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    The EU Clinical Trials Register currently displays   40628   clinical trials with a EudraCT protocol, of which   6628   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2015-003652-52
    Sponsor's Protocol Code Number:991
    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:2016-07-15
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2015-003652-52
    A.3Full title of the trial
    An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID)
    Estudio multicéntrico, prospectivo, sin enmascaramiento de la seguridad, la eficacia clínica y las propiedades farmacocinéticas de la inmunoglobulina humana normal para la administración intravenosa de BT595 como tratamiento de restitución en pacientes con inmunodeficiencia primaria (IP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open label, prospective, multicenter study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT595 as replacement therapy in patients with primary immunodeficiency disease (PID)
    Estudio multicéntrico, prospectivo, sin enmascaramiento de la seguridad, la eficacia clínica y las propiedades farmacocinéticas de la inmunoglobulina humana normal para la administración intravenosa de BT595 como tratamiento de restitución en pacientes con inmunodeficiencia primaria (IP)
    A.4.1Sponsor's protocol code number991
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBiotest AG
    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 supportBiotest AG
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiotest AG
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street AddressLandsteinerstraße 5
    B.5.3.2Town/ cityDreieich
    B.5.3.3Post code63303
    B.5.4Telephone number+4961038015127
    B.5.5Fax number+496103801341
    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 nameIgG Next Generation
    D.3.2Product code BT595
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeBT595
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN (IV)
    D.3.9.4EV Substance CodeSUB12041MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    Replacement therapy in patients with primary immunodeficiency disease (PID)
    Tratamiento de restitución en pacientes con inmunodeficiencia primaria (IP)
    E.1.1.1Medical condition in easily understood language
    Replacement therapy in patients in which the body does not produce enough of the protein called immunoglobulins or antibodies
    Tratamiento de restitución en pacientes cuyo cuerpo no produce suficientes proteinas llamadas inmunoglobulinas o anticuerpos
    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 19.0
    E.1.2Level PT
    E.1.2Classification code 10064859
    E.1.2Term Primary immunodeficiency syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 this study is to demonstrate that the rate of acute serious bacterial infections (i.e., the mean number of acute serious bacterial infections per subject year) is less than 1.0 to provide substantial evidence of efficacy.
    El objetivo principal de este estudio es demostrar que la tasa de infecciones bacterianas agudas graves (por ejemplo, el número medio de infecciones bacterianas agudas graves por año sujeto) es inferior a 1,0 para proporcionar una prueba sustancial de la eficacia.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study, in addition to further efficacy assessments, are to evaluate the safety and pharmacokinetic characteristics of BT595.
    Los objetivos secundarios de este estudio, además de llevar a cabo más evaluaciones sobre la eficacia, son evaluar la seguridad y las características farmacocinéticas del BT595.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a) Written informed consent/assent obtained from subjects/subjects’ parent(s) or legally acceptable representative indicating that they understand the purpose of and procedures required for the study and are willing to participate in it.
    b) Male or female, aged 2 through 75 years.
    c) Diagnosis of PID with impaired antibody production, i.e.:
    - Diagnosis of common variable immunodeficiency (CVID) as defined by the European Society for Immunodeficiencies (ESID)/Pan American Group for Immunodeficiency (PAGID) diagnostic criteria.
    - X linked agammaglobulinemia (XLA) as defined by ESID/PAGID diagnostic criteria.
    d) Established replacement therapy with any intravenous immunoglobulin (IVIg) reference preparation during the previous 6 months, including documentation of immunoglobulin G (IgG) trough levels.
    e) Established replacement therapy with a single IVIg reference preparation for at least 3 months prior to treatment start with BT595 at a 3 or 4 week schedule with a constant IVIg dose that did not change by ±20% of the mean dose, regular dosage intervals, and at least 1 IgG trough level of ≥5 g/L during the previous 3 months.
    a) Obtener el consentimiento informado o el asentimiento por escrito de los sujetos, sus progenitores o el representante legal autorizado que indica que entienden el propósito y los procedimientos necesarios para el estudio y que están dispuestos a participar en él.
    b) Hombres y mujeres de 2 a 75 años de edad.
    c) Diagnóstico de inmunodeficiencia primaria con producción deficiente de anticuerpos, por ejemplo:
    - Diagnóstico de inmunodeficiencia común variable (IDCV), de acuerdo a los criterios diagnósticos de la Sociedad Europea para Inmunodeficiencias (ESID) o del Grupo Panamericano para Inmunodeficiencias (PAGID).
