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    Summary
    EudraCT Number:2018-003519-24
    Sponsor's Protocol Code Number:ALN-TTR02-008
    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:2019-04-10
    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 number2018-003519-24
    A.3Full title of the trial
    An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients with Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) with Disease Progression Post-Orthotopic Liver Transplant
    Título del protocolo Estudio abierto para evaluar la seguridad, la eficacia y la farmacocinética (FC) de patisirán-LPN en pacientes con amiloidosis hereditaria por transtiretina (amiloidosis hATTR) y progresión de la enfermedad después de un trasplante ortotópico de hígado
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Patisiran-LNP in Patients with hATTR Amyloidosis Disease Progression Post-Liver Transplant
    Patisiran-NPL en Pacientes con progresion de la enfermedad de amiloidosis hATTR después de un transplante de higado.
    A.4.1Sponsor's protocol code numberALN-TTR02-008
    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 organisationAlnylam Pharmaceuticals, Inc
    B.5.2Functional name of contact pointClinical Trial Information Line
    B.5.3 Address:
    B.5.3.1Street Address300 Third Street
    B.5.3.2Town/ cityCambridge MA
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018772569526
    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 Yes
    D.2.1.1.1Trade name Onpattro
    D.2.1.1.2Name of the Marketing Authorisation holderAlnylam Netherlands B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 namepatisiran-LNP
    D.3.2Product code ALN-TTR02
    D.3.4Pharmaceutical form 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 INNPATISIRAN
    D.3.9.2Current sponsor codeALN-TTR02
    D.3.9.4EV Substance CodeSUB189946
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis)
    amiloidosis hereditaria por transtiretina (amiloidosis hATTR).
    E.1.1.1Medical condition in easily understood language
    hATTR 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.
    hATTR es una enfermedad hereditaria causada por una agregacion de proteinas en el corazon y el sistema nervioso. Conduce a disfuncion cardiaca,gastrointestinal y vejiga y daños en los nervios
    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 20.0
    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 20.0
    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 evaluate the TTR reduction of patisiran-LNP in hATTR amyloidosis patients with disease progression after OLT
    Evaluar la reducción de la TTR con patisirán-LPN en pacientes con amiloidosis hATTR y progresión de la enfermedad después de un trasplante ortotópico de hígado (THO).
    E.2.2Secondary objectives of the trial
    - To evaluate the effect of patisiran-LNP on neuropathy
    - To evaluate the effect of patisiran-LNP on patient reported outcomes including QoL, activities of daily living and autonomic neuropathy symptoms
    - To characterize the effect on nutritional status
    -Evaluar el efecto de patisirán-LPN sobre la neuropatía.
    -Evaluar el efecto de patisirán-LPN sobre resultados comunicados por los pacientes, como calidad de vida, actividades cotidianas y síntomas de neuropatía vegetativa.
    -Determinar el efecto sobre el estado nutricional.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -18 years of age or older
    -Received an orthotopic liver transplant for treatment of hATTR amyloidosis at least 1 year prior
    -Increase in polyneuropathy disability post-liver transplant as measured by polyneuropathy disability (PND) score.
    -Karnofsky performance status of ≥70%
    -Edad de 18 años o más.
    -Recepción de un TOH para tratar una amiloidosis por hATTR documentada al menos 1 año antes.
    -Aumento documentado de la Puntuación de discapacidad por polineuropatía en comparación con una evaluación previa al trasplante hepático O aumento documentado de la puntuación PND.
    -Estado funcional de Karnofsky ≥ 70%
    E.4Principal exclusion criteria
    -Currently taking tafamidis, doxycycline, diflunisal, or tauroursodeoxycholic acid
    -Liver allograft rejection episodes or abnormal LFTs suggestive of possible allograft rejection in the past 6 months
    -Abnormal liver function tests
    -Known leptomeningeal amyloidosis
    -New York Heart Association (NYHA) classification of >2
    -PND score IV (wheelchair bound or bedridden)
    - Prior organ transplant other than liver transplant
    -Hospitalization for congestive heart failure or arrhythmia in the past 3 months
    -Tratamiento activo con tafamidís, doxiciclina o ácido tauroursodesoxicólico.
    -Episodios de rechazo del aloinjerto hepático (crónico, agudo o subagudo) o anomalías en las PFH indicativas de un posible rechazo del aloinjerto en los seis meses previos a la fecha de obtención del consentimiento informado.
    -Resultados anormales en las pruebas de función hepática.
    -Amiloidosis leptomeníngea conocida.
    -Clase > 2 de la New York Heart Association (NYHA) en la visita de selección.
    -Puntuación PND de IV (confinado en una silla de ruedas o encamado).
    - Transplante de otros organos que no sea el higado
    -Hospitalización por insuficiencia cardíaca congestiva o arritmia en los tres meses previos
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline in serum TTR levels
    Reducción porcentual media de la TTR.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 6, Month 12
    Mes 6, Mes 12
    E.5.2Secondary end point(s)
    -Change from baseline in Neuropathy Impairment Score (NIS)
    -Change from baseline in quality of life and disability as assessed by Norfolk Quality of Life (QoL-DN), Rasch-built Overall Disability Scale (R-ODS), and Autonomic Symptoms Questionnaire (COMPASS 31)
    -Change from baseline in modified Body Mass Index (mBMI)
    -Puntuación NIS (Puntuación de deterioro neurológico).
    -Medidas de calidad de vida comunicadas por los pacientes (Norfolk QoL-DN), actividades cotidianas (R-ODS) y síntomas vegetativos (COMPASS-31).
    -Estado nutricional (IMCm).
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months (this is applicable to all 3 of the secondary end points)
    12 meses ( esto se aplica a los 3 puntos finales secundarios)
    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 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 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 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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    Ultina visita del ultimo paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    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 years1
    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 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: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    After participation in the trial, patients will be treated with the current standard therapy or with patisiran if it is commercially available in their respective country at that time.
    Después de la participación en el ensayo, los pacientes serán tratados con la terapia estándar actual o con patisiran si está disponible comercialmente en sus respectivos países en ese momento.
    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 Decision2019-03-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-10-20
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