Clinical Trials Register

Summary
EudraCT Number:	2009-010548-32
Sponsor's Protocol Code Number:	CLBH589H2101
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:	2012-03-06
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

2009-010548-32

A.3

Full title of the trial

A phase Ib/IIb, open-label, multi-center study of oral Panobinostat (LBH589) administered with 5-Azacitidine (Vidaza) in adult patients with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) or acute myeloid leukemia (AML)

Studio di Fase Ib/IIb, in aperto, multicentrico, con Panobinostat (LBH589) somministrato per via orale in associazione a 5-Azacitidina (Vidaza) in pazienti adulti con sindromi mielodisplastiche (MDS), leucemia mielomonocitica cronica (CMML) o leucemia mieloide acuta(AML)

A.3.1

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

A.4.1

Sponsor's protocol code number

CLBH589H2101

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT00946647

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	+39 02 96541
B.5.5	Fax number	+39 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	panobinostat
D.3.2	Product code	LBH589
D.3.4	Pharmaceutical form	Capsule, hard
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	PANOBINOSTAT
D.3.9.1	CAS number	404950-80-7
D.3.9.2	Current sponsor code	LBH589
D.3.9.4	EV Substance Code	SUB31049
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: 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	Yes
D.2.1.1.1	Trade name	VIDAZA*1FL 100MG
D.2.1.1.2	Name of the Marketing Authorisation holder	CELGENE EUROPE LIMITED
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/07/509 (AML); EU/3/01/084 (myelodysplastic)
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Powder for suspension for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	AZACITIDINE
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/l milligram(s)/litre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
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	panobinostat
D.3.2	Product code	LBH589
D.3.4	Pharmaceutical form	Capsule, hard
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	PANOBINOSTAT
D.3.9.1	CAS number	404950-80-7
D.3.9.2	Current sponsor code	LBH589
D.3.9.4	EV Substance Code	SUB31049
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	20
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

Myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia(CMML), or acute myeloid leukemia (AML)

Sindromi mielodisplastiche (MDS), leucemia mielomonocitica cronica (CMML) o leucemia mieloide acuta (AML)

E.1.1.1

Medical condition in easily understood language

Myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia(CMML), or acute myeloid leukemia (AML)

Sindromi mielodisplastiche (MDS), leucemia mielomonocitica cronica (CMML) o leucemia mieloide acuta (AML)

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	14.1
E.1.2	Level	PT
E.1.2	Classification code	10028533
E.1.2	Term	Myelodysplastic syndrome
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	14.1
E.1.2	Level	LLT
E.1.2	Classification code	10000886
E.1.2	Term	Acute myeloid leukemia
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	14.1
E.1.2	Level	PT
E.1.2	Classification code	10009018
E.1.2	Term	Chronic myelomonocytic leukaemia
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

As of amendment 2, primary objective of the phase Ib has been met. The primary objective of the Phase IIb part is to assess preliminary efficacy of treatment with the panobinostat and 5-Aza combination at RPIID relative to treatment with single agent 5-Aza through the assessment of composite CR (CR or CRi or bone marrow CR)

Con l'emendamento 2, Valutare l’efficacia preliminare del trattamento con l’associazione di panobinostat e 5-Aza alla RPIID rispetto al trattamento con 5-Aza in monoterapia mediante la determinazione della CR composita (CR o Cri o CR del midollo osseo).

E.2.2

Secondary objectives of the trial

•To assess preliminary efficacy of treatment with the panobinostat and 5-Aza combination at RPIID relative to treatment with single agent 5- Aza through the assessment of clinical response other than the composite CR specified in the primary objective, 1-year survival, and time to progression (TTP); •To characterize the safety and tolerability of panobinostat at RPIID in combination with 5-AZA, as well as, 5-AZA alone in the target patient population.

- Valutare l’efficacia preliminare del trattamento con l’associazione di panobinostat e 5-Aza alla RPIID rispetto al trattamento con 5-Aza in monoterapia mediante la determinazione della risposta clinica diversa dalla CR composita specificata nell’obiettivo primario, sopravvivenza a 1 anno e tempo alla progressione (TTP); - Caratterizzare la sicurezza d’impiego e la tollerabilità di panobinostat alla RPIID somministrato in associazione a 5-Aza e di 5-Aza in monoterapia, nelle popolazioni target di pazienti.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Adult patients (age ≥ 18 years) who are candidates for treatment with 5-Aza and present with one of the following: • intermediate-2 or high-risk myelodysplastic syndromes according to the International Prognostic Scoring System (IPSS). OR • AML with multilineage dysplasia and maximum of 30% blasts (former RAEB-T according to FAB) OR • chronic myelomonocytic leukemia (CMML) • ECOG performance status ≤ 2 • Patients must have the following laboratory values unless elevations are considered due to MDS or leukemia: AST/SGOT and/or ALT/SGPT ≤ 2.5 x ULN; serum creatinine ≤ 1.5 x ULN; serum bilirubin (total and direct) ≤ 2 x ULN; electrolyte panel within normal ranges (WNL) for the institution. • Negative pregnancy test • Clinically euthyroid (hypothyroidism corrected with supplementation is permitted). • Written informed consent obtained prior to any screening procedures

1. Pazienti adulti (età > 18 anni) che sono candidati al trattamento con 5-Aza e che presentano uno dei riscontri seguenti: Sindromi mielodisplastiche a rischio intermedio-2 o ad alto rischio in base all’International Prognostic Scoring Systema (IPSS), OPPURE AML con displasia multilinea e un massimo di 30% di blasti (precedente RAEB-T in base a FAB), OPPURE Leucemia mielomonocitica cronica (CMML); 2. ECOG performance status ≤ 2; 3. I pazienti devono presentare i seguenti valori di laboratorio a meno che gli innalzamenti siano considerati dovuti alla MDS o alla leucemia. AST//SGOT e/o ALT/SGPT ≤ 2,5 x ULN; creatininemia ≤ 1,5 x ULN; bilirubinemia (totale e diretta) ≤2 x ULN; profilo elettrolitico entro i limiti della norma (WNL) per il laboratorio; 4. Test di gravidanza negativo; 5. Condizione di eutiroidismo clinico (è consentito l’ipotiroidismo corretto con supplementi); 6. Consenso informato scritto ottenuto prima di qualsiasi procedura di screening.

E.4

Principal exclusion criteria

• Planned hematopoietic stem-cell transplantation (HSCT) • Patients with therapy-related MDS • Patients with therapy-related AML and/or relapsed/refractory AML • Clinical symptoms suggesting CNS leukemia • Concurrent therapy with any other investigational agent • Prior treatment with deacetylase inhibitor(s) • Prior treatment with 5-Azacytidine or 5-aza-2'-deoxycytidine • Time windows for prior therapies: Last dose of therapy, including cytokines and/or retinoids, immunotherapy, low-dose ara-C, investigational agent less than 28 days with the exception of hydroxyurea (24 hours) prior to receipt of study medication or AEs that have not recovered at least to NCI CTCAE Grade 1. • Patients with impaired cardiac function including any of the following: • Complete left bundle branch block or use of a permanent cardiac pacemaker, congenital long QT syndrome, history or presence of ventricular tachyarrhythmia, clinically significant resting bradycardia (<50 beats per minute), QTcF > 460 ms on screening ECG, or right bundle branch block + left anterior hemiblock (bifascicular block) • Presence of unstable atrial fibrillation (ventricular response rate >100 bpm). Patients with stable atrial fibrillation are eligible provided they do not meet the other cardiac exclusion criteria • Previous history of angina pectoris or acute MI within 6 months • Screening LVEF <45% by echocardiography or MUGA • Other clinically significant heart disease (e.g. uncontrolled hypertension or history of poor compliance with an antihypertensive regimen). • Drugs which may cause QT prolongation and the treatment cannot be discontinued or switched to a different medication prior to starting study drug. • Any of concurrent severe and/or uncontrolled medical conditions which could compromise participation in the study. For example: • Uncontrolled diabetes • Active or uncontrolled infection • Uncontrolled hypothyroidism • Acute or chronic liver or renal disease • Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral panobinostat (e.g., ulcerative diseases, diarrhea, malabsorption syndrome, or small bowel resection). • HIV, Hepatitis B/C infection according to the medical history (testing will not be performed). • Female patients who are pregnant or breast feeding or patients of childbearing potential (WOCBP) not willing to use a double barrier method of contraception during the study and for 3 months following the last dose of study drug. • Male patients whose sexual partner(s) are WOCBP who are not willing to use a double barrier method of contraception, one of which includes a condom, during the study and for 3 months after the end of treatment. • Suspected hypersensitivity to 5-Aza or Mannitol • Inability to swallow capsules • Unwilling or unable to comply with the protocol • Patient has evidence of clinically significant mucosal or internal bleeding

