Clinical Trials Register

Summary
EudraCT Number:	2010-022636-37
Sponsor's Protocol Code Number:	AT1001-012
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:	2012-03-13
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

2010-022636-37

A.3

Full title of the trial

A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT

Studio randomizzato in aperto per confrontare l'efficacia e la sicurezza di AT1001 e della terapia enzimatica sostitutiva (ERT) in pazienti affetti dalla malattia di Fabry e con mutazioni del gene GLA AT1001-responsivo precedentemente trattati con ERT

A.3.2

Name or abbreviated title of the trial where available

AT1001-012

A.4.1

Sponsor's protocol code number

AT1001-012

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	AMICUS THERAPEUTICS, INC
B.1.3.4	Country	United States
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	GlaxoSmithKline UK Ltd
B.4.2	Country	United Kingdom
B.4.1	Name of organisation providing support	Amicus Therapeutics, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Amicus Therapeutics
B.5.2	Functional name of contact point	Patient Advocacy
B.5.3	Address:
B.5.3.1	Street Address	30 A Upper High Street
B.5.3.2	Town/ city	Thame Oxon
B.5.3.3	Post code	0X9 3EX
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	001 609 662-2000
B.5.5	Fax number	001 609 662-5010
B.5.6	E-mail	clinicaltrials@amicustherapeutics.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/06/368
D.3 Description of the IMP
D.3.1	Product name	migalastat hydrocloride
D.3.2	Product code	AT1001
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	migalastat hydrocloride
D.3.9.1	CAS number	75172-81-5
D.3.9.2	Current sponsor code	AT1001
D.3.9.3	Other descriptive name	1-deoxygalactonojirimycin hydrochloride
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	150
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	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	REPLAGAL
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/3/00/002
D.3 Description of the IMP
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.3.8	INN - Proposed INN	Agalsidase alfa
D.3.9.1	CAS number	104138-64-9
D.3.9.2	Current sponsor code	NA
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/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1
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	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	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	Yes
D.3.11.13.1	Other medicinal product type	alfa galattosidasi A proteina umana prodotta in una linea cellulare umana tramite ingegneria geneti
D.IMP: 3
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	FABRAZYME
D.2.1.1.2	Name of the Marketing Authorisation holder	GENZYME Srl
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/3/00/003
D.3 Description of the IMP
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.3.8	INN - Proposed INN	Agalsidase beta
D.3.9.1	CAS number	104138-64-9
D.3.9.2	Current sponsor code	NA
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/ml milligram(s)/millilitre
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	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	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	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	Yes
D.3.11.13.1	Other medicinal product type	Agalsidasi beta e' una forma ricombinante dell'alfa galattosidasi A umana.

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Fabry disease

Malattia di Fabry

E.1.1.1

Medical condition in easily understood language

People with Fabry disease have a genetic disorder that prevents their
bodies from breaking down a particular lipid (a fat-like substance) in
their cells.

Le persone con malattia di Fabry hanno un disturbo genetico che impedisce al loro organismo di scomporre un particolare lipide (una sostanza simile ai grassi)all'interno delle
loro cellule

E.1.1.2

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

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	10016016
E.1.2	Term	Fabry's disease
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

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

The primary efficacy parameter is the change from baseline in glomerular filtration rate (GFR) as assessed by plasma clearance of iohexol ("iohexol GFR").

Il parametro primario di efficacia è la variazione rispetto al basale del
tasso di filtrazione glomerulare (GFR) come valutato dalla clearance plasmatica di ioexolo ("GFR ioexolo").

E.2.2

Secondary objectives of the trial

Change from baseline in:Estimated GFR (eGFR); 24-hour urine protein; left ventricular mass and ejection fraction; urine globotriaosylceramide; WBC α-galactosidase A activity; patient reported assessment of pain and Quality of Life as assessed by the BPI short form and Short Form 36;Composite clinical outcome, as assessed by time to the first occurrence of the following events and number of subjects who experience any of the following events:Renal events:A decrease in iohexol GFR ≥ 15 mL/min/1.73 m2, with the decreased GFR < 90 mL/min/1.73 m2 relative to baseline; An increase in 24-hour urine protein ≥ 33% , with the increased protein ≥ 300 mg relative to baseline.Cardiac events:Myocardial infarction;Unstable cardiac angina;New symptomatic arrhythmia requiring anti-arrhythmic medication, direct current cardioversion, pacemaker, or defibrillator implantation; Congestive heart failure, NYHA class III or IV.Cerebrovascular events:Stroke; Transient ischemic attack/Death.

