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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   41235   clinical trials with a EudraCT protocol, of which   6758   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


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    Summary
    EudraCT Number:2011-001362-18
    Sponsor's Protocol Code Number:GS-US-205-0160
    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:2011-12-21
    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 number2011-001362-18
    A.3Full title of the trial
    Open-Label Phase 3 Trial to Evaluate the Safety of Aztreonam 75 mg
    Powder and Solvent for Nebuliser Solution/Aztreonam for Inhalation Solution (AZLI) in Children with Cystic Fibrosis (CF) and Chronic Pseudomonas aeruginosa (PA) in the Lower Airways
    Estudio abierto en fase 3 para evaluar la seguridad de aztreonam 75 mg en polvo y disolvente para solución para inhalación por nebulizador / aztreonam para solución para inhalación (AZLI) en niños con fibrosis quística (FQ) e infección crónica por Pseudomonas aeruginosa (PA) en las vías respiratorias bajas.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the Safety of Aztreonam for Inhalation in Children with Cystic Fibrosis and Chronic Infection of the Airways by Pseudomonas aeruginosa bacteria
    Estudio de seguridad del aztreonam inhalado en niños/as con fibrosis quística e infección crónica por la la bacteria Pseudomonas Aeruginosa en las vías respiratorias.
    A.3.2Name or abbreviated title of the trial where available
    PALS (Pediatric Aztreonam Lysine Safety)
    PALS (Seguridad pediátrica del aztreonam lisina)
    A.4.1Sponsor's protocol code numberGS-US-205-0160
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01404234
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/117/2011
    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 Information
    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+441223897496
    B.5.5Fax number+441223897284
    B.5.6E-mailclinical.trials@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
    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.3.1Product nameAztreonam solución para inhalación
    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.3.9.1CAS number 78110-38-0
    D.3.9.2Current sponsor codeAZLI
    D.3.9.3Other descriptive nameN/A
    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 Yes
    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
    Cystic fibrosis and chronic infection of lower respiratory tract with
    Pseudomonas aeruginosa
    Fibrosis quística e infección crónica de las vías respiratorias bajas por Pseudomonas aeruginosa
    E.1.1.1Medical condition in easily understood language
    Cystic fibrosis and chronic lung infection with a bacterium (germ) called
    Pseudomonas aeruginosa
    Fibrosis quística e infección crónica pulmonar por la bacteria llamada
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10068288
    E.1.2Term Cystic fibrosis pulmonary exacerbation
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10070608
    E.1.2Term Infective pulmonary exacerbation of cystic fibrosis
    E.1.2System Organ Class 10021881 - Infections and infestations
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate safety of treatment with AZLI 75 mg 3 times daily (TID) for 3 courses of therapy (28 days on/28 days off) in female and male children less than 13 years of age with CF and chronic PA infection/colonization.
    Evaluar la seguridad del tratamiento con 3 dosis diarias (TID) de 75 mg de AZLI durante 3 ciclos de tratamiento (28 días de tratamiento / 28 días de reposo) en niños y niñas de menos de 13 años con FQ e infección / colonización crónica por PA.
    E.2.2Secondary objectives of the trial
    N/A
    N/A
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ? Aged 12 years or less.
    ? Diagnosis of CF as determined by the 1997 CF Consensus Conference criteria: sweat chloride level ? 60 mEq/L by quantitative pilocarpine iontophoresis; or a genotype with 2 identifiable mutations consistent with CF; or an abnormal nasal transepithelial potential difference (NPD), and 1 or more clinical features consistent with CF.
    ? Documented positive lower respiratory tract culture for PA at the screening visit plus 2 documented positive lower respiratory tract cultures for PA within 12 months prior to study entry (start of treatment).
    ? Clinically stable with no evidence of significant respiratory symptoms or, if obtained for clinical evaluation, no chest radiograph findings at screening that would require administration of IV antipseudomonal antibiotics, oxygen supplementation, or hospitalization.
    ? Niños o niñas con una edad máxima de 12 años.
    ? Diagnóstico de FQ determinado según los criterios de la Conferencia de Consenso sobre la Fibrosis Quística de 1997: Cloruro en el sudor ? 60 mEq/l en la prueba cuantitativa de iontoforesis con pilocarpina; o prueba anómala de la diferencia de potencial nasal transepitelial (NPD); o genotipo con 2 mutaciones identificables coherentes con un diagnóstico de FQ; y 1 o más de los rasgos clínicos coherentes con la FQ.
    ? Cultivo de las vías respiratorias bajas documentado como positivo para PA en la visita de selección más otros 2 cultivos documentados como positivos durante los 12 meses anteriores a la inclusión en el estudio.
    ? Clínicamente estable y sin signos de síntomas respiratorios importantes o, si se obtiene para una evaluación clínica, ausencia de hallazgos radiológicos en la selección que requiriesen la administración de antibióticos i.v. contra Pseudomonas, suplementación de oxígeno u hospitalización.
    E.4Principal exclusion criteria
    ? Use of IV or inhaled antipseudomonal antibiotics within 14 days of study entry (start of treatment).
