Clinical Trials Register

Summary
EudraCT Number:	2020-001543-94
Sponsor's Protocol Code Number:	AML2020
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:	2020-11-06
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

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2020-001543-94

A.3

Full title of the trial

Tagraxofusp in Patients with CD123+ or with Blastic Plasmacytoid Dendritic Cell Neoplasm Immunophenotype-like Acute Myeloid Leukemia.

Tagraxofusp nei pazienti affetti da Leucemia Mieloide Acuta CD123+ o con immunofenotipo BPDCN.

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Study aimed at evaluating the efficacy of Tagraxofusp, as a possible targeted therapy, in patients with Acute Myeloid Leukemia, for whom previous chemotherapy treatments have proved to be ineffective.

Studio volto a valutare l'efficacia del Tagraxofusp, come possibile terapia mirata, in pazienti affetti da Leucemia Mieloide Acuta, per i quali i trattamenti chemioterapici precedenti si siano mostrati poco efficaci.

A.3.2

Name or abbreviated title of the trial where available

AML2020

A.4.1

Sponsor's protocol code number

AML2020

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	FONDAZIONE GIMEMA (GRUPPO ITALIANO MALATTIE EMATOLOGICHE DELL' ADULTO) FRANCO MANDELLI ONLUS
B.1.3.4	Country	Italy
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Stemline Therapeutics, Inc.
B.4.2	Country	United States
B.4.1	Name of organisation providing support	Fondazione GIMEMA (Gruppo Italiano Malattie EMatologiche dell'Adulto) Franco Mandelli Onlus
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Fondazione GIMEMA
B.5.2	Functional name of contact point	Centro Dati Dati
B.5.3	Address:
B.5.3.1	Street Address	via Casilina,5
B.5.3.2	Town/ city	Roma
B.5.3.3	Post code	00182
B.5.3.4	Country	Italy
B.5.4	Telephone number	0670390526
B.5.5	Fax number	0670390540
B.5.6	E-mail	gimema@gimema.it

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	SL - 401
D.3 Description of the IMP
D.3.1	Product name	Elzonris™ (tagraxofusp-erzs)
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.1	CAS number	2055491-00-2
D.3.9.2	Current sponsor code	NA
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1000
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another 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.1.1

Medical condition(s) being investigated

Acute Myeloid Leukemia CD123 + or with BPDCN immunophenotype

Leucemia Mieloide Acuta CD123+ o con immunofenotipo BPDCN

E.1.1.1

Medical condition in easily understood language

AML disease that originates in the bone marrow (myeloid) and progresses rapidly (acute). Accumulation in the marrow of immature cells cause the inability to produce healthy cells and the pathology.

AML malattia che origina nel midollo osseo (mieloide) e progredisce velocemente (acuta).L'accumulo nel midollo di cellule immature provoca l'incapacità di produrre cellule sane e quindi la patologia.

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10000880
E.1.2	Term	Acute myeloid leukaemia
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10000880
E.1.2	Term	Acute myeloid leukaemia
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

Evaluate the activity of tagraxofusp, in terms PR, CR or CRi, in patients with CD123+ or BlasticPlasmacytoid Dendritic Cell Neoplasm-like phenotype (BPDCN-IF) Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML).

Valutare l’attività di tagraxofusp in termini di PR, CR o CRi nei pazienti affetti da Leucemia Mieloide Acuta recidivata/refrattaria con CD123+ o con immunofenotipo BPDCN.

E.2.2

Secondary objectives of the trial

To estimate in the two cohorts (BPDCN-IF and CD123+ AML):

1) Safety
2) Overall Survival (OS)
3) Event Free Survival (EFS)
4) Disease Free Survival (DFS)
5) Cumulative incidence of relapse (CIR)
6) Response rate and survival outcomes according to baseline clinical and biological characteristics
7) Evaluation of the activity of the treatment as a bridge to transplant

Valutare nelle due coorti (AML CD123+ e BPDCN-IF):
1. Sicurezza
2. Overall Survival (OS)
3. Event Free Survival (EFS)
4. Disease Free Survival (DFS)
5. Cumulative incidence of relapse (CIR)
6. Tasso di risposta e risultati sulla sopravvivenza in base alle caratteristiche cliniche e biologiche al baseline.

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

Pharmacogenetics
Version: 1.0
Date: 31/03/2020
Title: Translational Research
Objectives: 1) To assess the incidence of commonly mutated genes with prognostic relevance (e.g. NPM1, FLT3) in the study population. 2) To assess Minimal Residual Disease after experimental treatment by multicolour flow cytometry and molecular analysis (if molecular marker available). 3) To perform a wide genomic analysis on BPDCN-like AMLs samples to identify recurrent genetic mutations and aberrant pathways in order to better characterize this rare biological entity. The use of BPDCN-like signature coupled with molecular evaluation may allow for a better selection of patients who may benefit from anti CD123 novel targeted therapy.

Farmacogenetica
Versione: 1.0
Data: 31/03/2020
Titolo: Ricerca Traslazionale
Obiettivi: 1) Per valutare l'incidenza di geni comunemente mutati con rilevanza prognostica (ad esempio NPM1, FLT3) nella popolazione in studio. 2) Valutare la Malattia Residua Minima dopo il trattamento sperimentale con citometria a flusso multicolore e analisi molecolare (se disponibile marcatore molecolare). 3) Eseguire un'ampia analisi genomica su campioni di AML simili a BPDCN per identificare la genetica ricorrente mutazioni e percorsi aberranti al fine di caratterizzare meglio questa rara entità biologica.L'uso di una firma simile a BPDCN unita a una valutazione molecolare può consentire una migliore selezione dei pazienti che possono beneficiare della nuova terapia mirata anti-CD123.

E.3

Principal inclusion criteria

1. The patient has evidence of AML in the peripheral blood and/or bone marrow with either BPDCN-IF [CD123/CD4/CD56 (+)] or with AML that is CD123+ but negative for either, or both, CD4 and CD56.
2. The patient is =18 years old.
3. The patient must be refractory to at least one previous line of conventional therapy (either high dose therapy or hypomethylating agents) or relapsed after receiving conventional therapy (a maximum of two previous line of therapy is admitted).
4. The patient has an Eastern Cooperative Oncology Group (ECOG) performance score (PS) of 0 to 2.
5. The patient has adequate baseline organ function, including cardiac, renal, and hepatic function:
a. Left ventricular ejection fraction (LVEF) =institutional lower limit of normal as measured by multigated acquisition (MUGA) scan or 2-dimensional (2-D) echocardiography(ECHO) within 21 days before start of therapy and no clinically significant abnormalities on a 12-lead electrocardiogram (ECG).
b. Serum creatinine =1.5 mg/dL (133 µmol/L).
c. Serum albumin =3.2 g/dL (32 g/L) (albumin infusions are not permitted to enable eligibility).
d. Bilirubin =1.5 mg/dL (26 µmol/L).
e. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =2.5 times the upper limit of normal (ULN).
6. If the patient is a woman of childbearing potential (WOCBP), she must have a negative serum or urine pregnancy test at screeningwithin 1 week before treatment.
7. The patient has signed informed consent before initiation of any study-specific procedures or treatment.
8. The patient is able to adhere to the study visit schedule and other protocol requirements, including follow-up for survival assessment.
9. The patient (male and female) agrees to use acceptable contraceptive methods for the duration of time on the study and continue to use acceptable contraceptive methods for 1 week after the last infusion of tagraxofusp.

1. Diagnosi inequivocabile di LMA simil-BPDCN [CD123/CD4/CD56(+)] o LMA positiva per CD123 ma negativa per uno, o entrambi, CD4 e CD56.
2. Il paziente ha = 18 anni.
3. Il paziente è refrattario ad almeno una precedente linea di trattamento convenzionale (terapia ad alte dosi o con agenti ipometilanti) oppure ha
recidivato dopo aver ricevuto terapia convenzionale (sono ammesse al massimo due precedenti linee di trattamento).
4. Il paziente presenta un ECOG performance score da 0 a 2.
5. Il paziente ha un’adeguata funzionalità cardiaca, renale ed epatica:
a. Frazione di eiezione ventricolare sinistra (LVEF) = limite inferiore di normalità, misurazione effettuata mediante MUGA scan o ecocardiografia
bidimensionale entro 21 giorni prima dell'inizio della terapia e nessuna anomalia clinicamente significativa su ECG a 12 derivazioni.
b. Creatinina sierica = 1,5 mg/dL (133 µmol/L).
c. Albumina sierica = 3,2 g/dL (32 g/L) (non sono autorizzate infusioni di albumina con lo scopo di consentire l’eleggibilità).
d. Bilirubina = 1,5 mg/dL (26 µmol/L).
e. Aspartato aminotransferasi (AST) e alanina aminotransferasi (ALT) = 2,5 x ULN.
6. Se la paziente è una donna potenzialmente fertile, lei deve avere un test di gravidanza (siero o urina) negativo allo screening entro una settimana
dall’inizio del trattamento.
7. Il paziente ha firmato il consenso informato prima di iniziare qualsiasi procedura o trattamento specifico dello studio.
8. Il paziente è in grado di aderire al programma delle visite di studio e ad altri requisiti del protocollo, incluso il follow-up per la valutazione della
sopravvivenza.
9. Il paziente (uomo o donna) accetta l’utilizzo di metodi contraccettivi adeguati per l’intera durata dello studio e per almeno una settimana
dall'ultima infusione di tagraxofusp.

E.4

Principal exclusion criteria

1. The patient has a diagnosis of acute promyelocytic leukemia (APL; FAB subtype M3).
2. The patient has persistent clinically significant toxicities of Grade=2 from previous chemotherapy (excluding alopecia, nausea, fatigue, and liver function tests [as mandated in the inclusion criteria]).
3. The patient has received treatment with chemotherapy, wide-field radiation, or biologic therapy within 14 days of study entry.
4. The patient has received treatment with an investigational agent within 14 days of study entry.
5. The patient has previously received treatment with tagraxofusp.
6. The patient has an active malignancy and/or cancer history (excluding antecedent MDS) that may confound the assessment of the study endpoints. Patients with a past cancer history (within 2 years of entry) with substantial potential for recurrence and/or ongoing active malignancy will be evaluated on a case by case basis. Patients with the following neoplastic diagnoses are eligible: non-melanoma skin cancer, carcinoma in situ, cervical intraepithelial neoplasia, organconfined prostate
cancer with no evidence of progressive disease.
7. The patient has clinically significant cardiovascular disease (eg, uncontrolled or any New York Heart Association Class 3 or 4 congestive heart failure, uncontrolled angina, history of myocardial infarction, unstable angina or stroke within 6 months before study entry, uncontrolled hypertension or clinically significant arrhythmias not controlled by medication).
8. The patient has uncontrolled, clinically significant pulmonary disease (eg, chronic obstructive pulmonary disease, pulmonary hypertension) that, in the opinion of the Investigator, would put the patient at significant risk for pulmonary complications during the study.
9. The patient has known active or suspected central nervous system (CNS) leukemia. If suspected, CNS leukemia should be ruled out with relevant imaging and/or examination of cerebrospinal fluid.
10. The patient is receiving immunosuppressive therapy – with the exception of low-dose prednisone (=10 mg/day) – for treatment or prophylaxis of graft-versus-host disease (GVHD). If the patient has been on immunosuppressive treatment or prophylaxis for GVHD, the treatment(s) must have been discontinued at least 14 days before study treatment and there must be no evidence of Grade =2 GVHD.
11. The patient has uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, disseminated intravascular coagulation, or psychiatric illness/social situations that would limit compliance with study requirements.
12. The patient is pregnant or breastfeeding.
13. The patient has known positive status for human immunodeficiency virus or active or chronic hepatitis B or hepatitis C (Patients with positive serology for HBV can be enrolled and must receive antiviral prophylaxis – i.e lamivudine or entcavir).
14. The patient is oxygen-dependent.
15. The patient has any medical condition that, in the opinion of the Investigator, places the patient at an unacceptably high risk for toxicities.
16. The patient has AML and requires more than 1 g/day of hydroxyurea. (Hydroxyureaispermittedatdoses of =1 g/day.)

1. Diagnosi di leucemia promielocitica acuta (APL, FAB sottotipo M3).
2. Il paziente presenta persistenti tossicità clinicamente significative di Grado = 2 in seguito a precedente chemioterapia (escluse alopecia, nausea, fatica e valori di funzionalità epatica [come richiesto nei criteri di inclusione]).
3. Il paziente ha ricevuto un trattamento con chemioterapia, terapia biologica o radiazioni wide-field nei 14 giorni precedenti l’ingresso nello studio.
4. Il paziente ha ricevuto un trattamento con farmaco sperimentale nei 14 giorni precedenti l’ingresso nello studio.
5. Il paziente è stato precedentemente trattato con tagraxofusp.
6. Il paziente ha una neoplasia attiva e/o una storia tumorale (escluso MDS antecedente) che può portare a valutazioni erronee degli end-points dello studio. I pazienti con una storia tumorale (nei 2 anni precedenti l'ingresso) con un sostanziale potenziale di recidiva e/o neoplasie attive in corso saranno valutati caso per caso. I pazienti con le seguenti diagnosi neoplastiche sono eleggibili: carcinoma cutaneo non melanoma, carcinoma in situ, neoplasia intraepiteliale cervicale, carcinoma prostatico organo-confinato senza evidenza di malattia progressiva.
7. Il paziente ha una patologia cardiovascolare clinicamente significativa (come Insufficienza cardiaca congestizia incontrollata o di classe NYHA 3 o 4, angina incontrollata, storia di infarto del miocardio, angina instabile o ictus nei 6 mesi precedenti l'ingresso nello studio, ipertensione incontrollata o aritmia clinicamente significativa non controllata da farmaci).
8. Il paziente ha una patologia polmonare incontrollata e clinicamente significativa (come broncopneumopatia cronica ostruttiva, ipertensione polmonare) che, secondo il parere dello Sperimentatore, potrebbe mettere il paziente ad un rischio significativo di complicanze polmonari durante lo studio.
9. Il paziente ha una leucemia attiva o sospetta del sistema nervoso centrale (SNC). Se sospetta, la leucemia SNC dovrebbe essere esclusa con imaging e/o esame del liquido cerebrospinale.
10. Il paziente sta ricevendo una terapia immunosoppressiva – con eccezione di prednisone a basso dosaggio (=10 mg / die) – per il trattamento o la profilassi della malattia da rigetto (GVHD). In caso, la somministrazione deve essere stata interrotta almeno 14 giorni prima dell’inizio del trattamento in studio e non deve esserci evidenza di GVHD di Grado = 2.
11. Il paziente ha una patologia incontrollata intercorrente tra cui, ad esempio, un’infezione incontrollata, una coagulazione intravascolare disseminata o una patologia psichiatrica/situazioni sociali che limiterebbero la conformità con i requisiti dello studio.
12. Donne in gravidanza o allattamento.
13. Il paziente presenta uno stato positivo noto per il virus dell’immunodeficienza umana o epatite B o C attiva o cronica (i pazienti con sierologia positiva per HBV possono essere arruolati e devono ricevere profilassi antivirale – i.e. lamivudina o entecavir).
14. Il paziente è dipendente dall'ossigeno.
15. Il paziente ha una qualsiasi condizione medica che, secondo il parere dello Sperimentatore, lo pone ad un rischio inaccettabilmente alto di tossicità.
16. Il paziente ha LMA e richiede più di 1 g/die di idrossiurea (consentita a dosi = 1 g/die).

