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    Summary
    EudraCT Number:2011-003825-81
    Sponsor's Protocol Code Number:HGT-FIR-086
    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:2011-11-21
    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 number2011-003825-81
    A.3Full title of the trial
    A Multicenter, Open-Label, Non-Randomized Study to Assess the Pharmacokinetics, Tolerability, and Safety of a Single Subcutaneous Administration of Icatibant in Children and Adolescents with Hereditary Angioedema
    Un estudio multicéntrico, abierto, no aleatorizado para evaluar la farmacocinética, tolerabilidad y seguridad de una única administración subcutánea de Icatibant en niños y adolescentes con angioedema hereditario?
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study intended to measure in the bodies of children and adolescents how drug Icatibant is absorbed and removed after a single administration (dose). The children and adolescents in the study will all have the disease called Hereditary Angioedema (which causes abnormal swelling of the body), this is because the drug icatibant is intended to treat Hereditary Angiodema. The study will also look for side effects that may be due to the drug
    Un ensayo clínico para medir la absorción y eliminación del farmaco Icatibant tras una única administración (dosis) en niños y adolescentes Los niños y adolescentes tendrán la enfermedad denominada Angioedema Hereditario (la cual causa la inflamación abnormal del cuerpo) y es por ello que el farmaco Icatibant pretende tratar el Angioedema Hereditario. El estudio revisará los efectos adversos que puedan ser debidos al framaco.
    A.4.1Sponsor's protocol code numberHGT-FIR-086
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01386658
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/238/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorShire Orphan Therapies, 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 supportShire Orphan Therapies, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Orphan Therapies, Inc
    B.5.2Functional name of contact pointBradley Bloom
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number1617349 0200
    B.5.5Fax number1781482 2956
    B.5.6E-mailbbloom@shire.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 Firazyr (icatibant) 30 mg solution for injection in pre filled syringe
    D.2.1.1.2Name of the Marketing Authorisation holderShire Orphan Therapies GmbH
    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/3/03/133
    D.3 Description of the IMP
    D.3.1Product nameFirazyr (icatibant) 30 mg solution for injection in pre filled syringe
    D.3.2Product code JE049
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNicatibant
    D.3.9.1CAS number 138614-30-9
    D.3.9.2Current sponsor codeJE049
    D.3.9.3Other descriptive nameICATIBANT ACETATE
    D.3.9.4EV Substance CodeSUB29718
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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) 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
    Hereditary Angioedema
    Angioedema Hereditario
    E.1.1.1Medical condition in easily understood language
    Hereditary angioedema (HAE) is a rare genetic condition, where the body has local swellings in various body parts including the hands, feet, face and airway, (throat).
    Angioedema Hereditario (AH) es una enfermedad genetica rara en la cual se produce inflamaciones localizadas en varias partes del cuerpo, incluyendo las manos, pies, cara y vias aereas (garganta).
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level PT
    E.1.2Classification code 10019860
    E.1.2Term Hereditary angioedema
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate the PK, tolerability, and safety of a single SC dose of icatibant in children and adolescents with HAE during an acute HAE attack.
    Investigar la farmacocinética (FC), la tolerabilidad y la seguridad de una una dosis subcutánea (s.c.) de icatibant en niños y adolescentes con angioedema hereditario (AEH) durante una crisis aguda de AEH.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of a single SC dose of icatibant in children and adolescents with HAE.
    To evaluate levels of reproductive hormones after a single SC dose of icatibant in children and adolescents with HAE.
    To evaluate the continued safety of icatibant in pubertal/postpubertal children after
    repeat exposure.
    To evaluate reproductive hormone levels in pubertal/postpubertal children after repeat exposure.
    To evaluate the efficacy of icatibant in pubertal/postpubertal children after repeat exposure.
    Evaluar la eficacia de una única dosis s.c. de icatibant en niños y adolescentes con AEH.
    Evaluar los niveles de hormonas reproductivas tras una única dosis s.c. de icatibant en niños y adolescentes con AEH.
    Evaluar la seguridad continuada del icatibant en niños puberales / pospuberales tras una exposición repetida.
    Evaluar los niveles de la hormona reproductiva en niños puberales / pospuberales tras una exposición repetida.
    Evaluar la eficacia del icatibant en niños puberales / pospuberales tras una exposición repetida.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Two through 17 years of age, inclusive (ie, from the second birthday until the day prior to the eighteenth birthday) at the time of the subjects first attack.
