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    Summary
    EudraCT Number:2019-000954-67
    Sponsor's Protocol Code Number:ATB200-07
    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:2021-06-17
    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 number2019-000954-67
    A.3Full title of the trial
    A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease
    Studio di estensione in aperto di fase 3 per valutare la sicurezza e l’efficacia a lungo termine di ATB200 somministrato per via endovenosa con AT2221 orale in soggetti adulti affetti dalla malattia di Pompe a insorgenza tardiva
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease
    Uno studio che valuta la sicurezza e l’efficacia a lungo termine per via endovenosa di ATB200 (EV) quando viene co-sommisnistrato con AT2221 orale in soggetti adulti con la malattia di Pompe
    A.3.2Name or abbreviated title of the trial where available
    AMICUS ATB200-07
    AMICUS ATB200-07
    A.4.1Sponsor's protocol code numberATB200-07
    A.5.4Other Identifiers
    Name:US INDNumber:127,387
    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 SponsorAMICUS THERAPEUTICS, 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 supportAmicus Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmicus Therapeutics, Inc.
    B.5.2Functional name of contact pointPatient advocacy
    B.5.3 Address:
    B.5.3.1Street Address1 Cedar Brook Drive
    B.5.3.2Town/ cityCranburry
    B.5.3.3Post codeNJ 08512
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016096622000
    B.5.5Fax number0016096625010
    B.5.6E-mailclinicaltrials@amicusrx.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/3/18/2000
    D.3 Description of the IMP
    D.3.1Product nameATB200
    D.3.2Product code [ATB200]
    D.3.4Pharmaceutical form Powder 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 INNcipaglucosidasi alfa
    D.3.9.1CAS number 420784-05-0
    D.3.9.2Current sponsor codeATB200
    D.3.9.4EV Substance CodeSUB21275
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number105
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 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/3/18/2129
    D.3 Description of the IMP
    D.3.1Product nameAT2221
    D.3.2Product code [AT2221]
    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 INNMIGLUSTAT
    D.3.9.1CAS number 72599-27-0
    D.3.9.2Current sponsor codeAT2221
    D.3.9.4EV Substance CodeSUB20049
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number65
    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
    Adult Subjects With Late Onset Pompe Disease (LOPD)
    Soggetti adulti con malattia di Pompe a insorgenza tardiva
    E.1.1.1Medical condition in easily understood language
    A change in the genetic material resulting in lower levels of an enzyme called acid alpha-glucosidase (GAA). The GAA enzyme helps the body to break down glycogen, a type of stored sugar.
    Un cambiamento nel materiale genetico che porta a livelli più bassi di un enzima chiamato acido alfa-glucosidasi. L'enzima aiuta l'organismo a scomporre il glicogeno,un tipo di zucchero immagazzinato.
    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 24.0
    E.1.2Level LLT
    E.1.2Classification code 10075702
    E.1.2Term Pompe's disease late onset
    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
    The primary objective of this study is to assess the long-term safety and tolerability of ATB200/AT2221 co-administration
    L’obiettivo primario di questo studio è valutare la sicurezza e la tollerabilità a lungo termine della co-somministrazione di ATB200/AT2221
    E.2.2Secondary objectives of the trial
    - to assess the long-term efficacy of ATB200/AT2221 co-administration on:
    • ambulatory function, as measured by the 6-minute walk test (6MWT) ; • pulmonary function, as measured by sitting forced vital capacity (FVC) (% predicted)
    • on muscle strength; • on health-related patient-reported outcomes; • motor function
    • overall clinical impression, as assessed by both physician and subject
    • measures of pulmonary function other than FVC (% predicted)
    • biomarkers of muscle injury and disease substrate
    - to assess the immunogenicity of ATB200/AT2221 co-administration
    - Valutare l’efficacia a lungo termine della co-somministrazione su:
    • funzione deambulatoria, misurata mediante il test del cammino di 6 minuti (6MWT); • funzione polmonare, misurata mediante la capacità vitale forzata (CVF) (% prevista) in posizione seduta;
    • forza muscolare; • esiti riferiti dal paziente correlati alla salute; • funzione motoria
    • impressione clinica globale valutata sia dal medico che dal soggetto
    • misure della funzione polmonare diverse dalla CVF (% prevista)
    • biomarcatori di lesione muscolare e substrato di malattia
    - Valutare l’immunogenicità della co-somministrazione di ATB200/AT2221
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject must provide signed informed consent prior to any studyrelated procedures being performed.
    2. Subject must have completed Study ATB200-03.
    Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of ATB200/AT2221 (eg, hospitalization for a car accident or emergency surgery) and which resulted in several consecutive missed doses may be eligible to participate in this study upon approval by the Amicus medical monitor.
    3. Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug.
    1. Il soggetto deve fornire il consenso informato firmato prima che venga eseguita qualsiasi procedura correlata allo studio.
