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    Summary
    EudraCT Number:2010-023909-35
    Sponsor's Protocol Code Number:BVT.BSSL-030
    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:2011-11-18
    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 number2010-023909-35
    A.3Full title of the trial
    A Prospective, Randomized, Double-Blind, Phase 3 Study Comparing rhBSSL and Placebo Added to Infant Formula or Pasteurized Breast Milk During 4 Weeks of Treatment in Preterm Infants Born Before Week 32 of Gestational Age
    Estudio de fase 3 prospectivo, aleatorizado, doble ciego para comparar rhBSSL y placebo añadidos al preparado para lactantes o a la leche materna pasteurizada durante 4 semanas de tratamiento administrado a lactantes prematuros nacidos antes de la semana 32 de gestación.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 3 clinical study investigating the effects of rhBSSL (an enzyme to help digestion of fat) added to pasteurized breast milk or formula in babies born prematurely.
    Un estudio de fase 3 para investigar los efectos de rhBSSL (una enzima que ayuda a la digestión de la grasa) añadida al preparado para lactantes o a la leche materna pasteurizada administrado a lactantes prematuros
    A.3.2Name or abbreviated title of the trial where available
    BVT.BSSL-030
    A.4.1Sponsor's protocol code numberBVT.BSSL-030
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/62/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSwedish Orphan Biovitrum AB (publ)
    B.1.3.4CountrySweden
    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 supportSwedish Orphan Biovitrum AB (publ)
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPPD
    B.5.2Functional name of contact pointMattias Oleszewski
    B.5.3 Address:
    B.5.3.1Street AddressStephanienstraße 55
    B.5.3.2Town/ cityKarlsruhe
    B.5.3.3Post code76133
    B.5.3.4CountryGermany
    B.5.4Telephone number496233 6070194
    B.5.5Fax number496233 6070195
    B.5.6E-mailMatthias.Oleszewski@ppdi.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 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 nameLipasa estimulada por sales biliares humana recombinante
    D.3.2Product code rhBSSL
    D.3.4Pharmaceutical form Powder for oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNalfa bucelipasa
    D.3.9.2Current sponsor coderhBSSL
    D.3.9.3Other descriptive namelipasa estimulada por sales biliares humana recombinante (rhBSSL)
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8700
    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.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 Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for oral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Prevention of growth retardation due to lack of bile salt-stimulated lipase in enteral nutrition
    Prevención del retraso del crecimiento debido a la carencia de lipasa estimulada por sales biliares en la nutrición enteral.
    E.1.1.1Medical condition in easily understood language
    Babies born prematurely.
    Recién nacido prematuro
    E.1.1.2Therapeutic area Body processes [G] - Digestive System and Oral Physiological Phenomena [G10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level PT
    E.1.2Classification code 10036590
    E.1.2Term Premature baby
    E.1.2System Organ Class 10036585 - Pregnancy, puerperium and perinatal conditions
    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 rhBSSL improves growth in preterm infants as compared with placebo when administered in infant formula or PBM.
    El objetivo principal de este estudio es demostrar que la rhBSSL mejora el crecimiento en lactantes prematuros en comparación con placebo cuando se administra al preparado para lactantes o LMP.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are
    - To determine the effect of rhBSSL treatment in shortening the time of hospital stay
    - To determine the effect of rhBSSL treatment in improving early development
    - To determine the effect of rhBSSL treatment in decreasing readmittance to hospital
    - To determine the effect of rhBSSL treatment in increasing the levels of DHA and AA
    - To compare the safety and tolerability of rhBSSL treatment with that of placebo treatment
    Los objetivos secundarios de este estudio son: - Determinar el efecto del tratamiento con rhBSSL en la reducción del tiempo de hospitalización - Determinar el efecto del tratamiento con rhBSSL en la mejoría del desarrollo inicial - Determinar el efecto del tratamiento con rhBSSL para disminuir el reingreso en el hospital - Determinar el efecto del tratamiento con rhBSSL en el aumento de las concentraciones de ADH y AA - Comparar la seguridad y la tolerabilidad del tratamiento con rhBSSL y el tratamiento con placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Preterm infant born before Week 32 of gestation.
    2. Preterm infant who is <33 weeks postmenstrual age at the time of randomization.
    3. Preterm infant who is AGA or SGA at birth.
    4. Preterm infant who is receiving food enterally (bottle or gavage tube) at a level of at least 100 mL/kg/day at randomization.
