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    Summary
    EudraCT Number:2010-023909-35
    Sponsor's Protocol Code Number:BVT.BSSL-030
    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:2012-03-01
    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 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
    Studio prospettico, randomizzato, in doppio cieco, di fase 3 per confrontare rhBSSL e placebo aggiunti a latte artificiale per neonati o latte materno pastorizzato durante 4 settimane di trattamento in neonati pretermine nati prima della 32ma settimana di eta' gestazionale
    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.
    Studio clinico di fase 3 per valutare l'effetto di rhBSSL (un enzima che aiuta la digestione dei grassi) aggiunto al latte materno pastorizzato o al latte artificiale in bambini nati prematuramente.
    A.3.2Name or abbreviated title of the trial where available
    BVT.BSSL-030
    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 Plan
    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 number+49 6233 6070194
    B.5.5Fax number+49 6233 6070195
    B.5.6E-mailMattias.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 nameRecombinant Human Bile Salt-stimulated Lipase
    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 IMPGastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBucelipase alfa
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor coderhBSSL
    D.3.9.3Other descriptive nameRecombinant Human Bile-salt-stimulated lipase (rhBSSL)
    D.3.9.4EV Substance CodeNA
    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
    Prevenzione del ritardo nella crescita dovuto alla mancanza della lipasi stimolata dal sale di bile nella nutrizione enterale
    E.1.1.1Medical condition in easily understood language
    Babies born prematurely.
    Bambini nati prematuri.
    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 14.1
    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.
    L`obiettivo primario di questo studio è quello di dimostrare che rhBSSL migliora la crescita dei neonati pretermine rispetto al placebo quando somministrata a latte artificiale o a latte materno pastorizzato.
    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
    Gli obiettivi secondari dello studio sono i seguenti: • Determinare l`effetto del trattamento con rhBSSL sull`abbreviazione dei tempi di degenza ospedaliera • Determinare l`effetto del trattamento con rhBSSL sul miglioramento del primo sviluppo • Determinare l`effetto del trattamento con rhBSSL sulla diminuzione di riammissione in ospedale • Determinare l`effetto del trattamento con rhBSSL sull`aumento dei livelli di acido docosaesaenoico (DHA) e acido arachidonico (AA) • Confrontare la sicurezza e la tollerabilità del trattamento con rhBSSL con quelle del trattamento 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. Neonato/a pretermine nato/a prima della 32ma settimana di gestazione. 2. Neonato/a pretermine che abbia meno (&lt;) di 33 settimane di età postmestruale al momento della randomizzazione. 3. Neonato/a pretermine che sia giusto/a per l`età gestazionale o piccolo/a per l`età gestazionale alla nascita. 4. Neonato/a pretermine che venga nutrito/a per via enterale (biberon o tubo sonda gastrica) con una dose pari ad almeno 100 mL/kg/die alla randomizzazione. 5. Neonato/a pretermine la cui alimentazione enterale consiste solo in latte artificiale o solo in latte materno pastorizzato al momento dell`arruolamento, e che ci si aspetti rimanga con tale regime alimentare di solo latte artificiale per 4 settimane, o solo con latte materno pastorizzato per almeno 2 settimane dopo l`inizio del trattamento. 6. Neonato/a pretermine che si prevede non riceva latte materno fresco per 4 settimane dopo l`inizio del trattamento. 7. Il consenso informato è ottenuto dal rappresentante giuridicamente accettabile del paziente. Verrà fatto il possibile per assicuraci di ottenere il consenso informato da entrambi i genitori. Ove ciò non sia possibile, saranno seguite le normative locali per definire il rappresentante giuridicamente accettabile.
