Clinical Trials Register

Summary
EudraCT Number:	2019-002621-30
Sponsor's Protocol Code Number:	M19-063
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-06-17
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2019-002621-30

A.3

Full title of the trial

A Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in combination with Azacitidine after allogeneic Stem Cell Transplantation in Subjects with Acute Myeloid Leukemia (AML) (VIALE-T)

Studio clinico randomizzato, in aperto di fase 3 per valutare la sicurezza e l’efficacia di venetoclax in
combinazione con azacitidina dopo trapianto allogenico di cellule staminali in soggetti affetti da leucemia
mieloide acuta (LMA) (VIALE-T)

A.3.1

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

Open Label 2-Arm Study of Venetoclax in Combination with Azacitidine Versus Best Supportive Care after Allogeneic Stem Cell Transplantation in Subjects with Acute Myeloid Leukemia (AML)

Studio in aperto a 2 bracci di venetoclax in combinazione con azacitidina rispetto alla migliore terapia di supporto dopo trapianto allogenico di cellule staminali in soggetti con leucemia mieloide acuta (LMA)

A.3.2

Name or abbreviated title of the trial where available

P3 Ven+Aza vs BSC in AML post allogneneic stem cell transplant

P3 Ven+Aza vs BSC in soggetti con LMA dopo trapianto allogenico di cellule staminali

A.4.1

Sponsor's protocol code number

M19-063

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	ABBVIE DEUTSCHLAND GMBH & CO. KG
B.1.3.4	Country	Germany
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	AbbVie Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	AbbVie Ltd.
B.5.2	Functional name of contact point	EU Clinical Trials Helpdesk
B.5.3	Address:
B.5.3.1	Street Address	Abbvie House, Vanwall Business Park
B.5.3.2	Town/ city	Maidenhead, Berkshire
B.5.3.3	Post code	SL6 4UB
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	00441628561090
B.5.5	Fax number	00441628461153
B.5.6	E-mail	eu-clinical-trials@abbvie.com

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	EU/3/16/1767
D.3 Description of the IMP
D.3.1	Product name	Venetoclax
D.3.2	Product code	[ABT-199 (GDC-0199)]
D.3.4	Pharmaceutical form	Film-coated 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.3.8	INN - Proposed INN	Venetoclax 10 mg
D.3.9.2	Current sponsor code	ABT-199 (GDC-0199)
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	10
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.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/16/1767
D.3 Description of the IMP
D.3.1	Product name	Venetoclax
D.3.2	Product code	[ABT-199 (GDC-0199)]
D.3.4	Pharmaceutical form	Film-coated 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.3.8	INN - Proposed INN	Venetoclax 50 mg
D.3.9.2	Current sponsor code	ABT-199 (GDC-0199)
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	50
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.IMP: 3
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	EU/3/16/1767
D.3 Description of the IMP
D.3.1	Product name	Venetoclax
D.3.2	Product code	[ABT-199 (GDC-0199)]
D.3.4	Pharmaceutical form	Film-coated 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.3.8	INN - Proposed INN	Venetoclax 100 mg
D.3.9.2	Current sponsor code	ABT-199 (GDC-0199)
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.IMP: 4
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	Vidaza®
D.2.1.1.2	Name of the Marketing Authorisation holder	Celgene Europe Ltd
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	Azacitidine
D.3.2	Product code	[n/a]
D.3.4	Pharmaceutical form	Powder for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	AZACITIDINA
D.3.9.1	CAS number	320-67-2
D.3.9.2	Current sponsor code	n/a
D.3.9.4	EV Substance Code	SUB05624MIG
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

Acute Myeloid Leukemia (AML)

Leucemia Mieloide Acuta (LMA)

E.1.1.1

Medical condition in easily understood language

Hematologic Malignancies

Neoplasia Ematologica

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

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.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Part I: To determine the recommended Phase 3 dose of venetoclax in combination with azacitidine in AML patients when given as maintenance therapy following allogeneic stem cell transplantation.

