Clinical Trials Register

Summary
EudraCT Number:	2016-002804-14
Sponsor's Protocol Code Number:	ZX008-1503
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:	2016-10-26
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

2016-002804-14

A.3

Full title of the trial

An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults with Dravet Syndrome

Ensayo de ampliación sin enmascaramiento para evaluar la seguridad a largo plazo de la solución oral ZX008 (clorhidrato de fenfluramina) como tratamiento complementario en niños y adultos jóvenes con síndrome de Dravet

A.3.1

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

Clinical Study to evaluate the safety and effectiveness of Fenfluramine as adjunct therapy in children and young adults with Dravet Syndrome

Ensayo clínico para evaluar la seguridad y la eficacia de Fenfluramina como tratamiento complementario en niños y adultos jóvenes con síndrome de Dravet.

A.4.1

Sponsor's protocol code number

ZX008-1503

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02823145

A.5.4

Other Identifiers

Name:

IND Number

Number:

125797

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	Zogenix International 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	Zogenix, 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	Zogenix, Inc.
B.5.2	Functional name of contact point	AJ Acker
B.5.3	Address:
B.5.3.1	Street Address	5858 Horton Street, Suite 455
B.5.3.2	Town/ city	Emeryville
B.5.3.3	Post code	CA 94608
B.5.3.4	Country	United States
B.5.4	Telephone number	+1510550 8331
B.5.5	Fax number	+1510550 8341
B.5.6	E-mail	ajacker@zogenix.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/3/13/1219
D.3 Description of the IMP
D.3.1	Product name	Fenfluramine hydrochloride (red)
D.3.2	Product code	ZX008
D.3.4	Pharmaceutical form	Oral solution
D.3.4.1	Specific paediatric formulation	Yes
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	FENFLURAMINE HYDROCHLORIDE
D.3.9.1	CAS number	404-82-0
D.3.9.2	Current sponsor code	ZX008
D.3.9.3	Other descriptive name	FENFLURAMINE HYDROCHLORIDE (red)
D.3.9.4	EV Substance Code	SUB02115MIG
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	2.5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	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/3/13/1219
D.3 Description of the IMP
D.3.1	Product name	Fenfluramine hydrochloride (colorless)
D.3.2	Product code	ZX008
D.3.4	Pharmaceutical form	Oral solution
D.3.4.1	Specific paediatric formulation	Yes
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	FENFLURAMINE HYDROCHLORIDE
D.3.9.1	CAS number	404-82-0
D.3.9.2	Current sponsor code	ZX008
D.3.9.3	Other descriptive name	FENFLURAMINE HYDROCHLORIDE (colorless)
D.3.9.4	EV Substance Code	SUB02115MIG
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	2.5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.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

Dravet's syndrome

Síndrome de Dravet

E.1.1.1

Medical condition in easily understood language

Dravet's syndrome

Síndrome de Dravet

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	19.0
E.1.2	Level	LLT
E.1.2	Classification code	10073682
E.1.2	Term	Dravet syndrome
E.1.2	System Organ Class	100000004850

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

•To assess the long-term safety and tolerability of ZX008.

•Evaluar la seguridad y tolerabilidad de ZX008 a largo plazo

E.2.2

Secondary objectives of the trial

•To assess the effect of ZX008 relative to the pre-ZX008 baseline on the following effectiveness measures:
-The change in the frequency of convulsive seizures
-The proportion of subjects who achieve a ≥40%, ≥50%, and ≥75% reduction in convulsive seizure frequency
-The longest convulsive seizure-free interval
-The percentage of convulsive seizure-free days
-The non-convulsive seizure frequency
-The convulsive + non-convulsive seizure frequency.
•To estimate the incidence of the following on subjects receiving ZX008:
-Use of rescue medication
-Hospitalization to treat seizures
-Status epilepticus.

Please refer to the Clinical Study Protocol, Section 2 Study Objectives and Endpoints for complete list of Secondary Endpoints

•Evaluar el efecto de ZX008 en relación con el período inicial previo a ZX008 en los siguientes parámetros de la efectividad:
-El cambio en la frecuencia de las crisis convulsivas
-El porcentaje de pacientes que logran una reducción de ≥ 40 %, ≥ 50 % y ≥ 75 % con respecto a la frecuencia de las crisis convulsivas
-El intervalo más largo sin crisis convulsivas
-El porcentaje de días sin crisis convulsivas
-La frecuencia de las crisis no convulsivas
-La frecuencia de las crisis convulsivas + no convulsivas
•Calcular la incidencia de los siguientes aspectos en pacientes que reciban ZX008:
-Uso de medicación de rescate
-Hospitalización para tratar las convulsiones
-Estado epiléptico

