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    Summary
    EudraCT Number:2013-002987-17
    Sponsor's Protocol Code Number:ALN-TTR02-004
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-10-01
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2013-002987-17
    A.3Full title of the trial
    APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of ALN-TTR02 in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)
    APOLLO: Estudio en fase III, randomizado, doble ciego,
    controlado con placebo, multicéntrico y multinacional para
    evaluar la eficacia y la seguridad de ALN-TTR02 en la
    polineuropatía mediada por la transtirretina (TTR)
    (polineuropatía amiloidótica familiar - PAF)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The Study of an Investigational Drug, ALN-TTR02, for the Treatment of
    Transthyretin (TTR)-Mediated Amyloidosis.
    El estudio de un fármaco en investigación, ALN-TTR02, para el tratamiento de
    Transtiretina (TTR) mediada por amiloidosis.
    A.4.1Sponsor's protocol code numberALN-TTR02-004
    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 organisationMedpace Finland OY
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressPormestarinrinne 8
    B.5.3.2Town/ cityHelsinki
    B.5.3.3Post code00160
    B.5.3.4CountryFinland
    B.5.4Telephone number+49896737916
    B.5.5Fax number+49896702100
    B.5.6E-mailregsubmissions@medpace.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/3/11/857
    D.3 Description of the IMP
    D.3.1Product nameALN-TTR02
    D.3.2Product code ALN-TTR02
    D.3.4Pharmaceutical form Solution for injection
    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 INNALN-18328
    D.3.9.2Current sponsor codeALN-18328
    D.3.9.3Other descriptive nameALN-18328
    D.3.9.4EV Substance CodeSUB31538
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Transthyretin mediated amyloidosis (ATTR)
    amiloidosis mediada por la transtirretina
    (ATTR)
    E.1.1.1Medical condition in easily understood language
    ATTR is a hereditary disease caused by protein aggregates in the heart
    and the nervous system. It leads to heart dysfunction, damages to the
    nerves, and gastrointestinal and bladder dysfunctions.
    ATTR es una enfermedad hereditaria causada por agregados de proteínas en el corazón y el SN. Esto conduce a la disfunción del corazón,los daños en los nervios, y disfunciones gastroint.y en la vejiga.
    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 16.0
    E.1.2Level PT
    E.1.2Classification code 10007509
    E.1.2Term Cardiac amyloidosis
    E.1.2System Organ Class 10007541 - Cardiac disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level PT
    E.1.2Classification code 10019889
    E.1.2Term Hereditary neuropathic amyloidosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the efficacy of ALN-TTR02
    Determinar la eficacia de ALN-TTR02
    E.2.2Secondary objectives of the trial
    To determine the effect of ALN-TTR02 on various clinical parameters
    Determinar el efecto de ALN-TTR02 en varios parámetros clínicos
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ? Male or female of 18 to 80 years of age (inclusive);
    ? Have a diagnosis of FAP
    ? Meet Neuropathy Impairment Score requirements and Karnofsky performance status requirements
    ? Have adequate complete blood counts and liver function tests
    ? Have adequate cardiac function
    ? Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)
    1.Cualquier sexo, de 18 a 80 años de edad (inclusive)
    2.Presentar diagnóstico de PAF
    3.alcanzar los requisitos de puntuación del deterioro Neuropático y los requisitos del estado de rendimiento de Karnofsky
    4.Tener recuentos sanguíneos completos adecuados y pruebas de función hepática.
    5.Tener la función cardíaca adecuada
    6.Seronegativo para los virus de las hepatitis B (VHB) y C
    (VHC);
    E.4Principal exclusion criteria
    ? Had a prior liver transplant or is planned to undergo liver transplant during the study period;
    ? Has untreated hypo- or hyperthyroidism;
    ? Has known human immunodeficiency virus (HIV) infection;
    ? Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
    ? Recently received an investigational agent or device
    ? Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid
    Tener un trasplante de hígado previo o tener planeado someterse a trasplante de hígado durante el período de estudio;
    ? Tener hipo o hipertiroidismo no tratado
    ? Tener el virus de inmunodeficiencia humana (VIH);
    ? Tener un tumor maligno dentro de los ultimos 2 años, excepto para el carcinoma de células basales o escamosas de la piel o carcinoma in situ del cuello del útero que se ha tratado con éxito;
    ? Haya recibido recientemente un agente o dispositivo de investigación
    ? Esté tomando diflunisal, tafamidis, doxiciclina o ácido tauroursodeoxicólico
    E.5 End points
    E.5.1Primary end point(s)
    The difference between the ALN TTR02 and placebo groups in the change from baseline of modified Neuropathy Impairment Score+7 (mNIS+7)
    la diferencia entre los
    grupos de ALN-TTR02 y placebo en cuanto a la variación de la
    puntuación NIS+7 modificada (mNIS+7) desde el momento basal
    E.5.1.1Timepoint(s) of evaluation of this end point
    The difference between the ALN TTR02 and placebo groups in the change from baseline of modified NIS+7 (mNIS+7) at 18 months
    la diferencia entre los
    grupos de ALN-TTR02 y placebo en cuanto a la variación de la
    puntuación NIS+7 modificada (mNIS+7) hasta el mes 18.
    E.5.2Secondary end point(s)
    The difference between ALN-TTR02 and placebo in the change from baseline in the following measurements:
    ? Quality of Life
    ? Modified Body Mass Index
    ? Autonomic function
    ? Muscle weakness as assessed by walking test
    la diferencia entre ALN-TTR02 y placebo en
    cuanto a la variación de las siguientes determinaciones desde el momento basal:
    . Calidad de vida
    . Indice de masa corporal modificado
    .Debilidad muscular evaluada por el test de caminata
    E.5.2.1Timepoint(s) of evaluation of this end point
    The difference between ALN-TTR02 and placebo in the change from baseline in the various clinical parameters at 18 months
    la diferencia entre ALN-TTR02 y placebo en
    cuanto a la variación de las siguientes determinaciones desde el
    momento basal hasta el mes 18:
    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 No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA14
    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
    Argentina
    Brazil
    France
    Germany
    Italy
    Mexico
    Netherlands
    Portugal
    Spain
    Sweden
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    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.1.1.1In 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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 100
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state27
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 143
    F.4.2.2In the whole clinical trial 200
    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, if any.
    De acuerdo con el tratamiento de pre-estudio, si lo hay.
    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 Decision2013-11-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-08-17
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