Part II:To evaluate the efficacy of venetoclax in combination with azacitidine to improve Relapse Free Survival (RFS) in AML patients compared to Best Supportive Care (BSC) when given as maintenance therapy following allogeneic stem cell transplantation

Parte I: Determinare la dose raccomandata di Fase 3 di venetoclax in combinazione con azacitidina in pazienti affetti da LMA quando somministrato come terapia di mantenimento a seguito di trapianto allogenico di cellule staminali

Parte II: Valutare l’efficacia di venetoclax in combinazione con azacitidina nel migliorare la sopravvivenza libera da recidiva (RFS) in pazienti con LMA rispetto alla miglior terapia di supporto (BSC) quando somministrato come terapia di mantenimento a seguito di trapianto allogenico di cellule staminali.

E.2.2

Secondary objectives of the trial

*To confirm the safety of venetoclax in combination with azacitidine after allogeneic transplantation in subjects diagnosed with AML.
*To determine the efficacy of venetoclax in combination with azacitidine on overall survival.
*To determine the effect of venetoclax in combination with azacitidine on the frequency and severity of GvHD.
*To determine the effect of venetoclax in combination with azacitidine on Quality of life (QoL).

• Confermare la sicurezza di venetoclax in combinazione con azacitidina dopo trapianto allogenico in soggetti con diagnosi di LMA.
• Determinare l’efficacia di venetoclax in combinazione con azacitidina sulla sopravvivenza globale.
• Determinare l’effetto di venetoclax in combinazione con azacitidina sulla frequenza e sulla gravità di GvHD.
• Determinare l’effetto di venetoclax in combinazione con azacitidina sulla Qualità di Vita (QoL).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Adult male or female = 18 years old for Part 1 and, male or female at least 12 years old for Part 2.
Subject must be diagnosed with AML by World Health Organization (WHO) criteria (2017) and either be planning for allogeneic stem cell transplantation or have received allogeneic stem cell transplantation within the past 14 days.
Blast percentage in bone marrow before transplant must be < 10%. Blast count in peripheral blood must be “0” and Blast percentage in bone marrow must be < 5% after transplant within 7 days prior to Cycle 1 Day 1.
Bilirubin = 3 × ULN. Cycle 1 Day 1 must occur within 28 to 200 days after transplant and no later than 60 days since meeting count recovery criteria post-transplant.
Post-transplant count recovery (within 7 days prior to Cycle 1 Day 1) criteria: ANC measured twice at least 2 days apart and within one week from Cycle 1 Day 1. S
ubjects should not have granulocyte-colony stimulating factor (G-CSF) treatment for 7 days before first ANC count. (ANC value for Part 1 = 1,500/µL and = 1,000/µL for Part 2); Platelets should be measured twice at least 2 days apart and within 1 week from Cycle 1 Day 1.
Subjects should not have a platelet transfusion for 7 days before first platelet count. (value for Part 1 = 80,000/µL and = 50,000/µL for Part 2). Adequate renal function as demonstrated by a creatinine clearance >30 mL/min; calculated by the Cockcroft Gault formula or measured by 24-hour urine collection and subjects 12-17 years old must have the equivalent as calculated by the Schwartz formula. Subjects = 17 years old must have a Karnofsky Performance Scale (KPS) score > 50 and subjects 12 - = 16 years old must have a Lansky Play Performance Scale score > 40.

Soggetti di ambo i sessi e di età = 18 anni per la Parte 1 e soggetti di ambo i sessi e di età pari ad almeno 12 anni per la Parte 2.
Il soggetto con diagnosi di LMA sulla base dei criteri OMS (Organizzazione Mondiale della Sanità, WHO; 2017) e per cui sia programmato un trapianto allogenico di cellule staminali o che sia stato sottoposto a trapianto allogenico di cellule staminali negli ultimi 14 giorni.
La percentuale di blasti nel midollo osseo prima del trapianto deve essere < 10%
La conta dei blasti nel sangue periferico deve essere pari a “0” e la percentuale di blasti nel midollo osseo deve essere < 5% dopo il trapianto nei 7 giorni precedenti il Ciclo 1 Giorno 1
Bilirubina = 3 × ULN. Il Ciclo 1 Giorno 1 deve avere luogo da 28 a 200 giorni dopo il trapianto e non oltre 60 giorni dal momento in cui risultino soddisfatti i criteri di recupero ematologico post-trapianto.
Criteri relativi al recupero ematologico post-trapianto (nei 7 giorni precedenti il Ciclo 1 Giorno 1): ANC misurato due volte con un intervallo di almeno 2 giorni nel corso della settimana precedente il Ciclo 1 Giorno 1. I soggetti non devono aver ricevuto trattamento con fattore di crescita stimolante le colonie di granulociti (G-CSF) nei 7 giorni precedenti la prima misurazione dei livelli di ANC (valore di ANC = 1.500/µL per la Parte 1 e = 1.000/µL per la Parte 2); Livello di piastrine misurato due volte con un intervallo di almeno 2 giorni nel corso della settimana precedente il Ciclo 1 Giorno 1. I soggetti non devono aver ricevuto una trasfusione di piastrine nei 7 giorni precedenti la prima misurazione dei livelli di piastrine (valore = 80.000/µL per la Parte 1 e = 50.000/µL per la Parte 2). Funzione renale adeguata confermata da valori di clearance della creatinina > 30 mL/min calcolata mediante la formula Cockcroft Gault oppure misurata su un campione di urine delle 24 ore. Nei soggetti di età compresa fra 12 e 17 anni per calcolare l’equivalente di tale valore verrà utilizzata la formula Schwartz. I soggetti di età = 17 anni devono avere un punteggio Karnofsky Performance Scale (KPS) > 50 e i soggetti di età 12 - = 16 anni devono avere un punteggio Lansky Play Performance Scale > 40.

E.4

Principal exclusion criteria

History of disease progression during prior treatment with venetoclax.
History of any other malignancy within 2 years prior to study entry, except for: Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast; Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent; Myelodysplastic Syndrome.
Known infection with HIV or history of being positive for hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
Presence of clinical or laboratory symptoms/signs of extramedullary myeloid malignancy.

Storia di progressione della malattia nel corso del trattamento pregresso con venetoclax.
Storie di qualsiasi altra neoplasia maligna nei 2 anni precedenti l’ingresso nello studio, ad eccezione dei seguenti: carcinoma in situ della cervice uterina oppure carcinoma in sito della mammella, trattati adeguatamente; carcinoma cutaneo basocellulare oppure carcinoma cutaneo squamocellulare localizzato; Pregressa neoplasia maligna localizzata e sottoposta a resezione chirurgica (oppure trattata con altre modalità) con intento curativo; sindrome mielodisplastica.
Nota infezione da HIV oppure storia di positività per l’infezione da virus dell’epatite B (HBV o dell’epatite C (HCV).
Presenza di sintomi clinici o di laboratorio/segni di neoplasia maligna mieloide extramidollare.

E.5 End points

E.5.1

Primary end point(s)

Part I: The primary endpoint is the frequency of DLTs of venetoclax in combination with azacitidine
Part II: Relapse-Free Survival (RFS)

Parte I: L’endpoint primario è rappresentato dalla frequenza di tossicità limitanti la dose (DLT) per venetoclax in combinazione con azacitidina
Parte II: Sopravvivenza libera da recidiva (RFS)

E.5.1.1

Timepoint(s) of evaluation of this end point

Part I: the first treatment cycle (28 days).
Part II:The time from randomization to the date of relapse or the date of death
from any cause, whichever occurs first.

Parte I: il primo ciclo di trattamento (28 giorni).
Parte II: Il tempo trascorso fra la randomizzazione e la data della recidiva o la data del decesso per qualsiasi causa, quale di questi eventi avvenga per primo

E.5.2

Secondary end point(s)

• Overall Survival (OS)
• GvHD-free, relapse free survival (GRFS).
• Time to deterioration in Global Health Status (GHS)/QoL
• GvHD rate at 90 days after randomization
• Fatigue Change in adult subjects
• Patient Reported Outcome (PRO) using Patient Reported Outcomes
• Minimal residual disease (MRD) response rate

· Sopravvivenza Globale (Overall Survival, OS)
· Sopravvivenza libera da GvHD (graft-versus-host-disease) e da recidiva (GvHD-free, relapse-free survival (GRFS)
· Tempo al peggioramento del parametro Global Health Status GHS/QoL
· Tasso di GvHD 90 giorni dopo la randomizzazione
· Variazione della faticabilità nei soggetti adulti
· Esiti segnalati dai pazienti (PRO) sulla base degli esiti segnalati dai pazienti
· Tasso di risposta relativa alla Malattia Residua Minima

E.5.2.1

Timepoint(s) of evaluation of this end point

Overall Survival (OS)-time from randomization to death(any reason)
GvHD-free,relapse free survival(GRFS)-time from randomization to a) disease relapse, b)occurrence or worsening of GvHD, or c)death(any reason)
Time to deterioration using GHS/QoL in adult subjects-randomization to either deterioration of =5 points based on EORTC QLQ-C30(v3)or death (any cause)
GvHD rate at 90 days after randomization-Grade 2 or higher for aGvHD and moderate/severe for cGvHD per investigator
Fatigue Change in adult subjects-using Patient Reported Outcomes Measurement Information System (PROMIS) Cancer Fatigue SF 7a. Difference between randomization and 6 months of treatment
MRD response rate-only among subjects with MRD > 10-3 at study start; response is MRD conversion to < 10-3 after treatment start.

· Sopravvivenza Globale (OS)
· Sopravvivenza libera da GvHD e da recidiva
· Tempo al peggioramento sulla base del punteggio GHS/QoL
· Tasso di GvHD 90 giorni dopo la randomizzazione-di Grado = 2 per aGvHD e di grado moderato/grave per cGvHD sulla base della valutazione del medico sperimentatore
· Variazione della faticabilità in soggetti adulti - sulla base degli esiti segnalati dai pazienti (Patient Reported Outcome, PRO) utilizzando lo strumento PROMIS (Patient Reported Outcomes Measurement Information System) Cancer Fatigue SF 7a. Differenza fra la randomizzazione e 6 mesi di trattamento
· Tasso di risposta MRD - solo fra i soggetti con MRD = 10–3 all’inizio dello studio; per risposta si intende la conversione a MRD < 10–3 dopo l’inizio del trattamento

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

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

Yes

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)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Best Supportive Care (BSC)

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

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Brazil

Canada

China

Israel

Japan

Korea, Republic of

Russian Federation

Taiwan

United States

Switzerland

E.8.7

Trial has a data monitoring committee

Yes

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 subject's last visit or date of the last follow-up contact, whichever is later.

L’ultima visita dell’ultimo soggetto oppure la data dell’ultimo contatto di follow-up, quale dei due avvenga per ultimo

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

Yes

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

325

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

424

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)

If a subject prematurely discontinues the study (withdrawal of informed consent), The procedures outlined for the end of treatment visit are to be completed as soon as possible, preferably within 2 weeks. In addition, if subject is willing, a 30-day follow-up phone call after the last dose of study drug for subjects in Part 1 and Part 2, Arm A or after study withdrawal for subjects in Part 2, Arm B may be completed to ensure all treatment-emergent AEs/SAEs have been resolved.

Se un soggetto interrompe lo studio in anticipo (ritiro del consenso),le procedure per la visita di fine trattamento dovranno essere completate prima possibile,preferibilmente entro 2 settimane.Inoltre d'accordo col soggetto potrà essere realizzato un follow-up telefonico a 30 giorni dopo l’ultima dose per i soggetti nella Parte1 e nella Parte2,BraccioA o dopo il ritiro dallo studio per i soggetti nella Parte2,BraccioB,al fine di verificare la risoluzione di tutti gli AE/SAE emergenti.

G. Investigator Networks to be involved in the Trial

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2020-01-07

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-03-05

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

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