Clinical Trials Register

Summary
EudraCT Number:	2015-002354-12
Sponsor's Protocol Code Number:	CMBG453X2101
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2018-08-20
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

2015-002354-12

A.3

Full title of the trial

A phase I-Ib/II, open-label, multi-center study of the safety and efficacy of MBG453 as single agent and in combination with PDR001 in adult patients with advanced malignancies

Studio di Fase I-Ib/II, in aperto, multicentrico, sulla sicurezza d’impiego e l’efficacia di MBG453 in monoterapia e in associazione a PDR001, somministrato in pazienti adulti con tumori in stadio avanzato

A.3.1

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

An interventional study of MBG453 alone and in combination with PDR001 in patients with advanced malignancies

Studio di Fase I-Ib/II, con MBG453 in monoterapia e in associazione a PDR001 in pazienti con tumori in stadio avanzato.

A.3.2

Name or abbreviated title of the trial where available

An interventional study of MBG453 alone and in combination with PDR001 in patients with advanced mal

Studio di Fase I-Ib/II, con MBG453 in monoterapia e in associazione a PDR001 in pazienti con tumori

A.4.1

Sponsor's protocol code number

CMBG453X2101

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 S.P.A.
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 S.p.A.
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	0296541
B.5.5	Fax number	029659066
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	MBG453
D.3.2	Product code	MBG453
D.3.4	Pharmaceutical form	Concentrate 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.3.9.2	Current sponsor code	MBG453
D.3.9.4	EV Substance Code	SUB178459
D.3.10	Strength
D.3.10.1	Concentration unit	mg/kg milligram(s)/kilogram
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	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
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	PDR001
D.3.2	Product code	PDR001
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.3.9.2	Current sponsor code	PDR001
D.3.9.4	EV Substance Code	SUB171710
D.3.10	Strength
D.3.10.1	Concentration unit	mg/kg milligram(s)/kilogram
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	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
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

Solid tumors

tumori solidi

E.1.1.1

Medical condition in easily understood language

Solid tumors

tumori solidi

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	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10053571
E.1.2	Term	Melanoma
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	20.1
E.1.2	Level	PT
E.1.2	Classification code	10073251
E.1.2	Term	Clear cell renal cell carcinoma
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	20.0
E.1.2	Level	HLT
E.1.2	Classification code	10029111
E.1.2	Term	Neoplasms unspecified malignancy and site unspecified NEC
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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10059515
E.1.2	Term	Non-small cell lung cancer metastatic
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

Phase I/Ib part
● To characterize the safety and tolerability of MBG453 single agent and in combination with PDR001 and to identify recommended doses for future studies
Phase I-Ib dose ranging part
● To further investigate the safety and tolerability of different doses of MBG453 alone or in combination with PDR001
Phase II part
● To estimate anti-tumor activity of MBG453 alone and in combination with PDR001

Parte di Fase I-Ib: Caratterizzare la sicurezza di impiego e la tollerabilità di MBG453 in monoterapia e in associazione a PDR001 e identificare le dosi raccomandate per gli studi futuri.
Fase I-Ib valutazione della dose: Valutare ulteriormente la sicurezza di impiego e la tollerabilità di dosi differenti di MBG453 in monoterapia o in associazione a PDR001
Fase II: Valutare l’attività antitumorale di MBG453 in monoterapia e in associazione a PDR001.

E.2.2

Secondary objectives of the trial

Phase I-Ib/II part
● To evaluate the preliminary ant-tumor activity of MBG453 single agent and in combination with PDR001
● To characterize the pharmacokinetic profile of MBG453 single agent and in combination with PDR001
● To assess emergence of anti-MBG453 and anti-PDR001 antibodies following one or more i.v. infusions of MBG453 single agent and in combination with PDR001
● To assess potential predictors of efficacy of MBG453 single agent and in combination with PDR001 in tumor sample
● To assess the pharmacodynamic effect of MBG453 single agent and in combination with PDR001 in tumor sample
● To describe the survival distribution of patients treated with MBG453 single agent and in combination with PDR001 for each disease group
Phase II (combination):
● To make an initial comparison for MBG453 and PDR001 administered in combination on a Q2W and Q4W dosing schedules.

