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    EudraCT Number:2015-001578-17
    Sponsor's Protocol Code Number:SHP-GCB-402
    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:2020-11-04
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2015-001578-17
    A.3Full title of the trial
    An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of
    Treatment with Velaglucerase alfa on Bone-related Pathology in
    Treatment-naïve Patients with Type 1 Gaucher Disease
    Studio di fase 4 in aperto, multicentrico, a braccio singolo per valutare l'effetto del trattamento con
    Velaglucerasi alfa sulla patologia ossea in pazienti affetti da malattia di Gaucher di tipo 1 mai trattati in precedenza
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease
    Studio in aperto di fase IV di valutazione di Velaglucerasi alfa nelle patologie ossee in pazienti adulti, affetti da malattia di Gaucher di tipo 1 mai trattati in precedenza.
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberSHP-GCB-402
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN00000000
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00000000
    A.5.3WHO Universal Trial Reference Number (UTRN)U0000-0000-0000
    A.5.4Other Identifiers
    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.1.3.4CountryUnited 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 supportShire Human Genetic Therapies, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire
    B.5.2Functional name of contact pointSteven A. Johnson Jr.
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number0017814821525
    B.5.5Fax number0000000
    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 Yes
    D. of the Marketing Authorisation holderSHIRE PHARMACEUTICALS IRELAND LIMITED
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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/3/10/752
    D.3 Description of the IMP
    D.3.1Product nameVelaglucerase alfa
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Powder for 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.3.8INN - Proposed INNvelaglucerasi alfa
    D.3.9.1CAS number 884604-91-5
    D.3.9.2Current sponsor codeGA-GCB
    D.3.9.4EV Substance CodeSUB31010
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    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 Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 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
    Gaucher Disease
    Malattia di Gaucher
    E.1.1.1Medical condition in easily understood language
    Gaucher disease is caused by a defective enzyme. Due to this, fatty substances build up in the cells of the body, especially in liver, spleen and bone marrow.
    La malatt di Gaucher è provocata da un enzima difettoso. A causa di questo difetto, sostanze grasse si accumulano nelle cell dell'organismo, in particolare nel fegato, nella milza e nel midollo osseo
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10075697
    E.1.2Term Gaucher's disease type I
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of VPRIV therapy (60 U/kg every other week [EOW]) in treatment-naïve patients with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment.
    Valutare l'effetto della terapia con VPRIV (60 U/kg a settimane alterne [EOW]) in pazienti mai trattati in precedenza affetti da malattia di Gaucher di tipo 1 sulla variazione rispetto al basale del punteggio Z della DMO (densità minerale ossea) nel tratto lombare della colonna (LS), misurato mediante assorbimento a raggi X a doppia energia (DXA) dopo 24 mesi di trattamento.
    E.2.2Secondary objectives of the trial
    To evaluate the effect of VPRIV therapy (60 U/kg EOW) on bone mineral density (BMD) in treatment-naïve patients with type 1 Gaucher disease as measured by the change from baseline in LS BMD Z-score at 12 months, and bone marrow burden (BMB) as measured by magnetic resonance imaging (MRI) of the LS and femur after 12 and 24 months of treatment.
    Valutare l'effetto della terapia con VPRIV (60 U/kg EOW) sulla Densità Minerale Ossea (DMO) in pazienti mai trattati in precedenza affetti da malattia di Gaucher di tipo 1 misurata in base alla variazione rispetto al basale del punteggio Z della DMO nella LS a 12 mesi, e al carico del midollo osseo (BMB) misurato mediante RMI della LS e del femore dopo 12 e 24 mesi di trattamento.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each patient must meet the following criteria to be enrolled in this study.
    1. The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient GCB activity in leukocytes (whole blood only) or cultured skin fibroblasts. Diagnosis by dry blood spot test is not sufficient. Diagnosis may be
    based on results obtained prior to screening if documented in the patient's medical history.
    2. Patients must have a LS BMD Z-score <-1 as measured by DXA during the screening phase.
    3. Patient is treatment-naïve, ie, has not received ERT or SRT in the 12 months prior to enrollment.
    4. The patient is =18 and =65 years of age.
    5. Female patients of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study. (See Section 6.8.3 for acceptable methods of contraception).
    6. The patient, or patient's legally authorized representative(s), if applicable, understands the nature, scope, and possible consequences of the study and has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
    7. The patient must be sufficiently cooperative to participate in this clinical study as judged by the investigator.
    Ogni paziente deve soddisfare i seguenti criteri per essere arruolato in questo studio.
    1. Il paziente presenta una diagnosi documentata di malattia di Gaucher di tipo 1, documentata mediante un’attività deficitaria di GCB nei leucociti (solo sangue intero) o fibroblasti cutanei coltivati. La diagnosi ottenuta dall'analisi di una goccia di sangue essiccato non è sufficiente. La diagnosi può basarsi sui risultati ottenuti prima dello screening, se documentata nell'anamnesi medica del paziente.
    2. I pazienti devono avere un punteggio Z della DMO della LS <-1 misurato mediante DXA durante la fase di screening.
    3. Il paziente è naïve al trattamento, ovvero, non è stato sottoposto a terapie ERT o SRT nei 12 mesi precedenti l'arruolamento.
    4. Il paziente ha un'età compresa tra =18 e =65 anni.
    5. Le pazienti di sesso femminile in età fertile devono accettare di usare un metodo contraccettivo clinicamente accettabile per tutta la durata dello studio (vedere la Sezione 6.8.3 per i metodi contraccettivi accettabili).
    6. Il paziente, o il/i rappresentante/i legalmente autorizzato/i del paziente, ove pertinente, comprende la natura, l'ambito e le possibili conseguenze dello studio e ha fornito il consenso informato scritto che è stato approvato dal Consiglio di Revisione Istituzionale/ Comitato Etico Indipendente (IRB/CEI).
    7. Il paziente deve essere sufficientemente cooperante nel partecipare a questo studio clinico secondo il giudizio dello sperimentatore.
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria will be excluded from the study.
    1. Neurological symptoms indicating that the patient has type 3 Gaucher disease.
    2. A significant comorbidity, which, as determined by the investigator, might affect study data or confound the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver disease, etc).
    3. Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with erythropoietin (or erythropoietin-like substances) during the past year.
    4. Structural, joint-associated bone damage of such extent and severity that the investigator deems it could impact participation in the study and assessment of relevant study endpoints (eg, pain).
    5. The patient is pregnant or lactating.
    6. The patient has had a splenectomy.
    7. The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device) within 30 days prior to study enrollment or at any time during the study.
    8. Severe vitamin D deficiency to the level that would be expected to result in osteomalacia (25-hydroxyvitamin D < 10 ng/mL [25 nmol/L]). If there is mild vitamin D insufficiency at screening (25-hydroxyvitamin D > 10 and < 30 ng/mL) treat with 4000 IU 25-hydroxyvitamin D per day for 1 month and rescreen.
    9. The patient has previously interrupted ERT for safety reasons.
    10. The patient has had hypersensitivity to the active substance or to any of the excipients.
    I pazienti che rispondono ai seguenti criteri saranno esclusi dallo studio.
    1. Sintomi neurologici indicanti che il paziente è affetto da malattia di Gaucher di tipo 3.
    2. Una comorbilità significativa che, secondo quanto stabilito dallo sperimentatore, potrebbe influire sui dati dello studio o confondere i risultati dello studio (per esempio tumori maligni, cirrosi biliare primitiva, malattia epatica autoimmune, ecc.).
    3. Qualsiasi trattamento specifico per l'osteoporosi (per esempio bifosfonati) o trattamento con eritropoietina (o sostanze simili all’eritropoietina) nell’anno precedente.
