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    Summary
    EudraCT Number:2013-000051-40
    Sponsor's Protocol Code Number:LTS13632
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-05-25
    Trial 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 number2013-000051-40
    A.3Full title of the trial
    A Long-Term Study to Assess the Ongoing Safety and Efficacy of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency

    Studio a lungo termine per valutare su base continua la sicurezza e l’efficacia della sfingomielinasi acida ricombinante umana in pazienti con deficit di sfingomielinasi acida

    Titolo aggiornato dello studio: Studio a lungo termine per valutare su base continua la sicurezza e l’efficacia 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
    A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency
    Studio a lungo termine sulla sfingomielinasi acida umana ricombinante in pazienti con deficit di sfingomielinasi acida

    Titolo aggiornato dello studio: Studio a lungo termine su olipudase alfa in pazienti con deficit di sfingomielinasi acida
    A.4.1Sponsor's protocol code numberLTS13632
    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 organisationGenzyme Europe B.V.
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street AddressGooimeer 10
    B.5.3.2Town/ cityNaarden
    B.5.3.3Post codeNL-1411 DD
    B.5.3.4CountryNetherlands
    B.5.6E-maileumedinfo@genzyme.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 (rhASM)
    D.3.2Product code GZ402665
    D.3.4Pharmaceutical form Powder for concentrate 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 INNOlipudase alfa
    D.3.9.1CAS number 927883-84-9
    D.3.9.2Current sponsor codeGZ402665
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
    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 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
    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 19.0
    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 study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term treatment with olipudase alfa
    L’obiettivo primario dello studio prevede l’acquisizione di dati relativi alla sicurezza di olipudase alfa in pazienti con deficit della sfingomielinasi acida (acid sphingomyelinase deficiency, ASMD) sottoposti a trattamento a lungo termine con olipudase alfa.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration
    Gli obiettivi secondari di questo studio prevedono l’acquisizione di dati relativi all’efficacia di olipudase alfa e la caratterizzazione farmacodinamica (pharmacodynamics, PD) e farmacocinetica (pharmacokinetics, PK) di olipudase alfa a seguito della somministrazione a lungo termine.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The patient completed the treatment period of a previous study of olipudase alfa with an acceptable safety profile in the opinion of the investigator and sponsor
    The patient and/or the patient's parent(s)/legal guardian(s) is willing and able to provide signed written informed consent.
    The patient who is female and of childbearing potential must have a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG).
    Female patients of childbearing potential and sexually mature 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 up to 15 days following their last dose of study drug
    Il paziente ha completato il periodo del trattamento di un precedente studio su olipudase alfa con un profilo di sicurezza accettabile secondo il parere dello sperimentatore e dello sponsor.
    • Il paziente e/o il/i genitore/i o il/i tutore/i legale/i del paziente devono essere disposti e in grado di fornire il consenso informato firmato.
    • Le donne potenzialmente fertili devono presentare un test di gravidanza sulle urine per la gonadotropina corionica umana beta (β-HCG) con esito negativo.
    • Le donne potenzialmente fertili e gli uomini sessualmente maturi devono essere disposti a praticare un’effettiva astinenza in linea con le proprie preferenze e il proprio stile di vita oppure utilizzare 2 metodi contraccettivi efficaci accettabili fino a 15 giorni dopo l’ultima dose del farmaco dello studio.
    E.4Principal exclusion criteria
    The patient has any new condition or worsening of an existing condition which in the opinion of the investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study.
    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 prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
    The patient is unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatotoxic (eg, 3 hydroxy 3 methylglutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, antidepressants, kava, echinacea) or may cause or prolong bleeding (eg, anticoagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng).
    The patient requires medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).
    The patient has an alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >250 IU/L or total bilirubin >1.5 mg/dL
    • Il paziente presenta un nuovo disturbo o il peggioramento del quadro clinico esistente che, a parere dello sperimentatore, potrebbe rendere il paziente non idoneo all’arruolamento oppure potrebbe interferire con la sua partecipazione allo studio o con la sua capacità di portarlo a termine.
    • Il paziente, a parere dello sperimentatore, non è in grado di aderire ai requisiti dello studio.
    • Il paziente non è disposto o in grado di astenersi dall’uso di alcolici nel giorno precedente e nei 3 giorni successivi a ciascuna infusione di olipudase alfa per tutta la durata del periodo del trattamento.
    • Il paziente non è disposto o in grado di evitare, nei 10 giorni precedenti e nei 3 giorni successivi le biopsie epatiche, l’uso di farmaci o integratori a base di erbe potenzialmente epatotossici (es. inibitori della 3-idrossi-3-metilglutaril coenzima A, eritromicina, acido valproico, antidepressivi, kava, echinacea) oppure che possono causare o prolungare il sanguinamento (es. anticoagulanti, ibuprofene, aspirina, integratori a base di aglio, ginkgo, ginseng).
    • Il paziente necessita di farmaco/i che può/possono ridurre l’attività di olipudase alfa (es. fluoxetina, clorpromazina, antidepressivi triciclici [es. imipramina, desipramina]).
    • Il paziente presenta valori di alanina aminotransferasi o aspartato aminotransferasi >250 IU/L o di bilirubina totale >1,5 mg/dL.
    E.5 End points
    E.5.1Primary end point(s)
    - Adverse events//treatment-emergent adverse events, including infusion-associated reactions.
    Physical examinations including neurologic examinations.
    Vital signs, echocardiograms and electrocardiograms.
    Clinical laboratory tests.
    Safety biomarkers.