Parte di Fase I/Ib/II:
-Valutare l’attività antitumorale preliminare di MBG453 in monoterapia e in associazione a PDR001.
-Caratterizzare il profilo PK di MBG453 in monoterapia e in associazione a PDR001.
-Valutare l’insorgenza di anticorpi anti-MBG453 e anti-PDR001.
-Valutare i possibili fattori predittivi dell’efficacia di MBG453 in monoterapia e dell’associazione di MBG453 e PDR001 in campioni tumorali.
-Valutare l’effetto PD di MBG453 in monoterapia e in associazione a PDR001 in campioni tumorali.
-Descrivere la distribuzione della sopravvivenza dei pazienti trattati con MBG453 in monoterapia e in associazione a PDR001 per ciascun gruppo di malattia.
Fase II (associazione):
-Effettuare un confronto iniziale di MBG453 e PDR001, in associazione secondo uno schema Q2W e Q4W.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Histologically documented advanced or metastatic solid tumors.
2. Phase I-Ib part (including dose ranging part): Patients with advanced/metastatic solid tumors, with measurable or non-measurable disease as determined by RECIST v1.1, who have progressed despite standard therapy or are intolerant of standard therapy, or for whom no standard therapy exists and who did not receive prior anti-PD1/PD-L1 treatment.
3. Phase II part (MBG453 single agent): Patients with advanced/metastatic solid tumors in the indication in which at least one confirmed PR or CR was seen during the dose escalation phase I part. Patients must have measurable disease as determined by RECIST v1.1, have progressed despite standard therapy or be intolerant to standard therapy.
4. Phase II part (MBG453 in combination PDR001): Patients with advanced/metastatic tumors in the below selected indications, with at least one measurable lesion as determined by RECIST v1.1, who have received standard therapy and are intolerant of standard therapy or have progressed following their last prior therapy.:
• Melanoma (anti-PD-1/PD-L1 therapy naïve or pre-treated)
• NSCLC (anti-PD-1/PD-L1 therapy naïve or pre-treated)
• RCC (anti-PD-1/PD-L1 therapy naïve or pre-treated)
5. Must have a site of disease amenable to biopsy, and be a candidate for tumor biopsy according to the treating institution’s guidelines. Patient must be willing to undergo a new tumor biopsy at screening/baseline, and during therapy on the study.
Other protocol-defined inclusion criteria may apply.

1.Conferma istologica di tumori solidi in stadio avanzato o metastatico.
2.Parte di Fase I-Ib (compresa la parte di valutazione della dose): Pazienti con tumori solidi in stadio avanzato/metastatico, con malattia misurabile o non misurabile, come determinato da RECIST versione 1.1, che hanno manifestato progressione nonostante terapia standard o sono intolleranti alla terapia standard o per i quali non esiste terapia standard e che non hanno ricevuto trattamento anti- PD1/PD-L1 precedente.
3. Parte di Fase II (MBG453 monoterapia): Pazienti con tumori solidi in stadio avanzato/metastatico, con patologia nella quale è stata osservata almeno una PR o una CR confermata durante la fase di incremento della dose nella parte di fase I. I pazienti devono avere malattia misurabile, come determinato da RECIST v1.1, devono aver manifestato progressione nonostante terapia standard o essere intolleranti alla terapia standard.
4. Parte di Fase II (MBG453 in associazione a PDR001): Pazienti con tumori in stadio avanzato/metastatico, nelle patologie selezionate indicate di seguito, con almeno una lesione misurabile come determinato da RECIST v1.1, che hanno ricevuto terapia standard e sono intolleranti alla terapia standard e hanno manifestato progressione dopo l’ultima terapia precedente:
-Melanoma (Naïve o precedentemente trattati con la terapia anti-PD-1/PD-L1 )
-NSCLC(Naïve o precedentemente trattati con la terapia anti-PD-1/PD-L1 )
-RCC (Naïve o precedentemente trattati con la terapia anti-PD-1/PD-L1 ).
5.Il paziente deve presentare una sede di malattia nella quale la biopsia è fattibile ed essere candidato alla biopsia tumorale eseguita in base alle linee guida del centro/ospedale. Il paziente deve essere disposto a sottoporsi a una nuova biopsia tumorale allo/al screening/basale e durante la terapia in questo studio.
Potrebbero essere applicati altri criteri di inclusione del protocollo definito.