    ? Presence of a condition or abnormality that would compromise the participant's safety or the quality of study data, in the opinion of the investigator.
    ? History of hypersensitivity/adverse reaction to aztreonam or beta- agonists.
    ? Use of any investigational drug within 30 days of study entry (start of treatment).
    ? Uso de antibióticos contra Pseudomonas inhalados o por vía intravenosa en los 14 días previos a la incorporación al estudio (inicio del tratamiento).
    ? Presencia de una afección o anomalía que, en opinión del investigador, ponga en peligro la seguridad del participante o la calidad de los datos del estudio.
    ? Historia de hipersensibilidad / reacción adversa al aztreonam o a los agonistas beta.
    ? Uso de cualquier fármaco en investigación durante los 30 días anteriores a la entrada en el estudio (inicio del tratamiento).
    E.5 End points
    E.5.1Primary end point(s)
    Percentage of subjects who have discontinued the study drug due to safety or tolerability reasons by Day 168.
    Porcentaje de sujetos al día 168 que hayan abandonado el tratamiento del estudio por motivos de seguridad o de tolerabilidad.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Between baseline (Day 1) and Day 168.
    Entre el comienzo del tratamiento (Día 1) y el día 168.
    E.5.2Secondary end point(s)
    For subjects aged 6 years or more:
    ? Change in FEV1 % predicted at end of each 28-day-on-treatment cycle of AZLI
    ? Change in CFQ-R respiratory symptoms scale (RSS) score at the end of each 28-day-on-treatment cycle of AZLI
    For all subjects:
    ? Change in PA sputum density at end of each 28-day-on-treatment cycle of AZLI
    ? Use of additional antipseudomonal antibiotics
    ? Number and duration of hospitalizations
    ? Number and time to PA exacerbations
    ? Study-drug induced bronchospasm
    ? Adverse event rates adjusted for study duration
    En pacientes ? 6 años de edad:
    ? Cambio en el VEF1 % estimado al final de cada ciclo de 28 días de tratamiento con AZLI.
    ? Cambio en la escala de síntomas (ESR) respiratorios del CFQ-R al final de cada ciclo de 28 días de tratamiento con AZLI.
    En todos los pacientes:
    ? Cambio en la densidad de PA en el esputo al final de cada ciclo de 28 días de tratamiento con AZLI.
    ? Utilización de antibióticos adicionales contra Pseudomonas.
    ? Número y duración de las hospitalizaciones.
    ? Número y tiempo transcurrido hasta las exacerbaciones de PA.
    ? Broncoespasmo inducido por el fármaco del estudio.
    ? Tasas de acontecimientos adversos ajustadas según la duración del estudio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    For subjects aged 6 years or more at Days 28, 84, and 140:
    ? Change from baseline in FEV1 % predicted
    ? Change from baseline in CFQ-R respiratory symptoms scale (RSS)
    score
    For all subjects at Days 28, 84 and 140:
    ? Change from baseline in PA sputum density
    For all subjects from baseline to end of treatment (Day 140):
    ? Study-drug induced bronchospasm
    For all subjects from screening to end of study (Day 168):
    ? Use of additional antipseudomonal antibiotics
    ? Number and duration of hospitalizations
    ? Number and time to PA exacerbations
    ? Adverse event rates adjusted for study duration
    En pacientes ? 6 años (días 28, 84 y 140):
    ? Cambio respecto al nivel inicial en el % predicho del VEF1
    ? Cambio respecto al nivel inicial en la puntuación en la escala de síntomas respiratorios de la CFQ-R.
    En todos los pacientes (días 28, 84 y 140):
    ? Cambio en la densidad de PA en el esputo
    En todos los pacientes desde el día 1 hasta el fin del tratamiento (día 140):
    ? Broncoespasmo inducido por el fármaco del estudio.
    En todos los pacientes desde la selección hasta el fin del estudio (día 168):
    ? Utilización de antibióticos adicionales contra Pseudomonas.
    ? Número y duración de las hospitalizaciones.
    ? Número y tiempo transcurrido hasta las exacerbaciones de PA.
    ? Tasas de acontecimientos adversos ajustadas según la duración 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 No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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.8.3 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 EEA21
    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
    France
    Germany
    Italy
    Poland
    Spain
    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
    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 Yes
    F.1.1Number of subjects for this age range: 60
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 48
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    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 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.3.3.6.1Details of subjects incapable of giving consent
    Infants and young children
    Bebés y niños.
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Adolescent girls of childbearing potential using contraception
    Chicas adolescentes potencialmente fértiles usando métodos anticonceptivos
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 60
    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)
    Upon completion of study participation, subjects will be referred back to the physician who referred them to the study for continuing care and treatment.
    Al final de su participación en el estudio, los sujetos serán referidos de nuevo al médico que les recomendó el estudio para continuar con su cuidado y tratamiento.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Cystic Fibrosis Foundation Therapeutics (CFFT) Therapeutics Development Network (TDN)
    G.4.3.4Network Country United States
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation European Cystic Fibrosis Society Clinical Trials Network (ECFS-CTN)
    G.4.3.4Network Country Denmark
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2012-01-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-04-03
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