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    Summary
    EudraCT Number:2017-001621-41
    Sponsor's Protocol Code Number:AC-011-IT
    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:2020-11-04
    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 number2017-001621-41
    A.3Full title of the trial
    A phase III randomized study of doxycycline and tauroursodeoxycholic acid (Doxy/TUDCA) plus standard supportive therapy versus standard supportive therapy alone in cardiac amyloidosis caused by transthyretin
    Studio clinico in aperto di fase III, randomizzato, per valutare l’efficacia di un trattamento con doxiciclina e acido taurodesossicolico (Doxy-TUDCA) in associazione alla terapia di supporto standard contro la sola terapia di supporto standard, in pazienti affetti da amiloidosi cardiaca da transtiretina.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment with doxycycline and tauroursodeoxycholic acid (Doxy/TUDCA) plus standard supportive therapy in cardiac amyloidosis caused by transthyretin
    Trattamento con con doxiciclina e acido taurodesossicolico (Doxy-TUDCA) in aggiunta alla terapia standard in pazienti affetti da amiloidosi cardiaca da transtiretina
    A.3.2Name or abbreviated title of the trial where available
    AmY-DoT
    AmY-DoT
    A.4.1Sponsor's protocol code numberAC-011-IT
    A.5.4Other Identifiers
    Name:NANumber:NA
    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 SponsorFONDAZIONE I.R.C.C.S. POLICLINICO SAN MATTEO
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSocietà Italiana per L'Amiloidosi ONLUS
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione IRCCS Policlinico San Matteo
    B.5.2Functional name of contact pointCentro per lo studio e la cura dell
    B.5.3 Address:
    B.5.3.1Street AddressP.le Golgi 19
    B.5.3.2Town/ city27100
    B.5.3.3Post codePavia
    B.5.3.4CountryItaly
    B.5.4Telephone number0382502994
    B.5.5Fax number0382502990
    B.5.6E-mailsegreteria.amiloidosi@smatteo.pv.it
    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 BASSADO - 100 MG COMPRESSE 10 COMPRESSE
    D.2.1.1.2Name of the Marketing Authorisation holderPFIZER ITALIA S.R.L.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameDoxiciclina
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDOXICICLINA
    D.3.9.1CAS number 564-25-0
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 Yes
    D.2.1.1.1Trade name TAURO - 250 MG CAPSULE RIGIDE 20 CAPSULE
    D.2.1.1.2Name of the Marketing Authorisation holderTEOFARMA S.R.L.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAcido tauroursodesossicolico
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNACIDO TAUROURSODESOSSICOLICO
    D.3.9.1CAS number 128-13-2
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    Cardiac amyloidosis caused by transthyretin
    Amiloidosi cardiaca da transtiretina
    E.1.1.1Medical condition in easily understood language
    Cardiac amyloidosis caused by transthyretin
    Amiloidosi cardiaca causata da transtiretina
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy (superiority) of Doxy/TUDCA in patients with cardiac ATTR
    amyloidosis,
    Valutare l'efficacia di Doxy/TUDCA nei pazienti con amiloidosi cardiaca da transtiretina.
    E.2.2Secondary objectives of the trial
    -To compare overall survival at 18 and 30 months of patients receiving Doxy/TUDCA in addition to standard supportive therapy with that of patients receiving standard supportive therapy alone.
    - To assess safety of Doxy/TUDCA by measuring the rate and grade of adverse events.
    - To assess changes in biochemical, echocardiographic, and clinical markers of disease severity during and after treatment.
    - To assess the impact of re-treatment with Doxy/TUDCA in patients who experience a major
    cardiac event after Doxy/TUDCA.
    - To assess discontinuation on biochemical, echocardiographic, and clinical markers of disease
    severity.
    - To assess the impact of treatment with Doxy/TUDCA in patients originally enrolled in the
    control arm who experience a “major cardiac event” and are then given Doxy/TUDCA on biochemical, echocardiographic, and clinical markers of disease severity.
    - confrontare la sopravvivenza globale a 18 e 30 mesi dei pazienti trattati Doxy/TUDCA in aggiunta alla terapia di supporto standard, rispetto ai
    pazienti trattati unicamente con terapia di supporto standard.
    - valutare la sicurezza di Doxy/TUDCA, misurando frequenza e grado degli eventi avversi.
    - valutare i cambiamenti negli esami biochimici, nell’esame ecocardiografico, e nei marcatori clinici di gravità della malattia, durante e dopo il trattamento.
