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    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.
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    Summary
    EudraCT Number:2016-004087-19
    Sponsor's Protocol Code Number:ALN-AT3SC-009
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2017-07-12
    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 ConcernedSpain - AEMPS
    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: estudio abierto, internacional, de cambio de tratamiento para evaluar la eficacia y la seguridad de la profilaxis con fitusiran en pacientes con hemofilia A y B que recibían previamente profilaxis con factores o agentes de puenteo
    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
    Un estudio de fitusiran (ALN-AT3SC) en pacientes con hemofilia A y B que recibían previamente profilaxis con factores o agentes de puenteo
    A.3.2Name or abbreviated title of the trial where available
    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.5.6E-mailclinicaltrials@alnylam.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 IMPSubcutaneous use
    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 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 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
    Hemofilia A o Hemofilia 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.
    La hemofilia es un trastorno hemorrágico hereditario en el que la sangre no coagula normalmente y puede dar lugar a sangrado interno en los músculos y articulaciones
    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 100000011915
    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 100000011913
    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.
    Caracterizar la frecuencia de episodios hemorrágicos durante el tratamiento con fitusiran, en comparación con la frecuencia de episodios hemorrágicos durante la profilaxis con factores o agentes de puenteo
    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.
    Caracterizar la frecuencia de episodios hemorrágicos espontáneos ,la frecuencia de episodios hemorrágicos articulares y la calidad de vida relacionada con la salud (CVRS) en pacientes mientras recibían tratamiento con fitusirán en comparación con recibir factores o agentes de puenteo.
    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
    Varones ≥ 12 años de edad
    Hemofilia A o B (demostrada por una determinación del laboratorio central de FVIII < 1% o FIX ≤ 2% en la selección) con o sin inhibidores
    Mínimo de 1 episodio hemorrágico en los 12 meses previos a la selección
    Actividad AT >= 60% en la selección
    Detalles documentados del tratamiento profiláctico para la hemofilia A o B con concentrados de factores o agentes de puenteo desde al menos 6 meses antes de la selección
    Disposición y capacidad para cumplir los requisitos del estudio y para dar su consentimiento informado por escrito, y su asentimiento en el caso de pacientes por debajo de la edad legal de consentimiento, conforme a los requisitos locales y nacionales.
    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
    Trastornos hemorrágicos coexistentes conocidos diferentes de la hemofilia A o B
    Presencia de una hepatopatía clínicamente significativa
    VIH positivo conocido con recuento de CD4 < 200 células/μl
    Pacientes con antecedentes de tromboembolismo arterial o venoso
    Filtración glomerular estimada ≤ 45 ml/min/1,73 m2 (usando la fórmula de la Modificación de la dieta de la enfermedad renal [ MDRD ]).
    Pacientes con trastorno trombofílico coexistente
    Pacientes con antecedentes de alergias múltiples a fármacos o antecedentes de reacción alérgica a un oligonucleótido o a GalNAc
    Pacientes con antecedentes de intolerancia a inyecciones SC.
    Pacientes con necesidad prevista de una intervención quirúrgica durante el estudio o intervención quirúrgica cuya realización esté programada durante el estudio
    Cualquier transtorno (p. ej., problema médico) que en opinión del investigador determine que el paciente no sería apto para recibir el tratamiento el día 1, o que podría interferir en el cumplimiento del estudio, la seguridad del paciente o la participación del paciente hasta completar el período de tratamiento del estudio
    E.5 End points
    E.5.1Primary end point(s)
    Annualized bleeding rate (ABR)
    Tasa anualizada de hemorragias (TAH)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Through 13 months
    Hasta 13 meses
    E.5.2Secondary end point(s)
    Annualized spontaneous bleeding rate

    Annualized joint bleeding rate

    Haem-A-QOL score
    Tasa anualizada de hemorragias espontáneas
    Tasa anualizada de hemorragias articulares
    puntuación Haem-A-QOL
    E.5.2.1Timepoint(s) of evaluation of this end point
    Through 13 months
    Hasta 13 meses
    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
    profilaxis práctica clinica habitual
    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 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
    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
    UVUP
    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 months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    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: 7
    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.
    El paciente debe proporcionar el consentimiendo informado firmado y el asentimiento en caso de pacientes menores de edad
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state13
    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.
    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 Decision2017-08-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-10
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
    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
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