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    EudraCT Number:2013-000620-34
    Sponsor's Protocol Code Number:IGSC-1103
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-07-30
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2013-000620-34
    A.3Full title of the trial
    A Multicentre Phase III Study on the Efficacy, Safety and Pharmacokinetics of LFB-IgSC in Patients with Primary Immunodeficiency (PID) Syndromes
    Studio multicentrico di fase III sull’efficacia, la sicurezza e la farmacocinetica di LFB-IgSC in pazienti con sindrome da immunodeficienza primaria (PID)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial with a subcutaneous immunoglobulin (LFB-IgSC) to evaluate its efficacy, its safety and its behaviour in human blood in patients with Primary Immunodeficiency (PID) syndromes
    Uno studio clinico con immunoglobulina sottocutanea (LFB-IgSC) per valutare la sua efficacia, sicurezza e comportamento nel sangue umano in pazienti con sindrome da immunodeficienza primaria (PID)
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberIGSC-1103
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/305/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorLFB Biotechnologies
    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 supportLFB Biotechnologies
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLFB Biotechnologies
    B.5.2Functional name of contact pointClinical trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address3 avenue des Tropiques - BP 40305
    B.5.3.2Town/ cityCourtaboeuf Cedex
    B.5.3.3Post code91958
    B.5.4Telephone number+33169827010
    B.5.5Fax number+33169827272
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameHuman Normal Immunoglobulin for subcutaneous administration
    D.3.2Product code LFB-IgSC
    D.3.4Pharmaceutical form Solution for infusion
    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 INNHuman Normal immunoglobulin
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN
    D.3.9.4EV Substance CodeSUB14196MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    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
    Primary Immunofediciency (PID) syndromes
    Sindrome da Immunodeficienza primaria (PID)
    E.1.1.1Medical condition in easily understood language
    Primary immune deficiency (PID) diseases are a group of potentially serious disorders in which inherited defects in the immune system lead to increased infections.
    Le malattie da immunodeficienza primaria (PID) sono un gruppo di disordini potenzialmente seri nel quale i difetti ereditari del sistema immunitario portano all’aumento delle infezioni.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level HLT
    E.1.2Classification code 10036700
    E.1.2Term Primary immunodeficiency syndromes
    E.1.2System Organ Class 100000004870
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to assess the efficacy of LFB-IgSC.
    L’obiettivo primario dello studio è valutare l’efficacia di LFB-IgSC.
    E.2.2Secondary objectives of the trial
    There are two secondary objectives:
    - To assess the safety of LFB-IgSC,
    - To assess the pharmacokinetic profile of LFB-IgSC over a one-week injection interval.
    I due obiettivi secondari sono:
    - Valutare la sicurezza di LFB-IgSC;
    - Valutare il profilo farmacocinetico di LFB-IgSC per un totale di una settimana nel periodo che intercorre tra un'iniezione e l'altra.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female aged from 2 to 70 years old
    - Primary immunodeficiency syndrome with predominant antibody deficiency as: X-linked agammaglobulinaemia (XLA), Common variable immunodeficiency (CVID), Other PID syndrome in which the main immunological defect is deficiency in IgG production.
    - Stable IgG therapy for at least 5 months before the study, with a constant dose ranging from 0.2 to 0.8 g/kg per month and with regular intervals of 3 to 4 weeks for IVIg and one week for SCIg.
    - At least 2 documented serum IgG trough levels ≥ 5 g/l with the previous IgG dosage regimen.
    - For women of childbearing potential, negative blood pregnancy test at enrolment and agreement to use a medically-acceptable method of contraception throughout the study.
    1. Modulo di consenso informato datato e firmato dal paziente o dal rappresentante legale da ottenersi prima di qualsiasi procedura relativa allo studio e alla somministrazione del prodotto sperimentale.
    2. Pazienti di entrambi i sessi in età compresa tra 2 e 70 anni.
    3. Diagnosi di sindrome da immunodeficienza con prevalenza di deficienza anticorpale, definita come:
     Agammaglobulinemia legata al cromosoma X (XLA);
     Immunodeficienza comune variabile (CVID);
     Qualsiasi altra sindrome PID caratterizzata da deficit della produzione di immunoglobuline G (IgG) quale deficit immunologico principale.
    4. Essere stati sottoposti a terapia stabile con IgG per almeno 5 mesi prima dello studio, con dose costante tra gli 0,2 e gli 0,8 g/kg al mese, con intervalli regolari da 3 a 4 settimane per l’immunoglobulina a infusione endovenosa (IVIg) ed una settimana per quella a infusione sottocutanea (SCIg).
    5. Almeno 2 concentrazioni minime documentate di IgG seriche con valore ≥ 5 g/l sulla base del regime di dosaggio di IgG precedente.
    6. Per le donne in età fertile, esito negativo del test di gravidanza sul sangue al momento dell’arruolamento e consenso all’uso di metodi contraccettivi accettabili per l’intero corso dello studio.
    E.4Principal exclusion criteria
    - History of severe allergy or serious adverse reaction to any IVIg, SCIg or an excipient of LFB-IgSC.
    - Patient with known antibodies to IgA.
    - Glomerular filtration rate < 80 ml/min/1.73m2 measured according to the Modified Diet in Renal Disease (MDRD) calculation.
    - Progressive hepatic disease that could worsen during the study.
    - Refusal of PK study (for adults only).
    - History of cardiac ischemia, cardiac insufficiency, cerebral ischemia, stroke, thrombotic events or pulmonary embolism.