1. Trapianto di cellule staminali ematopoietiche (HSCT) pianificato;2. Pazienti con MDS correlata alla terapia. 3. Pazienti con AML correlata alla terapia e/o AML in recidiva/refrattaria;4. Sintomi clinici suggestivi di leucemia del SNC;5. Terapia concomitante con qualsiasi altro farmaco sperimentale;6. Terapia precedente con inibitore(i) della deacetilasi;7. Terapia precedente con 5-Azacitidina o 5-aza-2’-deossicitidina;8. Intervallo di tempo per le terapie precedenti: ultima dose di terapia, comprese citochine e/o retinoidi, immunoterapia, basse dosi di ara-C, farmaci sperimentali, con l’eccezione di idrossiurea (24 ore): inferiore a 28 giorni dall’inizio della somministrazione del trattamento in studio o presenza di eventi avversi (AE) che non si sono risolti almeno a NCI CTCAE Grado 1;9. Pazienti con compromissione della funzionalità cardiaca compresi uno qualsiasi dei riscontri seguenti:Blocco di branca sinistra completo o impiego di pacemaker cardiaco permanente, sindrome del QT lungo congenita, evidenza pregressa o attuale di tachiaritmia ventricolare, bradicardia a riposo clinicamente rilevante (< 50 battiti per minuto), QTcF > 460 ms all’ECG di screening o blocco di branca destro + emiblocco anteriore sinistro (blocco bifascicolare) Presenza di fibrillazione atriale instabile (frequenza di risposta ventricolare > 100 battiti/minuto). I pazienti con fibrillazione atriale stabile sono eleggibili ammesso che non presentino gli altri criteri di esclusione cardiaci Evidenza pregressa di angina pectoris o infarto miocardico acuto entro i 6 mesi precedenti LVEF allo screening < 45% mediante ecocardiografia o MUGA Altre cardiopatie clinicamente rilevanti (ad es: ipertensione arteriosa non controllata o anamnesi positiva per scarsa aderenza al trattamento antipertensivo);10. Farmaci che possono determinare prolungamento del QT e che non possono essere sospesi o sostituiti con un altro farmaco prima dell’inizio della somministrazione del trattamento in studio;11. Qualsiasi patologia concomitante grave e/o non controllata che possa compromettere la partecipazione allo studio;12. Compromissione della funzionalità gastrointestinale (GI) o patologia gastrointestinale che possa alterare significativamente l’assorbimento di panobinostat orale (ad es: patologia ulcerosa, diarrea, sindrome da malassorbimento o resezione dell’intestino tenue);13. Infezioni da HIV, epatite B/C in base all’anamnesi (non saranno eseguiti i test);14. Donne in gravidanza o allattamento o donne potenzialmente fertili che non desiderano impiegare un metodo contraccettivo di doppia barriera durante lo studio e nei 3 mesi successivi all’ultima somministrazione del trattamento in studio;15. Pazienti di sesso maschile, con partner potenzialmente fertili, che non desiderano impiegare un metodo contraccettivo di doppia barriera, uno dei quali deve essere il preservativo, durante lo studio e nei 3 mesi successivi alla fine del trattamento;16. Sospetta ipersensibilità a 5-Aza o Mannitolo;17. Pazienti che non possono deglutire le capsule;18. Incapacità o non volontà di aderire al protocollo;19. Pazienti con evidenza di sanguinamento delle mucose o sanguinamento interno clinicamente rilevante.

E.5 End points

E.5.1

Primary end point(s)

As of amendment 2, primary endpoint is Composite CR (CR or CRi or bone marrow CR)

Con l'emendamento 2, CR composita (CR o Cri o CR del midollo osseo)

E.5.1.1

Timepoint(s) of evaluation of this end point

Every 8 weeks and at end of trial

Ogni 8 settimane e alla fine dello studio

E.5.2

Secondary end point(s)

•Clinical response for AML: PR; for MDS/CMML: PR and Hematologic Improvement (HI) •Overall response (CR or CRi or bone marrow CR or PR) •1-year survival •Time to progression (TTP) based on the Guidelines for Implementation of IWG response criteria in AML, MDS and CMML according to Cheson 2003 and 2006 Post-text supplement 1 •Type, duration, frequency and relatedness of Adverse Events (AE). AE severity will be assessed according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 3.0) •Laboratory (biochemistry, hematology) •ECG monitoring (central review by eRT)

- Risposta clinica per AML: PR; per MDS/CMML: PR e miglioramento ematologico (HI). - Risposta globale (CR or CRi o CR del midollo osseo o PR). - Sopravvivenza a 1 anno. - Tempo alla progressione (TTP) in base alle linee-guida per l’implementazione dei criteri di risposta IWG nelle AML, MDS e CMML in base a Cheson 2003 e 2006 (Post-text supplement 1). - Tipo, durata, frequenza e correlazione degli eventi avversi (AE). Inoltre, la gravità degli AE sarà stabilita in base al National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 3.0). - Esami di laboratorio (biochimica, ematologia) - Monitoraggio ECG (revisione centralizzata mediante eRT)

E.5.2.1

Timepoint(s) of evaluation of this end point

As per visit schedules

Come programmato per visita

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Explore if gene mutation status of specific target genes known are associated with outcome

Esplorare se lo status di mutazione di specifici geni bersaglio è associato con l'esito

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

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

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

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

LVLS

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:

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

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)

As clinical practice and followed for the evaluation of survival

Come da pratica clinica e seguiti per la valutazione della sopravvivenza

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

2012-05-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-02-06

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2019-04-29

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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