Clinical Trials Register

Summary
EudraCT Number:	2012-000769-19
Sponsor's Protocol Code Number:	CBEZ235Z2401
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-09-13
Trial results	View 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

2012-000769-19

A.3

Full title of the trial

Randomized phase II study of BEZ235 or everolimus in advanced pancreatic neuroendocrine tumors

Studio randomizzato di Fase II con BEZ235 o everolimus in pazienti con tumore neuroendocrino pancreatico in stadio avanzato

A.3.1

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

Safety and efficacy of BEZ235 in patients with pancreatic neuroendocrine
tumors (pNET)

Sicurezza e efficacia di BEZ235 in pazienti con tumore neuroendocrino pancreatico in stadio avanzato (pNET)

A.4.1

Sponsor's protocol code number

CBEZ235Z2401

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	NOVARTIS FARMA
B.1.3.4	Country	Italy
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	Novartis Pharma Services AG
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	NOVARTIS FARMA
B.5.2	Functional name of contact point	Drug Regulatory Affairs
B.5.3	Address:
B.5.3.1	Street Address	Largo Umberto Boccioni, 1
B.5.3.2	Town/ city	ORIGGIO
B.5.3.3	Post code	21040
B.5.3.4	Country	Italy
B.5.4	Telephone number	02-96541
B.5.5	Fax number	02-9659066
B.5.6	E-mail	info.studiclinici@novartis.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	BEZ235
D.3.2	Product code	BEZ235
D.3.4	Pharmaceutical form	Powder for oral suspension
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	BEZ235
D.3.9.1	CAS number	1028385-32-1
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	200
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	BEZ235
D.3.2	Product code	BEZ235
D.3.4	Pharmaceutical form	Powder for oral suspension
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	BEZ235
D.3.9.1	CAS number	1028385-32-1
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	300
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	BEZ235
D.3.2	Product code	BEZ235
D.3.4	Pharmaceutical form	Powder for oral suspension
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	BEZ235
D.3.9.1	CAS number	1028385-32-1
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	400
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Comparator
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	AFINITOR*30CPR 5MG
D.2.1.1.2	Name of the Marketing Authorisation holder	NOVARTIS FARMA SpA
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.3.4	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.3.8	INN - Proposed INN	EVEROLIMUS
D.3.9.1	CAS number	159351-69-6
D.3.9.4	EV Substance Code	SUB02065MIG
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)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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: 5
D.1.2 and D.1.3	IMP Role	Comparator
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	AFINITOR*30CPR 10MG
D.2.1.1.2	Name of the Marketing Authorisation holder	NOVARTIS FARMA SpA
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.3.4	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.3.8	INN - Proposed INN	EVEROLIMUS
D.3.9.1	CAS number	159351-69-6
D.3.9.4	EV Substance Code	SUB02065MIG
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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

Adult patient in advanced pancreatic neuroendocrine tumors

Pazienti adulti con tumore neuroendocrino pancreatico in stadio avanzato

E.1.1.1

Medical condition in easily understood language

Adult patient with pancreatic neuroendocrine tumors pNET

Pazienti adulti con tumore neuroendocrino pancreatico pNET

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	15.0
E.1.2	Level	LLT
E.1.2	Classification code	10062476
E.1.2	Term	Neuroendocrine tumor
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

To assess the treatment effect of BEZ235 relative to everolimus on progression free survival in patients with advanced pancreatic neuroendocrine tumors who have not been previously treated with an mTOR inhibitor.

Valutare l’efficacia del trattamento con BEZ235 in confronto a everolimus in termini di sopravvivenza libera da progressione (PFS) in pazienti con tumore neuroendocrino pancreatico che non sono stati precedentemente trattati con un inibitore di mTOR.

E.2.2

Secondary objectives of the trial

To determine the safety and tolerability of BEZ235 and everolimus; -To estimate the objective response rate of BEZ235 and everolimus; -To estimate overall survival of BEZ235 and everolimus; -To estimate time to treatment failure of BEZ235 and everolimus.

