Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2020-005736-30
    Sponsor's Protocol Code Number:CDAII-LUSPA_FA02-2020
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2023-02-06
    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 number2020-005736-30
    A.3Full title of the trial
    A phase II, multicenter, open label study to evaluate the efficacy and safety of Luspatercept (ACE-536) in adult patients with Congenital Dyserythropoietic Anemia type Il (CDA ll).
    Studio di Fase 2, aperto, multicentrico per determinare l'efficacia e la sicurezza di Luspatercept (ACE-536) in soggetti adulti affetti da Anemia Diseritropoietica Congenita tipo II (CDA II).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The study is a Phase 2, open-label, multicenter study to determine the efficacy and safety of Luspatercept (ACE-536) in adults with congenital dyserythropoietic anemia type II (CDA II).
    Lo studio è uno studio di Fase 2, aperto, multicentrico per determinare l'efficacia e la sicurezza di Luspatercept (ACE-536) in soggetti adulti affetti da Anemia Diseritropoietica Congenita tipo II (CDA II).
    A.3.2Name or abbreviated title of the trial where available
    CDAII-LUSPATERCEPT
    CDAII-LUSPATERCEPT
    A.4.1Sponsor's protocol code numberCDAII-LUSPA_FA02-2020
    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 per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia
    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 supportFOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportBristol Myers Squibb
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia
    B.5.2Functional name of contact pointTrial Center
    B.5.3 Address:
    B.5.3.1Street AddressVia G. Garibaldi, 7/3 C
    B.5.3.2Town/ cityGenova
    B.5.3.3Post code16124
    B.5.3.4CountryItaly
    B.5.4Telephone number+393332408777
    B.5.6E-mailantonia.gigante@foranemia.org
    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 Reblozyl
    D.2.1.1.2Name of the Marketing Authorisation holderEU/1/20/1452/001
    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/3/14/1331
    D.3 Description of the IMP
    D.3.1Product nameLuspatercept
    D.3.2Product code [ACE-536]
    D.3.4Pharmaceutical form Lyophilisate for 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 INNLuspatercept
    D.3.9.1CAS number 1373715-00-4
    D.3.9.2Current sponsor codeACE-536
    D.3.9.3Other descriptive nameLuspatercept
    D.3.9.4EV Substance CodeSUB189400
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number37.5
    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.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
    Congenital Dyserythropoietic Anemia type II (CDAII)
    Anemia Diseritropoietica Congenita tipo II (CDA II)
    E.1.1.1Medical condition in easily understood language
    Blood disorder that requires infrequent blood transfusions to treat disease-related anemia.
    Disturbo del sangue che richiede trasfusioni di sangue non frequenti per il trattamento dell’anemia correlata alla malattia.
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10081457
    E.1.2Term Congenital dyserythropoietic anaemia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10081457
    E.1.2Term Congenital dyserythropoietic anaemia
    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
    - in transfusion dependent patients: the proportion of patients achieving any reduction of transfusion burden for at least 12 weeks during the treatment period;
    - in non-transfusion dependent patients: the proportion of subjects who achieve a mean hemoglobin increase by 1.0 g/dl sustained for 12 weeks within the first 24 weeks (in the absence of RBC transfusions).