    5. Preterm infant whose enteral feeding consists of only infant formula or only PBM at the time of inclusion, and who are expected to remain on only infant formula for 4 weeks, or only PBM for at least 2 weeks following treatment initiation.
    6. Preterm infant who is expected not to receive any fresh breast milk for 4 weeks following treatment initiation.
    7. Informed consent is obtained from the patient's legally acceptable representative. Every effort will be made to ensure informed consent is obtained from both parents. Where this is not possible, the local regulations will be followed in the definition of legally acceptable representative.
    1. Lactante prematuro nacido antes de la semana 32 de gestación. 2. Lactante prematuro con una edad posmenstrual <33 semanas en el momento de la aleatorización. 3. Lactante prematuro que es PEG o AEG al nacer. 4. Lactante prematuro que recibe al menos 100 ml/kg/día de alimentación enteral (por biberón o sonda) en el momento de la aleatorización. 5. Lactante prematuro cuya alimentación enteral consiste únicamente en preparado para lactantes o LMP en el momento de la inclusión y que se prevé que seguirá con preparado para lactantes solo durante 4 semanas, o con LMP sola durante al menos 2 semanas tras el inicio del tratamiento. 6. Lactante prematuro que no se espera que reciba leche materna fresca durante las 4 semanas siguientes al inicio del tratamiento. 7. El consentimiento informado se obtendrá del representante legal del paciente. Se hará todo lo posible para conseguir obtener el consentimiento informado de los dos padres. Si no fuera posible, se seguirá la normativa local en la definición de representante legal.
    E.4Principal exclusion criteria
    1. Expected stay at the hospital is less than 4 weeks from the first dose of study drug.
    2. Currently receiving mechanical ventilation via endotracheal tube (continuous positive airway pressure [CPAP] or high-flow nasal cannula are not criteria for exclusion).
    3. Require 30% oxygen (if on CPAP or in head box) or >0.5 L/min oxygen.
    4. Evidence of severe brain disease or damage, including grade III or IV peri- or intraventricular hemorrhage, meningitis or hydrocephalus, grade III or IV intracranial hemorrhage, or periventricular leukomalacia.
    5. Presence of major dysmorphology or congenital abnormalities that are likely to affect growth and/or development.
    6. Current clinical evidence of hemodynamically significant persistent ductus arteriosus.
    7. Clinical evidence of sepsis (including low or high white blood cell count and/or low platelet count and bacteriologically proven evidence of systemic infection). This should be based on the investigator's opinion and available local laboratory reference ranges. Patients should not have received antibiotics in preceding 48 hours except where these are being administered for prophylaxis as per routine unit protocols (eg, antifungal prophylaxis with fluconazole).
    8. Evidence of congenital infection (eg, cytomegalovirus).
    9. Previous or current diagnosis of necrotizing enterocolitis (Bell's stage 2 or greater).
    10. Prior or current treatment with corticosteroids, except hydrocortisone.
    11. Presence of any condition that in the opinion of the investigator makes the patient unsuitable for inclusion.
    12. Enrolled in another concurrent clinical intervention study.
    1. La estancia prevista en el hospital es inferior a 4 semanas desde la primera dosis del fármaco del estudio.
    2. Ventilación mecánica actual por sonda endotraqueal (la presión positiva continua en las vías respiratorias [PPCVR] o la cánula nasal de alto flujo no son criterios de exclusión).
    3. Necesidad de oxígeno 30% (si está con PPCVR o campana) o de >0,5 l/min de oxígeno.
    4. Signos de enfermedad o daño cerebral grave, incluidas hemorragia peri o interventricular de grado III o IV, meningitis o hidrocefalia, hemorragia intracraneal de grado III o IV o leucomalacia periventricular.
    5. Presencia de malformaciones o anomalías congénitas que probablemente afecten al crecimiento, al desarrollo o a ambos.
    6. Signos clínicos actuales de conducto arterioso persistente de importancia hemodinámica.
    7. Signos clínicos de sepsis (incluido recuento leucocitario bajo o alto y/o recuento plaquetario bajo y pruebas bacteriológicas de infección sistémica). Esto debe basarse en la opinión del investigador y en los intervalos de referencia del laboratorio local disponibles. Los pacientes no deben haber recibido antibióticos en las 48 horas precedentes, a menos que se administren como profilaxis de acuerdo con los protocolos habituales de la unidad (p. ej., profilaxis antimicótica con fluconazol).
