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    Summary
    EudraCT Number:2016-004087-19
    Sponsor's Protocol Code Number:ALN-AT3SC-009
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2018-10-19
    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 number2016-004087-19
    A.3Full title of the trial
    ATLAS-PPX trial: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis.
    ATLAS-PPX: Studio di switch in aperto, multinazionale, per descrivere l'efficacia e la sicurezza della profilassi con fitusiran in pazienti affetti da emofilia A e B che hanno ricevuto in precedenza profilassi con fattore o agente bypassante
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of fitusiran (ALN-AT3SC) in hemophilia A and B patients previously receiving factor or bypassing agent prophylaxis
    Studio di fitusiran (ALN-AT3SC) in pazienti affetti da emofilia A e B che hanno ricevuto in precedenza profilassi con fattore o agente bypassante
    A.3.2Name or abbreviated title of the trial where available
    ATLAS-PPX
    ATLAS-PPX
    A.4.1Sponsor's protocol code numberALN-AT3SC-009
    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 SponsorALNYLAM PHARMACEUTICALS, INC.
    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 supportAlnylam Pharmaceuticals, Inc.
    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 pointDorota Jusza
    B.5.3 Address:
    B.5.3.1Street AddressTrinity Park III, Domaniewska 49
    B.5.3.2Town/ cityWarsaw
    B.5.3.3Post code02-672
    B.5.3.4CountryPoland
    B.5.4Telephone number0048781811343
    B.5.5Fax number00
    B.5.6E-mailDorota.Jusza@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 ALN-AT3SC
    D.3.4Pharmaceutical form Solution for injection
    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 INNfitusiran
    D.3.9.1CAS number 1609016-97-8
    D.3.9.2Current sponsor codeALN-AT3SC
    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
    Emofilia A, Emofilia B
    Hemophilia A or Hemophilia 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 un disordine emorragico ereditato in cui il sangue non coaugula normalmente e può provocare sanguinamenti interni 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 10060614
    E.1.2Term Hemophilia B (Factor IX)
    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 10060613
    E.1.2Term Hemophilia A (Factor VIII)
    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 frequency of bleeding episodes while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor or bypassing agent prophylaxis.
    Caratterizzare la frequenza degli episodi di sanguinamento durante l’assunzione del trattamento con fitusiran, in relazione alla frequenza degli episodi di sanguinamento durante l’assunzione della profilassi con fattore o agente bypassante.
    E.2.2Secondary objectives of the trial
    To characterize the frequency of spontaneous bleeding episodes, the frequency of joint bleeding episodes in patients, and health related quality of life (HRQOL) in patients while receiving fitusiran treatment, relative to receiving factor or bypassing agent prophylaxis.
    Caratterizzare la frequenza degli episodi di sanguinamento spontaneo, la frequenza degli episodi di sanguinamento articolare nei pazienti e la qualità di vita correlata allo stato di salute (HRQOL) nei pazienti durante l'assunzione del trattamento con fitusiran, in relazione all’assunzione della profilassi con fattore o agente bypassante.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Males ≥12 years of age.
    - Severe hemophilia A or B (evidenced by a central laboratory FVIII <1% or FIX level ≤2% at Screening) with or without inhibitors
    - At least 1 bleeding episode in the last 12 months
    - AT activity ≥60% at Screening
    - Documented details of prophylactic treatment with factor concentrates or bypassing agents for the treatment of hemophilia A or B for at least 6 months
    - Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of patients under the age of legal consent
    - Soggetti di sesso maschile di ≥12 anni d'età.
    - Emofilia A o B grave (FVIII <1% o livello FIX ≤2% allo Screening dimostrato da un laboratorio centrale) con o senza inibitori
    - Almeno un episodio di sanguinamento negli ultimi 12 mesi
    - Attività AT ≥60% allo Screening
    - Documentazione dettagliata di trattamento profilattico con concentrati di fattore o agenti bypassanti per il trattamento dell’emofilia A o B per almeno 6 mesi
    - Volontà e capacità di aderire ai requisiti dello studio e di fornire il consenso informato scritto e l'assenso nel caso di pazienti al di sotto dell'età per fornire il consenso legale
    E.4Principal exclusion criteria
    - Patients with known co-existing bleeding disorders other than hemophilia A or B
    - Patients with clinically significant liver disease
    - Patients known to be HIV positive and have a CD4 count <200 cells/μL
    - Patients with a history of arterial or venous thromboembolism
    - Estimated glomerular filtration rate ≤45 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula)
    - Patients with a co-existing thrombophilic disorder
    - Patients with a history of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
    - Patients with a history of intolerance to SC injection(s)
    - Patients with an anticipated or planned need for surgery during the study
    - Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement
    - Pazienti con problemi di sanguinamento noti concomitanti diversi dall'emofilia A o B
    - Pazienti con malattia epatica clinicamente significativa
    - Pazienti con positività nota all'HIV e una conta CD4 <200 cellule/μl
    - Pazienti con anamnesi di tromboembolia arteriosa o venosa
    - Velocità di filtrazione glomerulare stimata ≤45 ml/minuto/1,73 m2 (utilizzando la formula di modifica della dieta nella malattia renale [Modification of Diet in Renal Disease, MDRD])
    - Pazienti con un disturbo trombofilico concomitante
    - Pazienti con anamnesi di molteplici allergie a farmaci o anamnesi di reazione allergica a un oligonucleotide o GalNAc
    - Pazienti con anamnesi di intolleranza alle iniezioni SC
    - Pazienti con necessità prevista o pianificata di intervento chirurgico nel corso dello studio
    - Qualsiasi altra condizione o comorbilità che renderebbe il paziente non idoneo all'arruolamento o potrebbe interferire con la partecipazione nello o con il completamento dello studio, secondo il giudizio dello Sperimentatore
    E.5 End points
    E.5.1Primary end point(s)
    Annualized bleeding rate (ABR)
    Tasso annuale di sanguinamento (ABR)
    E.5.1.1Timepoint(s) of evaluation of this end point
    through 13 months
    13 mesi
    E.5.2Secondary end point(s)
    - Annualized spontaneous bleeding rate
    - Annualized joint bleeding rate
    - Haem-A-QOL score
    - Tasso annuale di sanguinamento spontaneo
    - Tasso annuale di sanguinamento articolare
    - Punteggio Haem-A-QOL
    E.5.2.1Timepoint(s) of evaluation of this end point
    through 13 months
    13 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
    Profilassi, Terapia standard
    Prophylaxis SOC
    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 EEA32
    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
    Brazil
    Bulgaria
    Canada
    China
    Denmark
    France
    Germany
    Hungary
    Ireland
    Israel
    Italy
    Japan
    Korea, Republic of
    Malaysia
    Netherlands
    Portugal
    Russian Federation
    South Africa
    Spain
    Switzerland
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    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: 1
    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: 7
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 78
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 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
    Subject must provide written informed consent and assent in the case of patients under the age of legal consent.
    Il soggetto deve fornire il consenso informato scritto e l'assenso in caso di pazienti al di sotto dell'età per fornire il consenso legale.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 32
    F.4.2.2In the whole clinical trial 90
    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)
    Consistent with pre-study treatment.
    Conforme al trattamento pre-studio.
    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 Decision2018-05-24
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
    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
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