Clinical Trials Register

Summary
EudraCT Number:	2019-003871-20
Sponsor's Protocol Code Number:	AML1819
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:	2021-05-24
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

2019-003871-20

A.3

Full title of the trial

Phase III study to assess the impact of gemtuzumab ozogamicin, in combination with standard chemotherapy, on the levels of minimal residual disease, and the role of glasdegib as a post-transplant maintenance, in adult patients, aged 18-60 years, with previously untreated, de novo, favorable-intermediate-risk acute myeloid leukemia.

Studio di fase III per determinare l’impatto di gemtuzumab ozogamicin, in associazione a chemioterapia standard, sui livelli di malattia minima residua, e il ruolo di glasdegib come mantenimento post-trapianto, in pazienti adulti, di età compresa tra 18 e 60 anni, affetti da Leucemia Mieloide Acuta non precedentemente trattata, di nuova diagnosi, a rischio favorevole o intermedio.

A.3.1

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

Study to determine the efficacy of Gemtuzumab Ozogamicin, together with standard chemotherapy, on minimal residual disease levels as well as the efficacy of Glasdegib as maintenance after transplantation, in adult patients with Acute Myeloid Leukemia (AML), a tumor characterized from an abnormal proliferation of blood cells present in the bone marrow in the form of immature myeloid "precursors", ie cells not yet differentiated, called blasts.

Studio volto a determinare l'efficacia di Gemtuzumab Ozogamicin, unito alla chemioterapia standard, sui livelli di malattia minima residua oltre che l'efficacia di Glasdegib come mantenimento dopo il trapianto, in pazienti adulti affetti da Leucemia Mieloide Acuta (AML), un tumore caratterizzato da una proliferazione anomala delle cellule del sangue presenti nel midollo osseo sotto forma di «precursori» mieloidi immaturi, cioè cellule non ancora differenziate,dette blasti.

A.3.2

Name or abbreviated title of the trial where available

AML1819

A.4.1

Sponsor's protocol code number

AML1819

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	Fondazione GIMEMA (Gruppo Italiano Malattie EMatologiche dell'Adulto) Franco Mandelli Onlus
B.4.2	Country	Italy
B.4.1	Name of organisation providing support	Pfizer S.R.L.
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 GIMEMA
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	Yes
D.2.1.1.1	Trade name	MYLOTARG 5 mg polvere per concentrato per soluzione per infusione
D.2.1.1.2	Name of the Marketing Authorisation holder	Pfizer Inc.
D.2.1.2	Country which granted the Marketing Authorisation	Italy
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Gemtuzumab Ozogamicin
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Powder for solution for infusion
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.3.8	INN - Proposed INN	Gemtuzumab Ozogamicin
D.3.9.1	CAS number	220578-59-6
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	Gemtuzumab Ozogamicin (GO)
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
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.IMP: 2
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Glasdegib
D.3.2	Product code	[PF-04449913]
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	Glasdegib
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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

Favorable-intermediate-risk Acute Myeloid Leukemia (AML)

Leucemia Mieloide Acuta (LMA) a rischio favorevole o intermedio

E.1.1.1

Medical condition in easily understood language

AML disease that originates in the bone marrow (myeloid) and progresses quickly (acute). If the blood cells, maturing, undergo a transformation in the tumor sense, we have the pathology.

AML malattia che origina nel midollo osseo (mieloide) e progredisce velocemente (acuta).Se le cellule del sangue, maturando, vanno incontro a una trasformazione in senso tumorale si ha 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

The study has two co-primary objectives:
1. Activity of gemtuzumab ozogamicin in combination with chemotherapy in terms of MRD negativity achievement;
2. Efficacy of glasdegib maintenance vs clinical observation

Lo studio ha due obiettivi co-primari:

1. L’attività di gemtuzumab ozogamicin, in associazione a chemioterapia in termini di ottenimento della negatività della malattia minima residua (MRD);

2. L’efficacia del mantenimento con glasdegib vs osservazione clinica

E.2.2

Secondary objectives of the trial

To assess:
1. Overall Survival (OS)
2. Event Free Survival (EFS)
3. Cumulative incidence of relapse (CIR)
4. Response rate after induction therapy
5. Safety: adverse events (AE) and serious AE (SAE)
6. OS, EFS, DFS and CIR in different risk groups
7. OS, EFS, DFS and CIR according to the MRD level at each evaluation step
8. Response rate, OS, EFS, DFS and CIR according to baseline characteristics
9. Quality of Life (QoL) evaluation

