    - The proportion of subjects with PA-negative cultures through 28 days post-treatment in the 14-day treatment group vs 28-day treatment group
    El objetivo principal de este estudio es :
    - Determinar la proporción de pacientes con erradicación de PA hasta 28 días después del tratamiento en el grupo de tratamiento de 14 días frente al grupo de tratamiento de 28 días
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 29
    Dia 29
    E.5.2Secondary end point(s)
    Time from primary eradication to PA recurrence over a 108-week post-treatment follow-up
    period
    - The proportion of subjects with PA-negative cultures through 28 days post-treatment in the 14-day treatment group vs historical pooled data for PA eradication at 28 days post-treatment in subjects treated with TNS
    - Time to PA recurrence for a sub-group of subjects matching the population in the TNS ELITE Study over a 108-week post-treatment follow-up period
    Tiempo desde la erradicación primaria hasta la recaída de PA durante el período de seguimiento de 108 semanas después del tratamiento.
    - Proporción de sujetos con cultivos PA negativos hasta 28 días después del tratamiento en el grupo de tratamiento de 14 días vs datos históricos agrupados para la erradicación de PA a los 28 días después del tratamiento en sujetos tratados con TNS
    - Tiempo hasta recurrencia de PA para un subgrupo de sujetos que coinciden con la población en el estudio TNS ELITE durante un período de seguimiento de 108 semanas después del tratamiento
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary objectives include the evaluations of the time to recurrence of PA from primary eradication over the 108-week post-treatment follow-up period. Primary eradication is defined as all PA-negative cultures through 28 days post AZLI treatment. Cultures will be obtained at week 16 and then at 12-week intervals during the follow-up time frame.
    Los objetivos secundarios incluyen las evaluaciones del tiempo desde la erradicación primaria hasta la recaída de PA durante el período de seguimiento de 108 semanas después del tratamiento. La erradicación primaria se define como todos los cultivos negativos para PA hasta 28 días después del tratamiento con AZLI. Los cultivos se obtendrán en la semana 16 y, posteriormente, a intervalos de 12 semanas durante el periodo de seguimiento.
    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 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 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.8.2.4Number of treatment arms in the trial2
    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 EEA46
    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
    Austria
    Belgium
    France
    Germany
    Greece
    Ireland
    Israel
    Italy
    Netherlands
    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
    Date of last study visit of last study subject
    Fecha de la última visita del último sujeto del estudio
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months11
    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: 140
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 28
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 56
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 56
    F.1.2Adults (18-64 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Infants and young children
    Lactantes y niños
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Women of childbearing potential using contraception
    Mujeres en edad fertil que usan métodos anticonceptivos
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 92
    F.4.2.2In the whole clinical trial 140
    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)
    After a patient has completed/terminated their study participation, long term care for the participant will remain the responsibility of their primary treating physicians.
    Después de que un sujeto haya completado/terminado la participación en el estudio, la responsabilidad del cuidado a largo plazo del participante seguirá siendo responsabilidad de su médico de atención primaria.
    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 Decision2017-08-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-07-12
    P. End of Trial
    P.End of Trial StatusOngoing
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