Clinical Trials Register

Summary
EudraCT Number:	2019-002217-19
Sponsor's Protocol Code Number:	M19-708
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
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

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2019-002217-19

A.3

Full title of the trial

Randomized, Open-label, 2-Arm, Multicenter, Phase 3 Study of Venetoclax and Azacitidine Versus Best Supportive Care as Maintenance Therapy for Patients with Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy (VIALE-M)

Studio Multicentrico di Fase 3 , Randomizzato, In Aperto, A 2 Bracci per Valutare Venetoclax in Associazione ad Azacitidina rispetto alla Miglior Terapia di Supporto come Terapia di Mantenimento in Pazienti Affetti da Leucemia Mieloide Acuta in Prima Remissione dopo Chemioterapia Convenzionale (VIALE-M)

A.3.1

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

Randomized, Open-label, 2-Arm, Multicenter, Study of Venetoclax and Azacitidine Versus Best Supportive Care as Maintenance Therapy for Patients with Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy

Studio Multicentrico , Randomizzato, In Aperto, A 2 Bracci per Valutare Venetoclax in Associazione ad Azacitidina rispetto alla Miglior Terapia di Supporto come Terapia di Mantenimento in Pazienti Affetti da Leucemia Mieloide Acuta in Prima Remissione dopo Chemioterapia Convenzionale

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

M19-708

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	na
D.3.9.3	Other descriptive name	na
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

Neoplasie ematologiche

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 AZA as maintenance therapy in subjects with AML who have achieved CR or CRi with conventional chemotherapy

Part II: To evaluate if venetoclax in combination with AZA as maintenance therapy improves relapse-free survival (RFS) comparing to BSC in subjects with AML who have achieved CR + CRi with conventional chemotherapy.

Parte I: Determinare la dose raccomandata di Fase 3 di venetoclax somministrato in combinazione con AZA come terapia di mantenimento in soggetti affetti da LMA che hanno ottenuto CR o CRi con la chemioterapia convenzionale
Parte II: Valutare se venetoclax in combinazione con AZA come terapia di mantenimento migliori la sopravvivenza libera da recidiva (relapse-free survival, RFS) rispetto a BSC in soggetti affetti da LMA che hanno ottenuto CR o CRi con la chemioterapia convenzionale.

E.2.2

Secondary objectives of the trial

•To evaluate if venetoclax in combination with AZA as maintenance therapy improves overall survival (OS) in comparison to BSC
•To evaluate the safety of venetoclax in combination with AZA as maintenance therapy in comparison to BSC
•To evaluate if venetoclax in combination with AZA as maintenance therapy improves the MRD conversion rate among subjects who are MRD-positive at study initiation in comparison to BSC
•To evaluate if venetoclax in combination with AZA as maintenance therapy compared to BSC delays time to deterioration in Global Health Status (GHS)/(QoL) score as measured by procedures outlined in the European Organisation for Research and Treatment of Cancer Quality-of-Life Questionnaire - Core 30 item (EORTC QLQ C30) scoring manual
•To evaluate if venetoclax in combination with AZA as maintenance therapy has impact on fatigue when compared to BSC based on PRO assessment of the Patient Reported Outcomes Measurement Information System(PROMIS)Fatigue Short Form (SF) 7a

Valutare se venetoclax in combinazione con AZA come terapia di mantenimento migliori la sopravvivenza globale (overall survival, OS) rispetto a BSC.Valutare la sicurezza di venetoclax in combinazione con AZA come terapia di mantenimento rispetto a BSC.Valutare se venetoclax in combinazione con AZA come terapia di mantenimento migliori il tasso di conversione a malattia residua minima rispetto a BSC fra i soggetti MRD positivi all’inizio dello studio.Valutare se venetoclax in combinazione con AZA come terapia di mantenimento rispetto a BSC prolunghi il tempo al peggioramento del punteggio GHS/(QoL) misurato in base alle procedure descritte nel manuale di assegnazione del punteggio per il questionario EORTC QLQ-C30. Valutare se venetoclax in combinazione con AZA come terapia di mantenimento produca un impatto sulla faticabilità rispetto a BSC sulla base degli esiti segnalati dai pazienti valutati mediante lo strumento Fatigue Short Form [SF] 7a del sistema PROMIS

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Subject must be = 18 years old, have newly diagnosed AML with intermediate or adverse risk cytogenetics, have confirmed CR or CRi following completion of planned induction and consolidation chemotherapy, have achieved first CR + CRi within 4 months of enrollment or no more than 75 days since last dose of conventional chemotherapy, and have an Eastern Cooperative Oncology Group (ECOG) performance status of = 2.
The key laboratory requirements are as follows:
•creatinine clearance = 30 mL/minute; calculated by the Cockcroft Gault formula or measured by 24-hour urine collection;
•bilirubin < 3.0 × upper limit of normal (ULN) (adequate liver function);
•absolute neutrophil count = 1,500/µL;
platelets = 100,000/mm3.

E.3 CRITERI DI INCLUSIONE PRINCIPALI (elencare i più importanti):

Italiano Il soggetto deve essere di età = 18 anni, presentare LMA di nuova diagnosi con rischio citogenetico intermedio oppure sfavorevole, avere conferma di CR + CRi dopo aver completato una chemioterapia programmata di induzione e consolidamento, aver ottenuto la prima CR o CRi nei 4 mesi precedenti l’arruolamento o non oltre 75 giorni dall’ultima dose della chemioterapia convenzionale, e presentare punteggio dello stato funzionale ECOG (Eastern Cooperative Oncology Group) pari a = 2.
I principali requisiti relativi ai valori di laboratorio sono i seguenti:
· clearance della creatinina = 30 mL/minuto; calcolata mediante formula Cockcroft Gault oppure misurata su campione delle urine delle 24 ore;
· bilirubina < 3,0 × il limite superiore della norma (ULN) (funzionalità epatica adeguata);
· conta assoluta dei neutrofili = 1.500/µL;
· piastrine = 100.000/mm3.

E.4

Principal exclusion criteria

History of APL.
History of active central nervous system involvement with AML.

Storia di LPA
Storia di interessamento attivo del sistema nervoso centrale associato a LMA

E.5 End points

E.5.1

Primary end point(s)

Part I: The primary endpoint is dose-limiting toxicities of venetoclax in combination with AZA

Part II:Relapse-Free Survival (RFS)

Parte I: L’endpoint primario è rappresentato dalle tossicità limitanti la dose per venetoclax in combinazione con AZA

Parte II: Sopravvivenza libera da recidiva (RFS)

E.5.1.1

Timepoint(s) of evaluation of this end point

Timepoint for part I: After approximately 20 subjects have been treated for at least 28 days

Timepoint for part II: The time from randomization to the date of relapse or the date of death from any cause, whichever comes first.

Tempistica per la parte I: Una volta che circa 20 soggetti siano stati trattati per almeno 28 giorni

Tempistica per la 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 is defined as the number of days from the date of randomization to the date of death.
•Minimal Residual Disease: The MRD conversion rate is defined as the proportion of subjects deemed MRD positive (= 10-3) at study initiation who converted to MRD of < 10-3 in the bone marrow after randomization or initiation of treatment.
•Patient-Reported Outcomes: GHS/QoL scale from the EORTC QLQ-C30 scores will be assessed and computed according to procedures outlined in EORTC QLQ-C30 scoring manual. Time to deterioration in GHS/QoL is defined as time from randomization to death from any cause or first-time decrease of = 5 points from baseline.
•Patient-Reported Outcomes: Fatigue will be assessed using the PROMIS Fatigue SF 7a global fatigue score and calculated according to the procedure outlined in the PROMIS fatigue scoring manual. The impact on fatigue score will be evaluated by the change from baseline at all scheduled visits.

· Sopravvivenza Globale: Per OS si intende il numero di giorni trascorsi dalla data della randomizzazione fino alla data del decesso.
· Malattia Residua Minima: Per tasso di conversione della MRD si intende la percentuale di soggetti considerati MRD-positivi (= 10–3) all’inizio dello studio che sono passati a un livello di MRD < 10–3 nel midollo osseo dopo la randomizzazione o l’inizio del trattamento.
· Esiti segnalati dai pazienti: i punteggi della scala GHS/QoL dello strumento EORTC QLQ-C30 saranno valutati e calcolati in base alle procedure descritte nel manuale di assegnazione del punteggio per EORTC QLQ-C30. Per tempo al peggioramento del punteggio GHS/QoL si intende il tempo trascorso dalla randomizzazione al decesso per qualsiasi causa o alla prima riduzione pari a = 5 punti rispetto al baseline.
· Esiti segnalati dai pazienti: La faticabilità sarà valutata sulla base del punteggio dello strumento PROMIS Fatigue SF 7a global fatigue e calcolati in base alle procedure descritte nel manuale di assegnazione del punteggio per lo strumento PROMIS fatigue. Per impatto sul punteggio relativo alla faticabilità si intende la variazione rispetto al baseline rilevata a tutte le visite programmate.

E.5.2.1

Timepoint(s) of evaluation of this end point

•Overall Survival: OS is defined as the number of days from the date of randomization to the date of death.
•The MRD conversion rate is defined as the proportion of subjects deemed MRD positive (= 10-3) at study initiation who converted to MRD of < 10-3 in the bone marrow after randomization or initiation of treatment.
•Time to deterioration in GHS/QoL is defined as time from randomization to death from any cause or first-time decrease of = 5 points from baseline.
•The impact on fatigue score will be evaluated by the change from baseline at all scheduled visits."

· Sopravvivenza Globale: Per OS si intende il numero di giorni trascorsi dalla data della randomizzazione fino alla data del decesso.
· Per tasso di conversione della MRD si intende la percentuale di soggetti considerati MRD-positivi (= 10–3) all’inizio dello studio che sono passati a un livello di MRD < 10–3 nel midollo osseo dopo la randomizzazione o l’inizio del trattamento.
· Per tempo al peggioramento del punteggio GHS/QoL si intende il tempo trascorso dalla randomizzazione al decesso per qualsiasi causa o alla prima riduzione pari a = 5 punti rispetto al baseline.
· L’impatto sul punteggio relativo alla faticabilità sarà valutato sulla base della variazione rispetto al baseline rilevata a tutte le visite programmate.

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)

Yes

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

Migliore terapia di supporto

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

Japan

Korea, Republic of

Russian Federation

Taiwan

Turkey

United States

France

Germany

Greece

Hungary

Italy

Spain

United Kingdom

Czechia

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

The end-of-study is defined as the date of the last subject's last visit or date of the last follow-up contact, whichever is later.

Per fine dello studio clinico si intende la data dell’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

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:

160

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

200

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

117

F.4.2.2

In the whole clinical trial

360

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 final 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.

Qualora un soggetto interrompa lo studio anticipatamente,le procedure descritte per la visita finale dovranno essere completate il prima possibile. In aggiunta,in accordo con il soggetto,ci potrà essere un contatto telefonico di FU 30 gg dopo l’ultima dose del medicinale sperimentale per i soggetti nella Parte 1 e 2, Braccio A o dopo il ritiro dallo studio per i soggetti nella Parte 2,Braccio B,al finediverificarechetuttigliAE/SAEemergentidaltrattamentosisianorisolti

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

2020-04-16

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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IMP with orphan designation in the indication
Orphan Designation Number:
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