    8. Signos de infección congénita (p. ej., citomegalovirus).
    9. Diagnóstico previo o actual de enterocolitis necrosante (estadio 2 de Bell o superior).
    10. Tratamiento previo o actual con corticosteroides, excepto hidrocortisona.
    11. Presencia de cualquier circunstancia que haga inadecuada la inclusión del paciente en opinión del investigador.
    12. Participación en otro estudio de intervención clínica concomitante.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy variable is growth velocity in grams per kilogram per day during 4 weeks of treatment.
    La variable de eficacia principal es la velocidad de crecimiento en gramos por kilogramo al día durante 4 semanas de tratamiento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and weekly to discharge then 3 and 12 month follow-up
    basal y a la semana del alta, luego seguimiento a los 3 y 12 meses.
    E.5.2Secondary end point(s)
    Change from Baseline in body weight (g) at 3 months
    Body weight (g) at 12 months' corrected age
    Change from Baseline in total body length (mm) at 4 weeks and 3 months
    Body length (mm) at 12 months' corrected age
    Time to readiness for discharge
    Time to discharge
    Change from Baseline in head circumference (mm) at 4 weeks and 3 months
    Head circumference (mm) at 12 months' corrected age
    Time from Baseline to 150 mL/kg/day of enteral feeding
    Readmission to hospital within 1 month of discharge
    Levels of DHA and AA at 4 weeks
    Adverse events (AEs)
    Physical examination
    Bayley Scale of Infant and Toddler Development III (cognitive)
    Bayley Scale of Infant and Toddler Development III (language)
    Bayley Scale of Infant and Toddler Development III (motor)
    Vomiting (frequency and volume)
    Vital signs (heart rate, blood pressure, body temperature)
    Laboratory variables amylase, bilirubin, aminotransferases, sodium and urea
    Levels of vitamin A and D
    Levels of rhBSSL antibodies
    - Cambio del peso corporal (g) respecto al basal a los 3 meses
    - Peso corporal (g) a una edad corregida de 12 meses
    - Cambio de la longitud corporal total (mm) respecto a la basal a las 4 semanas y los
    3 meses
    - Longitud corporal (mm) a una edad corregida de 12 meses
    - Tiempo hasta estar en disposición de recibir el alta
    - Tiempo hasta el alta
    - Cambio del perímetro cefálico (mm) respecto al basal a las 4 semanas y los 3 meses
    - Perímetro cefálico (mm) a una edad corregida de 12 meses
    - Tiempo desde el momento basal hasta la toma de 150 ml/kg/día de alimentación enteral
    - Reingreso en el hospital en el mes siguiente al alta
    - Niveles de ADH y AA a las 4 semanas
    - Acontecimientos adversos (AA).
    - Exploración física
    - Escala de Bayley de desarrollo infantil III (cognitivo)
    - Escala de Bayley de desarrollo infantil III (lenguaje)
    - Escala de Bayley de desarrollo infantil III (motor)
    - Vómitos (frecuencia y volumen)
    - Constantes vitales (presión arterial, frecuencia cardiaca, temperatura corporal)
    - Variables de laboratorio: amilasa, bilirrubina, aminotransferasas, sodio y urea
    - Concentraciones de vitamina A y D
    - Niveles de anticuerpos anti-rhBSSL
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline and weekly to discharge then 3 and 12 month follow-up
    basal y a la semana del alta, luego seguimiento a los 3 y 12 meses.
    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 No
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA70
    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
    Belgium
    Czech Republic
    France
    Germany
    Hungary
    Italy
    Poland
    Russian Federation
    Spain
    Sweden
    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
    The end of the study is defined as the date on which the last patient completes the last visit (includes follow up visits).
    El final del estudio se define como la fecha en la que el último paciente completa la última visita (incluye las visitas de seguimiento).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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 years2
    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: 432
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
    F.1.1.2.1Number of subjects for this age range: 432
    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) No
    F.1.1.6Adolescents (12-17 years) No
    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 No
    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
    preterm infants born before week 32 of gestational age
    Recién nacidos prematuros nacidos antes de la semana 32 de gestación
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state55
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 390
    F.4.2.2In the whole clinical trial 432
    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)
    Normal treatment of that condition
    Tratamiento normal de la enfermedad
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation N/A
    G.4.3.4Network Country Spain
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-06-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-06-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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