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    Summary
    EudraCT Number:2011-000032-28
    Sponsor's Protocol Code Number:LAL-CL03
    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:2012-09-03
    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 number2011-000032-28
    A.3Full title of the trial
    AN OPEN LABEL, MULTICENTER, DOSE ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SBC-102 IN CHILDREN WITH GROWTH FAILURE DUE TO LYSOSOMAL ACID LIPASE DEFICIENCY
    Studio in aperto, multicentrico, a dosi crescenti per la valutazione della sicurezza, tollerabilita', efficacia, farmacocinetica e farmacodinamica di SBC-102 nei bambini con ritardo nella crescita dovuto a deficit di lipasi acida lisosomiale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A reasarch study to look at the safety, effectiveness and the effects on the body of a new drug, SBC-102, in children with growth problems caused by a deficiency in the enzyme that break down fats
    Studio clinico per valutare la sicurezza, efficacia e gli effetti sul corpo di un nuovo farmaco, SBC-102, nei bambini con ritardo della crescita causati da un deficit nell'enzima che separa i grassi
    A.3.2Name or abbreviated title of the trial where available
    SBC-102 in Children with Growth Failure due to LYSOSOMAL ACID LIPASE DEFICIENCY
    SBC-102 in bambini con ritardo della crescita(LAL)
    A.4.1Sponsor's protocol code numberLAL-CL03
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01371825
    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 SponsorSYNAGEVA BIOPHARMA CORP.
    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 supportSynageva BioPharma Corp.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPremier Research Group srl
    B.5.2Functional name of contact pointStart-up
    B.5.3 Address:
    B.5.3.1Street AddressVia Winckelmann 2
    B.5.3.2Town/ citymilano
    B.5.3.3Post code20146
    B.5.3.4CountryItaly
    B.5.4Telephone number0248959768
    B.5.5Fax number0248959776
    B.5.6E-mailstefania.giovanelli@premier-research.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/827
    D.3 Description of the IMP
    D.3.1Product namerecombinant human lysosomal acid lipase (rhLAL)
    D.3.2Product code SBC-102
    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.3.9.1CAS number 1276027-63-4
    D.3.9.2Current sponsor codeSBC-102
    D.3.9.3Other descriptive namerecombinant human lysosomal acid lipase (rhLAL)
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 Yes
    D.3.11.13.1Other medicinal product typericombinante umano della lipasi acida lisosomiale (rhLAL)
    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
    Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).
    ritardo nella crescita dovuto a deficit di lipasi acida lisosomiale (malattia di Wolman)
    E.1.1.1Medical condition in easily understood language
    Lysosomal Acid Lipase (LAL) Deficiency
    Deficit della Liapsi Acida Lisosomiale (LAL)
    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 15.0
    E.1.2Level HLT
    E.1.2Classification code 10024579
    E.1.2Term Lysosomal storage disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.0
    E.1.2Level HLGT
    E.1.2Classification code 10021605
    E.1.2Term Inborn errors of metabolism
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.0
    E.1.2Level SOC
    E.1.2Classification code 10027433
    E.1.2Term Metabolism and nutrition disorders
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate the effect of SBC-102
    therapy on survival at 12 months of age in children with growth failure
    due to LAL Deficiency.
    L’obiettivo primario del presente studio consiste nel valutare gli effetti della terapia con SBC-102 sulla sopravvivenza all’età di 12 mesi nei bambini affetti da ritardo nella crescita causato da deficit di LAL.
    E.2.2Secondary objectives of the trial
    (1) to evaluate the safety and tolerability of SBC-102 in children with
    growth failure due to LAL Deficiency;
    (2) to evaluate the effect of SBC-102 therapy on survival beyond 12
    months of age in children with growth failure due to LAL Deficiency;
    (3) to evaluate the effects of SBC-102 on growth parameters in children
    with growth failure due to LAL Deficiency;
    (4) to evaluate the effects of SBC-102 on hepatomegaly, splenomegaly,
    and liver function in children with growth failure due to LAL Deficiency;
    (5) to determine the effects of SBC 102 on hematological parameters in
    children with growth failure due to LAL Deficiency; and
    (6) to characterize the PK of SBC 102 delivered by intravenous (IV)
    infusion.
    (1) valutare la sicurezza e la tollerabilità di SBC-102 nei bambini affetti da ritardo nella crescita causato da deficit di LAL; (2) valutare gli effetti della terapia con SBC-102 sulla sopravvivenza oltre l’età di 12 mesi nei bambini affetti da ritardo nella crescita causato da deficit di LAL; (3) valutare gli effetti di SBC-102 sui parametri di crescita dei bambini affetti da ritardo nella crescita causato da deficit di LAL; (4) valutare gli effetti di SBC-102 sull’epatomegalia, splenomegalia e funzione epatica nei bambini affetti da ritardo nella crescita causato da deficit di LAL; (5) determinare gli effetti di SBC-102 sui parametri ematologici nei bambini affetti da ritardo nella crescita causato da deficit di LAL; (6) caratterizzare la farmacocinetica di SBC-102 somministrato mediante infusione endovenosa (IV).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject's parent or legal guardian understands the full nature and
    purpose of the study, including possible risks and side effects, and
    provides written informed consent/permission prior to any study
    procedures being performed
    2. Male or female child with a documented decreased LAL activity
    XML File Identifier: xHVSKdDb30v7HfyciGRPYEL0jt0=
    Page 10/21
    relative to the normal range of the lab performing the assay or
    documented result of molecular genetic testing (2 mutations) confirming
    a diagnosis of LAL Deficiency
    3. Growth failure with onset before 6 months of age, as defined in the
    protocol
    1. Un genitore o il tutore legale del soggetto comprende appieno la natura e le finalità dello studio, inclusi i possibili rischi ed effetti avversi, e presta per iscritto il proprio consenso o assenso informato prima dell’esecuzione di qualsiasi procedura dello studio.
    2. Il bambino, di sesso maschile o femminile, presenta documentazione attestante una minore attività della LAL rispetto al normale intervallo del laboratorio che effettua il test o il risultato di un test genetico-molecolare (2 mutazioni) che confermi la diagnosi di deficit di LAL.
    3. Insorgenza prima dei 6 mesi di età di ritardo nella crescita, definito come:
    diminuzione di peso in almeno due dei principali percentili di una tabella standard di peso per età (WFA) dell’Organizzazione Mondiale della Salute (OMS): 1°, 3°, 5°, 10°, 25°, 50°, 75°, 90°, 95°, 97°, 99°;
    O
    peso corporeo in kg al di sotto del 10° percentile di una tabella standard di peso per età dell’OMS E nessun aumento di peso nelle due settimane precedenti lo screening;
    O
    perdita di peso corporeo &gt;5% in soggetti di età superiore a 2 settimane.
    E.4Principal exclusion criteria
    1. Clinically important concurrent disease or co-morbidities which, in the
    opinion of the Investigator and Sponsor, would interfere with study
    participation, including, but not restricted to, congestive heart failure,
    ongoing circulatory collapse requiring inotropic support, acute or chronic
    renal failure, additional severe congenital abnormality, or other
    extenuating circumstances such as life-threatening under nutrition or
    rapidly progressive liver disease.
    2. Subject is >24 months of age. (Note: Subjects >8 months of age on
    the date of first infusion will not be eligible for the primary efficacy
    analysis.)
    3. Has received an investigational medicinal product other than SBC-102
    within 14 days prior to the first dose of SBC-102 in this study.
    4. Myeloablative preparation, or other systemic pre-transplant
    conditioning, for hematopoietic stem cell or liver transplantation.
    5. Previous hematopoietic stem cell or liver transplant.
    6. Known hypersensitivity to eggs.
    1. Malattie concomitanti, rilevanti dal punto di vista clinico, o comorbilità che, in base all’opinione dello Sperimentatore o dello Sponsor, possano interferire con la partecipazione allo studio, includendo, senza pretesa di esaustività, insufficienza cardiaca congestizia, collasso circolatorio in corso che richieda terapia inotropa, insufficienza renale acuta o cronica, ulteriore anomalia congenita grave o altre circostanze aggravanti, quali malnutrizione potenzialmente mortale o malattia epatica in rapido avanzamento.
    2. Età del soggetto &gt;24 mesi. Nota: i soggetti di età &gt;8 mesi alla data della prima infusione non saranno eleggibili per l’analisi di efficacia primaria.
    3. Al soggetto è stato somministrato un prodotto medicinale sperimentale diverso da SBC-102 nei 14 giorni antecedenti alla prima dose di SBC-102 nell’ambito del presente studio.
    4. Preparazione mieloablativa, o altre condizioni sistemiche antecedenti al trapianto, per il trapianto di fegato o di cellule staminali ematopoietiche.
    5. Precedente trapianto di fegato o di cellule staminali ematopoietiche.
    6. Ipersensibilità nota alle uova.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy: proportion of subjects surviving to 12 months of age
    Efficacia:percentuale di soggetti sopravvissuti all’età di 12 mesi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    continuously, see protocol
    continuo, vedere protocollo
    E.5.2Secondary end point(s)
    Efficacy: (1) changes from baseline in percentiles and/or z-scores for
    WFA, WFL/WFH, and LFA/HFA and the corresponding growth status
    indicators of underweight, wasting, and stunting, as well as changes
    from baseline in z scores for head circumference-for-age (HCFA) and
    mid-upper arm circumference-for-age (MUACFA);
    (2) changes from baseline in aspartate aminotransferase (AST) and ALT;
    (3) normalization of hemoglobin levels without requirement for blood
    transfusion; and
    (4) change from baseline in serum ferritin.
    Safety: incidence of AEs, SAEs, and IRRs; changes from baseline clinical
    laboratory tests; changes in vital signs during and post-infusion relative
    to pre-infusion values; physical examination findings; use of concomitant
    medications/therapies; and characterization of ADAs
    Pharmacokinetics: maximum observed serum concentration (Cmax) and
    apparent serum clearance (CL), as data permit. The impact of ADAs on
    SBC-102 PK will also be explored.
    Efficacia: (1) variazioni dal basale dei percentili e/o dei punteggi z per WFA, WFL/WFH e LFA/HFA e dei corrispondenti indicatori di stato della crescita di sottopeso, deperimento e ritardo nella crescita, così come variazioni dal basale dei punteggi z per circonferenza della testa per età (HCFA), circonferenza del braccio per età (MUACFA); (2) variazioni dal basale di aspartato amino transferasi (AST) e ALT; (3) normalizzazione dei livelli di emoglobina senza necessità di trasfusione sanguigna; (4) variazione dal basale dei livelli di ferritina sierica.
    Sicurezza:l’incidenza di eventi avversi (AE), gravi eventi avversi (SAE) e IRR; variazioni dal basale dei test clinici di laboratorio; variazioni dei parametri vitali (pressione arteriosa, frequenza cardiaca e respiratoria e temperatura corporea) durante e dopo l’infusione rispetto ai valori pre-infusione; risultati dell’esame obiettivo; uso di farmaci o terapie concomitanti; caratterizzazione degli ADA.
    Farmacocinetica: concentrazione plasmatica massima osservata (Cmax) e la clearance plasmatica apparente (CL). Si valuterà anche l’impatto degli ADA sulla farmacocinetica di SBC-102.
    E.5.2.1Timepoint(s) of evaluation of this end point
    continuously, see protocol
    continuo, vedere protocollo
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Taiwan
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 10
    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: 1
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 7
    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
    Newborn and very young patients
    Neonati e pazienti molto giovani
    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 6
    F.4.2.2In the whole clinical trial 10
    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)
    Once the study has been completed patients may be offered the
    opportunity to continue receiving SBC-102 upon commercial availability
    (if local marketing approval has been received) or under a
    compassionate use program if the overall risk/benefit ratio of
    continuation of therapy with SBC-102 remain favorable.
    Una volta che lo studio e' stato completato, ai pazienti può essere offerta l'opportunità di continuare a ricevere SBC-102 sino alla disponibilità commerciale (se è stata ottenuta l'approvazione all'immissione in commercio) o sotto un programma di uso compassionevole se il rapporto rischio/beneficio per il proseguio della terapia con SBC-102 rimarrà favorevole.
    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 Decision2012-08-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-07-09
    P. End of Trial
    P.End of Trial StatusCompleted
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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