    - valutare l'impatto del ri-trattamento con Doxy/TUDCA sugli esami biochimici, ecocardiografici e sui marker clinici di gravità della malattia nei pazienti che presentano un importante evento cardiaco dopo la sospensione di Doxy/TUDCA.
    - valutare l'impatto della discontinuazione sugli esami biochimici, ecocardiografici e sui marker clinici della malattia.
    - valutare l'impatto del trattamento con Doxy/TUDCA su marcatori biochimici, ecografici e clinici di gravità della malattia nei pazienti originariamente arruolati nel gruppo di contro
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Diagnosis of ATTRwt amyloidosis, or ATTRm p.Ile68Leu and p.Val122Ile;
    - 18 years or older;
    - cardiac involvement (mean left ventricular wall thickness >12 mm in the absence of other causes);
    - history of occurrence of at least 1 event of symptomatic heart failure;
    - stable diuretic dosage for at least 2 weeks before treatment initiation;
    - agree to practice effective methods of contraception where necessary
    - voluntary written consent
    - diagnosi di amiloidosi ATTRwt, o ATTRm p.Ile68Leu e p.Val122Ile.
    - età maggiore di 18 anni
    - coinvolgimento cardiaco (spessore della parete ventricolare media >12 mm assenza di altre cause)
    - storia di insorgenza di almeno un caso di insufficienza cardiaca sintomatica
    - dosaggio diuretico stabile per almeno due settimane prima di iniziare il trattamento
    - utilizzo di adeguato metodo contraccettivo, ove necessario
    - consenso informato scritto
    E.4Principal exclusion criteria
    - Non-ATTR amyloidosis
    - pregnant or nursing women
    - uncontrolled bacterial, viral, fungal, HIV, HBV, or HCV infection;
    - presence of other active malignancy with
    - known allergy to tetracycline;
    - treatment with drugs potentially affecting doxycycline absorption
    - significant acute gastrointestinal symptoms;
    - active peptic ulceration and/or esophageal reflux disease
    - amiloidosi non-ATTR
    - donne in gravidanza o in allattamento
    - infezione non controllata
    - presenza di altri tumori maligni attivi
    - allergia nota alle tetracicline
    - trattamento con farmaci che potenzialmente influenzano l'assorbimento della doxiciclina
    - sintomi gastrointestinali acuti significativi
    - ulcera peptica attiva e/o malattia da reflusso esofageo
    E.5 End points
    E.5.1Primary end point(s)
    Major cardiac events
    Eventi cardiaci maggiori
    E.5.1.1Timepoint(s) of evaluation of this end point
    18 months
    18 mesi
    E.5.2Secondary end point(s)
    Overall survival; Adverse events equal or higher grade 3 (CTCAE v 5.0); Changes in BNP, NT-proBNP, cTnI, mLVW, EF, MCF, GLS, 6MWT
    Sopravvivenza globale; Eventi avversi grado 3 o superiore (CTCAE v 5.0); Cambiamenti nei valori di BNP, NT-proBNP, cTnI, spessore mLVW, EF, MCF, GLS, 6MWT
    E.5.2.1Timepoint(s) of evaluation of this end point
    18 and 30 months; At each visit; After 6, 12, 18 months during treatment and at 6, 12 months after the end of treatment
    18 e 30 mesi; Ad ogni visita; A 6, 12 e 18 mesi durante il trattamento, e 6 e 12 mesi dopo la sospensione del trattamento rispetto al basale
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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 Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3.1Comparator description
    Terapia di supporto standard
    Standard supportive therapy
    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 concerned4
    E.8.5The trial involves multiple Member States No
    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 Information not present in EudraCT
    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
    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 months0
    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 months0
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 66
    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 state102
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 102
    F.4.2.2In the whole clinical trial 102
    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)
    Access to treatment with Doxy/TUDCA will be granted to experimental arm patients who experience a "major cardiac event" off-treatment during the follow-up period and to ontrol arm patients after they meet the primary endpoint.
    L'accesso al trattamento con Doxy/TUDCA sarà concesso a pazienti randomizzati nel braccio sperimentale che presentano un evento cardiaco importante durante il periodo di follow-up “off-therapy” e ai pazienti randomizzati nel braccio di controllo dopo che abbiano raggiunto l'end-point primario.
    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 Decision2017-11-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-05
    P. End of Trial
    P.End of Trial StatusOngoing
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