    O bien
    - Agammaglobulinemia ligada al cromosoma X (ALX) de acuerdo a los criterios diagnósticos de ESID o PAGID.
    d) Tratamiento de restitución establecido con cualquier preparado de referencia de inmunoglobulina por vía intravenosa (IgIV) durante los seis meses previos, incluida la documentación de los niveles mínimos de inmunoglobulina G (IgG).
    e) Tratamiento de restitución establecido con un solo preparado de referencia de IgIV durante como mínimo tres meses antes de iniciar el tratamiento con BT595 según un calendario de tratamiento de 3 o 4 semanas a una dosis constante de IgIV, que no ha cambiado en un ±20 % de la dosis media, a un intervalo posológico regular y al menos un nivel mínimo de 1 IgG ≥5 g/l durante los tres meses previos.
    E.4Principal exclusion criteria
    a) Pregnancy or unreliable contraceptive measures or lactation period (females only).
    b) Known intolerance to immunoglobulins or comparable substances (e.g., vaccination reaction).
    c) Known intolerance to proteins of human origin or known allergic reactions to components of the study product.
    d) Participation in another clinical study within 30 days before entering the study or during the study and/or previous participation in this study.
    e) Employee or direct relative of an employee of the Contract Research Organization, the study site, or Biotest.
    f) Acquired medical conditions known to cause secondary immune deficiency, such as chronic lymphatic leukemia, lymphoma, multiple myeloma, as well as protein losing enteropathies and hypoalbuminemia.
    g) Other medical condition, laboratory finding, or physical examination finding that precludes participation.
    h) Recent febrile illness that precludes or delays participation.
    i) Active infection and receiving antibiotic therapy for the treatment of this infection at the time of screening. Note: if the subject is deemed to be a screen failure due to a nonserious active infection requiring antibiotic therapy, the subject may be rescreened after the initial screening.
    j) Therapy with systemic steroids or other immunosuppressant drugs at the time of enrollment (current daily use of corticosteroids, i.e., >10 mg prednisone equivalent/day for >30 days. Intermittent corticosteroid use during the study is allowable, if medically necessary).
    k) History of thrombotic events (including myocardial infarction, cerebral vascular accident [including stroke], pulmonary embolism, and deep vein thrombosis) within the 6 months before treatment start with BT595 or the presence of significant risk factors for thrombotic events.
    l) Therapy with live attenuated virus vaccines within 3 months before start of the study.
    m) Selective, absolute immunoglobulin A (IgA) deficiency or known antibodies to IgA.
    n) Positive diagnosis of hepatitis B or hepatitis C.
    o) Positive HIV test.
    p) History of drug or alcohol abuse within the 12 months before treatment start with BT595.
    q) Inability or lacking motivation to participate in the study.
    a) Embarazo, métodos anticonceptivos no fiables o estar en periodo de lactancia (solo las mujeres).
    b) Intolerancia conocida a las inmunoglobulinas o sustancias comparables (por ejemplo, reacción a la vacuna).
    c) Intolerancia conocida a las proteínas de origen humano o reacciones alérgicas conocidas a los componentes del producto en estudio.
    d) Participar en otro estudio clínico en los 30 días previos a la inclusión en el estudio o durante el estudio o haber participado previamente en este estudio.
    e) Ser empleado o familiar directo de algún empleado de la organización de investigación por contrato, del centro del estudio o de Biotest.
    f) Enfermedades adquiridas que se sabe que causan inmunodeficiencias secundarias tales como leucemia linfática crónica, linfoma, mieloma múltiple, así como hipoalbuminemia y enteropatías con pérdida de proteínas.
    g) Otras enfermedades, hallazgos de laboratorio o hallazgos en la exploración física que excluyen la participación.
    h) Enfermedad febril reciente que excluye o retrasa la participación.
    i) Infección activa tratada con un tratamiento antibiótico durante la fase de selección. Nota: si el sujeto se considera no apto a causa de una infección activa leve que requiere tratamiento antibiótico, puede volver a pasar el proceso de selección una vez finalizada la selección inicial.
    j) Terapia con esteroides sistémicos u otros fármacos inmunodepresores durante la fase de inclusión en el estudio (consumo actual diario de corticoesteroides, por ejemplo >10 mg de prednisona al día o equivalente durante >30 días. Se admite la administración intermitente de corticoesteroides durante el estudio si es necesario desde el punto de vista médico).