Por favor, refiéranse al Protocolo , sección 2, objetivos del estudio y criterios de valoración para la lista completa de los criterios de valoración secundarios

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject is aged 2 to 18 years inclusive, as of the day of the core study Screening Visit. Subject is male or non-pregnant, non-lactating female. Female subjects of childbearing potential must not be pregnant or breast-feeding. Female subjects of childbearing potential must have a negative urine pregnancy test. Subjects of childbearing or child-fathering potential must be willing to use medically acceptable forms of birth control, which includes abstinence, while being treated on this study and for 30 days after the last dose of study drug.
2. Subject has satisfactorily completed the core study in the opinion of the investigator and the sponsor.
3. Subject has documented medical history to support a clinical diagnosis of Dravet syndrome, where convulsive seizures are not completely controlled by current antiepileptic drugs.
4. Subject has been informed of the nature of the study and informed consent has been obtained from the legally responsible parent/guardian.
5. Subject has provided assent in accordance with IRB/IEC requirements, if capable.
6. Subject’s caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability.
7. Subject’s parent/caregiver has been compliant with diary completion during the core study, in the opinion of the investigator (eg, at least 90% compliant).
8. Subjects entering from study ZX008-1504 must be receiving a therapeutically relevant and stable dose of clobazam, valproic acid, and stiripentol (Cohort 1 Dose Regimen 3 and Cohort 2 only) for at least 4 weeks prior to screening and are expected to remain stable throughout the study.

1.El paciente tiene entre 2 y 18 años, ambos inclusive, en la fecha de la visita de selección del ensayo principal. El paciente es un hombre o una mujer que no está embarazada ni en período de lactancia. Las participantes de sexo femenino en edad fértil no deben estar embarazadas ni en período de lactancia. Las participantes en edad fértil deben tener una prueba de embarazo en orina negativa. Los pacientes con capacidad para concebir o engendrar hijos deben estar dispuestos a utilizar métodos médicamente aceptables de regulación de la natalidad, lo que incluye la abstinencia, mientras se reciba tratamiento en este ensayo y hasta 30 días después de la última dosis del fármaco del ensayo.
2.El paciente ha completado satisfactoriamente el ensayo principal en opinión del investigador y el promotor.
3.El paciente tiene antecedentes médicos documentados que respalden el diagnóstico clínico de síndrome de Dravet en el que las crisis convulsivas no estén del todo controladas con los antiepilépticos actuales.
4.Se ha informado al paciente del carácter del ensayo y se ha obtenido el consentimiento informado por parte del progenitor o tutor legalmente responsable.
5.El paciente ha otorgado su asentimiento de acuerdo con los requisitos del Consejo de Revisión Institucional /Comité Ético de Investigación Clínica (CEIC), si está capacitado.
6.El cuidador del paciente está dispuesto y tiene capacidad para rellenar el diario y cumplir el calendario de visitas y las normas para la contabilidad del fármaco del ensayo.
7.Paciente cuyo progenitor o cuidador haya cumplimentado debidamente el diario durante el ensayo principal en opinión del investigador (p. ej., cumplimiento de al menos el 90 %).
8.Los pacientes que accedan desde el ensayo ZX008-1504 deben estar recibiendo una dosis terapéuticamente relevante y estable de clobazam, ácido valproico y estiripentol (pauta posológica 3 de la cohorte 1 y cohorte 2 únicamente) durante al menos 4 semanas antes de la selección. Se prevé que los pacientes permanezcan estables a lo largo del ensayo.