    2. Il soggetto deve aver completato lo studio ATB200-03.
    Nota: i soggetti che sono stati costretti a ritirarsi dallo studio ATB200-03 per motivi logistici non correlati all’efficacia o alla sicurezza di ATB200/AT2221 (ad es. ricovero ospedaliero per un incidente automobilistico o un intervento chirurgico d’urgenza) e che hanno determinato la mancata assunzione di diverse dosi consecutive possono essere idonei a partecipare a questo studio previa approvazione del responsabile del monitoraggio medico di Amicus.
    3. I soggetti di sesso femminile in età fertile e i soggetti di sesso maschile devono acconsentire all’uso di metodi contraccettivi accettabili dal punto di vista medico durante lo studio e nei 90 giorni successivi all’ultima dose del farmaco in studio
    E.4Principal exclusion criteria
    1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
    2. Subject has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms.
    3. Subject, if female, is pregnant or breastfeeding.
    4. Subject, whether male or female, is planning to conceive a child during the study.
    1. Il soggetto intende ricevere la terapia genica o partecipare a un altro studio interventistico per la malattia di Pompe.
    2. Il soggetto presenta una condizione medica oppure un’altra situazione estenuante che, a giudizio dello sperimentatore o del responsabile del monitoraggio medico, potrebbe costituire un rischio ingiustificato per il soggetto stesso oppure compromettere la sua capacità di ottemperare ai requisiti del protocollo o avere un impatto negativo sugli stessi. Ciò include la depressione clinica (diagnosticata da uno psichiatra o altro professionista della salute mentale) con sintomi non controllati o scarsamente controllati.
    3. Il soggetto, se di sesso femminile, è in stato di gravidanza o allatta al seno.
    4. Il soggetto, a prescindere dal sesso, ha in programma di concepire un bambino durante lo studio
    E.5 End points
    E.5.1Primary end point(s)
    Long-term safety: incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug, frequency and severity of immediate and late IARs, and any abnormalities noted in other safety assessments (eg,
    clinical laboratory tests, ECGs, vital signs). Immunogenicity to ATB200 will also be described.
    Il profilo di sicurezza a lungo termine di ATB200/AT2221 sarà caratterizzato usando l’incidenza di eventi avversi emergenti dal trattamento (TEAE), eventi avversi seri (SAE) ed EA che portano all’interruzione del farmaco in studio, frequenza e gravità di IAR immediate e tardive ed eventuali anomalie riscontrate in altre valutazioni della sicurezza (ad es. esami clinici di laboratorio, ECG, segni vitali). Sarà descritta anche l’immunogenicità ad ATB200.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Every study visit until regulatory approval or marketing authorization and/or commercialization in the participating subject's country, or study termination by the sponsor
    Ogni visita di studio fino all’approvazione regolatoria o all’autorizzazione all’immissione in commercio e/o alla commercializzazione nel Paese del soggetto partecipante o all’interruzione dello studio da parte dello sponsor
    E.5.2Secondary end point(s)
    Efficacy:
    • change from baseline in: • 6-minute walk distance (6MWD); • sitting FVC (% predicted); • in the manual muscle test score for the lower extremities; • in the total score for the PROMIS – physical function; • in the total score for the PROMIS – fatigue; • in the following variables related to motor function:
    - GSGC total score; - time to complete the 10-meter walk (ie, assessment of gait) of the GSGC test; - time to complete the 4-stair climb of the GSGC test; - time to complete the Gower's maneuver of the GSGC test; - time to arise from a chair as part of the GSGC test; - change from baseline in the time to complete the TUG test
    • change from baseline in the following variables related to muscle strength:
    - manual muscle test score for the upper extremities; - manual muscle test total score (upper and lower extremities combined); - quantitative muscle test value (kg) for the upper extremities; - quantitative muscle test value (kg) for the lower extremities; - quantitative muscle test total value (kg) (upper and lower extremities combined)
    • change from baseline in the following variables from patient-reported outcome measures: - total score for the PROMIS – dyspnea; - total score for the PROMIS – upper extremity; - R-PAct Scale total score; - EQ-5D-5L health status
    • actual value of the subject's functional status (improving, stable, or declining) pertaining to the effects of study drug in the following areas of life, as measured by the SGIC: - overall physical well-being; - effort of breathing; - muscle strength; - muscle function; - ability to move around; - activities of daily living; - energy level; - level of muscular pain
    • actual value of the subject's functional status (improving, stable, or declining), as measured by the PGIC
    • change from baseline in the following measures of pulmonary function, as follows: - sitting SVC (% predicted); - MIP (cmH2O); - MIP (% predicted); - MEP (cmH2O); - MEP (% predicted); - SNIP (cmH2O)

    Pharmacodynamic endpoints are as follows: • change from baseline in serum CK level; • change from baseline in urinary Hex4 level
    Gli endpoint di efficacia sono i seguenti:
    • variazione rispetto al basale: • nella distanza percorsa a piedi in 6 minuti (6MWD); • nella CVF da seduto (% prevista); • nel punteggio del test muscolare manuale per le estremità inferiori; • nel punteggio totale per il PROMIS - funzione fisica; • nel punteggio totale per il PROMIS – affaticamento; • nelle seguenti variabili correlate alla funzione motoria:
    - punteggio GSGC totale, - tempo necessario per percorrere a piedi 10 metri (ovvero, valutazione della deambulazione) nel test GSGC, - tempo necessario per salire 4 gradini durante il test GSGC, -tempo necessario per eseguire la manovra di Gower del test GSGC, - tempo necessario per alzarsi da una sedia nell’ambito del test GSGC, -variazione rispetto al basale nel tempo necessario per completare il test TUG.
    • variazione rispetto al basale nelle seguenti variabili correlate alla forza muscolare:
    -punteggio del test muscolare manuale per le estremità superiori; -punteggio totale del test muscolare manuale (estremità superiori e inferiori combinate); - valore del test muscolare quantitativo (kg) per le estremità superiori; -valore del test muscolare quantitativo (kg) per le estremità inferiori; -valore totale (in kg) del test muscolare quantitativo (estremità superiori e inferiori combinate)
    • variazione rispetto al basale nelle seguenti variabili delle misure degli esiti riferiti dal paziente:
    -punteggio totale per il PROMIS – dispnea; -punteggio totale per il PROMIS - estremità superiori;-punteggio totale della Scala R-PAct;-stato di salute valutato mediante il questionario EQ-5D-5L
    • valore effettivo dello stato funzionale del soggetto (migliorato, stabile o peggiorato) in relazione agli effetti del farmaco in studio nei seguenti settori della vita, misurato mediante la SGIC: - benessere fisico generale, -sforzo respiratorio, -forza muscolare, -funzione muscolare, -capacità di muoversi, -attività della vita quotidiana-livello energetico, -livello di dolore muscolare
    • valore effettivo dello stato funzionale del soggetto (migliorato, stabile o peggiorato), misurato mediante la PGIC
    • variazione rispetto al basale nelle seguenti misure della funzione polmonare, come segue: CVL da seduto (% prevista),PIM (cmH2O), PIM (% prevista).PEM (cmH2O),PEM (% prevista), SNIP (cmH2O)

    Gli endpoint farmacodinamici sono i seguenti: - variazione rispetto al basale nel livello sierico di CK; -variazione rispetto al basale nel livello urinario di Hex4
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Efficacy: Week 12, 26; after Week 26, every 26 weeks until regulatory approval or marketing authorization and/or commercialization in the participating subject's country, or study termination by the sponsor.
    - Pharmacodynamic: Week 2, 4, 6, 12, 26; after Week 26, every 26 weeks until regulatory approval or marketing authorization and/or commercialization in the participating subject's country, or study termination by the sponsor.
    - Efficacia: Settimana 12, 26; dopo settimana 26, ogni 26 settimane fino all’approvazione regolatoria o all’autorizzazione all’immissione in commercio e/o alla commercializzazione nel Paese del soggetto partecipante o all’interruzione dello studio da parte dello sponsor.
    - Farmacodinamica: Settimana 2, 4, 6, 12, 26; dopo settimana 26, ogni 26 settimane fino all’approvazione regolatoria o all’autorizzazione all’immissione in commercio e/o alla commercializzazione nel Paese del soggetto partecipante o all’interruzione dello studio da parte dello sponsor.
    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 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 Yes
    E.6.13.1Other scope of the trial description
    - Effect of ATB200/AT2221 co-administration on biomarkers of muscle injury and disease substrate - Immunogenecity of ATB200/AT2221 co-administration
    Effetto della co-somministrazione di ATB200 / AT2221 sui biomarcatori dei muscoli substrato di lesioni e malattie - Immunogenecità della co-somministrazione di ATB200 / AT2221
    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 No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Bosnia and Herzegovina
    Canada
    Japan
    Korea, Democratic People's Republic of
    Korea, Republic of
    Taiwan
    United States
    Austria
    Belgium
    Bulgaria
    Denmark
    France
    Germany
    Greece
    Hungary
    Italy
    Netherlands
    Poland
    Romania
    Slovakia
    Slovenia
    Sweden
    United Kingdom
    Argentina
    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
    LPLV
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 120
    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)
    Treatment with open-Label ATB200/AT2221 is planned to continue until regulatory approval or marketing authorization and/or commercialization in the participating study subject's respective country, or study termination by the sponsor. After participation in the trial and in case of no marketing authorization and/or commercialization, patients will have the option to be treated with the current standard of care therapy
    Il trattamento con ATB200/AT2221 in aperto continuerà fino all'approvazione regolatoria o all'autorizzazione all'immissione in commercio e/o alla commercializzazione nel rispettivo paese del soggetto partecipante di studio o alla conclusione dello studio da parte dello sponsor. Dopo la partecipazione allo studio e in caso di mancata autorizzazione all'immissione in commercio e/o commercializzazione,i pazienti avranno la possibilità di essere trattati con l'attuale standard di terapia terapeutica
    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 Decision2020-03-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-01
    P. End of Trial
    P.End of Trial StatusOngoing
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