    - Any additional cause of immunodeficiency, other than a primary immunodeficiency, (such as acquired immunodeficiency or malignancy of lymphoid cells).
    - Allogenic haematopoietic stem cell transplantation.
    - Need for routine premedication before SCIg infusions (excluding dermal anaesthetics).
    - Need for long-term therapy with corticosteroids or prophylactic antibiotics during the study.
    - Immunosuppressive agents, including anti-CD20 antibodies, during the last 6 months.
    Anamnesi di allergia o gravi reazioni avverse a qualsiasi IVIg, SCIg o ad un eccipiente di LFB-IgSC;
    2. Pazienti con presenza nota di anticorpi contro l’immunoglobulina A (IgA);
    3. Velocità di filtrazione glomerulare <80 ml/min/1,73 m2, misurata in base al calcolo ottenuto mediante la formula MDRD (Modified Diet in Renal Disease, modifica della dieta nella malattia renale);
    4. Patologia epatica progressiva con rischio di peggioramento nel corso dello studio;
    5. Mancato consenso alla partecipazione allo studio farmacocinetico (solo per i pazienti adulti);
    6. Anamnesi di ischemia cardiaca, insufficienza cardiaca, ischemia cerebrale, ictus, eventi trombotici o embolia polmonare;
    7. Qualsiasi ulteriore causa di immunodeficienza, oltre all’immunodeficienza primaria, quali immunodeficienza acquisita o malignità delle cellule linfoidi;
    8. Trapianto allogenico di cellule staminali emapoietiche;
    9. Necessità di una premedicazione di routine prima delle infusioni SCIg, fatta eccezione per gli anestetici cutanei;
    10. Necessità di terapia a lungo termine a base di corticosteroidi o antibiotici profilattici nel corso dello studio;
    11. Impiego di agenti immunosoppressori, compresi gli anticorpi anti-CD20, nel corso degli ultimi 6 mesi;
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the number of serious bacterial infections (SBI) per patient and per year (annualized rate) as defined:
    - Bacteraemia or sepsis
    - Bacterial meningitis
    - Osteomyelitis / septic arthritis
    - Bacterial pneumonia
    - Visceral abscess
    L’endpoint primario consiste nel definire il numero di infezioni batteriche gravi (SBI) per paziente e per anno (tasso annualizzato) quali:
    - Batteriemia o sepsi;
    - Meningite batterica;
    - Osteomielite / artrite settica;
    - Polmonite batterica;
    - Ascesso viscerale.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the course of the study
    Durante il corso dello studio
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints
    - Rate of infections (serious or non-serious) per patient and per year
    - Number of days missed from work or school due to infections,
    - Number of days of hospitalization related to infection,
    - Number of days with use of antibiotics,
    - Number of fever episodes (>38°C) related to infections,
    - Total IgG trough levels will be assessed every month over a period of 6 months starting at week 21. These IgG trough levels will be compared to trough levels obtained with the previous immunoglobulin (IVIg or SCIg). In addition, distribution of IgG sub-classes will be described.
    Safety endpoints
    - Number of patients who have presented an AE and number of AEs:
    For all AEs reported during the study
    For IMP related AEs
    For infusional AEs i.e., AEs that begin during or within 72 hours after an infusion
    For local reactions to IMP
    For serious AEs
    - Percentage of site-infusions with site reaction
    - Changes in vital signs from before infusions to after infusions.
    Endpoint secondari di efficacia:
    -Tasso di infezioni gravi e non, per paziente e per anno;
    - Giorni di assenza dal lavoro o da scuola a causa delle infezioni;
    - Giorni di ricovero correlati all’infezione;
    - Giorni di assunzione degli antibiotici;
    - Numero di episodi febbrili (temperatura 38 °C) correlati alle infezioni;
    - I livelli totali delle concentrazioni minime di IgG verranno misurati mensilmente in un lasso di tempo di 6 mesi a partire dalla settimana 21. Tali livelli verranno confrontati con i livelli di concentrazioni minime ottenuti con le precedenti immunoglobuline ricevute dal paziente (IVIg o SCIg). Inoltre, verrà descritta la distribuzione delle sottoclassi di IgG.

    Endpoint di sicurezza:
    - Numero di pazienti che hanno sperimentato un evento avverso (AE) e numero di eventi avversi:
     Per tutti gli eventi avversi registrati durante lo studio;
     Per gli eventi avversi correlati al prodotto medicinale sperimentale;
     Per gli eventi avversi correlati all'infusione, vale a dire AE verificatisi durante l'infusione o nel corso delle 72 ore successive;
     Per le reazioni locali al farmaco sperimentale;
     Per gli eventi avversi gravi.
    -Percentuale delle infusioni per sito rispetto alle reazioni per sito;
    -Variazioni nei parametri vitali, prima e dopo le infusioni.
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Total IgG trough levels: every month over a period of 6 months starting at W21.
    - All other secondary endpoints: throughout the course of the study
    IgG totale attraverso i livelli: ogni mese per un periodo di 6 mesi iniziando a settimana 21
    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 No
    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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E. trial type description
    Phase III with first administration to humans
    Fase III con prima somministrazione negli umani
    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 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) 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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    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
    United Kingdom
    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
    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 months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    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: 25
    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. of subjects for this age range: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 13
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 50
    F.4.2.2In the whole clinical trial 55
    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)
    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 Decision2013-08-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-29
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2014-03-14
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