    2. Documented diagnosis of HAE Type I or II. Diagnosis must be confirmed by documented immunogenic and/or functional C1-INH deficiency results (<50% of normal levels). Diagnosis may be on the basis of historic data or by diagnostic testing conducted at the time of screening.
    3. Informed consent (and subject assent as appropriate) signed by the subjects parent(s) or legal guardian(s).
    1. Dos a 17 años de edad, ambas edades incluidas (es decir, desde el segundo cumpleaños hasta el día antes de cumplir 18 años) en el momento en que se ha producido la primera crisis del paciente.
    2. Diagnóstico documentado de AEH tipo I o II. El diagnóstico debe ser confirmado mediante deficiencia inmunógena documentada (por debajo del límite de normalidad) y/o funcional (<50% de los niveles normales) de C1-INH. El diagnóstico puede realizarse a partir de datos históricos o pruebas de diagnóstico efectuadas en el momento de la selección.
    3. Consentimiento informado (y consentimiento del paciente según corresponda) firmado por los padres o tutores legales del paciente.
    E.4Principal exclusion criteria
    1. Diagnosis of angioedema other than HAE.
    2. Participation in another clinical study during the 30 days prior to treatment.
    3. Any known factor/disease that might interfere with the treatment compliance, study conduct or result interpretation.
    4. Congenital or acquired cardiac anomalies that interfere significantly with cardiac function.
    5. Treatment with ACE inhibitors within 7 days prior to treatment.
    6. Use of hormonal contraception within the 90 days prior to treatment.
    7. Androgen use (eg, stanozolol, danazol, oxandrolone, methyltestosterone, testosterone) within the 90 days prior to treatment.
    8. The subject is pregnant or breastfeeding.
    1.Diagnóstico de angioedema que no sea AEH.
    2.Participación en otro estudio clínico durante 30 días antes del tratamiento.
    3.Cualquier factor o enfermedad conocidos que puedan interferir con el cumplimiento del tratamiento, la realización del estudio o la interpretación del resultado.
    4.Anomalías cardíacas congénitas o adquiridas que interfieran significativamente con la función cardíaca.
    5.Tratamiento con inhibidores de la enzima de conversión de la angiotensina (ECA) durante 7 días antes del tratamiento.
    6.Uso de anticoncepción hormonal durante los 90 días antes del tratamiento.
    7.Uso de andrógenos (por ejemplo, estanozolol, danazol, oxandrolona, metiltestosterona o testosterona) durante 90 días antes del tratamiento.
    8.Paciente embarazada o en lactancia.
    E.5 End points
    E.5.1Primary end point(s)
    The PK profile of icatibant after a single SC injection in pediatric subjects treated for acute attacks of HAE.
    The tolerability and safety of SC icatibant as assessed by injection site reactions, AEs, vital signs, ECG recordings, physical examination, clinical laboratory parameters (serum chemistry [including liver function tests], hematology, urinalysis), reproductive hormone levels, and immunogenicity (presence of anti-icatibant antibodies).
    El perfil FC del icatibant tras una sola inyección s.c. en pacientes pediátricos tratados por crisis agudas de AEH.
    La tolerabilidad y la seguridad del icatibant s.c. que se evalúan a partir de las reacciones producidas en el lugar de la inyección, los acontecimientos adversos (AA), las constantes vitales, los registros de electrocardiograma (ECG), las exploraciones físicas, los parámetros de laboratorio clínicos (bioquímica [incluidas las pruebas funcionales hepáticas], hematología y análisis de orina), los niveles de la hormona reproductiva y la capacidad inmunógena (anticuerpos antiicatibant).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Post-Treatment Day 1 though Day 8
    Follow-up Day 9 through Day 90 (±7 days)
    Two additional icatibant-treated attacks for a total of 3 icatibant-treated attacks
    (Pubertal/postpubertal subjects only)
    Treatment Day 1
    Post-Treatment Day 1 though Day 8
    Follow-up Day 9 through Day 90 (±7 days)
    Día 1 al 8 tras el tratamiento.
    Seguimiento el día 9 al 90 (±7 días)
    Dos adiccionales crisis tartadas con icatibant para un total de 3 crisis tratadas con icatibant
    (Sujetos Puberales/postpuberales únicamente)
    Tratamiento día 1
    Día 1 al 8 tras el tratamiento.
    Seguimiento el día 9 al 90 (±7 días)
    E.5.2Secondary end point(s)
    For all subjects (2 to 17 years of age): investigator assessment and scoring of cutaneous, abdominal, and laryngeal symptoms of acute HAE attacks by an investigator-rated symptom score.
