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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   42567   clinical trials with a EudraCT protocol, of which   7008   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:2011-003825-81
    Sponsor's Protocol Code Number:HGT-FIR-086
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-11-08
    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 ConcernedGermany - BfArM
    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
    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

    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/142/2013
    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 pointKevin Anderson
    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 number1781482 9652
    B.5.6E-mailkanderson@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
    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).
    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 16.0
    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
    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 the effect on reproductive hormone levels 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 repeated exposures.
    - To evaulate the efficacy of icatibant in the treatment of acute HAE attacks in pubertal/postpubertal children after repeated exposures.
    - To evaluate the effect on reproductive hormone levels in pubertal/postpubertal children after repeated exposure.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Two to <18 years of age (ie, from the second birthday through the day prior to the eighteenth birthday) at the time of enrollment and treatment with icatibant.
    - Prepubertal and pubertal/postpubertal subjects experiencing an acute cutaneous, abdominal, or laryngeal HAE attack treated with icatibant as part of this study.
    - Pubertal/postpubertal subjects with HAE who are treated with icatibant as part of this study, but not during an attack.
    2. Documented diagnosis of HAE type I or II. Diagnosis may be on the basis of historical data: family history, characteristic attack manifestations, recurrent attacks, historical C1-INH deficiency, and exclusion of other forms of angioedema. Inclusion will be permitted initially based on medical history only if a clear diagnosis has been made based on all of the preceding criteria. However, the diagnosis must be confirmed prior to treatment by documented immunogenic and/or functional C1-INH deficiency (C1-INH protein level below the lower limit of normal and/or functional level <50% of normal) as performed by the Sponsor’s central laboratory.
    3. Informed consent (and subject assent as appropriate) signed by the subject’s parent(s) or legal guardian(s).
    E.4Principal exclusion criteria
    1. Diagnosis of angioedema other than HAE.
    2. Participation in another clinical trial that involves use of any investigational product (drug or device) within 30 days prior to study enrollment or at any time during the study.
    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 angiotensin converting enzyme (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. Pregnancy or breastfeeding.
    9. A physical condition that interferes with pubertal status determination.
    E.5 End points
    E.5.1Primary end point(s)
    The PK profile of icatibant after a single SC injection in pediatric subjects (in prepubertal children with an acute attack of HAE and pubertal/postpubertal children with or without an acute attack 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).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Post-Treatment Day 1 through Day 8 (±1 days)
    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 through Day 8 (±1 days)
    Follow-up Day 9 through Day 90 (±7 days)
    E.5.2Secondary end point(s)
    Only for subjects treated with icatibant during an HAE attack

    Time to onset of relief of symptoms and time to minimal symptoms, as measured by investigator- and subject-reported outcomes
    • For subjects 2 to less than 18 years of age: investigator assessment and scoring of cutaneous, abdominal and laryngeal symptoms of acute HAE attacks by an investigator-rated symptom score.
    • For subjects 4 years of age and older: subject self-assessment of HAE related pain using the Faces Pain Scale-Revised (FPS-R).
    • For subjects less than 4 years of age: investigator assessment of HAE-related pain (cutaneous, abdominal and laryngeal) using a validated pain scale (Faces, Legs, Activity, Cry, and Consolability [FLACC].
    Incidence of rescue medication use.
    Proportion of subjects with worsened intensity of clinical HAE symptoms between 2 and 4 hours after treatment with icatibant.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Post-Treatment Day 1 through Day 8 (±1 days)
    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 through Day 8 (±1 days)
    Follow-up Day 9 through Day 90 (±7 days)
    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 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 concerned2
    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 tenth prepubertal subject and the twentieth pubertal/postpubertal subject have completed the 6-month follow-up after the initial treatment, and the tenth pubertal/postpubertal subject has completed the 6-month follow-up after his or her third and final treatment for an attack, the study will be closed.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months3
    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: 30
    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: 10
    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 2011-11-08. 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Paediatric subjects aged from 2 to 17 years
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    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
    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 Decision2011-12-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-03-12
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