    - nei pazienti trasfusione dipendenti, la percentuale di pazienti che ha ottenuto una riduzione del carico trasfusionale per almeno 12 settimane durante il periodo di trattamento;
    - nei pazienti non trasfusione dipendenti, la percentuale di pazienti che raggiunge un aumento medio dell'emoglobina di 1.0g/dL per 12 settimane entro le prime 24 settimane, in assenza di trasfusione di globuli rossi
    E.2.2Secondary objectives of the trial
    Proportion of transfusion dependent subjects who achieve>=50%transfusion burden reduction over12and24weeks during treatment period as well duration of response
    Proportion of transfusion dependent subjects who achieve transfusion independency within 12 weeks and the duration of transfusion independence response up to week 48
    Mean change in Hb level in NTD patients from baseline to week24
    Main changes in biomarkers of erythropoiesis, markers of hemolysis, iron metabolism including Hepcidin and bone metabolism from baseline to Week 24
    Mean change from baseline in LIC from baseline to Week48
    Mean change from baseline in mean daily dose of ICT used
    Relative change (%) from baseline in serum ferritin level to Week 24 [serum levels were drawn at the baseline and at week 24. Levels were analyzed for serum ferritin measured in mg/L]
    Duration of reduction in transfusion burden or transfusion independence
    Duration of response in non-TD patients
    Ancillary studies on red cell before/after treatment
    % di soggetti che raggiungono la riduzione>=50% del carico trasfusionale in 12 e 24 sett durante il trattamento nonché la durata della risposta (nei TD)
    % di soggetti che raggiungono trasfusione indipendenza entro 12 sett e la durata di indipendenza trasfusionale
    Risposta fino a sett 48
    Variazione media del livello di emoglobina glicata nei soggetti NTD da basale a sett 24
    Principali cambiamenti nei biomarcatori dell'eritropoiesi, marcatori emolisi, metab ferro compreso sTfr1, epcidina e metab osseo da basale a sett 24
    Variazione media della LIC da basale a sett 24
    Variazione media rispetto al basale della dose media giornaliera di TIC utilizzata
    Variazione relativa(%) rispetto al basale del livello di ferritina sierica alla sett 24 [livelli sierici misurati al basale e alla settimana 24. Livelli della ferritina sierica misurati in mg/L]
    Durata della riduzione delle trasfusioni o indipendenza trasfusionale
    Durata di risposta nei pz NTD
    Studi ausiliari su eritrociti pre/post trattamento
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Life quality
    Version: 1.3
    Date: 04/08/2022
    Title: QoL
    Objectives: use of questionnaire sf36 to increase knowledge of the Qol respect to the disease

    Qualita' della vita
    Versione: 1.3
    Data: 04/08/2022
    Titolo: QoL
    Obiettivi: utilizzo del questionario sf36 per ottenere in formazioni sulla qualità di vita rispetto alla malattia
    E.3Principal inclusion criteria
    - Males or female patients >= 18 years of age at the time of signing the informed consent document (ICF).
    - Subject must understand and voluntarily sign an informed consent form (ICF) prior to any study-related assessments/procedures being conducted.
    - Biallelic causative mutations in SEC23B gene alone or associated with other gene variants (e.g., globin gene defects).
    - Monoallelic causative mutations in SEC23B gene in presence of hypoglycosylation of band 3 and/or associated with other gene variants (e.g., globin gene defects).
    - Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 or 1
    - Non-transfusion dependent (NTD) patients, defined as having received < 1/0 unit of RBCs within 8 weeks prior to Cycle 1 Day 1.
    - Or transfusion dependent patients, defined as requiring ³ 3 units of RBCs every 8 weeks (confirmed over 6 months prior to Cycle 1 Day 1).
    - Anemia, defined as: (i) mean Hb concentration < 10.9 g/dl of 2 measurements (one performed within one day prior to Cycle 1 Day 1 and the other performed during the screening period) in non-transfusion dependent patients, or (ii) transfusion dependent.
    - Splenectomized or non-splenectomized patients are both eligible.
    - avere >= 18 anni di età al momento della sottoscrizione del documento di consenso informato;
    - sottoscrivere il documento di consenso informato;
    - avere mutazioni causali bialleliche nel gene SEC23B;
    - mutazioni causative monoalleliche nel gene SEC23B in presenza di ipoglicosilazione della banda 3 e/o associate ad altre varianti genetiche (ad es. difetti del gene globinico);
    - performance status: punteggio ECOG (Eastern Cooperative Oncology Group) pari a 0 o 1;
    - essere pazienti non trasfusione dipendenti (NTD), definiti come soggetti che hanno ricevuto <1/0 unità di globuli rossi entro 8 settimane prima del ciclo 1 giorno 1;
    - essere pazienti trasfusione dipendenti, definiti come soggetti che richiedono> =3 unità di globuli rossi ogni 8 settimane (confermato oltre 6 mesi prima del ciclo 1 giorno 1);
    -L’Anemia è definita come:(i) concentrazione media di Hb <10.0 g/dl di 2 misurazioni (uno eseguita entro un giorno prima del ciclo 1 giorno 1 e l'altro eseguito durante il periodo discreening) in pazienti dipendenti non trasfusione,o (ii) trasfusione dipendente.