    - The time to onset of relief of symptoms, defined as the earliest time at which a 20% improvement is seen in the average post-treatment score with no worsening of any single component score.
    - The time to minimal symptoms, defined as the earliest time post-treatment when all symptoms are either mild or absent based on the investigator-rated symptom score.
    For subjects >=4 years of age only: subject self-assessment of HAE-related pain using the Faces Pain Scale-Revised (FPS-R). The time to onset of relief of symptoms, defined as the earliest time at which a 20% improvement is seen in the post-treatment score.
    For subjects <4 years of age only: investigator assessment of HAE-related pain using the Faces, Legs, Activity, Cry, and Consolability (FLACC) scale. The time to onset of relief of symptoms, defined as the earliest time at which a 20% improvement is seen in the total post-treatment score.
    The incidence of rescue medication use.
    The proportion of subjects with worsened intensity of clinical HAE symptoms between 2 and 4 hours after treatment with SC icatibant using investigator-rated symptom scores.
    Para todos los pacientes (de 2 a 17 años de edad): evaluación y puntuación por parte de un investigador de los síntomas cutáneos, abdominales y laríngeos de las crisis agudas de AEH a partir de una puntuación de los síntomas clasificada por un investigador.
    El tiempo que transcurre hasta el inicio del alivio de los síntomas, que se define como el momento inicial en el que se observa una mejoría de un 20% en la puntuación media después del tratamiento sin empeoramiento en la puntuación de ningún componente.
    - El tiempo que transcurre hasta que los síntomas son mínimos, que se define como el momento inicial después del tratamiento en el que todos los síntomas son leves o inexistentes, de acuerdo con la puntuación de los síntomas clasificada por el investigador.
    Para pacientes >=4 años de edad solo: autoevaluación por parte del paciente del dolor relacionado con el AEH mediante la escala de dolor de caras-revisada (FPS-R, Faces Pain Scale-Revised). El tiempo que transcurre hasta el inicio del alivio de los síntomas, que se define como el momento inicial en el que se observa una mejoría de un 20% en la puntuación después del tratamiento.
    Para pacientes <4 años de edad solo: evaluación por parte del investigador del dolor relacionado con el AEH mediante la escala de dolor FLACC (cara, piernas, actividad, llanto y consuelo). El tiempo que transcurre hasta el inicio del alivio de los síntomas, que se define como el momento inicial en el que se observa una mejoría de un 20% en la puntuación total después del tratamiento.
    La incidencia del uso de medicación de rescate.
    La proporción de pacientes en los que se observa una intensificación de los síntomas de AEH clínicos entre 2 y 4 horas después del tratamiento con icatibant s.c. de acuerdo con la puntuación de síntomas clasificada por el investigador.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Post-Treatment Day 1 though Day 8
    Follow-up Day 9 through Day 90 (±7 days)
    Two additional icatibant-treated attacks for a total of 3 icatibant-treated attacks
    (Pubertal/postpubertal subjects only)
    Treatment Day 1
    Post-Treatment Day 1 though Day 8
    Follow-up Day 9 through Day 90 (±7 days)
    Día 1 al 8 tras el tratamiento.
    Seguimiento el día 9 al 90 (±7 días)
    Dos adiccionales crisis tartadas con icatibant para un total de 3 crisis tratadas con icatibant
    (Sujetos Puberales/postpuberales únicamente)
    Tratamiento día 1
    Día 1 al 8 tras el tratamiento.
    Seguimiento el día 9 al 90 (±7 días)
    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 Yes
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Canada
    Germany
    Hungary
    Israel
    Italy
    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
    Once the sixteenth prepubertal subject and the twentieth pubertal/postpubertal subject has completed Day 90 follow-up after treatment for an initial attack, and the fifteenth pubertal/postpubertal subject has completed the Day 90 follow-up after the third and final treatment, the study will be closed.
    Una vez el decimosexto sujeto prepuberal y el vigésimo sujeto puberal/pospuberal ha completado el día 90 de seguimiento tras el tratamiento de la crisis inicial y el decimoquinto sujeto puberal/pospuberal ha completado el día 90 de seguimiento tras el tercer y ultimo tratamiento, el estudio será cerrado.
    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 months0
    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 months0
    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: 36
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 16
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2011-11-21. Yes
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 36
    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)
    The normal treatment will be used
    Tratamiento habitual
    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 Decision2012-03-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-03-06
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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