E.4

Principal exclusion criteria

1. Presence of symptomatic central nervous system (CNS) metastases.
2. History of severe hypersensitivity reactions to other monoclonal antibodies (mAbs).
3. Human Immunodeficiency Virus (HIV), HBV (Hepatitis B Virus) or HCV (Hepatitis C Virus) infection.
4. Active autoimmune disease or a documented history of autoimmune disease, including ulcerative colitis and Crohn’s disease or any condition that requires systemic steroids.
5. Systemic steroid therapy or any immunosuppressive therapy (≥10mg/day prednisone or equivalent).
6. Use of any vaccines against infectious diseases (e.g. varicella, pneumococcus) within 4 weeks of initiation of study treatment.
7. Pre-treatment with anti-CTLA4 antibodies in combination with any other antibody or drug specifically targeting T-cell co-stimulation or checkpoint pathway.
8. Participation in an interventional, investigational non-immunotherapy study within 2 weeks of the first dose of study treatment.
9. Prior participation in an interventional, investigational cancer vaccine or immunotherapy study except for an anti-PD-1/PD-L1 study.
Other protocol-defined exclusion criteria may apply.

1. Presenza di una metastasi sintomatica al sistema nervoso centrale (SNC).
2. Anamnesi positiva per reazioni da ipersensibilità gravi ad altri anticorpi monoclonali (MAK, MAB).
3. Infezione da virus dell'Immunodeficienza Umana (HIV), da HBV (virus dell'epatite B) o da HCV (Virus dell'epatite C).
4. Malattia autoimmune in fase attiva o anamnesi documentata di malattia autoimmune, comprese colite ulcerosa e malattia di Crohn o qualsiasi condizione che richieda terapia sistemica con corticosteroidi.
5. Terapia corticosteroidea sistemica o qualsiasi terapia immunosoppressiva (≥ 10mg / die di prednisone o equivalente).
6. Impiego di qualsiasi vaccino per malattie infettive (ad esempio varicella, pneumococco) entro 4 settimane dall’inizio del trattamento in studio.
7. Trattamento precedente con anticorpi anti-CTLA4 in associazione a qualsiasi altro anticorpo o farmaco con target specifico per la co-stimolazione dei linfociti T o dei checkpoint.
8. Partecipazione a uno studio sperimentale interventistico (non immunoterapia) entro 2 settimane prima della prima dose del trattamento in studio.
9. Partecipazione precedente a uno studio interventistico sperimentale con un vaccino antitumorale o immunoterapia, a eccezione degli studi con anti-PD-1 o anti-PD-L1.
Si possono applicare altri criteri di esclusione dal protocollo.