    k) Antecedentes de episodios trombóticos (como infarto de miocardio, accidente cerebrovascular [incluido el ictus], embolia pulmonar y trombosis venosa profunda) en los seis meses previos al inicio del tratamiento con BT595 o la presencia de importantes factores de riesgo que favorezcan episodios trombóticos.
    l) Terapia con vacunas elaboradas con microbios vivos atenuados en los tres meses previos al inicio del estudio.
    m) Déficit absoluto y selectivo de inmunoglobulina A (IgA) o de anticuerpos conocidos frente a IgA.
    n) Diagnóstico definitivo de hepatitis B o hepatitis C.
    o) Resultado positivo en la prueba del VIH.
    p) Antecedentes de abuso de drogas o alcohol en los doce meses previos al inicio del tratamiento del estudio con BT595.
    q) Incapacidad o falta de motivación para participar en el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Rate of acute serious bacterial infections
    (i.e., the mean number of acute serious bacterial infections per subject year).
    Acute serious bacterial infections include:
    • Bacteremia or sepsis
    • Bacterial meningitis
    • Osteomyelitis/septic arthritis
    • Bacterial pneumonia
    • Visceral abscess
    Tasa de infecciones bacterianas agudas graves
    (por ejemplo, el número medio de infecciones bacterianas agudas graves por año sujeto).
    Las infecciones bacterianas agudas graves incluyen:
    • Bacteriemia o sepsis
    • Meningitis bacteriana
    • Osteomielitis/ artritis séptica
    • Neumonía bacteriana
    • Absceso visceral
    E.5.1.1Timepoint(s) of evaluation of this end point
    after 12 months/per subject
    después de 12 meses/por sujeto
    E.5.2Secondary end point(s)
    • IgG trough levels (total IgG) before each infusion
    • Rate of any infections (number per year)
    • Rate of nonserious infections (number per year)
    • Time to resolution of infections
    • Antibiotic treatment (number of days antibiotic treatment received per month)
    • Rate of time lost from school/work due to infections (number of days per month) and their treatment (number of days treatment per month)
    • Hospitalization (number of days per month overall and due to infection)
    • Fever episodes (number of days per year)
    • Changes in health-related quality of life: Pediatric subjects (2 17 years, inclusive) will complete the Pediatric Quality of Life (Peds QL™) Measurement Model (child self report and/or parent proxy report). Additionally, all adult subjects will complete the EuroQol Five Dimension (EQ 5D 3L™) Health Questionnaire and pediatric subjects (4 17 years, inclusive) will complete the youth version of the EQ 5D™ (EQ 5D Y™) Health Questionnaire (child self report or proxy report).
    • Niveles mínimos de IgG (IgG total) antes de cada infusión
    • Tasa de cualquier infección (número por año)
    • Tasa de infecciones leves (número por año)
    • Tiempo de resolución de las infecciones
    • Tratamiento antibiótico (número de días al mes de tratamiento antibiótico)
    • Tasa de tiempo perdido de escolarización o trabajo a causa de las infecciones (número de días al mes) y su tratamiento (número de días de tratamiento al mes)
    • Hospitalización (número total de días al mes y a causa de la infección)
    • Episodios febriles (número de días al año)
    • Cambios en la calidad de vida relacionada con la salud: Los sujetos pediátricos (2-17 años, inclusive) completarán el modelo de medición de la calidad de vida pediátrica (Peds QL™) (cumplimentado por el niño y/o por sus progenitores). Además, todos los sujetos adultos cumplimentarán el cuestionario sobre la salud EuroQol de cinco dimensiones (EQ 5D 3L™) y los sujetos pediátricos (4-17 años, inclusive) la versión juvenil del cuestionario sobre la salud EQ 5D™ (EQ 5D Y™) (cumplimentado por el niño y/o por sus progenitores).
    E.5.2.1Timepoint(s) of evaluation of this end point
    after 12 months/per subject
    después de 12 meses/por sujeto
    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. 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 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Russian Federation
    United Kingdom
    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
    Última Visita del Ultimo Paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months22
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months22
    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: 20
    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: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 35
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    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)
    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 Decision2016-09-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-09-13
    P. End of Trial
    P.End of Trial StatusCompleted
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