- Determinare la sicurezza e la tollerabilità di BEZ235 ed everolimus; - Valutare il tasso di risposta obiettiva di BEZ235 ed everolimus; - Valutare la sopravvivenza globale con BEZ235 ed everolimus; - Valutare il tempo al fallimento del trattamento di BEZ235 ed everolimus.

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

PHARMACOGENETIC:
Vers:00
Date:2012/05/17
Title:N.A. (Complementary study included in the protocol)
Objectives:Please see section protocol ''7.2.3 Biomarkers''

PHARMACOGENOMIC:
Vers:00
Date:2012/05/17
Title:N.A. (Complementary study included in the protocol)
Objectives:Please see section protocol ''7.2.3 Biomarkers''

FARMACOGENETICA:
Vers:00
Data:2012/05/17
Titolo:N.A. (Studio complementare inserito all'interno del protocollo)
Obiettivi:Per favore vedere paragrafo ''7.2.3 Biomarkers'' del protocollo

FARMACOGENOMICA:
Vers:00
Data:2012/05/17
Titolo:N.A. (Studio complementare inserito all'interno del protocollo)
Obiettivi:Per favore vedere paragrafo ''7.2.3 Biomarkers'' del protocollo

E.3

Principal inclusion criteria

Advanced histologically confirmed well differentiated pancreatic neuroendocrine tumor - Progressive disease within the last 12 months - Measurable disease per RECIST Version 1.0 determined by multiphase MRI or triphasic CT Other protocol-defined inclusion criteria may apply

1.Conferma istologica di tumore neuroendocrino pancreatico (pNET) ben differenziato in stadio avanzato; 2.Documentazione radiologica di progressione della malattia nei 12 mesi precedenti la randomizzazione.Se il paziente ha ricevuto terapia antitumorale durante gli ultimi 12 mesi, la progressione radiologica deve essere documentata durante o dopo la terapia; 3. Malattia misurabile secondo RECIST Versione 1.0 mediante RMN multifase o TAC trifasica; 4.WHO performance status <= 2; 5.Consenso informato scritto ottenuto prima di qualsiasi procedura di screening; 6.Pazienti di età >= 18 anni il giorno in cui forniscono il proprio consenso allo studio; 7.I pazienti devono presentare un’adeguata funzionalità midollare e d’organo, definita da: Conta assoluta dei neutrofili (ANC) >= 1,5 x 109/L, Piastrine >= 100 x 109/L, Emoglobina >= 9,0 g/dL, INR < 1,3, ALT e AST <= 2,5 x ULN o <= 5 x ULN in presenza di metastasi epatiche, Bilirubina sierica totale <= 1,5 x ULN, Creatinina sierica <= 1,5 x ULN, Glicemia a digiuno <= 140 mg/dL, HbA1c <= 8%, Colesterolo sierico a digiuno <= 300 mg/dL OPPURE <= 7,75 mmol/L E trigliceridi a digiuno <= 2,5 x ULN. Per maggiori informazioni vedere par. 5.2 del protocollo.

E.4

Principal exclusion criteria

- Prior treatment with mTOR or PI3K inhibitors - Patients with more than 2 prior systemic treatment regimens - Previous cytotoxic chemotherapy, targeted therapy, or biotherapy within the last 4 weeks Other protocol-defined inclusion/exclusion criteria may apply

1.Pazienti con carcinoma neuroendocrino scarsamente differenziato, carcinoma neuroendocrino ad alto grado, adenocarcinoide, ‘goblet cell carcinoma’ e carcinoma a piccole cellule; 2.Trattamento precedente con inibitori di PI3K e/o di mTOR; 3.Pazienti sottoposti a più di 2 precedenti regimi di trattamento sistemico; 4.Pazienti con ipersensibilità nota, intolleranza e/o controindicazioni a uno dei trattamenti in studio; 5.Pazienti in trattamento con qualsiasi altro farmaco antitumorale concomitante a eccezione di SSA, durante lo studio; 6.Pazienti sottoposti a trattamenti antineoplastici sistemici nei 28 giorni precedenti l’inizio del trattamento in studio; 7.Pazienti sottoposti a radioterapia a campo esteso o irradiazione di >= 25% del midollo osseo <= 28 giorni o radioterapia a campo ristretto come terapia palliativa <= 14 giorni prima dell’inizio del trattamento in studio o che non si sono ripresi dagli effetti collaterali di tale terapia; 8.Pazienti sottoposti a intervento chirurgico maggiore nei 14 giorni precedenti l’inizio del trattamento in studio che non si sono ripresi dagli effetti collaterali di tale procedura; 9.Embolizzazione dell’arteria epatica o crioablazione o ablazione con radiofrequenza di metastasi epatiche nei 2 mesi precedenti l’arruolamento; 10.Pazienti che presentano un’altra neoplasia o hanno avuto una neoplasia nei 3 anni precedenti l’arruolamento nello studio; 11.Anamnesi positiva/evidenza di cardiopatia grave e/o non controllata che può compromettere la partecipazione del paziente allo studio; 12.Pazienti con ipertensione arteriosa non adeguatamente controllata; 13.Pazienti con diabete mellito non controllato; 14.Pazienti con compromissione della funzionalità gastrointestinale o con gastropatie che possono alterare significativamente l’assorbimento di BEZ235; 15.Pazienti che sono stati sottoposti a terapia con dosi elevate di corticosteroidi sistemici o altri farmaci immunosoppressori all’inizio del trattamento in studio; 17.Consumo di arance di Siviglia (arance amare), pompelmo, ibridi del pompelmo, pomelo e altri frutti esotici del genere Citrus durante i 7 giorni precedenti l’inizio del trattamento in studio.Il succo d’arancia è consentito; 18.Pazienti immunocompromessi compresi i pazienti con sieropositività nota per infezione da HIV; 19.Pazienti con diarrea di Grado >= 2.20. Pazienti con altra condizione clinica concomitante grave e/o non controllata che, secondo l’opinione dello sperimentatore, possa controindicare la partecipazione del paziente allo studio clinico. Per maggiori informazioni vedere par. 5.3 del protocollo.

E.5 End points

E.5.1

Primary end point(s)

Progression free survival based on investigator assessment

Sopravvivenza libera da progressione basata sulla valutazione dello sperimentatore

E.5.1.1

Timepoint(s) of evaluation of this end point

up to approx 18 months

Fino a circa 18 mesi

E.5.2

Secondary end point(s)

Frequency and severity of adverse events;other safety data as considered appropriate; - Objective Response Rate (best overall response); - Overall survival; - Time to treatment failure

- Incidenza e gravità degli eventi avversi; altri dati di sicurezza d’impiego ritenuti appropriati; - Tasso di risposta obiettiva (miglior risposta globale); - Sopravvivenza globale; - Tempo al fallimento del trattamento.

E.5.2.1

Timepoint(s) of evaluation of this end point

Up to approx 18 months for all secondary endpoint except for overall survival up to approx 30 months

Fino a circa 18 mesi per tutti gli endpoint secondari ad eccezione della sopravvivenza globale fino a circa 30 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

Yes

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

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

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

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

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

Korea, Republic of

New Zealand

Russian Federation

Switzerland

United States

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

The end of study (last patient last visit) will occur after all patients have completed their last assessment as per protocol. The final analysis will be conducted after all enrolled patients have completed the study

La fine dello studio avverrà dopo che tutti i pazienti abbiano completato la loro ultima valutazione prevista dal protocollo. L'analisi finale sarà condotta dopo che tutti i pazienti arruolati avranno completato lo studio.

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

Number of subjects for this age range:

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:

110

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

140

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)

After discontinuing study treatment further treatment is left to the physician's discretion, no cross over to the BEZ235 arm will be allowed.

Sarà discrezione del medico decidere la terapia successiva alla sospensione del trattamento in studio. Non è consentito il cross-over al braccio BEZ235.

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

2012-09-24

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-09-06

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2014-08-19

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