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    The EU Clinical Trials Register currently displays   42517   clinical trials with a EudraCT protocol, of which   7000   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2011-003825-81
    Sponsor's Protocol Code Number:HGT-FIR-086
    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:2012-03-19
    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 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
    Studio multicentrico, open-label, non randomizzato per la valutazione di farmacocinetica, tollerabilita' e sicurezza di una singola somministrazione sottocutanea di icatibant in bambini e adolescenti con angioedema ereditario.
    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 Angiodema 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
    Lo studio clinico ha lo scopo di conoscere come Icatibant viene assorbito ed eliminato dopo una singola somministrazione nell’organismo dei bambini e adolescenti. I bambini e gli adolescenti nello studio saranno affetti da AEE (che provoca un anomalo gonfiore del corpo),Icatibant ha lo scopo di trattare l’angioedema ereditario. Lo studio ha lo scopo di analizzare anche gli effetti collaterali che possono essere causati dal farmaco
    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 HUMAN GENETIC 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 number1 617 349 0200
    B.5.5Fax number1 781 482 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*SC 1SIR 30MG 3ML10MG/M
    D.2.1.1.2Name of the Marketing Authorisation holderSHIRE ITALIA SpA
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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.3.4Pharmaceutical form Solution for injection
    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 ACETATE
    D.3.9.1CAS number 138614-30-9
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameNA
    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 ereditario
    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).
    L'angioedema ereditario (HAE) è una rara malattia genetica, dove si manifesta una tumefazione locale in varie parti del corpo incluse le mani, i piedi, viso e delle vie aeree (gola)
    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 14.1
    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
    Studiare la farmacocinetica (PK), tollerabilità e sicurezza di una dose sottocutanea (SC) singola di icatibant nei bambini e negli adolescenti con angioedema ereditario (HAE) durante un attacco acuto della malattia
    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
    Valutare l'efficacia di una dose SC singola di icatibant in bambini e adolescenti con HAE. Valutare i livelli degli ormoni riproduttivi dopo una dose SC singola di icatibant in bambini e adolescenti con HAE
    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 subject's 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 subject's parent(s) or legal guardian(s).
    1.Età compresa fra 2 e 17 anni, estremi compresi (ossia dal compimento del secondo anno al giorno prima del compimento dei diciotto anni) al momento del primo attacco del paziente. 2.Diagnosi documentata di HAE tipo I o II. La diagnosi deve essere confermata mediante deficit C1-INH immunogenico documentato (al di sotto del limite inferiore a quello normale) e/o funzionale (&lt; 50% dei livelli normali). La diagnosi può essere fatta sulla base di dati storici o mediante test diagnostici condotti al momento dello screening. 3.Consenso informato (e assenso del paziente se pertinente) firmato dai genitori o dai tutori legali del paziente.
    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.Diagnosi di angioedema diverso da HAE. 2.Partecipazione ad altro studio clinico durante i 30 giorni precedenti il trattamento. 3.Qualsivoglia fattore/malattia noti che possano interferire con l'aderenza al trattamento, la conduzione dello studio e l'interpretazione dei risultati. 4.Anomalie cardiache congenite o acquisite che interferiscano in modo significativo con la funzionalità cardiaca. 5.Trattamento con inibitori dell'enzima di conversione dell'angiotensina (ACE) entro 7 giorni prima del trattamento. 6.Uso di contraccettivi ormonali entro i 90 giorni precedenti il trattamento. 7.Uso di androgeni (per es., stanozololo, danazolo, oxandrolone, metiltestosterone, testosterone) entro i 90 giorni precedenti il trattamento. 8.Paziente in stato di gravidanza o di allattamento al seno.
    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).
    Il profilo PK di icatibant dopo una singola iniezione SC nei pazienti pediatrici trattati per attacchi acuti di HAE. La tollerabilità e la sicurezza di icatibant SC valutate mediante reazioni al sito di iniezione, eventi avversi (AE), parametri vitali, elettrocardiogramma (ECG), esame fisico, parametri clinici di laboratorio (chimica sierica [compresi test di funzionalità epatica], ematologia, analisi delle urine), livelli degli ormoni riproduttivi e immunogenicità (anticorpi anti-icatibant).
    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)
    Post-trattamento Giorno 1 giorno al 8 °giorno Follow-up giorno 9 al giorno 90 (± 7 giorni) Due ulteriori trattamenti di un attacco per un totale di 3 trattamenti di un attacco (solamente per soggetti puberale/postpuberale) Trattamento giorno 1 Post-trattamento Giorno 1 giorno al 8 ° giorno Follow-up giorno 9 al giorno 90 (± 7 giorni)
    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 posttreatment 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 HAErelated 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 HAErelated 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.
    •Per tutti i pazienti (età da 2 a 17 anni): valutazione e scoring da parte dello sperimentatore dei sintomi cutanei, addominali e laringei negli attacchi acuti di HAE mediante punteggio dei sintomi attribuito dallo sperimentatore. -Momento di esordio o sollievo dei sintomi, definito come primo momento in cui è stato osservato un 20% di miglioramento del punteggio medio post-trattamento senza peggioramento di un componente singolo del punteggio. -Momento di presentazione di sintomi minimi, definito come primo momento post-trattamento in cui tutti i sintomi sono lievi o assenti sulla base di un punteggio dei sintomi attribuito dallo sperimentatore. •Solo per i pazienti di età ≥ 4 anni: autovalutazione del paziente del dolore correlato a HAE mediante Faces Pain Scale-Revised (FPS-R) (Scala delle facce [R]). Momento dell'inizio del sollievo dei sintomi, definito come primo momento in cui è stato evidenziato un 20% di miglioramento del punteggio post-trattamento. •Solo per i pazienti di età < 4 anni: valutazione da parte dello sperimentatore del dolore correlato a HAE mediante la scala FLACC (volto, gambe, attività, pianto e consolabilità). Momento dell'inizio del sollievo dei sintomi, definito come primo momento in cui è stato evidenziato un 20% di miglioramento del punteggio complessivo post-trattamento. •Incidenza dell'uso di farmaci di salvataggio. •Proporzione di pazienti con peggioramento dell'intensità dei sintomi clinici di HAE fra 2 e 4 ore dopo il trattamento con icatibant SC mediante punteggio dei sintomi attribuito dallo sperimentatore
    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)
    Post-trattamento Giorno 1 giorno al 8 °giorno Follow-up giorno 9 al giorno 90 (± 7 giorni) Due ulteriori trattamenti di un attacco per un totale di 3 trattamenti di un attacco (solamente per soggetti puberale/postpuberale) Trattamento giorno 1 Post-trattamento Giorno 1 giorno al 8 ° giorno Follow-up giorno 9 al giorno 90 (± 7 giorni)
    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 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 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
    Israel
    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
    Dopo che il 16 paz prepuberale e il 20 puberale/postpuberale avranno completato il FU 90 gg dal trattamento dell'attacco iniziale e il 15 paz puberale/postpuberale avrà completato il FU di 90 gg dopo il terzo trattamento lo studio sarà concluso
    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 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 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
    Sarà utilizzato il trattamento normale
    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 Decision2012-05-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-22
    P. End of Trial
    P.End of Trial StatusCompleted
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