Valutare:
1. Overall Survival (OS);
2. Event Free Survival (EFS);
3. Cumulative incidence of relapse (CIR);
4. Percentuale di risposta dopo la terapia d’induzione;
5. Sicurezza: eventi avversi (AE) ed eventi avversi gravi (SAE);
6. OS, EFS, DFS (Disease Free Survival) e CIR nei differenti gruppi di rischio;
7. OS, EFS, DFS e CIR secondo il livello di MRD ad ogni step di valutazione;
8. Percentuale di risposta, OS, EFS, DFS e CIR secondo le caratteristiche al baseline;
9. Valutazione della Qualità di vita (QoL).

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: 18/11/2019
Title: Translational research
Objectives: MRD assessment on peripheral blood:In patients with RUNX1-RUNX1T1, CBFB-MYH11 and mutated NPM1 AML, MRD assessment is recommend in peripheral blood (PB) and bone marrow (BM) since peripheral blood can provide even better prognostic information than the bone marrow source. The prospective objective is to learn when using MPFC for MRD assessment, - PB analysis has the same prognostic role than BM and; - the combined assessment of MRD in the two sources may increase our ability to stratify patients in terms of risk, thus improving the role of MRD monitoring and promoting early and more effective therapeutic intervention.

Life quality
Version: 1.0
Date: 18/11/2019
Title: Quality of life and other Patient Reported Outcomes Assessment
Objectives: To describe and compare QoL profiles over time between patients randomized to either glasdegib maintenance or clinical observation.To describe longitudinal QoL profile of AML patients since study entry. -To investigate the prognostic value of baseline QoL parameters for the percentage of MRD negativity after consolidation in patients treated in induction and consolidation with GO. - To investigate the prognostic value of baseline QoL parameters for overall survival (OS), event-free survival (EFS), disease-free survival (DFS) and the cumulative incidence of relapse (CIR). - To compare the reporting of patients’ symptom severity and QoL after induction, between AML patients and their treating physicians.

Farmacogenetica
Versione: 1.0
Data: 18/11/2019
Titolo: Ricerca Traslazionale
Obiettivi: Valutazione MRD su sangue periferico: Nei pazienti con RUNX1-RUNX1T1, CBFB-MYH11 e NPM1 AML mutata, la valutazione MRD è raccomandata nel sangue periferico (PB) e nel midollo osseo (BM) poiché il sangue periferico può fornire informazioni prognostiche persino migliori rispetto alla fonte del midollo osseo. L'obiettivo prospettico è quando si utilizza MPFC per la valutazione MRD, - L'analisi PB ha lo stesso ruolo prognostico di BM e; - la valutazione combinata della MRD nelle due fonti può aumentare la nostra capacità di stratificare i pazienti in termini di rischio, migliorando così il ruolo del monitoraggio della MRD e promuovendo interventi terapeutici precoci e più efficaci.

Qualita' della vita
Versione: 1.0
Data: 18/11/2019
Titolo: Qualità della vita e altre valutazioni dei risultati riportate dai pazienti
Obiettivi: Descrivere e confrontare i profili QoL nel tempo tra pazienti randomizzati al mantenimento con glasdegib o all'osservazione clinica.Descrivere il profilo QoL longitudinale dei pazienti con LMA dall'entrata in studio. -Per studiare il valore prognostico dei parametri QoL basali per la percentuale di negatività della MRD dopo consolidamento in pazienti trattati in induzione e consolidamento con GO. - Studiare il valore prognostico dei parametri QoL basali per la sopravvivenza globale (OS), la sopravvivenza libera da eventi (EFS), la sopravvivenza libera da malattia (DFS) e l'incidenza cumulativa di recidiva (CIR). - Confrontare la segnalazione della gravità dei sintomi dei pazienti e la QoL dopo l'induzione, tra i pazienti con LMA e i loro medici curanti.

E.3

Principal inclusion criteria

1 Signed written informed consent according to ICH/EU/GCP and national/local laws
2 Patients aged between 18 and 60 years
3 Patients previously untreated for their AML by other chemotherapeutic agents (except for no more than 14 days HU) or radiotherapy
4 Unequivocal diagnosis of de novo AML according to WHO diagnostic criteria (at least 20% blasts in the bone marrow), other than acute promyelocytic leukemia, documented by bone marrow aspiration (or biopsy in case of dry tap) (not supervening after other myeloproliferative disease or myelodysplastic syndromes of = 6 months duration)
5 Patients with favorable-intermediate AML according to ELN 2017 (except for FLT3-ITD/TKD positive AML)
6 WHO performance status 0-3
7 Adequate renal (serum creatinine = 2 x the institutional ULN) and liver (total serum bilirubin = 2 x ULN; serum ALT and AST = 2.5 x ULN) function, unless considered due to organ leukemic involvement
8 Left Ventricular Ejection Fraction (LVEF) = 50%, as determined by echocardiogram
9 Absence of severe concomitant neurological or psychiatric diseases and congestive heart failure or active uncontrolled infection
10 Absence of any psychological, familial, sociological and geographical condition potentially hampering compliance with the study protocol and the follow-up schedule.
11 Women of childbearing potential with a negative serum pregnancy test within 48 hrs prior to administration of chemotherapy. Post-menopausal women with amenorrhoic for at least 12 months to be considered of non-childbearing potential. Male and female patients agreed to employ an effective barrier method of birth control throughout the study and for at least 6 months following discontinuation of study drug.

1. Firma del consenso informato scritto in accordo con le normative ICH/EU/GCP e le leggi nazionali;
2. Pazienti di età compresa tra i 18 ed i 60 anni;
3. Pazienti affetti da LMA non precedentemente trattata con altri agenti chemioterapici (con l’eccezione di non più di 14 giorni di idrossiurea) o radioterapia;
4. Diagnosi inequivocabile di LMA all’esordio non trattata, secondo i criteri diagnostici WHO (almeno il 20% di blasti nel midollo osseo), con l’esclusione della leucemia acuta promielocitica, documentata da aspirato midollare (o biopsia in caso di punctio sicca) e non sopraggiunta dopo altre patologie mieloproliferative o sindromi mielodisplastiche di durata maggiore di sei mesi;
5. Pazienti con una LMA favorevole o intermedia secondo ELN 2017 (eccetto FLT3-ITD/TKD positivi);
6. Performance status WHO 0-3;
7. Funzionalità renale appropriata (creatinina sierica = 2 x ULN) ed epatica (bilirubina sierica totale = 2 x ULN; ALT e AST sieriche = 2,5 x ULN), se non considerate come dovute alla patologia;
8. Frazione di eiezione ventricolare sinistra (LVEF) = 50%, determinata con ecocardiogramma;
9. Assenza di gravi patologie psichiatriche o neurologiche concomitanti ed insufficienza cardiaca congestizia o infezione attiva non controllata;
10. Assenza di qualsiasi condizione psicologica, familiare, sociologica e geografica che potenzialmente ostacoli l’aderenza al protocollo di studio ed al programma di follow-up.
11. Le donne fertili devono avere un test sierico di gravidanza negativo entro le quarantotto ore precedenti la somministrazione della chemioterapia. Le donne in post-menopausa devono essere amenorroiche per almeno dodici mesi al fine di essere considerate non potenzialmente fertili. I pazienti maschi e femmine devono acconsentire all’utilizzo di un efficace metodo anticoncezionale di barriera durante tutto lo studio e per almeno i sei mesi successivi l’interruzione del trattamento in studio.

E.4

Principal exclusion criteria

1. Patients already treated for their AML by other chemotherapeutic agents (except for no more than 14 days HU) or radiotherapy
2. Acute promyelocytic leukemia
3. Blast crisis of chronic myeloid leukemia
4. FLT3-ITD/TKD positive AML
5. AML supervening after other myeloproliferative disease = 6 months duration
6. AML supervening after antecedent myelodysplastic syndromes
7. Therapy-related AML
8. Other active or progressive malignant diseases.
9. Inadequate renal or liver function (see no. 7 Inclusion )
10. Severe heart failure requiring diuretics
11. Ejection fraction < 50%
12. Uncontrolled infections
13. HIV positive serology
14. Severe concomitant neurological or psychiatric diseases
15. Patients who are pregnant or adults of reproductive potential not employing an effective method of birth control.