    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 degenza ospedaliera prevista è inferiore a 4 settimane dalla prima dose del farmaco in studio. 2. Attualmente sottoposto/a a ventilazione meccanica tramite tubo endotracheale (pressione positiva continua delle vie aeree [CPAP] o cannula nasale a flusso elevato, non sono criteri di esclusione). 3. Richieda ossigeno ≥30% (se in CPAP o in scatola d`afflusso) o&gt; 0,5 L/min di ossigeno. 4. Evidenza di grave malattia cerebrale o danni, compresi emorragia peri-o intraventricolare di grado III o IV, meningite o idrocefalo, emorragia intracranica di grado III o IV o leucomalacia periventricolare. 5. Presenza di grandi dimorfismi o anomalie congenite che possono influenzare la crescita e/o lo sviluppo. 6. Attuale evidenza clinica di dotto arterioso persistente emodinamicamente significativo. 7. Evidenza clinica di sepsi (compreso bassa o alta conta dei globuli bianchi e/o bassa conta di piastrine e evidenza di infezioni sistemiche comprovata batteriologicamente). Ciò dovrebbe basarsi sul parere dello sperimentatore e sui valori di riferimento disponibili dal laboratorio locale. I pazienti non devono aver ricevuto antibiotici nelle precedenti 48 ore, salvo che questi siano stati somministrati secondo protocolli interni per profilassi (ad esempio, profilassi antifungina con fluconazolo). 8. Evidenza di infezione congenita (ad esempio, citomegalovirus). 9. Diagnosi precedente o attuale di enterocolite necrotizzante (stadio di Bell 2 o superiore). 10. Trattamento precedente o corrente con corticosteroidi, ad eccezione di idrocortisone. 11. Presenza di qualsiasi condizione che a giudizio dello sperimentatore renda il/la paziente non idoneo/a all`inclusione. 12. Arruolamento in un altro studio clinico 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 variabile primaria di efficacia è la velocità di crescita in grammi per chilogrammo al giorno durante 4 settimane di trattamento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and weekly to dischaege then 3 and 12 month follow-up
    Al baseline e settimanalmente sino alla dimissione, quindi al terzo e dodicesimo mese di follow-up.
    E.5.2Secondary end point(s)
    The study has the following secondary efficacy endpoints: • 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 The study has the following secondary safety endpoints: • Adverse events • 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) • Routine laboratory variables including amylase, bilirubin, aminotransferases, sodium and urea • Levels of vitamin A and D • Levels of rhBSSL antibodies
    Lo studio ha i seguenti endpoint secondari di efficacia: • Variazione rispetto al basale del peso corporeo (g) a 3 mesi • Peso corporeo (g) all`età corretta di 12 mesi • Variazione rispetto al basale della lunghezza totale del corpo (mm) a 4 settimane e 3 mesi • Lunghezza del corpo (mm) all`età corretta di 12 mesi • Tempo fino alla preparazione per la dimissione • Tempo fino alla dimissione • Variazione rispetto al basale della circonferenza cranica (mm) a 4 settimane e 3 mesi • Circonferenza cranica (mm) all`età corretta di 12 mesi • Tempo dal basale a 150 mL/kg/die di alimentazione enterale • Riammissione in ospedale entro 1 mese dalla dimissione • Livelli di DHA e AA a 4 settimane Lo studio ha i seguenti endpoint secondari di sicurezza: • Eventi avversi • Esame obbiettivo • Scala di Bayley per lo sviluppo infantile III (cognitivo) • Scala di Bayley per lo sviluppo infantile III (linguaggio) • Scala di Bayley per lo sviluppo infantile III (motorio) • Vomito (frequenza e volume) • Segni vitali (frequenza cardiaca, pressione sanguigna, temperatura corporea) • Variabili di laboratorio di routine tra cui amilasi, bilirubina, aminotransferasi, sodio e urea • Livelli di vitamina A e D • Livelli di anticorpi all’rhBSSL
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline and weekly to dischaege then 3 and 12 month follow-up
    Al baseline e settimanalmente sino alla dimissione, quindi al terzo e dodicesimo mese di follow-up.
    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 concerned8
    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
    Russian Federation
    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
    LPLV (includes follow up visits)
    LVLP (visite di follow up incluse)
    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
    Neonato/a pretermine nato/a prima della 32ma settimana di gestazione
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state43
    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
    Normale trattamento per la condizione
    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 Decision2011-10-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-04-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-07-03
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