E.5 End points

E.5.1

Primary end point(s)

Evaluate the activity of tagraxofusp in patients with CD123+ or BlasticPlasmacytoid Dendritic Cell Neoplasm-like phenotype (BPDCN-IF) Relapsed/Refractory (R/R) Acute Myeloid Leukemia(AML). The activity of the study drug will be evaluated in terms of PR, CR or CRi after four cycles.

Valutare l'attività di tagraxofusp in pazienti con Leucemia mieloide acuta con cellule dendritiche CD123 + o Fenotipo Blastico Plasmacitoide simile alla neoplasia (BPDCN-IF) recidivante / refrattaria (R / R). L'attività del farmaco in studio sarà valutata in termini di PR, CR o CRi dopo quattro cicli.

E.5.1.1

Timepoint(s) of evaluation of this end point

After 4 cycles of therapy.

Dopo 4 cicli di terapia.

E.5.2

Secondary end point(s)

To estimate in the two cohorts (BPDCN-IF and CD123+ AML):
1) Rate of adverse events (AE) and serious AE (SAE)
2) Overall Survival (OS) at 24 months
3) Event Free Survival (EFS) at 24 months
4) Disease Free Survival (DFS) at 24 months from response assessment
5) Cumulative incidence of relapse (CIR) at 24 months from response assessment
6) Response rate, OS, EFS, DFS and CIR according to baseline characteristics such as age, performance status, white blood cell (WBC), morphology, cytogenetic and molecular features.
7) Percentage of patients undergoing allogeneic stem cell transplantation in MRD-negative CR

Per stimare nelle due coorti (BPDCN-IF e CD123 + AML):
1) Tasso di eventi avversi (AE) e eventi avversi gravi (SAE)
2) Sopravvivenza globale (OS) a 24 mesi
3) Event Free Survival (EFS) a 24 mesi
4) Disease Free Survival (DFS) a 24 mesi dalla valutazione della risposta
5) Incidenza cumulativa di recidiva (CIR) a 24 mesi dalla valutazione della risposta
6) Tasso di risposta, OS, EFS, DFS e CIR in base alle caratteristiche di base come l'età, stato delle prestazioni, globuli bianchi (WBC), morfologia, caratteristiche citogenetiche e molecolari.
7) Percentuale di pazienti sottoposti a trapianto di cellule staminali allogeniche in CR MRD negativo

E.5.2.1

Timepoint(s) of evaluation of this end point

At 24 months.

A 24 mesi.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.7

Trial has a data monitoring committee

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

Last Visit of the Last Subject

Ultima Visita dell'ultimo Paziente

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

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)

Patients will continue to be followed according to the normal care activity provided by good clinical practice.

I Pazienti continueranno ad essere seguiti secondo la normale attività assistenziale prevista dalla buona pratica clinica.

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	Fondazione GIMEMA (Gruppo Italiano Malattie EMatologiche dell'Adulto)
G.4.3.4	Network Country	Italy

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2020-12-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-10-26

P. End of Trial
P.	End of Trial Status	Ongoing

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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