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    Summary
    EudraCT Number:2015-000371-26
    Sponsor's Protocol Code Number:DFI12712
    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:2018-02-06
    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 number2015-000371-26
    A.3Full title of the trial
    A phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency
    Studio di fase 2/3, multicentrico, randomizzato, in doppio cieco, controllato con placebo, a dosi ripetute, con comparazione di dose per valutare l’efficacia, la sicurezza, la farmacodinamica e la farmacocinetica di olipudase alfa in pazienti con deficit di sfingomielinasi acida
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency
    Sttudio di efficacia, sicurezza, farmacodinamica e farmacocinetica di Olipudase alfa in pazienti con deficit di sfingomielinasi acida.
    A.3.2Name or abbreviated title of the trial where available
    ASCEND
    ASCEND
    A.4.1Sponsor's protocol code numberDFI12712
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02004691
    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 SponsorGENZYME CORPORATION
    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 supportGENZYME CORPORATION
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSANOFI S.P.A.
    B.5.2Functional name of contact pointCONTACT POINT
    B.5.3 Address:
    B.5.3.1Street AddressVIALE BODIO, 37/B
    B.5.3.2Town/ cityMILANO
    B.5.3.3Post code20158
    B.5.3.4CountryItaly
    B.5.4Telephone number800226343
    B.5.5Fax number0239394168
    B.5.6E-mailinformazioni.medicoscientifiche@sanofi-aventis.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/3/01/056
    D.3 Description of the IMP
    D.3.1Product nameOLIPUDASE ALFA
    D.3.2Product code GZ402665
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOLIPUDASE ALFA
    D.3.9.1CAS number 927883-84-9
    D.3.9.2Current sponsor codeGZ402665
    D.3.9.4EV Substance CodeSUB75088
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee 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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
    Pazienti con deficit di sfingomielinasi acida (malattia di Niemann-Pick di Tipo B)
    E.1.1.1Medical condition in easily understood language
    Patients with Niemann-Pick Type B disease
    pazienti con malattia di Niemann- Pick di Tipo B
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10041515
    E.1.2Term Sphingomyelin lipidosis
    E.1.2System Organ Class 100000004850
    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 this phase 2/3 study is to evaluate the efficacy of different doses of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult patients with acid sphingomyelinase deficiency (ASMD) by assessing separately, changes in spleen volume as measured by abdominal magnetic resonance imaging (MRI) and infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide.
    L’obiettivo primario di questo studio di fase 2/3 è quello di valutare l’efficacia di differenti dosi di olipudase alfa (ricombinante umano della sfingomielinasi acida) somministrate per via endovenosa una volta ogni 2 settimane per 52 settimane in pazienti adulti affetti da deficit di sfingomielinasi acida (ASMD) valutando separatamente la variazione del volume della milza misurato attraverso risonanza magnetica (RM) e della malattia polmonare infiltrativa con test di funzionalità polmonare, capacità di diffusione polmonare del monossido di carbonio (DLCO)
    E.2.2Secondary objectives of the trial
    To confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks. To characterize the effect of olipudase alfa on liver volume after 52 weeks of study drug administration. To characterize the effect of olipudase alfa on the splenomegaly-related symptom score after 52 weeks of study drug administration To characterize the effect of olipudase alfa on platelet count after 52 weeks of study drug administration. To characterize the effect of olipudase alfa after 52 weeks of study drug administration on a symptom-based composite score composed of 4 symptom domains (pain, fatigue, dyspnea, and abdominal complaints).
    • Confermare la sicurezza di olipudase alfa somministrato per via endovenosa ogni 2 settimane per 52 settimane
    • Caratterizzare l’effetto di olipudase alfa sul volume epatico dopo 52 settimane di somministrazione del farmaco in studio

    • Caratterizzare l’effetto di olipudase alfa sul punteggio MF-SAF modificato correlato ai sintomi di splenomegalia dopo 52 settimane di somministrazione del farmaco in studio

    • Caratterizzare l’effetto di olipudase alfa sulla conta piastrinica dopo 52 settimane di somministrazione del farmaco in studio
    • Caratterizzare l’effetto di olipudase alfa dopo 52 settimane di somministrazione del farmaco in studio con un punteggio composito basato sui sintomi, ottenuto tramite un diario elettronico comprendente 4 aree sintomatiche (dolore, affaticamento, dispnea e fastidi addominali)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The patient is willing and able to provide signed written informed consent. The patient is male or female aged 18 years or older. The patient has documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes; and a clinical diagnosis consistent with Niemann-Pick disease type B (NPD B). The patient has diffuse capacity of the lung for carbon monoxide ≤70% of the predicted normal value. The patient has a spleen volume ≥6 multiples of normal (MN) measured by MRI; patients who have had partial splenectomy will be allowed if the procedure was performed ≥1 year before screening/baseline and the residual spleen volume is ≥6 MN. Female patients of childbearing potential must have a negative serum pregnancy test for beta-human chorionic gonadotropin (β-HCG). Female patients of childbearing potential and male patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception.