    4. Danno osseo strutturale, associato alle articolazioni di tale entità e gravità, che lo sperimentatore ritenga possa influire sulla partecipazione allo studio e sulle valutazioni di endpoint rilevanti dello studio (per esempio, dolore).
    5. La paziente è in stato di gravidanza o allatta al seno.
    6. Il paziente è stato sottoposto a una splenectomia.
    7. Il paziente è arruolato in un altro studio clinico che comporta sperimentazioni cliniche o l'uso di qualsiasi prodotto sperimentale (farmaco o dispositivo) entro i 30 giorni precedenti all'arruolamento nello studio o in qualsiasi momento durante lo studio.
    8. Grave carenza di vitamina D a un livello che potrebbe causare osteomalacia (25-Idrossivitamina D <10 ng/mL [25 nmol/L]). Se è presente una lieve insufficienza di vitamina D allo screening (25-Idrossivitamina D > 10 e <30 ng/mL), trattare con 4000 IU di 25-Idrossivitamina D al giorno per 1 mese ed eseguire nuovamente lo screening.
    9. Il paziente ha precedentemente interrotto l'ERT per motivi di sicurezza.
    10. il paziente ha manifestato ipersensibilità al principio attivo o a uno qualsiasi degli eccipienti.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to 24 months (Week 103 [end of study]) in LS BMD Z-score as measured by DXA. Assessments will be performed at the screening visit and Weeks 25, 51,77, and 103 (end of study).
    Variazione rispetto al basale a 24 mesi (Settimana 103 [fine dello studio] del punteggio Z della DMO della LS, misurato tramite DXA. Le valutazioni saranno svolte alla visita di screening e alle Settimane 25, 51, 77 e 103 (fine dello studio)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of study).
    Le valutazioni saranno svolte alla visita di screening e alle Settimane 25, 51, 77 e 103 (fine dello studio).
    E.5.2Secondary end point(s)
    The key secondary endpoints of this study are:
    • Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in BMB score (MRI of lumbar spine and femur). Assessments will be
    performed at the baseline visit and Weeks 25, 51, 77, and 103 (end of study).
    • Change from baseline to 12 months (Week 51) in LS BMD Z-score.
    Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of study).
    I principali endpoint secondari di questo studio sono:
    • Variazione rispetto al basale a 12 mesi (Settimana 51) e a 24 mesi (Settimana 103 [fine dello studio]) nel punteggio BMB (RMI di tratto lombare e femore). Le valutazioni saranno svolte alla visita basale e alle Settimane 25, 51, 77 e 103 (fine dello studio).
    • Variazione rispetto al basale a 12 mesi (Settimana 51) dello Z -score della DMO dell' LS.
    Le valutazioni saranno svolte alla visita di screening e alle Settimane 25, 51, 77 e 103 (fine dello studio).
    E.5.2.1Timepoint(s) of evaluation of this end point
    . Assessments will be performed at the baseline visit and Weeks 25, 51, 77, and 103 (end of study).
    . Assessments will be performed at the screening visit and Weeks 25, 51,77, and 103 (end ofstudy).
    Le valutazioni saranno svolte alla visita basale e alle Settimane 25, 51, 77 e 103 (fine dello studio).
    Le valutazioni saranno svolte alla visita di screening e alle Settimane 25, 51, 77 e 103 (fine dello studio).
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E. trial design description
    studio non controllato ma in aperto
    study not controlled but open
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    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 EEA7
    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 Information not present in EudraCT
    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
    United States
    E.8.7Trial has a data monitoring committee No
    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 years3
    E.8.9.1In the Member State concerned months5
    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 months5
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 40
    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 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 13
    F.4.2.2In the whole clinical trial 40
    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)
    At the end of the study the patient’s doctor or primary care doctor will discuss the options for continued care
    Al termine dello studio il medico del paziente o il medico di base discuteranno le opzioni per la prosecuzione dei trattamenti
    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 Decision2016-12-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-18
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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