    - Splenectomizzati o non-splenectomizzati pazienti sono entrambi ammissibili.
    E.4Principal exclusion criteria
    1. Any clinically significant pulmonary (including pulmonary hypertension), cardiovascular, endocrine, neurologic, hepatic (including cirrhosis and liver failure), gastrointestinal, infectious, immunological (including clinically significant allo- or auto-immunization) or genitourinary disease considered by the investigator as not adequately controlled prior to Cycle 1 Day 1.
    2. Patients with history of cancer, or suffering from evolutive cancer, or in remission with treatment stopped less than 2 years before.
    3. Anemia from other causes.
    4. Symptomatic splenomegaly.
    5. Thromboembolic events = grade 3 according to the National Cancer lnstitute-Common Terminology Criteria for Adverse Events (NCl-CTCAE) v.4 .0 (current active minor version) within 12 months prior to Cycle 1 Day 1.
    6. Ejection fraction < 50% by echocardiogram, MUGA or cardiac MRI.
    7. Uncontrolled hypertension defined as systolic blood pressure (BP) = 150 mm Hg or diastolic BP =95 mm Hg.
    8. QTc > 450 msec on screening ECG.
    9. Proteinuria = Grade 2.
    10. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.
    11. Treatment with another investigational drug or device, or approved therapy for investigational use 28 days prior to Cycle 1 Day 1, or if the half-life of the previous investigational product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer.
    12. Transfusion event within 7 days prior to Cycle 1 Day 1.
    13. Splenectomy within 56 days prior to Cycle 1 Day 1.
    14. Major surgery (except splenectomy) within 28 days prior to Cycle 1 Day 1. Patients must have completely recovered from any previous surgery prior to Cycle 1 Day 1.
    15. lron chelation therapy initiated within 56 days prior to Cycle 1 Day 1.
    16. Cytotoxic agents, systemic corticosteroids, immunosuppressants, or anticoagulant therapy such as warfarin or heparin within 28 days prior to Cycle 1 Day 1 (prophylactic aspirin up to 100 mg/d is permitted).
    17. Evidence of active hepatitis C (HCV) infection, or active infectious hepatitis B, or known positive human immunodeficiency virus (HIV)
    1. qualsiasi malattia polmonare clinicamente significativa (inclusa l'ipertensione polmonare), cardiovascolare, endocrina, neurologica, epatica (inclusa cirrosi e insufficienza epatica), gastrointestinale, infettiva, immunologica (inclusa allo o
    autoimmunizzazione clinicamente significativa) o genitourinaria considerata dallo sperimentatore come non adeguatamente controllato prima del ciclo 1 giorno 1;
    2. storia di cancro, o di cancro evolutiva, o in remissione da meno di 2 anni;
    3. anemia da altre cause;
    4. splenomegalia sintomatica;
    5. eventi tromboembolici di grado >= 3 secondo il National Cancer dell'lstituto -Comune Term i Nology Criteri di eventi avversi (NSC -CTCAE) v.4 . 0 (versione secondaria attiva corrente) nei 12 mesi prima del ciclo 1 giorno 1;
    6. frazione di eiezione <50% tramite ecocardiogramma, MUGA o risonanza magnetica cardiaca;
    7. ipertensione non controllata definita come pressione arteriosa sistolica (PA) >= 150 mm Hg o distolica (PD) >= 95 mm Hg;
    8. QTc> 450 msec all'ECG di screening;
    9. proteinuria >= Grado 2;
    10. qualsiasi infezione attiva che richiede terapia antibiotica parenterale entro 28 giorni
    precedenti al ciclo 1 giorno 1 o antibiotici per via orale entro 14 giorni dal ciclo 1 giorno 1;
    11. trattamento con un altro farmaco o dispositivo sperimentale o terapia approvata per uso
    sperimentale 28 giorni prima del Ciclo 1 Giorno 1, o se l'emivita del precedente prodotto
    sperimentale è nota, entro 5 volte l'emivita precedente al Ciclo 1 Giorno 1, qualunque sia il più lungo;
    12. evento trasfusione nei 7 giorni prima del ciclo 1 giorno 1;
    13. splenectomia entro 56 giorni prima del ciclo 1 giorno 1
    14. chirurgia maggiore (eccetto splenectomia) 28 giorni prima del Ciclo 1 Giorno 1. I soggetti
    devono aver completamente recuperato da qualsiasi precedente intervento chirurgico