    • Il paziente è disposto ed è in grado di fornire il consenso informato scritto e firmato.
    • Il paziente è maschio o femmina di età pari o superiore a 18 anni.
    • Il paziente ha documentata carenza di sfingomielinasi acida, come valutato sulla base dei leucociti periferici, fibroblasti in coltura, o linfociti; e una diagnosi clinica coerente con NPD B.
    • Il paziente ha capacità di diffusione polmonare del monossido di carbonio (DLCO) ≤ 70% del normale valore predittivo.
    • Il paziente ha un volume della milza ≥6 volte il volume normale (MN) misurato tramite risonanza magnetica; saranno ammessi i pazienti che hanno avuto splenectomia parziale, se la procedura è stata eseguita ≥1 anno prima dello screening/basale e il volume della milza residuo è ≥6 MN.
    • Pazienti di sesso femminile in età fertile con test di gravidanza negativo per la beta-gonadotropina corionica umana sierica (β HCG).
    • Pazienti di sesso femminile in età fertile e pazienti di sesso maschile devono essere disposti a praticare la vera astinenza in linea con il loro stile di vita preferito e solito, o utilizzare 2 metodi efficaci accettabili di contraccezione.
    E.4Principal exclusion criteria
    The patient has received an investigational drug within 30 days before study enrollment. The patient has a medical condition, including significant intercurrent illness; significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or <40% left ventricular ejection fraction by echocardiogram); active hepatitis B or hepatitis C, or infection with human immunodeficiency virus (HIV); cirrhosis (as determined by clinical evaluation or liver biopsy); malignancy diagnosed within the past 5 years (other than basal cell carcinoma), or any other extenuating circumstance that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities. The patient has a platelet count <60,000/μL (based on the average of 2 samples obtained at least 12 hours but no longer than 24 hours apart). The patient has an international normalized ratio (INR) >1.5. The patient has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >250 IU/L or total bilirubin >1.5 mg/dL (except for patients with Gilbert's syndrome). The patient has had a major organ transplant (eg, bone marrow or liver). The patient is scheduled during the study for in-patient hospitalization including elective surgery and excluding the liver biopsies required per protocol. The patient, in the opinion of the investigator, is unable to adhere to the requirements of the study. The patient is unwilling or unable to abstain from the use of alcohol for 1 day before and 3 days after each study drug infusion. Testing for blood alcohol levels will not be required. The patient is unwilling or unable to avoid 10 days before and 3 days after the protocol scheduled liver biopsies that are required at screening/baseline and at week 52, the use of medications or herbal supplements that are potentially hepatotoxic (e.g., 3-hydroxy-3-methyl glutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, anti-depressants, kava, echinacea) and/or may cause or prolong bleeding (e.g., anti-coagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng). The patient requires medications that may decrease olipudase alfa activity (e.g., fluoxetine, chlorpromazine, tricyclic antidepressants [e.g., imipramine, or desipramine]). The patient requires use of invasive ventilatory support. The patient requires use of noninvasive ventilator support while awake for longer than 12 hours daily. The patient is breast-feeding.
    • Il paziente ha ricevuto un farmaco sperimentale nei 30 giorni precedenti l'arruolamento nello studio.
    • Il paziente ha una condizione medica, tra cui significative malattie concomitanti; significativa malattia cardiaca (ad esempio, aritmia clinicamente significativa, ipertensione polmonare moderata o grave, disfunzione valvolare, o frazione di eiezione ventricolare sinistra <40% valutata con ecocardiogramma [ECHO]); epatite B attiva o epatite C, o infezione da virus dell'immunodeficienza umana (HIV); cirrosi (diagnosticata tramite valutazione clinica o biopsia epatica); tumore maligno diagnosticato negli ultimi 5 anni (diverso da carcinoma a cellule basali), o qualsiasi altra circostanza che possa interferire in modo significativo con la compliance allo studio, comprese le valutazioni previste e le attività di follow-up
    • Il paziente ha una conta piastrinica <60 x 103/μL (basata sulla media di 2 campioni ottenuti a distanza di almeno 12 ore, ma non più di 24 ore).
    • Il paziente ha un valore INR >1.5.
    • Il paziente ha valori di alanina aminotransferasi (ALT) o aspartato aminotransferasi (AST) >250 UI/L o bilirubina totale >1.5 mg/dl (eccetto i pazienti con sindrome di Gilbert).
    • Il paziente ha avuto un importante trapianto d'organo (per esempio, di midollo osseo o del fegato).
    • Ospedalizzazioni del paziente programmate durante lo studio, compresa la chirurgia elettiva ed escluse le biopsie epatiche richieste dal protocollo.
    • Il paziente, a giudizio dello sperimentatore, non è in grado di rispettare i requisiti dello studio.
    • Il paziente non vuole o non è capace di astenersi dall'uso di alcol per 1 giorno prima e 3 giorni dopo ogni infusione del farmaco in studio. Non sarà richiesto testare i livelli di alcol nel sangue.
    • Il paziente non vuole o non può evitare l'uso di farmaci o integratori a base di erbe, potenzialmente epatotossici (ad esempio, inibitori della 3-idrossi-3-metilglutaril coenzima A reduttasi, eritromicina, acido valproico, antidepressivi, kava, echinacea) e/o capaci di causare o prolungare il sanguinamento (ad esempio, anti-coagulanti, ibuprofene, aspirina, supplementi d'aglio, ginkgo, ginseng) 10 giorni prima e 3 giorni dopo le biopsie epatiche, che sono necessarie allo screening/basale e alla settimana 52 .
    • Il paziente necessita di farmaci che possono ridurre l'attività di olipudase alfa (ad esempio, fluoxetina, clorpromazina, antidepressivi triciclici [ad esempio, imipramina, o desipramina]).
    • Il paziente richiede l'utilizzo di un supporto ventilatorio invasivo.
    • Il paziente richiede l'utilizzo di supporto ventilatorio non invasivo durante la veglia per più di 12 ore al giorno.
    • La paziente è in allattamento.
    E.5 End points
    E.5.1Primary end point(s)
    Percentage change in spleen volume. - Percentage change in diffusing capacity of the lung for carbon monoxide
    variazione percentuale del volume della milza - Variazione percentuale della capacità polmonare di diffusione del monossido di carbonio
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to Week 52
    Dal basale alla settimana 52.
    E.5.2Secondary end point(s)
    - Percentage change in liver volume - Change in splenomegaly-related symptom score - Percentage change in platelet count - Change in symptom based composite score composed of 4 symptom domains (pain, fatigue, dyspnea, and abdominal complaints) - Number of adverse events
    - Variazione percentuale del volume della milza; - Variazioni del punteggio relativo ai sintomi correlati alla splenomegalia; Variazione percentuale della conta piastrinica; - Variazione nel punteggio composito basato sui sintomi in 4 ambiti (dolore, stanchezza, dispnea e disturbi addominali); - Numero di Eventi Avversi
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Percentage change in liver volume - Time Frame: Baseline to Week 52 - Change in splenomegaly-related symptom score - Time Frame: Baseline to Week 52 - Percentage change in platelet count - Time Frame: Baseline to Week 52 - Change in symptom based composite score composed of 4 symptom domains (pain, fatigue, dyspnea, and abdominal complaints). - Time Frame: Baseline to Week 52 - Number of adverse events - Time Frame: Baseline to approximately 5 years
    - Variazione percentuale del volume della milza - Finestra temporale: dal basale alla settimana 52; - Variazioni del punteggio relativo ai sintomi correlati alla splenomegalia - Finestra temporale: dal basale alla settimana 52; - Variazione percentuale della conta piastrinica - Finestra temporale: dal basale alla settimana 52; - Variazione nel punteggio composito basato sui sintomi in 4 ambiti (dolore, stanchezza, dispnea e disturbi addominali) - Finestra temporale: dal basale alla settimana 52; - Numero di Eventi Avversi - finestra temporale: dal basale a circa 5 anni
    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 Yes
    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) Yes
    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 trial3
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Argentina
    Australia
    Brazil
    Chile
    France
    Germany
    Israel
    Italy
    Japan
    Netherlands
    Portugal
    Spain
    Tunisia
    Turkey
    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
    LPLV
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    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 years7
    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 No
    F.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 34
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 Yes
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 35
    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)
    As part of the trial, all patients will receive active treatment in the extension treatment period (ETP) which will last up to 4 years. Subsequently, depending on trial results and whether a marketing authorisation for olipudase alfa is granted, the sponsor will decide to either transition patients to commercial treatment or continue patients on investigational treatment or to discontinue treatment.
    In qualità di partecipantio allo studio clinico, tutti i pazienti riceveranno il trattamento attivo nel periodo di estensione del trattamento (ETP) che durerà fino a 4 anni. In seguito, sulla base dei risultati dello studio e laddove sarà garantita l'autorizzazione all'immissione in commercio per olipudase alfa, il Promotore deciderà se assegnare ai pazienti il trattamento in commercio, se far loro proseguire il trattamento sperimentale e se far interrompere il trattamento.
    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 Decision2015-10-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-03-08
    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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