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    Summary
    EudraCT Number:2018-002915-93
    Sponsor's Protocol Code Number:ACE-536-LTFU-001
    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:2021-06-15
    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 number2018-002915-93
    A.3Full title of the trial
    A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other Luspatercept (ACE-536) clinical trials.
    Studio di rollover di Fase 3B, in aperto, a braccio singolo per valutare la sicurezza a lungo termine in soggetti che hanno partecipato ad altre sperimentazioni cliniche con luspatercept (ACE-536).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other Luspatercept (ACE-536) clinical trials.
    Studio di rollover di Fase 3B, in aperto, a braccio singolo per valutare la sicurezza a lungo termine in soggetti che hanno partecipato ad altre sperimentazioni cliniche con luspatercept (ACE-536).
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberACE-536-LTFU-001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04064060
    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 SponsorCELGENE CORPORATION
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinical Trial Disclosure
    B.5.3 Address:
    B.5.3.1Street Address86 Morris Avenue
    B.5.3.2Town/ citySummit, New Jersey
    B.5.3.3Post code07901
    B.5.3.4CountryUnited States
    B.5.4Telephone number0019137096862
    B.5.5Fax number000000
    B.5.6E-mailClinicalTrialDisclosure@celgene.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.2.1.1.1Trade name Reblozyl
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe B.V.
    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;EU/3/14/1300
    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.3.9.4EV Substance CodeSUB189400
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number38
    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/3/14/1331; EU/3/14/1300
    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.3.9.4EV Substance CodeSUB179621
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number38
    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
    Prior participation on a clinical trial of luspatercept (ACE-536) in protocols eligible for participation in this study ACE-536-LTFU-001 with the following medical conditions:
    - myelodysplastic syndrome (MDS)
    - beta (ß)-thalassemia (THAL)
    - myelofibrosis (MF)
    Precedente partecipazione a sperimentazioni cliniche con (ACE-536) in protocolli elegibili per la partecipazione a questo studio ACE-536-LTFU-001 con le seguenti condizioni cliniche:
    - sindrome mielodisplastica (MDS)
    - beta (ß)-talassemia (THAL)
    - mielofibrosi (MF)
    E.1.1.1Medical condition in easily understood language
    myelodysplastic syndrome (MDS); beta (ß)-thalassemia (THAL); myelofibrosis (MF)
    Sindrome mielodisplastica (MDS), beta (ß)-talassemia (THAL); mielofibrosi (MF)
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10074356
    E.1.2Term Non-transfusion dependent thalassemia
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10068361
    E.1.2Term MDS
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10054658
    E.1.2Term Thalassemia
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074689
    E.1.2Term Post polycythemia vera myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074690
    E.1.2Term Post essential thrombocythemia myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10074691
    E.1.2Term Post polycythaemia vera myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074692
    E.1.2Term Post essential thrombocythaemia myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10028537
    E.1.2Term Myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077161
    E.1.2Term Primary myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10002272
    E.1.2Term Anemia
    E.1.2System Organ Class 100000004851
    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 long-term safety (including progression to acute myeloid leukemia (AML) and/or other malignancies/pre-malignancies) of luspatercept in subjects who have participated in other luspatercept clinical trials
    Valutare la sicurezza a lungo termine (inclusa la progressione a leucemia mieloide acuta [AML] e/o altri tumori maligni/premaligni) di luspatercept in soggetti che hanno partecipato ad altre sperimentazioni cliniche con luspatercept
    E.2.2Secondary objectives of the trial
    To follow subjects for overall survival
    Seguire la sopravvivenza globale dei soggetti
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is = 18 years at the time of signing the informed consent form (ICF).
    2. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
    3. Subject has been participating in a luspatercept trial and continues to fulfill all the requirements of the parent protocol and the subject has been either:
    a. Assigned to luspatercept treatment, continues to receive clinical benefit in the opinion of the investigator and should continue to receive luspatercept treatment, OR
    b. Assigned to placebo arm in the parent protocol (at the time of unblinding or in follow-up) and should cross over to luspatercept treatment, OR
    c. Assigned to the Follow-up Phase of the parent protocol, previously treated with luspatercept or placebo in the parent protocol who shall continue into Long-term Post-treatment Follow-up Phase in the rollover study until the follow-up commitments are met (unless requirements are met as per parent protocol to crossover to luspatercept treatment).
    4. Subject understands and voluntarily signs an informed consent document prior to any study-related assessments or procedures being conducted.
    5. Subject demonstrates compliance, as assessed by the investigator, with the parent study protocol requirements.
    6. Applies to on treatment subjects only- females of childbearing potential (FCBP) defined as a sexually mature woman who:
    1) has achieved menarche at some point,
    2) has not undergone a hysterectomy or bilateral oophorectomy, or
    3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) and must:
    a. Have two negative pregnancy tests as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy.
    This applies even if the subject practices true abstinence* from heterosexual contact.
    b. Either commit to true abstinence* from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy.
    7. Applies to on treatment subjects only- Male subjects must:
    a. Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy.
    * True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [eg, calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception).
    1. Il soggetto ha = 18 anni al momento della sottoscrizione del modulo di consenso informato (ICF).
    2. Il soggetto intende ed è in grado di attenersi al programma di visite dello studio e agli altri requisiti del protocollo.
    3. Il soggetto ha partecipato a una sperimentazione con luspatercept e continua a soddisfare tutti i requisiti del protocollo primario e il soggetto:
    a. è stato assegnato al trattamento con luspatercept, continua — secondo il parere dello sperimentatore — a manifestare benefici clinici e deve continuare a ricevere il trattamento con luspatercept, OPPURE
    b. è stato assegnato al braccio con placebo nel protocollo primario (al momento dell'apertura del cieco o del follow-up) e deve passare al trattamento con luspatercept, OPPURE
    c. è stato assegnato alla fase di follow-up del protocollo primario, è stato precedentemente trattato con luspatercept o placebo nell’ambito del protocollo primario e deve proseguire la fase di follow-up post-trattamento a lungo termine nello studio di rollover fino a quando gli impegni di follow-up siano soddisfatti (a meno che siano soddisfatti i requisiti del protocollo primario per il passaggio al trattamento con luspatercept).
    4. Il soggetto comprende e sottoscrive volontariamente un documento di consenso informato prima che venga effettuata qualsiasi valutazione o procedura legata allo studio.
    5. Il soggetto dimostra di aderire, in base al giudizio dello sperimentatore, ai requisiti del protocollo dello studio primario.
    6. Si applica soltanto ai soggetti in trattamento - Soggetti di sesso femminile in età fertile (FCBP), definiti come donne che abbiano raggiunto la maturità sessuale e che:
    1) abbiano raggiunto il menarca in un dato momento, 2) non sono state sottoposte a isterectomia o a ooforectomia bilaterale oppure 3) non si trovano in stato di post-menopausa naturale (l'amenorrea dovuta a terapia per il cancro non esclude la potenziale fertilità) da almeno 24 mesi consecutivi (ovvero hanno avuto cicli mestruali in un momento qualsiasi dei precedenti 24 mesi consecutivi) e devono:
    a. presentare due test di gravidanza negativi verificati dallo sperimentatore prima di iniziare la terapia in studio. Accettare di sottoporsi a test di gravidanza continuativi nel corso dello studio e dopo la conclusione della terapia in studio. Questo vale anche nel caso in cui il soggetto pratichi l'astinenza totale* dal contatto eterosessuale.
    b. Impegnarsi all'astinenza totale* dal contatto eterosessuale (che deve essere riesaminata con cadenza mensile e con documentazione originale) o accettare di usare ed essere in grado di attenersi a un metodo di contraccezione altamente efficace senza interruzione 35 giorni prima di iniziare il trattamento con il prodotto sperimentale (IP), durante la terapia in studio (incluse le interruzioni di dosaggio) e per 84 giorni dopo l'interruzione della terapia in studio.
    7. Si applica soltanto ai soggetti in trattamento - Soggetti di sesso maschile devono:
    a. praticare l'astinenza totale* (che deve essere riesaminata con cadenza mensile) o accettare di utilizzare il preservativo durante il contatto sessuale con donne in gravidanza o donne in età fertile nel corso del periodo di partecipazione allo studio, durante le interruzioni della dose e per almeno 84 giorni dopo la sospensione del prodotto sperimentale anche nel caso di precedente vasectomia dall’esito positivo.
    * L'astinenza totale è accettabile quando ciò è in linea con lo stile di vita preferito e abituale del soggetto. (L'astinenza periodica [come i metodi del calendario, dell’ovulazione, sintotermico, post-ovulazione] e il coito interrotto non sono considerati metodi contraccettivi accettabili).
    E.4Principal exclusion criteria
    1. Applies to on treatment subjects only- Concomitant use of any medications/procedures that are prohibited in the parent luspatercept protocol.
    2. Subject has met one or more criteria for study discontinuation as stipulated in the parent luspatercept protocol.
    3. First luspatercept transition visit into rollover study > 21 days after end of study (EOS) visit (last dose/visit in case of no EOS visit) of the parent luspatercept study with the exception of those subjects already in
    the Post-treatment Follow up Phase from the parent study. Note-Subject with current dose delays from the parent protocol during the Transition Phase, will continue in the rollover protocol regardless of the delay.
    4. Applies to on treatment subjects only- Pregnant or breastfeeding females.
    5. Subject has any significant medical condition, laboratory abnormality, psychiatric illness, or is considered vulnerable by local regulations (eg, imprisoned or institutionalized) that would prevent the subject from participating in the study.
    6. Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
    7. Subject has any condition that confounds the ability to interpret data from the study.
    1. Si applica soltanto ai soggetti in trattamento - Eventuale uso di medicinali/procedure concomitanti vietati dal protocollo primario con luspatercept.
    2. Soddisfacimento da parte dei soggetti di uno o più criteri per l'interruzione dello studio definiti dal protocollo primario con luspatercept.
    3. Prima visita per la transizione a luspatercept nello studio di rollover dopo > 21 giorni dalla visita di fine studio (EOS) dello studio primario con luspatercept (ultima dose/visita in caso di assenza della visita di fine studio) ad eccezione di coloro già in fase di follow-up post-trattamento dallo studio primario. Nota: i soggetti che durante la Fase di transizione presentano dei ritardi nell’assunzione della dose attuale, passeranno al protocollo di rollover indipendentemente dal ritardo.
    4. Si applica soltanto ai soggetti in trattamento - Donne in gravidanza o in allattamento.
    5. Se il soggetto presenta una qualsiasi condizione medica significativa, anomalie di laboratorio, malattia psichiatrica o condizione di vulnerabilità secondo la normativa locale (per esempio, detenuto o istituzionalizzato) che possa impedire al soggetto di partecipare allo studio.
    6. Il soggetto presenta una qualsiasi condizione, inclusa la presenza di anomalie di laboratorio, che lo/la espone a rischi inaccettabili qualora dovesse partecipare allo studio.
    7. Il soggetto presenta una condizione che interferisce con la capacità di interpretare i dati dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    1) Adverse events (AEs) - Type, frequency, severity of AEs, relationship of treatment emergent adverse events to luspatercept.
    2) Progression to high/very high risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only) - Number and percentage of subjects progressing to high/very high risk MDS or AML.
    3) Development of other malignancies/pre-malignancies - Number and percentage of subjects developing other malignancies/premalignancies.
    1) Eventi avversi (AE) - Tipo, frequenza, gravità degli AE, correlazione tra gli eventi avversi emergenti dal trattamento e luspatercept.
    2) Progressione a sindrome mielodisplastica (MDS) ad alto/altissimo rischio o alla leucemia mieloide acuta (AML) (solo MDS e mielofibrosi [MF]) - Numero e percentuale di soggetti che mostrano progressione a MDS o AML ad alto/altissimo rischio.
    3) Sviluppo di altri tumori maligni/premaligni - Numero e percentuale di soggetti che sviluppano altri tumori maligni/premaligni
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) Adverse events (AEs) - Timeframe: Enrollment to 42 days post last dose
    2) Progression to high/very high risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only) - Timeframe: Enrollment to Longterm Post-treatment Follow-up
    3) Development of other malignancies/pre-malignancies - Timeframe: Enrollment to Long-term Post-treatment Follow-up
    1) Eventi avversi (AE) - Intervallo di tempo: Arruolamento a 42 giorni dopo l’ultima dose
    2) Progressione a sindrome mielodisplastica (MDS) ad alto rischio/a rischio molto alto o a leucemia mieloide acuta (AML) (solo MDS e mielofibrosi [MF]) - Intervallo di tempo: Arruolamento al follow-up post-trattamento a lungo termine
    3) Sviluppo di altri tumori maligni/pre-maligni - Intervallo di tempo: Arruolamento al follow-up post-trattamento a lungo termine
    E.5.2Secondary end point(s)
    Overall survival - Time from date of randomization until death from any cause
    Sopravvivenza globale - Periodo dalla data di randomizzazione fino al decesso per qualsiasi causa
    E.5.2.1Timepoint(s) of evaluation of this end point
    Enrollment to Long-term Post-treatment Follow-up
    Arruolamento al follow-up post-trattamento a lungo termine
    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 No
    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 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) Yes
    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 concerned19
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA151
    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
    Austria
    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
    Rollover study will be terminated, and subjects will discontinue from study when all subjects fulfill 5years from Dose 1 of parent protocol or 3years of post-treatment from last dose of parent protocol, whichever occurs later. End of Trial is defined as either the date of last visit of last subject to complete the post-treat FU, or date of receipt of last data point from last subject needed for primary, secondary and/or exploratory analysis, as per protocol, whichever is the later date
    Lo studio di rollover verrà interrotto e i sog interromperanno la part allo studio quando tutti i sog avranno completato 5 anni dalla Dose1 del prot primario o 3anni di post-trattamento dall'ultima dose del prot primario, a seconda di quale ev si verifichi più tardi. La fine della sper è definita come data dell'ult visita dell'ult sog che completa il fup post-trat o la data di ricezione un'an prim, sec e/o espl, a seconda di quale delle due date sia posteriore
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months5
    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: 223
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 519
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 430
    F.4.2.2In the whole clinical trial 742
    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)
    Subjects who received at least one dose of IP should undergo EOT and 42d Follow-up evaluations after last dose of IP or EOT Visit, whichever occurs later. Subjects will be followed every 6mths thereafter for
    monitoring&overall survival from date of last dose of IP until death, withdrawal of consent for further follow-up, or until study termination, or until subject is lost to follow-up, or until parent luspatercept
    protocol requirements are met. Subjects will receive SoC treatment post study.
    I sog che hanno ric almeno 1dose di IP devono essere sott a EOT e alle val di FUP a 42g dopo l'ultima dose dell’IP o la visita EOT, a sec di quale si verifichi per ul. I sog saranno seguiti ogni 6mesi succ per il monit e la soprav globale dalla data dell'ultima dose di IP fino a decesso, riti del cons per ult FUP, o fino all’int dello st, o fino a quando il sog è perso al FUP, o fino a quando i requisiti del prot prim con luspatercept vengono sod. Il sog riceverà trattamento SoC dopo lo st
    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 Decision2019-05-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-20
    P. End of Trial
    P.End of Trial StatusOngoing
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