    prima del ciclo 1 giorno 1,
    15. terapia ferro chelante iniziata meno di 56 giorni prima del ciclo 1 giorno 1;
    16. agenti citotossici, corticosteroidisistemici, immunosoppressori, o terapia anticoagulante
    quali warfarin o eparina entro 28 giorni prima del ciclo 1 giorno 1 (aspirina profilattica fino a 100 m/d è consentita);
    17. Evidenza di infezione attiva da epatite C (HCV) o epatite B infettiva attiva o virus dell'immunodeficienza umana (HIV)
    E.5 End points
    E.5.1Primary end point(s)
    - in transfusion dependent patients: the proportion of patients achieving any reduction of transfusion burden for at least 12 weeks during the treatment period;
    - in non-transfusion dependent patients: the proportion of subjects who achieve a mean hemoglobin increase by 1.0 g/dl sustained for 12 weeks within the first 24 weeks (in the absence of RBC transfusions).
    - nei pazienti trasfusione dipendenti, la percentuale di pazienti che ha ottenuto una riduzione del carico trasfusionale per almeno 12 settimane durante il periodo di trattamento;
    - nei pazienti non trasfusione dipendenti, la percentuale di pazienti che raggiunge un aumento medio dell'emoglobina di 1.0g/dL per 12 settimane entro le prime 24 settimane, in assenza di trasfusione di globuli rossi
    E.5.1.1Timepoint(s) of evaluation of this end point
    - in transfusion dependent patients at least 12 weeks during the treatment period
    - in non-transfusion dependent patients for 12 weeks within the first 24 weeks
    - nei pazienti trasfusione dipendenti almeno 12 settimane durante il periodo di trattamento
    - nei pazienti non trasfusione dipendenti per 12 settimane entro le prime 24 settimane
    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 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 concerned8
    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
    End of trial defined as the date all subjects completed at least 48 weeks in this study starting on the first dose of IP or stopping early, or the date the last subject data required for a primary assessment was received , secondary and/or exploratory analysis, as previously specified in the protocol, if more recent.
    La Fine della sperimentazione definita come la data in cui tutti i soggetti hanno completato almeno 48 settimane in questo studio a partire dalla prima dose di IP o hanno interrotto anticipatamente, o la data di ricezione dell'ultimo dato dall'ultimo soggetto, dato necessario per un'analisi primaria, secondaria e/o esplorativa, come preliminarmente specificato nel protocollo, a seconda di quale delle due date sia la più recente.
    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 months4
    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 months4
    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: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 40
    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)
    The following assessments will be performed,:
    - Iron chelating therapies - record on an ongoing basis up to End of Study
    - Post-treatment medications/therapies - such as surgery, radiation, systemic or any other therapy for the subject's disease - record on an ongoing basis up to End of Study.
    - Post-treatment procedures - record on an ongoing basis up to End of Study.
    - Transfusion assessment.

    Adverse event assessment.
    Verranno effettuate le seguenti valutazioni:
    - Terapie chelanti del ferro - da valutare in base dell'andamento fino alla fine dello studio
    - Farmaci / terapie post-trattamento - come chirurgia, radioterapia, sistemica o qualsiasi altra terapia per la malattia del soggetto - da valutare in base dell'andamento fino alla fine dello studio.
    - Procedure post-trattamento - da valutare in base dell'andamento fino alla fine dello studio.
    - Valutazione trasfusionale.
    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 Decision2023-05-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-07-17
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Wed May 28 20:32:32 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA