Clinical Trials Register

Summary
EudraCT Number:	2011-004378-27
Sponsor's Protocol Code Number:	CAFQ056A2299
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:	2012-03-02
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

2011-004378-27

A.3

Full title of the trial

An open-label treatment study to evaluate the safety, tolerability and efficacy of AFQ056 in Parkinson's patients with L-dopa induced dyskinesias

Studio in aperto per valutare la sicurezza, la tollerabilita' e l'™efficacia di AFQ056 in pazienti affetti da malattia di Parkinson con discinesie indotte da L-dopa

A.3.1

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

Open-label, Long-term Safety Extension Study of AFQ056 in Parkinson's Patients With L-dopa Induced Dyskinesias

Studio di estensione, in aperto, a lungo termine, di sicurezza, tollerabilita' e efficacia in pazienti affetti da malattia di Parkinson con discinesie indotte da L-dopa

A.4.1

Sponsor's protocol code number

CAFQ056A2299

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01491932

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
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	NA
D.3.2	Product code	AFQ056
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	mavoglurant
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	NA
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	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: 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	NA
D.3.2	Product code	AFQ056
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	mavoglurant
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	NA
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	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	NA
D.3.2	Product code	AFQ056
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	mavoglurant
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	NA
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	75
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	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	NA
D.3.2	Product code	AFQ056
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	mavoglurant
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.9.3	Other descriptive name	NA
D.3.9.4	EV Substance Code	NA
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	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

L-dopa induced dyskinesias in patients with Parkinson's disease

Pazienti affetti da malattia di Parkinson con discinesie indotte da L-dopa

E.1.1.1

Medical condition in easily understood language

Parkinson's Patients With L-dopa Induced Dyskinesias

Pazienti con Parkinson con discinesie indotte da L-dopa

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.1
E.1.2	Level	HLT
E.1.2	Classification code	10013929
E.1.2	Term	Dyskinesias and movement disorders NEC
E.1.2	System Organ Class	10029205 - Nervous system disorders

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 evaluate the long-term safety and tolerability of AFQ056 in patients with PD-LID as assessed by •Incidence and severity of adverse events and serious adverse events •Changes in vital signs, laboratory assessments, and ECGs •Changes in underlying symptoms of PD as measured by the UPDRS (Unified Parkinson's Disease Rating Scale) part III (Motor Examination) and AEs potentially related to an exacerbation of the movement disorder of PD

Valutare la sicurezza e la tollerabilità a lungo termine di AFQ056 in pazienti con PD-LID definite tramite: • Incidenza e severità degli eventi avversi e degli eventi avversi seri • Variazioni nei segni vitali, nelle valutazioni di laboratorio e negli elettrocardiogrammi • Variazioni nei sintomi sottostanti della malattia di Parkinson, misurati tramite o Scala UPDRS (Unified Parkinson’s Disease Rating Scale) parte III (Esame motorio) o Eventi avversi potenzialmente correlati ad una esacerbazione del disordine di movimento della malattia di Parkinson

E.2.2

Secondary objectives of the trial

To evaluate the anti-dyskinetic efficacy of AFQ056 treatment in patients with PD-LID on dyskinesia as assessed by •mAIMS (modified Abnormal Involuntary Movement Scale) total score •the Revised Lang-Fahn Activities of Daily Living Dyskinesia Scale (LFADLDS) patient and caregiver versions •items 32, 33 and 34 of Part IV of the UPDRS

Valutare l’efficacia anti-discinetica del trattamento con AFQ056 in pazienti con discinesie indotte da Ldopa definita tramite : • Punteggio totale della scala mAIMS (modified Abnormal Involuntary Movement Scale) • Scala Revised Lang-Fahn Activities of Daily Living Dyskinesia Scale (LFADLDS) versione del paziente e del caregiver • Item 32, 33 e 34 della UPDRS Parte IV.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Patients who have completed a previous AFQ056A study or are eligible as defined in the core study protocol - Outpatients - Patients who have a primary caregiver willing and able to assess the condition of the patient throughout the study in accordance with protocol requirements Other protocol-defined inclusion criteria may apply

pazienti eleggibili per l’inclusione in questo studio devono soddisfare tutti i seguenti criteri: • Pazienti che hanno completato lo studio principale o che sono eleggibili secondo quanto definito nel protocollo dello studio principale • Maschi e femmine • Pazienti ambulatoriali • Consenso informato firmato prima che venga effettuata qualsiasi valutazione e prima che venga assunto il farmaco in studio

E.4

Principal exclusion criteria

-Atypical or secondary form of Parkinson's disease -History of surgical treatment for PD including deep brain stimulation -Advanced, severe, or unstable disease (other than PD) -History of malignancy -Evidence of dementia -Untreated/ineffectively treated mental disorders -Treatment with certain prohibited medications -Abnormal lab values or heart abnormalities -Pregnant or nursing women Other protocol-defined exclusion criteria may apply

I pazienti che soddisfano uno qualsiasi dei seguenti criteri al momento della prima visita (Visita di Screening per i pazienti del Gruppo 2 e Visita Basale per i pazienti del Gruppo 1, rispettivamente, a meno che non venga specificata un’altra visita) non saranno eleggibili per l’inclusione nel presente studio. • Malattia di Parkinson di forma atipica o secondaria • Storia di trattamento chirurgico per la PD, compresa la stimolazione cerebrale profonda • Punteggio 5 nella fase ON nella scala Modified Hoehn and Yahr Staging • Qualsiasi patologia (diversa dalla PD) avanzata, severa o instabile • Evidenza di demenza (o MMSE ≤ 26) • Disordine depressivo maggiore non trattato o trattato in modo non efficace; pazienti che attualmente manifestano allucinazioni/psicosi che richiedono trattamento anti-psicotico, e/o stati confusionali (DSM-IVR, Diagnostic and Statistical Manual of Mental Disorders, 4° edizione, revisionata) • Trattamento con uno qualsiasi dei seguenti: o forti o moderati inibitori di CYP3A4 entro 1 settimana prima della visita basale o forti o moderati induttori di CYP3A4 entro 1 settimana prima della visita basale o warfarina o digossina entro 1 settimana prima della visita basale o amantadina entro 3 giorni prima della visita basale o metoclopramide entro 3 giorni prima della visita basale o agenti neurolettici tipici entro 1 settimana prima della visita basale Per maggiori dettagli consultare i paragrafi 4.1 e 4.2 del protocollo originale.

E.5 End points

E.5.1

Primary end point(s)

a)Incidence rate of adverse events including serious adverse events b)Severity of adverse events including serious adverse events c)Change in vital signs d)Changes in hematology/blood chemistry and urinalysis laboratory evaluations e)Change in ECGs f)Change in Unified Parkinson's Disease Rating Scale (UPDRS) part III scores g)Incidence of AEs to an exacerbation of the underlying movement disorder Parkinson's disease

a)Tasso di incidenza di eventi avversi compreso gli eventi avversi seri b)Severità degli eventi avversi compresi gli eventi avversi seri c)Cambiamenti nei segni vitali d)Cambiamenti nelle valutazioni di laboratorio (ematologia, biochimica, analisi delle urine...) e)Cambiamenti nell'ECGs f)Cambiamenti nella''Unified Parkinson's Disease Rating Scale (UPDRS) part III scores'' g)Tasso d'incidenza degli AEs riferiti ad una escerbazione dei disordini dei movimenti nella malattia di Parkinson

E.5.1.1

Timepoint(s) of evaluation of this end point

a) and b)Monitored for the duration of the study (anticipated to be an average of 3 years) c)Assessed at Day -14 to -3, Day 1, Weeks 1, 2, 4, 8, 12, Months 6, 9, 12, every 6 months thereafter. If a patient discontinues in between these visits, this will be assessed at the time of discontinuation. d) and e)Assessed at Day -14 to -3, Day 1,Weeks 4, 8, 12, Months 6, 9, 12, every 6 months thereafter. If a patient discontinues in between these visits, these will be assessed at the time of discontinuation. f)Assessed at Day 1, Weeks 4, 8, 12, Months 6, 9, 12, every 6 months thereafter. If a patient discontinues in between these visits, this will be assessed at the time of discontinuation. g)Monitored for the duration of the study (anticipated to be an average of 3 years)

a) e b)Controllato per tutta la durata dello studio (circa 3 anni) c)Valutato dal Giorno -14 al -3, G 1, Settimane 1, 2, 4, 8, 12, Mesi 6, 9, 12, ogni 6 mesi quindi. Se un paziente discontinuasse fra queste visite, la valutazione è prevista al momento della discontinuazione. d)ed e)valutazione dal Giorno -14 al -3, Giorno 1,Settimane 4, 8, 12, Mesi 6, 9,12, ogni 6 mesi quindi. Se un paziente discontinuasse fra queste visite, la valutazione è prevista al momento della discontinuazione. f)Valutazione al Giorno 1, Settimane 4, 8, 12, Mesi 6, 9, 12, ogni 6 mesi quindi. Se un paziente discontinuasse fra queste visite, la valutazione é prevista al momento della discontinuazione. g)Controllato per tutta la durata dello studio (previsto per circa 3 anni)

E.5.2

Secondary end point(s)

a)Change in mAIMS (modified Abnormal Involuntary Movement Scale) total score b)Change in Revised Lang-Fahn Activities of Daily Living Dyskinesia Scale (LFADLDS) scores (patient and caregiver versions) c)Change in score for items 32, 33, and 34 of Part IV of the UPDRS d)Change in Mini Mental State Exam (MMSE) score e)Change in the Scales for outcomes in Parkinson's disease – Psychiatric Complications (SCOPA-PC) score f)Proportion of patients who have suicidal ideation and behavior as mapped to Columbia Classification Algorithm for Suicide assessment (CCASA) using data from Columbia-Suicide Severity Rating Scale (C-SSRS)

a)Cambiamenti nella mAIMS (modified Abnormal Involuntary Movement Scale) total score b)Cambiamenti nella Revised Lang-Fahn Activities of Daily Living Dyskinesia Scale (LFADLDS) scores (versione paziente e caregiver) c)Cambiamenti nella ''score per items 32, 33, e 34 della Part IV della UPDRS d)Cambiamenti nel''Mini Mental State Exam (MMSE) score e)Cambiamenti nelle ''Scales for outcomes in Parkinson's disease – Psychiatric Complications (SCOPA-PC) score'' f)Percentuale di pazienti che hanno ideazione e comportamento suicidi come tracciato nella ''CCASA'' utilizando dati dalla ''Columbia-Suicide Severity Rating Scale (C-SSRS)''

E.5.2.1

Timepoint(s) of evaluation of this end point

a)Assessed at Day 1, Weeks 1, 2, 4, 8, 12, Months 6, 9, 12, every 6 months thereafter. b)Assessed at Day 1, Weeks 4, 12, Months 6, 9, 12, every 6 months thereafter. c) and e)Assessed at Day 1, Weeks 4, 8, 12, Months 6, 9, 12, every 6 months thereafter. d)Assessed at Day -14 to -3, Day 1 (only if not done in the respective core study), Months 6, 12, every 6 months thereafter. a), b), c), d) and e) If a patient discontinues in between these visits, this will be assessed at the time of discontinuation. f)monitored for the duration of the study (anticipated to be an average of 3 years)

a)Valutazione al Giorno 1, Settimane 1, 2, 4, 8, 12, Mesi 6, 9, 12, ogni 6 quindi. b)Valutazione al Giorno 1, Settimane 4, 12, Mesi 6, 9, 12, ogni 6 mesi quindi. c) ed e)Valutazione al Giorno 1, Settimane 4, 8, 12, Mesi 6, 9, 12, ogni 6 mesi quindi. d)Valutazione dal Giorno -14 al -3, Giorno 1 (soltanto se non già valutato negli studi core), Mesi 6, 12, quindi ogni 6 mesi. a), b), c), d) ed e) Se un paziente discontinuasse fra queste visite, queste valutazione verrano eseguite al momento della discontinuazione. f)controllato per tutta la durata dello studio(previsto per circa 3 anni)

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

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

Tolerability

tollerabilità

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

E.8.1.1

Randomised

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

Information not present in EudraCT

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

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

Canada

United States

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

LVLS : 17.NOV.2015

LVLS : 17.11.2015

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)

Expecting the study to run until the drug become available on the market. The study will be complete when all patients receiving study drug have completed their final visit (Visit 199). The investigator must provide follow-up medical care for all patients who are prematurely withdrawn from the study, or must refer them for appropriate ongoing care

Lo studio dovrebbe coprire i pazienti fino alla disponibilità del farmaco sul mercato. Lo studio sarà completo quando tutti i pazienti che ricevono il farmaco in esperimentazione avranno completato la loro visita finale (visita 199). Lo esperimentatore deve fornire l'assistenza medica necessaria a tutti i pazienti che si ritirano anticipatamente dallo studio, o deve indirizzarli ad una terapia appropiata.

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

2012-03-27

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-02-23

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2013-09-02

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