1. Pazienti la cui LMA sia già stata trattata con altri agenti chemioterapici (con l’eccezione di non più di 14 giorni di idrossiurea) o radioterapia;
2. Leucemia acuta promielocitica;
3. Crisi blastica di leucemia mieloide cronica;
4. LMA FLT3-ITD/TKD positiva;
5. LMA manifestatasi dopo un’altra patologia mieloproliferativa della durata di più di sei mesi;
6. LMA manifestatasi dopo una precedente sindrome mielodisplastica;
7. LMA correlata alla terapia;
8. Altre patologie maligne in progressione;
9. Funzionalità renale ed epatica inadeguata (vedi criterio di inclusione n°7);
10. Grave deficit cardiaco che necessiti di diuretici;
11. Frazione di eiezione <50%;
12. Infezioni non controllate;
13. Sierologia HIV positiva
14. Gravi patologie neurologiche o psichiatriche concomitanti;
15. Pazienti in gravidanza o adulti potenzialmente fertili che non utilizzino metodi anticoncezionali.

E.5 End points

E.5.1

Primary end point(s)

Co-Primary
1. Percentage of MRD negativity after consolidation in patients treated in induction and consolidation with GO;
2. Disease Free Survival (DFS) in patients randomized to glasdegib maintenance or clinical observation

Co-primari
1. Percentuale di negatività della MRD dopo consolidamento in pazienti trattati in induzione e consolidamento con GO;
2. Sopravvivenza libera da malattia (DFS) in pazienti randomizzati al mantenimento con glasdegib o all'osservazione clinica

E.5.1.1

Timepoint(s) of evaluation of this end point

1. After the consolidation phase for patients treated with Gemtuzumab Ozogamicin during the induction and consolidation phases.
2. At the end of the study.

1. Dopo la fase di consolidamento per i pazienti trattati con Gemtuzumab Ozogamicin durante le fasi di induzione e consolidamento.
2. Alla fine dello studio.

E.5.2

Secondary end point(s)

Secondary study end-points are:
1. Overall Survival (OS) at 24 months
2. Event Free Survival (EFS) at 24 months
3. Cumulative incidence of relapse (CIR) at 24 months
4. Response rate in terms of patients who achieve CR after induction therapy
5. Safety in terms of number and type of adverse events (AE) and serious AE (SAE)
6. OS, EFS, DFS and CIR in favorable and intermediate risk groups
7. OS, EFS, DFS and CIR according to the MRD level after induction and consolidation
8. Response rate, OS, EFS, DFS and CIR according to morphology, cytogenetic and molecular baseline characteristics.
9. To estimate mean trajectories over time of pre-selected QoL scales of the EORTC QLQ-C30 questionnaire.

Gli endpoint di studio secondari sono:
1. Sopravvivenza globale (OS) a 24 mesi
2. Event Free Survival (EFS) a 24 mesi
3. Incidenza cumulativa di recidiva (CIR) a 24 mesi
4. Tasso di risposta in termini di pazienti che ottengono CR dopo terapia di induzione
5. Sicurezza in termini di numero e tipo di eventi avversi (AE) e eventi avversi gravi (SAE)
6. OS, EFS, DFS e CIR in gruppi a rischio favorevole e intermedio
7. Sistema operativo, EFS, DFS e CIR in base al livello MRD dopo induzione e consolidamento
8. Tasso di risposta, OS, EFS, DFS e CIR in base alle caratteristiche morfologiche, citogenetiche e basali molecolari.
9. Per stimare le traiettorie medie nel tempo delle scale QoL preselezionate del questionario EORTC QLQ-C30.

E.5.2.1

Timepoint(s) of evaluation of this end point

1. 24 months after starting the study;
2. 24 months after starting the study;
3. 24 months after obtaining the remission;
4. After induction therapy;
5. During the entire course of the study;
6. 24 months after starting the study;
7. 24 months after starting the study;
8. 24 months after starting the study;
9. From the baseline to the end of the study.

1. A 24 mesi dall'inizio dello studio;
2. A 24 mesi dall'inizio dello studio;
3. A 24 mesi dall'ottenimento della remissione;
4. Dopo la terapia di induzione;
5. Durante l'intero corso dello studio;
6. A 24 mesi dall'inizio dello studio;
7. A 24 mesi dall'inizio dello studio;
8. A 24 mesi dall'inizio dello studio;
9. Dal baseline alla fine dello studio.

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

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 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:

414

F.1.3

Elderly (>=65 years)

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

414

F.4.2

For a multinational trial

F.4.2.1

In the EEA

414

F.4.2.2

In the whole clinical trial

414

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 normal care 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) Franco Mandelli Onlus
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

2020-02-10

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-02-26

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

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IMP with orphan designation in the indication
Orphan Designation Number:
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