E.5 End points

E.5.1

Primary end point(s)

Phase I/Ib parts:
• The incidence and severity of Aes and SAEs, including changes in
laboratory parameters, vital signs and ECGs
• Dose interruptions, reductions and dose intensity
• The incidence of DLTs during the first cycle of treatment with single
agent MBG453
• The incidence of DLTs during the first and second cycle of treatment
with MBG453 in combination with PDR001
Phase I-Ib dose ranging part:
• Incidence and severity of AEs and SAEs, including changes in
laboratory parameters, vital signs and ECGs
• Dose interruptions, reductions and dose intensity
Phase II part:
• Overall response rate (ORR) per RECIST v1.1

Parte di Fase I/Ib:
-Incidenza e gravità degli AEs e degli SAEs, comprese le alterazioni dei valori di laboratorio, dei segni vitali e degli ECG.
-Interruzione, riduzione e intensità della dose
-Incidenza di DLTs durante il primo ed il secondo ciclo di trattamento in monoterapia con MBG453
-Incidenza di DLTs durante il primo ed il secondo ciclo di trattamento con MBG453 in associazione a PDR001
Parte di Fase I/Ib valutazione della dose:
-Incidenza e gravità degli AEs e degli SAEs, comprese le alterazioni dei valori di laboratorio, dei segni vitali e degli ECG.
-Interruzione, riduzione e intensità della dose
Parte di Fase II:
-Tasso di risposta globale per RECIST v1.1

E.5.1.1

Timepoint(s) of evaluation of this end point

Phase I/Ib parts:
• Up to 90 days after last dose or longer
• During a treatment cycle (28 days)
• End of cycle 1
• End of cycle 1 and end of cycle 2
Phase I-Ib dose ranging part:
• Up to 90 days after last dose or longer
• During a treatment cycle (28 days)
Phase II part:
• Every 8 weeks until week 40, and then every 12 weeks until progression of disease

Parte di Fase I/Ib:
-fino a 90 giorni dopo l’ultima dose o più a lungo
-durante il ciclo di trattamento (28 giorni)
-fine del Ciclo 1
-Fine del ciclo 1 e fine del ciclo 2
Parte di Fase I/Ib valutazione della dose:
- fino a 90 giorni dopo l’ultima dose o più a lungo
-durante il ciclo di trattamento (28 giorni)
Parte di Fase II
-Ogni 8 settimane fino a 40 settimane, e poi ogni 12 settimane fino a progressione della malattia

E.5.2

Secondary end point(s)

• Best Overall Response (BOR), Progression Free Survival (PFS) and DOR per RECIST v1.1; ORR and PFS per irRC
• Serum PK parameters (e.g., AUC, Cmax, Tmax, half-life); Serum concentration vs. time profiles
• Presence and/or concentration of anti-MBG453 and anti-PDR001 antibodies
• Assess potential associations between expression of PD-L1 and other immunological markers such as, but not restricted to TIM-3, CD8, FoxP3 and anti-tumor activity
• Tumor Infiltrating Lymphocytes (TIL) counts and expression of immune-related genes (RNA/protein)
• Overall survival (OS)
Phase II part (combination):
• Incidence and severity of AEs and SAEs, including changes in laboratory parameters, vital signs and ECGs
• Dose interruptions, reductions and dose intensity
• Serum PK parameters (e.g., AUC, Cmax, Tmax, half-life); Serum concentration vs. time profiles
• ORR, BOR, PFS and DOR per RECIST v1.1; ORR and PFS per irRC

Parte di Fase I/Ib:
-Migliore risposta globale, sopravvivenza libera da progressione e DOR per RECIST v1.1; tasso di risposta globale e sopravvivenza libera da progressione per irRC
-paramentri PK nel siero (esempio AUC, Cmax, Tmax, emivita); concentrazione sierica vs. profili temporali
-presenza e/i concentrazione di anticorpi anti-MBG453 e anti-PDR001
-Valutare associazioni potenziali tra espressione di PD-L1 e altri marcatori immunologici quali, ma non delimitate a TIM-3, CD8, FoxP3 e l'attività antitumorale
-conta dei linfociti infiltranti il tumore (TIL) e di espressione di geni immuno-correlati (RNA / proteine)
-La sopravvivenza globale (OS)
Fase II parte (associazione):
-L'incidenza e la gravità di Aes e di SAEs, comprese le alterazioni dei valori di laboratorio, dei segni vitali e degli ECG.
- Interruzione, riduzione e intensità della dose
-I parametri farmacocinetici siero (ad esempio, l'AUC, Cmax, Tmax, tempo di dimezzamento); Siero concentrazione vs. profili temporali
-Tasso di risposta globale, Migliore risposta globale, sopravvivenza libera da progressione e DOR per RECIST v1.1; Tasso di risposta globale e sopravvivenza libera da progressione per irRC.

