Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   39363   clinical trials with a EudraCT protocol, of which   6448   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2018-002880-25
    Sponsor's Protocol Code Number:LTE15174
    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:2021-01-29
    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 number2018-002880-25
    A.3Full title of the trial
    ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX
    ATLAS-OLE: Studio in aperto per valutare l’efficacia e la sicurezza a lungo termine di Fitusiran in pazienti affetti da emofilia A o B, con o senza anticorpi inibitori del fattore VIII o IX
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX
    Studio per valutare l’efficacia e la sicurezza a lungo termine di Fitusiran in pazienti con emofilia A o B, con o senza anticorpi inibitori del fattore VIII o IX
    A.3.2Name or abbreviated title of the trial where available
    ATLAS-OLE
    ATLAS-OLE
    A.4.1Sponsor's protocol code numberLTE15174
    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)U1111-1210-0018
    A.5.4Other Identifiers
    Name:IND 125632Number:IND 125632
    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 organisationPPD Global
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street Address929 North Front Street
    B.5.3.2Town/ cityWilmington
    B.5.3.3Post code28401
    B.5.3.4CountryUnited States
    B.5.4Telephone number+498957877131
    B.5.5Fax number+498957877400
    B.5.6E-mailSaban.Musoski@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 Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1297; EU/3/14/1298
    D.3 Description of the IMP
    D.3.1Product nameFitusiran
    D.3.2Product code [SAR439774]
    D.3.4Pharmaceutical form Concentrate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1609016-97-8
    D.3.9.2Current sponsor codeSAR439774
    D.3.9.4EV Substance CodeSUB130859
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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.IMP: 2
    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/3/14/1297; EU/3/14/1298 D.2.6 Has the IMP been
    D.3 Description of the IMP
    D.3.1Product nameFitusiran
    D.3.2Product code SAR439774
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1609016-97-8
    D.3.9.2Current sponsor codeSAR439774
    D.3.9.4EV Substance CodeSUB130859
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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
    Hemophilia A or Hemophilia B
    Emofilia A o Emofilia B
    E.1.1.1Medical condition in easily understood language
    Hemophilia is an inherited bleeding disorder in which the blood does not clot normally and can result in internal bleeding into the muscles and joints.
    L'emofilia è una malattia emorragica ereditaria in cui il sangue non si coagula normalmente e può provocare emorragie interne nei muscoli e nelle articolazioni.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10060613
    E.1.2Term Hemophilia A (Factor VIII)
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10060614
    E.1.2Term Hemophilia B (Factor IX)
    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
    To characterize the long-term safety and tolerability of fitusiran
    Caratterizzare sicurezza e tollerabilità a lungo termine di fitusiran
    E.2.2Secondary objectives of the trial
    To characterize the long-term efficacy of fitusiran as assessed by the
    frequency of:
    - Bleeding episodes,
    - Spontaneous bleeding episodes,
    - Target joint bleeding episodes.
    To characterize the effects of fitusiran on health-related quality of life
    measures in participants =17 years of age.
    Caratterizzare l'efficacia a lungo termine di fitusiran valutata in base alla frequenza di:
    - episodi di sanguinamento,
    - episodi di sanguinamento spontaneo,
    - episodi di sanguinamento delle articolazioni target.
    Caratterizzare gli effetti di fitusiran sulle misure della qualità di vita correlata allo stato di salute nei partecipanti di età = 17 anni.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Age
    01. Participant must be at least 12 years of age inclusive, at the time of signing the informed consent
    Type of participant and disease characteristics
    02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial
    Sex
    03. Male
    A) There are no contraceptive requirements for this study except where required by local regulations.
    Informed Consent
    04. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized
    representative.
    I partecipanti sono eleggibili per l’inclusione nello studio solo se soddisfano tutti i criteri seguenti:
    Età
    01. Il partecipante deve avere un’età di almeno 12 anni compiuti al momento della firma del consenso informato

    Tipo di partecipante e caratteristiche della malattia
    02. Partecipanti con emofilia A o B grave che hanno completato una sperimentazione clinica con fitusiran di Fase 3

    Sesso
    03. Maschile
    A) Non vi sono requisiti di contraccezione per questo studio, ad eccezione dei casi in cui previsto dalle normative locali.

    Consenso informato
    04. Essere in grado di fornire un consenso informato firmato che include il rispetto dei requisiti e delle limitazioni elencati nel modulo di consenso informato e in questo protocollo. Nei Paesi in cui la maggiore età legale è al di sopra dei 18 anni, deve essere firmato un ICF specifico anche dal rappresentante legale del partecipante.
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteria
    apply:
    Medical conditions
    01. Completion of a surgical procedure within 14 days prior to screening,
    or currently receiving additional factor concentrate or BPA infusion for
    postoperative hemostasis
    Prior/concomitant therapy
    02. Current participation in ITI
    03. Current use of factor concentrates or BPAs as regularly administered
    prophylaxis designed to prevent spontaneous bleeding episodes
    04. Use of compounds other than factor concentrates or BPAs for
    hemophilia treatment
    Prior/concurrent clinical study experience
    05. Received an investigational drug or device, other than fitusiran,
    within 30 days of anticipated IMP administration or 5 half-lives of the
    IMP, whichever is longer
    06. Current or prior participation in a gene therapy trial
    Diagnostic assessments
    07. ALT and/or AST >1.5× upper limit of normal reference range (ULN)
    for patients who are naïve to fitusiran at study start; ALT and/or AST
    >5× ULN for patients who were in the fitusiran arm in the parent study
    Other exclusions
    08. Individuals accommodated in an institution because of regulatory or
    legal order; prisoners or participants who are legally institutionalized
    09. Any country-related specific regulation that would prevent the
    participant from entering the study
    10. Participant not suitable for participation, whatever the reason, as
    judged by the Investigator, including medical or clinical conditions, or
    participants potentially at risk of noncompliance to study procedures
    11. Participants who are dependent on the Sponsor or Investigator (as
    defined in section 1.61 of the International Council for Harmonisation
    (ICH)-Good Clinical Practice (GCP) E6)
    12. Participants are employees of the clinical study site or other
    individuals directly involved in the conduct of the study, or immediate
    family members of such individuals
    13. Any specific situation during study implementation/course that may
    rise ethics considerations
    14. Sensitivity to any of the study interventions, or components thereof,
    or drug or other allergy that, in the opinion of the Investigator,
    contraindicates participation in the study
    I partecipanti sono esclusi dallo studio se soddisfano uno dei seguenti criteri:

    Condizioni mediche
    01. Completamento di procedura chirurgica entro i 14 giorni precedenti lo Screening o attuale trattamento con infusione aggiuntiva di concentrato di fattore o BPA per emostasi postoperatoria

    Terapia precedente/concomitante
    02. Attuale partecipazione a terapia di induzione della tolleranza immunologica (ITI)
    03. Attuale uso di concentrati di fattore o BPA come profilassi regolarmente somministrata selezionata per prevenire episodi di sanguinamento spontaneo
    04. Uso di composti diversi dai concentrati di fattore o BPA per il trattamento dell'emofilia

    Esperienza precedente/concomitante in studi clinici
    05. Trattamento con un farmaco o dispositivo sperimentale, diverso da fitusiran, nei 30 giorni precedenti la somministrazione prevista dell'IMP o entro 5 emivite dell'IMP, a seconda di quale sia il periodo più lungo
    06. Partecipazione attuale o passata in una sperimentazione con terapia genica

    Valutazioni diagnostiche
    07. ALT e/o AST > 1,5 × il limite superiore della norma (ULN) per i pazienti naïve a fitusiran all'inizio dello studio; ALT e/o AST > 5 × ULN per i pazienti che erano nel braccio trattato con fitusiran nello studio originario.

    Altre esclusioni
    08. Soggetti che risiedono in un istituto a causa di un provvedimento normativo o legale; detenuti o partecipanti che sono istituzionalizzati per disposizione di legge
    09. Qualsiasi normativa specifica del Paese che impedisca al partecipante di entrare nello studio
    10. Partecipante non idoneo alla partecipazione, secondo il giudizio dello sperimentatore, qualunque sia la ragione, comprese condizioni mediche o cliniche, o partecipanti potenzialmente a rischio di non conformità alle procedure dello studio
    11. Partecipanti che dipendono dallo Sponsor o dallo sperimentatore (come definito nella sezione 1.61 delle norme di buona pratica clinica (GCP), descritte dalle linee guida E6 della Conferenza Internazionale sull’Armonizzazione (ICH))
    12. Partecipanti che sono dipendenti del centro dello studio clinico o qualsiasi altro soggetto direttamente coinvolto nella conduzione dello studio o familiari diretti di tali soggetti
    13. Qualsiasi situazione specifica durante l'attuazione/lo svolgimento dello studio che possa sollevare considerazioni dal punto di vista etico
    14. Sensibilità a uno qualsiasi degli interventi dello studio, o relativi componenti, o allergia a farmaci o altre allergie che, secondo il parere dello sperimentatore, costituiscano una controindicazione alla partecipazione allo studio
    E.5 End points
    E.5.1Primary end point(s)
    Incidence, severity, relatedness, and seriousness of AEs, and laboratory assessments
    Incidenza, gravità, correlazione e serietà degli AE e valutazioni di laboratorio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Through duration of the study (up to 55 months)
    Per tutta la durata dello studio (fino a 55 mesi)
    E.5.2Secondary end point(s)
    Annualized bleeding rate in the treatment period.
    Annualized spontaneous bleeding rate in the treatment period.
    Annualized joint bleeding rate in the treatment period.
    Change in haemophilia quality of life questionnaire for adults physical
    health score and total score in the treatment period (in participants =17
    years of age).
    Tasso annuale di sanguinamento nel periodo di trattamento.
    Tasso annuale di sanguinamento spontaneo nel periodo di trattamento.
    Tasso annuale di sanguinamento articolare nel periodo di trattamento.
    Variazione nel questionario sulla qualità di vita specifico per l'emofilia a livello di punteggio di salute fisica dell'adulto e punteggio totale durante il periodo di trattamento (nei partecipanti = 17 anni di età).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Through duration of treatment (up to 48 months)
    Per tutta la durata del trattamento (fino a 48 mesi)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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 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 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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Rescue therapy per standard of Care
    Rescue therapy per standard of Care
    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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA40
    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
    Australia
    Bulgaria
    Canada
    China
    Denmark
    France
    Germany
    Hungary
    Iceland
    India
    Ireland
    Italy
    Japan
    Korea, Democratic People's Republic of
    Malaysia
    Netherlands
    Portugal
    Russian Federation
    South Africa
    Spain
    Taiwan
    Turkey
    Ukraine
    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 Patient Last Visit (LPLV)
    Ultima visita dell’ultimo paziente (LPLV)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days3
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 22
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 210
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 12
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 244
    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
    Nessuno
    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 Decision2019-04-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-06-18
    P. End of Trial
    P.End of Trial StatusOngoing
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA