Clinical Trials Register

Summary
EudraCT Number:	2012-002333-11
Sponsor's Protocol Code Number:	R727-CL-1118
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-11-29
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

Spain - AEMPS

A.2

EudraCT number

2012-002333-11

A.3

Full title of the trial

A Randomized, Double-Blind Study of the Efficacy and Safety of REGN727 Added-on to Rosuvastatin versus Ezetimibe Added-on to Rosuvastatin versus Rosuvastatin Dose Increase in Patients Who are Not Controlled on Rosuvastatin

A.3.1

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

Efficacy and Safety of REGN727 added-on to Rosuvastatin versus Ezetimibe added-on to Rosuvastatin versus Rosuvastatin dose increase in patients who are not controlled on Rosuvastatin

Eficacia y la seguridad de REGN727 añadido a rosuvastatina en comparación con ezetimiba y añadido a rosuvastatina en comparación con el aumento de la dosis de rosuvastatina en pacientes que no están controlados con rosuvastatina

A.3.2

Name or abbreviated title of the trial where available

Odyssey Options II

A.4.1

Sponsor's protocol code number

R727-CL-1118

A.5.4

Other Identifiers

Name:

IND number

Number:

105574

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	Regeneron Pharmaceuticals, 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	Regeneron Pharmaceuticals, 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	Regeneron Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	Stephen Donahue, MD
B.5.3	Address:
B.5.3.1	Street Address	777 Old Saw Mill River Road
B.5.3.2	Town/ city	Tarrytown, NY
B.5.3.3	Post code	10591
B.5.3.4	Country	United States
B.5.4	Telephone number	0019148475672
B.5.6	E-mail	stephen.donahue@regeneron.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	REGN727/SAR236553
D.3.2	Product code	REGN727
D.3.4	Pharmaceutical form	Solution 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	REGN 727
D.3.9.1	CAS number	1245916-14-6
D.3.9.2	Current sponsor code	REGN727 (SAR236553)
D.3.9.3	Other descriptive name	REGN727 (SAR236553)
D.3.9.4	EV Substance Code	SUB74847
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	75 to 150
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	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	CRESTOR
D.2.1.1.2	Name of the Marketing Authorisation holder	ASTRA ZENECA
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Rosuvastatin
D.3.2	Product code	Rosuvastatin
D.3.4	Pharmaceutical form	Capsule
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	Rosuvastatin
D.3.9.1	CAS number	147098-20-2
D.3.9.2	Current sponsor code	Rosuvastatin
D.3.9.3	Other descriptive name	Rosuvastatin
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	10 to 40
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	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	Ezetrol
D.2.1.1.2	Name of the Marketing Authorisation holder	Merck Sharp & Dohme
D.2.1.2	Country which granted the Marketing Authorisation	United Kingdom
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	Ezetimibe
D.3.2	Product code	EZE
D.3.4	Pharmaceutical form	Capsule
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	Ezetimibe (sp)
D.3.9.1	CAS number	163222-33-1
D.3.9.2	Current sponsor code	EZE
D.3.9.3	Other descriptive name	Ezetimibe (sp)
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Injection
D.8.4	Route of administration of the placebo	Subcutaneous use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule
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

Patients with Non-familial hypercholesterolemia or heterozygous familial hypercholesterolemia (heFH) at high CV risk who are not adequately controlled with rosuvastatin (10mg or 20mg) with or without other lipid-modifying therapy (LMT) (excluding EZE)

Pacientes con un riesgo CV elevado, con hipercolesterolemia no familiar o hipercolesterolemia familiar heterocigota (HFH) que no están controlados adecuadamente con atorvastatina (20 mg o 40 mg), con otro TML o sin él (a excepción de la EZE).

E.1.1.1

Medical condition in easily understood language

High blood cholesterol level

Colesterol elevado en sangre

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	10020603
E.1.2	Term	Hypercholesterolaemia
E.1.2	System Organ Class	10027433 - Metabolism and nutrition 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 reduction of LDL-C by REGN727 as add-on therapy to rosuvastatin in comparison with EZE as add-on therapy to rosuvastatin, and in comparison with doubling the rosuvastatin dose, after 24 weeks of treatment in patients with hypercholesterolemia at high CV risk.

Es analizar la reducción del C-LDL producido por REGN727 como tratamiento complementario de atorvastatina, en comparación con EZE como tratamiento complementario a la atorvastatina, en comparación con el doble de la dosis de atorvastatina o en comparación con un cambio de tratamiento, de atorvastatina a rosuvastatina, transcurridas 24 semanas de tratamiento en pacientes con hipercolesterolemia con un riesgo CV elevado

E.2.2

Secondary objectives of the trial

- To evaluate the reduction of LDL-C by REGN727 75 mg as add-on therapy to rosuvastatin in comparison with EZE as add-on therapy to rosuvastatin, or in comparison with doubling of the rosuvastatin dose after 12 weeks of treatment
- To evaluate the effect of REGN727 on other lipid parameters (eg, ApoB, non-HDL-C, total-C, Lp[a], HDL-C, TG levels, ApoA-1)
- To evaluate the safety and tolerability of REGN727
- To evaluate the development of anti-REGN727 antibodies

-Evaluar la disminución que produce REGN727 75 mg del C-LDL como tratamiento complementario de la atorvastatina, en comparación con EZE como tratamiento complementario a la atorvastatina, en comparación con el doble de la dosis de atorvastatina o en comparación con un cambio de tratamiento de atorvastatina a rosuvastatina, transcurridas 12 semanas de tratamiento.
-Evaluar el efecto de REGN727 en otros parámetros lipídicos (p. ej., Apo B, C-no-HDL, C-total, Lp(a), C-HDL, niveles de TG y niveles de Apo A-1).
-Evaluar la seguridad y la tolerabilidad de REGN727.
-Evaluar el desarrollo de anticuerpos anti-REGN727.

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

Title: A Randomized, Double-Blind Study of the Efficacy and Safety of REGN727 Added-on to Rosuvastatin versus Ezetimibe Added-on to Rosuvastatin versus Rosuvastatin Dose Increase in Patients Who are Not Controlled on Rosuvastatin.
Objectives: to identify genetic associations with clinical or biomarker response to PCSK9 inhibition, hyperlipidemia, or CVD. If needed, samples may also be used to identify markers associated with toxicity.

Estudio randomizado, doble ciego para evaluar la eficacia y la seguridad de REGN727 añadido a rosuvastatina en comparación con ezetimiba y añadido a rosuvastatina en comparación con el aumento de la dosis de rosuvastatina en pacientes que no están controlados con rosuvastatina
Objetivos: identificar asociaciones genéticas con la respuesta clínica o biomarcador para PCSK9 inhibición, hiperlipidemia, o CVD. Si es necesario, las muestras también se puede utilizar para identificar los marcadores asociados con la toxicidad.

E.3

Principal inclusion criteria

A patient must meet either 1a or 1b to be eligible for inclusion in the study:
a. Patients with screening (visit 1) LDL-C ?70 mg/dL (1.81 mmol/L) who are not adequately controlled with a 10 mg or 20 mg stable daily dose of rosuvastatin for at least 4 weeks before the screening visit (visit 1), with or without other LMT, excluding EZE. Patients with heFH* or non-FH must also have a history of documented CHD (defined below), or non-CHD CVD (defined below) or diabetes mellitus with target organ damage.
OR
b. Patients with screening (visit 1) LDL-C ?100 mg/dL (2.59 mmol/L) who are not adequately controlled with a 10 mg or 20 mg stable daily dose of rosuvastatin for at least 4 weeks before the screening visit (visit 1), with or without other LMT (excluding EZE). Patients must also have heFH*, or have non-FH, without CHD or non-CHD CVD, but with a calculated 10-year fatal CVD risk SCORE ?5%, or with moderate CKD, or with diabetes mellitus but no target organ damage.

1. El paciente debe cumplir el criterio 1a o 1b para ser apto para su inclusión en el estudio:
a. Pacientes que en el screening (Visita 1) presenten valores de C-LDL ? 70 mg/dl (1,8 mmol/l) que no estén siendo controlados adecuadamente con una dosis diaria estable de 20 mg o 40 mg de atorvastatina durante un mínimo de 4 semanas antes de la visita del screening (Visita 1), con otro TML o sin él (a excepción de EZE). Los pacientes con HFH* o HNF también deben tener antecedentes de CPC documentada (definida a continuación) o ECV NO-CPC (definida a continuación) o diabetes mellitus con lesión orgánica.
O
b. Pacientes que en el screening (Visita 1) presenten valores de C-LDL ? 100 mg/dl (2,59 mmol/l) que no estén siendo controlados adecuadamente con una dosis diaria de 20 mg o 40 mg de atorvastatina durante un mínimo de 4 semanas antes de la visita del screening (Visita 1), con otro TML o sin él (a excepción de EZE). Los pacientes también deben tener HFH* o HNF, sin CPC o ECV NO-CPC, pero con un riesgo de ECV mortal calculado en 10 años ? 5 % o una NPC o diabetes mellitus sin lesión orgánica.

E.4

Principal exclusion criteria

LDL-C <70 mg/dL (<1.81 mmol/L) in patients with CVD
LDL-C <100 mg/dL (<2.59 mmol/L) in patients without CVD, but with other risk factors
LDL-C >250 mg/dL or TG >400 mg/dL
Homozygous FH or patients receiving plasmapheresis CV event within 3 months prior to screening
NYHA Class III or IV heart failure
History of hemorrhagic stroke
Uncontrolled endocrine disease known to influence serum lipids
Any clinically significant abnormality that in the judgment of the investigator would preclude safe completion of the study or constrain endpoints assessment such as major systemic diseases, patients with short life expectancy, patients who cannot tolerate subcutaneous injections.
Use of ezetimibe or red yeast rice products within 4 weeks of screening
Use of systemic corticosteroids or use of fibrates, other than fenofibrate within 6 weeks of the screening
eGFR <30 mL/min/1.73m2
ALT or AST aminotransferase >3 x upper limit of normal (ULN)
CPK >3 x ULN
TSH < lower limit of normal (LLN)
Hypersensitivity to monoclonal antibody therapeutics
Pregnant or breast-feeding women
Women of childbearing potential with no effective contraceptive method of birth control

Paciente con C-LDL < 70 mg/dl con antecedentes de CPC o ECV NO-CPC documentadas
Paciente con C-LDL < 100 mg/dl y sin antecedentes de CPC o ECV NO-CPC documentadas, pero con otros factores de riesgo
LDL-C >250 mg/dL o TG >400 mg/dL
HF homocigótica o pacientes que se hayan sometido a una plasmaféresis en los 2 meses anteriores al screening.
insuficiencia cardiaca (NYHA clase III o IV) en los últimos 12 meses
Antecedentes conocidos de ictus hemorrágico
Presencia de cualquier enfermedad endocrina no controlada
Cualquier anomalía clínicamente significativa identificada en el momento del screening que, a juicio del Investigador o cualquier Investigador adjunto, impida la realización segura del estudio o limite la evaluación de los criterios de valoración, como por ejemplo, enfermedades sistémicas graves o pacientes con una esperanza de vida corta, o se consideren que son incapaces de administrarse o tolerar las inyecciones durante un período largo de tiempo.
Uso de ezetimibe o productos a base de arroz de levadura roja en las 4 semanas previas a la visita del screening
Uso de corticoesteroides sistémicos o fibratos que no sean fenofibratos en las 6 semanas previas a la visita del screening
FGe < 30 ml/min/1,73 m2
ALAT o ASAT > 3 x límite superior de normalidad (LSN)
CPK > 3 x LSN
TSH < límite inferior de normalidad (LIN)
Hipersensibilidad conocida a medicamentos con anticuerpos monoclonales
Mujeres embarazadas o en período de lactancia.
Mujeres en edad fértil que no estén protegidas con un método anticonceptivo eficaz y/o que no estén dispuestas o no puedan someterse a una prueba de embarazo.

E.5 End points

E.5.1

Primary end point(s)

The percent change in calculated LDL-C from baseline to week 24.

El parámetro principal de la eficacia es el cambio porcentual del C-LDL calculado desde el inicio hasta la Semana 24.

E.5.1.1

Timepoint(s) of evaluation of this end point

From baseline to week 24

Desde el inicio hasta la Semana 24.

E.5.2

Secondary end point(s)

- The percent change in calculated LDL-C from baseline to week 12.
- The percent change in ApoB from baseline to week 24.
- The percent change in non-HDL-C from baseline to week 24.
- The percent change in total-C from baseline to week 24.
- The percent change in ApoB from baseline to week 12.
- The percent change in non-HDL-C from baseline to week 12.
- The percent change in total-C from baseline to week 12.
- The proportion of patients reaching LDL-C goal at week 24.
- The percent change in Lp(a) from baseline to week 24.
- The percent change in HDL-C from baseline to week 24.
- The percent change in HDL-C from baseline to week 12.
- The percent change in Lp(a) from baseline to week 12.
- The percent change in fasting TG from baseline to week 24.
- The percent change in fasting TG from baseline to week 12.
- The percent change in ApoA-1 from baseline to week 24.
- The percent change in ApoA-1 from baseline to week 12.

-Cambio porcentual del C-LDL calculado desde el inicio hasta la Semana 12.
-Cambio porcentual del nivel de Apo B desde el inicio hasta la Semana 24.
-Cambio porcentual del C-no-HDL desde el inicio hasta la Semana 24.
-Cambio porcentual del C-total desde el inicio hasta la Semana 24.
-Cambio porcentual del nivel de Apo B desde el inicio hasta la Semana 12.
-Cambio porcentual del C-no-HDL desde el inicio hasta la Semana 12.
-Cambio porcentual del C-total desde el inicio hasta la Semana 12.
-La proporción de pacientes que alcancen el nivel de C-LDL objetivo en la Semana 24.
-Cambio porcentual de la Lp(a) desde el inicio hasta la Semana 24.
-Cambio porcentual del nivel de C-HDL desde el inicio hasta la Semana 24.
-Cambio porcentual del nivel de C-HDL desde el inicio hasta la Semana 12.
-Cambio porcentual de la Lp(a) desde el inicio hasta la Semana 12.
-Cambio porcentual de TG en ayunas desde el inicio hasta la Semana 24.
-Cambio porcentual de TG en ayunas desde el inicio hasta la Semana 12.
-Cambio porcentual del nivel de Apo A-1 desde el inicio hasta la Semana 24.
-Cambio porcentual del nivel de Apo A-1 desde el inicio hasta la Semana 12.

E.5.2.1

Timepoint(s) of evaluation of this end point

From baseline to week 12 or week 24

Desde el inicio hasta la Semana 24.

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

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

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

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

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

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Canada

France

Germany

Italy

Mexico

New Zealand

Spain

United Kingdom

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

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:

300

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

361

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

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

2013-01-23

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2013-01-11

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2014-05-09

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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