Clinical Trials Register

Summary
EudraCT Number:	2013-002318-11
Sponsor's Protocol Code Number:	109MS306
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2015-05-08
Trial 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

2013-002318-11

A.3

Full title of the trial

Open-Label, Randomized, Multicenter, Multiple-Dose, Active Controlled, Parallel-Group, Efficacy and Safety Study of BG00012 in Children From 10 to Less Than 18 Years of Age With Relapsing-Remitting Multiple Sclerosis

Estudio abierto, aleatorizado, multicéntrico, de múltiples dosis, comparativo con fármaco activo y con grupos paralelos, sobre la eficacia y la seguridad de BG00012 en niños de 10 a menos de 18 años de edad con esclerosis múltiple recurrente-remitente

A.3.1

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

Phase 3 Efficacy and Safety Study of BG00012 in Pediatric Subjects With relapsing-remitting multiple sclerosis (RRMS).

Un estudio sobre la seguridad y eficacia de BG00012 en sujetos pediatricos con esclerosis múltiple recurrente-remitente

A.3.2

Name or abbreviated title of the trial where available

CONNECT

A.4.1

Sponsor's protocol code number

109MS306

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/027/2013

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Biogen Idec Research Limited
B.1.3.4	Country	United Kingdom
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	Biogen Idec Research Limited
B.4.2	Country	United Kingdom
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Biogen Idec Research Limited
B.5.2	Functional name of contact point	Medical Director
B.5.3	Address:
B.5.3.1	Street Address	Innovation House, 70 Norden Road
B.5.3.2	Town/ city	Maidenhead
B.5.3.3	Post code	SL6 4AY
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	3491806 2000
B.5.6	E-mail	clinicaltrials@biogenidec.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	BG00012
D.3.2	Product code	BG00012
D.3.4	Pharmaceutical form	Gastro-resistant 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	DIMETHYL FUMARATE
D.3.9.1	CAS number	624-49-7
D.3.9.2	Current sponsor code	BG00012
D.3.9.3	Other descriptive name	DIMETHYL FUMARATE
D.3.9.4	EV Substance Code	SUB13608MIG
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	120
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	AVONEX
D.2.1.1.2	Name of the Marketing Authorisation holder	Biogen Idec Ltd
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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	Avonex
D.3.4	Pharmaceutical form	Injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intramuscular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Interferon beta-1a
D.3.9.1	CAS number	220581-49-7
D.3.9.3	Other descriptive name	INTERFERON BETA-1A
D.3.9.4	EV Substance Code	SUB12440MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	7.5 to 30
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.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

Relapsing-Remitting Multiple Sclerosis

Esclerosis múltiple recurrente-remitente

E.1.1.1

Medical condition in easily understood language

Multiple Sclerosis

Esclerosis múltiple

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	18.1
E.1.2	Level	SOC
E.1.2	Classification code	10029205
E.1.2	Term	Nervous system disorders
E.1.2	System Organ Class	10029205 - Nervous system disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	18.1
E.1.2	Level	PT
E.1.2	Classification code	10063399
E.1.2	Term	Relapsing-remitting multiple sclerosis
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

The main objectives of the study are as follows:
? To evaluate the safety, tolerability, and effect on the disease course of BG00012 (dimethyl fumarate) in pediatric participants with RRMS, as compared with a disease modifying treatment.
? To assess pharmacokinetic (PK) and pharmacodynamic (PD) parameters in a representative subset of pediatric participants
? To assess health outcomes and evolution of disability

Los principales objetivos del estudio son los siguientes:
? Evaluar la seguridad, tolerabilidad y el efecto de BG00012 en el desarrollo de la enfermedad en niños con EMRR en comparación con un tratamiento modificador de la enfermedad.
? Evaluar los parámetros farmacocinéticos (FC) y farmacodinámicos (FD) en un subgrupo representativo de niños afectados.
? Evaluar los efectos sobre la salud y la evolución de la discapacidad.

E.2.2

Secondary objectives of the trial

Not Applicable

No aplica

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

key inclusion criteria:
- Must have a body weight of ?30 kg.
- Must have a diagnosis of RRMS (consensus definition for pediatric RRMS [Krupp 2007]).
- Must be ambulatory with a baseline EDSS score between 0 and 5.5, inclusive.
- Must have experienced at least 1 relapse within the last 12 months prior to Day 1, or at least 2 relapses within the last 24 months prior to Day 1, with a prior brain MRI demonstrating lesions consistent with MS, or show evidence of Gd enhancing lesions of the brain on an MRI performed within the 6 weeks prior to Day 1.
- Must be neurologically stable, with no evidence of relapse within 50 days prior to Day 1 and no evidence of corticosteroid treatment within 30 days prior to Day 1.
- Subjects of childbearing potential who are sexually active must be willing to practice effective contraception during the study and be willing and able to continue contraception for at least 30 days after their final dose of study treatment.

Criterios de inclusión clave:
- Debe tener un peso corporal de ?30 kg.
- Debe tener un diagnóstico de la EMRR (definición de consenso para la EMRR pediátricos [Krupp 2007]).
- Debe ser ambulatoria con una puntuación EDSS basal entre 0 y 5.5, ambos inclusive.
- Deben haber experimentado al menos 1 recaída en los 12 meses anteriores al Día 1 o al menos 2 recaídas en los 24 meses previos al día 1, con una RM cerebral anterior que muestre lesiones coherentes con la EM, o evidencia de lesiones potenciadas con Gd del cerebro en una RM realizada durante las 6 semanas anteriores al Día 1.
- Deben estar estables neurológicamente, sin evidencias de recaída en un periodo de 50 días antes del Día 1 y sin evidencias de haber seguido tratamiento con corticosteroides en los 30 días anteriores al Día 1.
- Las pacientes en edad fértil y que sean sexualmente activas deben utilizar métodos anticonceptivos eficaces durante el estudio, así como estar dispuestas a, y ser capaces de, continuar con ellas durante al menos 30 días después de su última dosis del tratamiento del estudio

E.4

Principal exclusion criteria

Key exclusion criteria:
- Primary progressive, secondary progressive, or progressive relapsing MS (as defined by [Lublin and Reingold 1996]). These conditions require the presence of continuous clinical disease worsening over a period of at least 3 months. Subjects with these conditions may also have superimposed relapses but are distinguished from relapsing remitting subjects by the lack of clinically stable periods or clinical improvement.
- Disorders mimicking MS, such as other demyelinating disorders (e.g., acute disseminated encephalomyelitis), systemic autoimmune disorders (e.g., Sjögren disease, lupus erythematosus), metabolic disorders (e.g., dystrophies), and infectious disorders.
- History of premalignant or malignant disease. Subjects with basal cell carcinoma that has been completely excised prior to screening will remain eligible.
- History of severe allergic or anaphylactic reactions, or known drug hypersensitivity to DMF or fumaric acid esters.
- History of abnormal laboratory results indicative of any significant endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, renal, and/or any other major disease that would preclude participation in a clinical study.
- History of clinically significant cardiovascular, pulmonary, GI, dermatologic, growth, developmental, psychiatric (including depression), neurologic (other than MS), and/or other major disease that would preclude participation in a clinical study.
- History of human immunodeficiency virus.
- An MS relapse that has occurred within 50 days prior to Day 1 AND/OR the subject has not stabilized from a previous relapse prior to Day 1.
- Other unspecified reasons that, in the opinion of the Investigator or Biogen Idec, make the subject unsuitable for enrollment.
- For the PD/PK subset of subjects: subjects unable to swallow the BG00012 capsule whole.
Key Treatment history
- Any previous treatment with Fumaderm (fumaric acid esters) or BG00012.
- Prior treatment with any of the following: total lymphoid irradiation, cladribine, T-cell or T-cell receptor vaccination, any therapeutic monoclonal antibody, with the exception of rituximab or natalizumab
- Prior treatment with any of the following medications within the 12 months prior to Day 1: mitoxantrone, cyclophosphamide, rituximab
- Prior treatment with any of the following medications or procedures within 6 months prior to Day 1: fingolimod, teriflunomide, natalizumab, cyclosporine, azathioprine, methotrexate, mycophenolate mofetil, laquinimod, IV immunoglobulin, plasmapheresis or cytapheresis
- Prior treatment with any of the following within 3 months prior to Day 1: glatiramer acetate, interferon-alpha, interferon ? (subjects who are positive for neutralizing antibodies to interferon-? may receive interferon-? treatment up to 2 weeks prior to Day 1)
- Treatment with any of the following medications within 30 days prior to Day 1:
- steroids (IV or oral corticosteroid treatment, including agents that may act through the corticosteroid pathway [e.g., low dose naltrexone]), 4-aminopyridine or related products (except subjects on a stable dose of controlled-release fampridine for 3 months)
NOTE: Other protocol-defined inclusion/exclusion criteria may apply

Criterios de exclusión clave:
-EM recurrente primaria progresiva, secundaria progresiva o progresiva (bajo la definición de [Lublin y Reingold 1996]). Estas enfermedades requieren la presencia de un empeoramiento clínico continuo de la enfermedad durante un periodo mínimo de 3 meses. Los pacientes con estas enfermedades también pueden acumular recaídas pero se diferencian de los pacientes con EM recurrente-remitente por la ausencia de periodos clínicamente estables o de mejora clínica.
-Trastornos que imitan a la EM, como otras afecciones desmielinizantes (por ejemplo, encefalomielitis aguda diseminada), trastornos autoinmunitarios sistémicos (por ejemplo, síndrome de Sjögren, lupus eritematoso), trastornos metabólicos (por ejemplo, distrofias) y trastornos infecciosos.
- Antecedentes de enfermedades precancerosas o tumores malignos. Los pacientes con carcinoma basocelular que se ha extirpado completamente antes de la selección siguen siendo aptos.
- Antecedentes de reacciones alérgicas o anafilácticas graves o hipersensibilidad conocida a DMF o ésteres de ácido fumárico.
-Antecedentes de resultados analíticos anómalos indicativos de cualquier endocrinopatía, hemopatía, hepatopatía, inmunopatía, metabolopatía, uropatía, nefropatía significativa y/u otros trastornos importantes que puedan ser motivo de exclusión para la participación en un estudio clínico.
-Antecedentes de enfermedad cardiovascular, pulmonar, gastrointestinal, dermatológica, del crecimiento, del desarrollo, psiquiátrica (incluyendo depresión), neurológica (diferente a la EM) clínicamente significativas y/u otros trastornos importantes que puedan ser motivo de exclusión para la participación en un estudio clínico.
-Antecedentes de infección por el virus de la inmunodeficiencia humana.
-Una recaída de la EM que haya tenido lugar en los 50 días anteriores al día 1; O BIEN, el paciente no se ha estabilizado desde una recaída anterior previa al Día 1.
-Otra razón no especificada que, en opinión del investigador o de Biogen Idec, haga que el paciente no sea apto para la inscripción.
-Para el subgrupo de pacientes para FD/FC: pacientes incapaces de ingerir la cápsula de BG00012 entera.
Historia de tratamiento clave:
- Cualquier tratamiento previo con Fumaderm® o BG00012.
- Tratamiento previo con cualquiera de los siguientes: Irradiación linfática total, Cladribina, Vacuna de linfocitos T o de receptores de linfocitos T, Cualquier anticuerpo monoclonal terapéutico, a excepción de rituximab o natalizumab
- Tratamiento previo en los 12 meses anteriores al día 1 con cualquiera de los siguientes medicamentos: Mitoxantrona, Ciclofosfamida, Rituximab.
- Tratamiento previo en los 6 meses anteriores al Día 1 con cualquiera de los siguientes medicamentos: Fingolimod, Teriflunomida, Natalizumab, Ciclosporina, Azatioprina, Metotrexato, Micofenolato de mofetilo, Laquinimod, Inmunoglobulina intravenosa, Plasmaféresis o citoféresis
- Tratamiento previo en los 3 meses anteriores al Día 1 con cualquiera de los siguientes medicamentos: Acetato de glatirámero, Interferón ?, Interferón ? (los pacientes que obtengan un resultado positivo para los anticuerpos neutralizadores del interferón ? pueden recibir el tratamiento con interferón ? hasta dos semanas antes del Día
-Tratamiento previo en los 30 días anteriores al día 1 con cualquiera de los siguientes medicamentos: Corticosteroides (tratamiento con corticosteroides por vía IV u oral, que incluye fármacos que pueden actuar mediante la vía de los corticosteroides [por ejemplo, naltrexona a bajas dosis]); 4-aminopiridina o productos relacionados (excepto los pacientes que tomen una dosis estable de fampridina de liberación controlada durante 3 meses)
NOTA: Se pueden aplicar otros criterios de inclusión / exclusión definidos en el protocolo

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint of the study is the proportion of participants free of new/newly enlarging T2 hyperintense lesions on brain MRI scans at Week 96.

El criterio de valoración principal del estudio es conocer la proporción de pacientes sin lesiones hiperintensas en T2 nuevas o aumentadas en RM cerebrales en la Semana 96.

E.5.1.1

Timepoint(s) of evaluation of this end point

Day 1 (baseline visit), weeks 24, 48, 72, 96

Día 1 (visita inicial) semanas 24, 48, 72 y 96.

E.5.2

Secondary end point(s)

The secondary endpoints of this study are as follows:
? Number of new/newly enlarging T2 hyperintense lesions on brain MRI scans at Weeks 24 and 96
? Proportion of participants free of new/newly enlarging T2 hyperintense lesions on brain MRI scans at Weeks 24 and 48
? Proportion of participants free of new MRI activity as measured by brain MRI scans (new MRI activity includes: Gd enhancing MRI lesion on brain MRI scans; new T2 MRI lesions on brain MRI scans and newly enlarging LRI lesions on brain MRI scans) at Weeks 24, 48, and 96
? Time to first relapse up to week 96
? Proportion of participants who do not experience relapse up to Week 96
? Annualized relapse rate at Weeks 48 and 96
? Number of participants that experience adverse events (AEs) and serious adverse events (SAEs), including prospective and follow up of flushing, nausea, abdominal pain, and diarrhea up to week 96
? Participants receiving BG00012 who consent to participate in the PD/PK subset - PK parameters derived from PK subset data analysis: area under the concentration time curve from time 0 to 10 hours (AUC0 10), maximum observed plasma concentration (Cmax), time to reach maximum observed plasma concentration (Tmax), lag time, elimination half-life (t½), apparent clearance (Cl/F), and apparent volume of distribution (V/F) up to week 4
? Participants receiving BG00012 who consent to participate in the PD/PK subset - PD parameters, derived from PD subset data analysis: changes in the nuclear factor (erythroid derived 2) like 2 activation pathway markers quinone 1 (NQO 1) and heme oxygenase 1 (HO 1)up to week 4
? Fatigue as measured by the Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue Scale scores (Multidimensional Fatigue Scale scores designed as a generic symptomspecific instrument to measure fatigue in patients with acute and chronic health conditions as well as healthy school and community populations.) up to week 96
? Quality of Life as measured by the PedsQL up to week 96
? Change from baseline to Week 96 in the Expanded Disability Status Scale (EDSS score; The EDSS measures the disability status of people with multiple sclerosis on a scale that ranges from 0 to 10. The first levels 1.0 to 4.5 refer to people with a high degree of ambulatory ability and the subsequent levels 5.0 to 9.5 refer to the loss of ambulatory ability. The range of main categories include (0) = normal neurologic exam? to (5) = ambulatory without aid or rest for 200 meters? disability severe enough to impair full daily activities? to (10) = death due to MS.) up to week 96
? Vital signs, electrocardiograms (ECGs) and changes in clinical laboratory data, including monitoring of liver function, renal function, hematologic and coagulation parameters up to week 96
? Other pre-specified outcomes

Los criterios de valoración secundarios de este estudio son los siguientes:
? Número de lesiones hiperintensas en T2 nuevas o aumentadas en RM cerebrales en las Semanas 24 y 96.
? La proporción de pacientes sin lesiones hiperintensas en T2 nuevas o aumentadas en RM cerebrales en las Semanas 24 y 48.
? Proporción de pacientes sin actividad nueva en la RM (es decir, sin lesiones potenciadas con gadolinio y sin lesiones en T2 nuevas o aumentadas en RM cerebrales) en las Semanas 24, 48 y 96.
? Tiempo hasta la primera recaída.
? Proporción de pacientes sin recaídas hasta la Semana 96.
? Tasa anual de recaídas en las Semanas 48 y 96.
? Incidencia de AA y acontecimientos adversos graves (AAG), incluyendo seguimiento posterior del rubor, náuseas, dolor abdominal y diarrea.
? Constantes vitales, electrocardiogramas (ECG) y variaciones en los datos de análisis clínicos, incluidos la función hepática y renal y los parámetros hematológicos y de coagulación.
? Parámetros FC de BG00012 provenientes del análisis de datos del subgrupo de FC: área bajo la curva de concentración plasmática y tiempo de 0 a 10 horas (ABC0-10), concentración plasmática máxima observada (Cmáx), tiempo hasta alcanzar la concentración plasmática máxima (Tmáx), periodo de latencia, semivida de eliminación (t½), aclaramiento aparente (Cl/F) y volumen de distribución aparente (V/F).
? Parámetros FD, provenientes del análisis de datos del subgrupo de FD: variaciones en los marcadores de la vía de activación del factor nuclear (derivado de eritroide 2) relacionado con el factor 2, la nicotinamida adenina dinucleótido fosfato [NAD(P)H] deshidrogenasa, quinona oxidorreductasa 1 (NQO-1) y la hemo oxigenasa 1 (HO-1).
? Medición del cansancio mediante las puntuaciones de la Escala multidimensional de cansancio según el PedsQL.
? Medición de la calidad de vida según el PedsQL.
? Cambio en la puntuación de la EDSS desde el inicio hasta la Semana 96.

E.5.2.1

Timepoint(s) of evaluation of this end point

The various secondary endpoints will be measured throughout the study as defined by the Schedule of Events. Specifically there are visits at Day 1 (baseline) weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, 96 and a safety follow-up visit at week 100. There is also a safety telephone call at week2.

Los diversos criterios de valoración secundarios se medirán a lo largo del estudio como lo define el Programa de Eventos. Especificamente hay visitas en el Día 1 (visita inicial) las semanas 4, 8, 12, 24, 36, 48, 60, 72, 84, 96 y una visita de seguimiento de seguridad en la semana 100. También hay una llamada telefónica seguridad en semana 2 .

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

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

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

Belgium

Canada

Croatia

Czech Republic

Denmark

France

Hungary

Israel

Italy

Poland

Portugal

Romania

Serbia

Spain

Sweden

Turkey

United Kingdom

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: The end of study is last subject, last visit for final collection of data for the primary outcome

LVLS: El fin del estudio es el último sujeto y la última visita para la recogida de datos para el resultado primario

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:

142

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

122

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

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

114

F.4.2.2

In the whole clinical trial

142

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)

Subjects who choose not to enroll in the extension study will have a Safety Follow-Up Visit 4 weeks after taking the final dose. Subjects who withdraw prematurely will complete the Early Withdrawal Visit and the Safety Follow-Up Visit 4 weeks after taking their final dose.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2016-03-02

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-06-05

P. End of Trial
P.	End of Trial Status	Ongoing

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Orphan Designation Number:
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