    - Liver biopsy (patients previously enrolled in DFI13412).
    Liver ultrasound/Doppler (patients previously enroiled in DFI13803).

    - Immune response assessments
    • Eventi avversi/eventi avversi emergenti dal trattamento, incluse le reazioni associate all’infusione.
    • Esami obiettivi, inclusi esami neurologici.
    • Misurazione dei segni vitali, Ecocardiografia e Elettrocardiogrammi
    • Analisi cliniche di laboratorio.
    • Biomarcatori di sicurezza.
    • Biopsia epatica (solo pazienti che hanno precedentemente partecipato allo studio DFI13412).
    • Ecografia epatica con Doppler (solo pazienti che hanno precedentemente partecipato allo studio DFI13803).
    • Valutazioni della risposta immunitaria.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to 5 years
    Dal basale a 5 anni
    E.5.2Secondary end point(s)
    - Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume
    - Pulmonary Imaging
    - Pulmonary function test
    - Hematology (hemoglobin and platelet count)
    - Lipid profile
    - Health outcome questionnaires
    - Hand X ray for bone age and bone maturation (pediatric patients)
    - Tanner staging (pediatric patients)
    - Linear patient growth by height Z -score (pediatric patients)
    • Risonanza magnetica per immagini (RMI) addominale per valutare i miglioramenti nel volume di milza e fegato.
    • Imaging polmonare
    • Test di funzionalità polmonare
    • Ematologia (emoglobina e conta piastrinica)
    • Profilo lipidico
    • Questionari sullo stato di salute
    • Radiografia della mano per la valutazione dell’età ossea e della maturazione ossea (pazienti pediatrici)
    • Stadiazione di Tanner (pazienti pediatrici)
    • Crescita lineare dei pazienti mediante Z-score per l’altezza (pazienti pediatrici).
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume - Baseline to 5 years
    - Pulmonary Imaging - Baseline to 5 years
    - Pulmonary function test - Baseline to 5 years
    - Hematology (hemoglobin and platelet count) - Baseline to 5 years
    - Lipid profile - Baseline to 5 years
    - Health outcome questionnaires - Baseline to 5 years
    - Hand X ray for bone age and bone maturation (pediatric patients) - Baseline to 5 years
    - Tanner staging (pediatric patients) - Baseline to 5 years
    - Linear patient growth by height Z -score (pediatric patients) - Baseline to 5 year
    Dal basale a 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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    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) No
    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) 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 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 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
    Brazil
    France
    Germany
    Italy
    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
    Last visit of the last subject
    ULTIMA VISITA ULTIMO PAZIENTE
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    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: 12
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric patients
    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 8
    F.4.2.2In the whole clinical trial 17
    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)
    None
    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 Decision2016-08-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-08-02
    P. End of Trial
    P.End of Trial StatusOngoing
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