E.5.2.1

Timepoint(s) of evaluation of this end point

Phase I/Ib parts:
• Every 8 weeks until week 40, and then every 12 weeks until progression of disease
• Cycle 1 and Cycle 3 Days 1,2,8,11 and 15. Cycles 2, 3, 4, 5 and 6 on
Day 1, and end of treatment
• Cycles 1 to 6 on Day 1 and end of treatment
• Screening and after Cycle 3 Day 1
• Screening and after Cycle 3 Day 1
• From time of start treatment until the date of death
Phase II part (combination):
• Up to 90 days after last dose or longer
• During a treatment cycle (28 days)
• Cycle 1 and Cycle 3 Days 1,2,8,11 and 15. Cycles 2, 3, 4, 5 and 6 on Day 1, and end of treatment
• Every 8 weeks until week

Parte di Fase I/Ib:
-ogni 8 settimane fino alla settimana 40, e poi ogni 12 settimane fino a progressione della malattia.
-Ciclo 1 e Ciclo 3 Giorno 1,2,8,11 e 15. Ciclo 2,3,4,5 e 6 al Giorno 1, e alla fine del trattamento.
-Ciclo 1 a 6 al Giorno 1 e alla fine del trattamento
-screening e dopo Ciclo 3 Giorno 1
-Screening e dopo Ciclo 3 Giorno 1
-dall’inizio del trattamento fino alla data di morte
Parte di Fase II (associazione):
-fino a 90 giorni dopo l’ultima dose o più a lungo
-durante il ciclo di trattamento (28 giorni)
-Ciclo 1 e Ciclo 3 Giorno 1,2,8,11 e 15. Ciclo 2,3,4,5 e 6 al Giorno 1 e alla fine del trattamento.
-Ogni 8 settimane fino alla settimana 40, e poi ogni 12 settimane fino a progressione della malattia

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

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

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)

Yes

E.7.1.1

First administration to humans

Yes

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

E.8.1.1

Randomised

E.8.1.2

Open

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)

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

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

Canada

Germany

Italy

Korea, Republic of

Netherlands

Singapore

Switzerland

Taiwan

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 the study will be when at least 80% of the patients have completed the survival follow-up period, or discontinued the study for any reason, and all patients have completed treatment as well as the 90 day safety follow-up period, or if the study is terminated early.

La fine dello studio sarà quando almeno l'80% dei pazienti ha completato il follow-up per il periodo di sopravvivenza, o per interruzione dello studio per qualsiasi motivo, e tutti i pazienti hanno completato il trattamento e il periodo di follow-up di sicurezza di 90 giorni, o lo studio termina in anticipo.

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

Information not present in EudraCT

F.1.1.2

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

Information not present in EudraCT

F.1.1.3

Newborns (0-27 days)

Information not present in EudraCT

F.1.1.4

Infants and toddlers (28 days-23 months)

Information not present in EudraCT

F.1.1.5

Children (2-11years)

Information not present in EudraCT

F.1.1.6

Adolescents (12-17 years)

Information not present in EudraCT

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

175

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

Yes

F.3.3.2

Women of child-bearing potential using contraception

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

100

F.4.2.2

In the whole clinical trial

250

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)

None

nessuno

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

2016-04-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-01-20

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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IMP with orphan designation in the indication
Orphan Designation Number:
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