Variaz dal basale del:GFR stimato;proteinuria a 24 ore;massa ventricolare sinistra e frazione di eiezione;escrezione urinaria di globotriaosiloceramide;dell’attività enzimatica dell’ß-galattosidasi A nella conta leucocitaria;valut riferita dal paz del dolore in base al questionario breve sul dolore BPI;qualità della vita riferita dal paz in base alla valut del questionario sullo stato di salute SF-36.L’esito clinico composito,in base alla valut del tempo alla prima insorgenza dei seguenti eventi:Eventi renali:Riduzione di GFR tramite ioexolo ≥ 15 ml/min/1,73 m2,con GFR ridotto<90 ml/min/1,73 m2 relativo al basale;Un aumento nella proteinuria a 24 ore ≥ 33%,con un aumento delle proteine ≥ 300 mg relativo al basale.Eventi cardiaci:Infarto del miocardio;Angina instabile;Nuova aritmia sintomatica che richiede tratt farmacologico antiaritmico,cardioversione elettrica diretta,impianto di pacemaker o defibrillatore;Scompenso cardiaco congestizio,classe III o IV secondo la NYHA.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Male or female, diagnosed with Fabry disease and between 16 and 74 years of age, inclusive at Visit 1; Confirmed GLA mutation shown to be responsive to AT1001 in the in vitro HEK-293 cell-based assay, or a confirmed GLA mutation that is non-testable in this assay, but has evidence to suggest the potential to respond to AT1001;Initiated treatment with ERT at least 12 months before Visit 2 ;Dose level and regimen of ERT have been stable for the 3 months before Visit 2 and is at least 80% of the currently labeled dose for this time period; GFR ≥ 30mL/min/1.73 m2 ;If taking angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) has been on a stable dose for at least the 4 weeks before Visit 1; If of reproductive potential, agree to use medically accepted methods of contraception throughout the duration of the study and for up to 30 days after last dose of protocol defined study medication: For sites in Italy: If of reproductive potential, agree to plan (or have their partner plan) with his/her physician a birth control strategy (or method) in order to avoid pregnancy throughout the duration of the study and for up to 30 days after last dose of protocol defined study medication;Able to provide written informed consent, and assent if applicable. Subjects under 18 years of age will provide written informed assent, and written informed consent will be provided by their parent or legal guardian

Uomini o donne, a cui e' stata diagnosticata la malattia di Fabry e con un’eta' compresa tra 16 e 74 anni di eta', inclusi alla Visita 1;Mutazione confermata del gene GLA che ha dimostrato di essere rispondente ad AT1001 nel saggio in vitro su cellule HEK-293 oppure mutazione confermata del gene GLA non esaminabile in questo saggio, ma con evidenza di essere potenzialmente rispondente ad AT1001; Trattamento iniziale con ERT almeno 12 mesi precedenti la Visita 2;Il livello di dosaggio e il regime di ERT si sono mantenuti stabili per i 3 mesi precedenti la Visita 2 e hanno costituito almeno l’80% della dose attualmente designata per questo periodo di tempo;GFR ≥ 30ml/min/1,73 m2;Se la dose di assunzione di inibitori dell’enzima di conversione dell’angiotensina (ACE) o di bloccanti del recettore dell’angiotensina (ARB) e' rimasta stabile per almeno 4 settimane precedenti la Visita 1;In caso di soggetti potenzialmente fertili, consenso a utilizzare metodi medicalmente accettati di contraccezione per tutta la durata dello studio e per massimo 30 giorni dopo l’ultima dose di farmaco in studio definito dal protocollo; per i centri in italia:se potenzialmente fertili, devono acconsentire a un piano (o fare pianificare al partner) con il loro medico una strategia (o metodo) di contraccezione al fine di evitare una gravidanza nel corso dell’intero studio e fino a 30 giorni dopo l’ultima dose del farmaco in studio definito dal protocollo;Capacita' di fornire consenso informato scritto e assenso, ove necessario. I soggetti di eta' inferiore a 18 anni forniranno assenso informato scritto e il consenso informato scritto sara' fornito da un genitore o da un tutore legale

E.4

Principal exclusion criteria

1. Undergone, or is scheduled to undergo, kidney transplantation or any other solid organ transplantation 2. On regular dialysis that is specifically for the treatment of chronic kidney disease 3. Had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Visit 1 4. Has clinically significant unstable cardiac disease in the opinion of the investigator (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure) 5. Is pregnant or breast-feeding 6. Has a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (e.g., miglustat, miglitol) 7. Has absolute contraindication to iohexol and/or inability to undergo iohexol GFR testing 8. Requires treatment with Glyset (miglitol), or Zavesca (miglustat) 9. Received any investigational/experimental drug, biologic or device within 30 days of Visit 1 10. Has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study 11. Is otherwise unsuitable for the study, in the opinion of the investigator

1. Sottoposto, o in programma di sottoporsi, a trapianto di reni o di qualsiasi altro organo solido 2. Sottoposto a dialisi regolare specificamente per il trattamento dell’insufficienza renale cronica 3. Precedente attacco ischemico transitorio documentato, ictus, angina instabile o infarto del miocardio nei 3 mesi precedenti la Visita 1 4. Cardiopatia instabile clinicamente significativa secondo il giudizio dello sperimentatore (ad es. cardiopatia che richieda trattamento attivo come aritmia sintomatica, angina instabile o scompenso cardiaco congestizio di classe III o IV secondo la NYHA (New York Heart Association) 5. Stato di gravidanza o allattamento al seno 6. Storia di allergia o sensibilita' ad AT1001 (inclusi gli eccipienti) o altri imino-zuccheri (ad es. miglustat, miglitolo) 7. Controindicazione assoluta a ioexolo e/o incapacita' a sottoporsi al test del GFR tramite ioexolo 8. Necessita' di trattamento con Glyset (miglitolo) o Zavesca (miglustat) 9. Ricezione di qualsiasi farmaco, agente biologico o dispositivo sperimentale/nuovo nei 30 giorni precedenti la Visita 1 10. Presenza di qualsiasi malattia o patologia intercorrente che possa precludere al soggetto di rispettare i requisiti del protocollo o suggerisca allo sperimentatore che il potenziale soggetto possa correre dei rischi inaccettabili derivanti dalla partecipazione allo studio 11. Inadeguatezza per qualsiasi altra ragione allo studio, secondo il giudizio dello sperimentatore

E.5 End points

E.5.1

Primary end point(s)

Change from baseline in glomerular filtration rate (GFR) as assessed by plasma clearance of iohexol (“iohexol GFR”).

Variazione dal basale del tasso di filtrazione glomerulare (GFR, Glomerular Filtration Rate) sulla base della valutazione della clearance plasmatica di ioexolo (''GFR tramite ioexolo'').

E.5.1.1

Timepoint(s) of evaluation of this end point

18 months

18 mesi

E.5.2

Secondary end point(s)

Change from baseline in:
-Estimated GFR; 24-hour urine protein; left ventricular mass and ejection
fraction; urine GL-3; WBC α-galactosidase A activity; patient reported
assessment of pain and Quality of Life as assessed by the BPI short form
and Short Form 36
-Composite clinical outcome, as assessed by time to the first occurrence
of the following events and number of subjects who experience any of
the following events:
Renal events: A decrease in iohexol GFR ≥ 15 mL/min/1.73 m2, with the
decreased GFR < 90 mL/min/1.73 m2 relative to baseline; An increase in
24-hour urine protein ≥ 33% , with the increased protein ≥ 300 mg
relative to baseline
Cardiac events: Myocardial infarction; Unstable cardiac angina; New
symptomatic arrhythmia requiring anti-arrhythmic medication, direct
current cardioversion, pacemaker, or defibrillator implantation;
Congestive heart failure, NYHA class III or IV
Cebrovascular events: Stroke; Transient ischemic attack/Death

Variazione dal basale del: GFR stimato; proteinuria a 24 ore; massa ventricolare sinistra e frazione di eiezione;escrezione urinaria di globotriaosiloceramide;dell’attivita' enzimatica dell’α-galattosidasi A nella conta leucocitaria; valutazione riferita dal paziente del dolore in base al questionario breve sul dolore BPI; qualita' della vita riferita dal paziente in base alla valutazione del questionario sullo stato di salute SF-36.L’esito clinico composito, in base alla valutazione del tempo alla prima insorgenza dei seguenti eventi: Eventi renali: Riduzione di GFR tramite ioexolo ≥ 15 ml/min/1,73 m2, con GFR ridotto < 90 ml/min/1,73 m2 relativo al basale; Un aumento nella proteinuria a 24 ore ≥ 33% , con un aumento delle proteine ≥ 300 mg relativo al basale. Eventi cardiaci:Infarto del miocardio;Angina instabile; Nuova aritmia sintomatica che richiede trattamento farmacologico antiaritmico, cardioversione elettrica diretta, impianto di pacemaker o defibrillatore; Scompenso cardiaco congestizio, classe III o IV secondo la NYHA. Eventi cerebrovascolari:Ictus ;Attacco ischemico transitorio/Decesso

E.5.2.1

Timepoint(s) of evaluation of this end point

18 months

18 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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

ERT

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

Argentina

Australia

Brazil

Japan

Mexico

Russian Federation

Switzerland

Taiwan

Turkey

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

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

Yes

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

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)

Normal Treatment for that Condition

Il trattamento normale per tale condizione.

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

2011-12-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-07-05

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2015-05-28

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