E.4

Principal exclusion criteria

1. Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study medication.
2. Subject has current or past history of cardiovascular or cerebrovascular disease, myocardial infarction or stroke.
3. Subject with current cardiac valvulopathy or pulmonary hypertension that the investigator, parent, IPCAB, IDSMC, or sponsor deems clinically significant and warrants discontinuation of study medication.
4. Subject has current or recent history of Anorexia Nervosa, bulimia, or depression within the prior year that required medical treatment or psychological treatment for a duration greater than 1 month.
5. Subject is at imminent risk of self-harm or harm to others, in the investigator’s opinion, based on clinical interview and responses provided on the Columbia-Suicide Severity Rating Scale (C-SSRS). Subjects must be excluded if they report suicidal behavior as measured by the C-SSRS Since Last Visit, which includes suicidal ideation with intent and plan (Item #5). If a subject reports suicidal ideation on Item 4 without specific plan, and the investigator feels that the subject is appropriate for the study considering the potential risks, the investigator must document appropriateness for inclusion, and discuss with the parent/caregiver to be alert to mood or behavioral changes, especially around times of dose adjustment.
6. Subject has a current or past history of glaucoma.
7. Subject has moderate or severe hepatic impairment. Asymptomatic subjects with mild hepatic impairment (elevated liver enzymes <3x upper limited of normal [ULN] and/or elevated bilirubin <2x ULN) may be entered into the study after review and approval by the Medical Monitor in conjunction with the sponsor, in consideration of comorbidities and concomitant medications.
8. Subject is receiving concomitant therapy with: centrally-acting anorectic agents; monoamine-oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; atomoxetine, or other centrally-acting noradrenergic agonist; cyproheptadine, and/or cytochrome P450 (CYP) 2D6/3A4/2B6 inhibitors/substrates (see Appendix 1). (Note: Short-term medication requirements will be handled on a per case basis by the Medical Monitor.)
9. Subject is currently taking carbamazepine, oxcarbamazepine, eslicarbazepine, phenobarbital, or phenytoin, or has taken any of these within the past 30 days, as maintenance therapy.
10. For subjects entering from core studies ZX008-1501, ZX008-1502, or ZX008-1504 (Cohort 1/Dose Regimens 1 &2): Subject is currently receiving or has received stiripentol in the past 21 days prior to core study Visit 1.
11. Subject is unwilling to refrain from large or daily servings of grapefruits and/or Seville oranges, and their juices beginning with Visit 1 and throughout the study.
12. Subject has positive result on urine THC Panel or whole blood CBD at Visit 1.
13. Subject is unwilling or unable to comply with scheduled visits, drug administration plan, laboratory tests, other study procedures, and study restrictions.
14. Subject has a clinically significant condition, or has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject.

1.Pacientes con hipersensibilidad conocida a la fenfluramina o a alguno de los excipientes de la medicación del ensayo.
2.Pacientes con enfermedades cardiovasculares o cerebrovasculares en curso o previas, infarto de miocardio o accidente cerebrovascular.
3.Pacientes con valvulopatía cardíaca o hipertensión pulmonar en curso que el investigador, progenitor, CAICP, CIVDS o promotor consideren de importancia clínica y que justifique la interrupción de la medicación del ensayo.
4.Pacientes con anorexia nerviosa, bulimia o depresión en curso o previas durante el año anterior que hayan requerido tratamiento médico o psicológico durante más de 1 mes.
5.Pacientes que, en opinión del investigador, se hallen en peligro inminente de autolesionarse o de lesionar a otras personas, según una entrevista clínica y las respuestas proporcionadas en la Escala de Evaluación del Riesgo de Suicidio de la Universidad de Columbia (C-SSRS). Debe excluirse a los pacientes que declaren haber tenido conductas suicidas según la C-SSRS desde la última visita, lo que incluye ideas suicidas con intención y planificación (ítem n.º 5). Si un paciente declara ideas suicidas en el ítem 4 sin un plan específico y el investigador considera que el paciente es adecuado para el ensayo después de sopesar los posibles riesgos, el investigador debe documentar la idoneidad para la inclusión e indicar al progenitor o al cuidador que se mantenga alerta ante cualquier cambio del estado de ánimo o conductual, especialmente alrededor de los momentos de ajuste de las dosis.
6.Pacientes con glaucoma en curso o previo.
7.Pacientes con insuficiencia hepática moderada o grave. Los pacientes asintomáticos con insuficiencia hepática leve (enzimas hepáticas elevadas < 3 veces el LSN o bilirrubina elevada < 2 veces el LSN) podrán ingresar en el ensayo tras la revisión y aprobación del monitor médico junto con el promotor, teniendo en cuenta las comorbilidades y los medicamentos concomitantes.
8.Pacientes que estén recibiendo tratamiento concomitante con: anorexígenos de acción central; inhibidores de la monoaminooxidasa; cualquier compuesto de acción central con niveles clínicamente apreciables de propiedades agonistas o antagonistas de la serotonina, incluida la inhibición de la recaptación de serotonina; atomoxetina u otro agonista noradrenérgico de acción central; ciproheptadina o inhibidores/sustratos del citocromo P450 (CYP) 2D6/3A4/2B6 (véase el Apéndice 1). (Nota: El monitor médico estudiará caso por caso los requisitos relativos a los tratamientos farmacológicos de corta duración).
9.Pacientes que estén tomando carbamazepina, oxcarbamazepina, eslicarbazepina, fenobarbital o fenitoína, o bien que hayan tomado alguno de estos medicamentos durante los últimos 30 días como terapia de mantenimiento.
10.Para pacientes que accedan desde los ensayos principales ZX008-1501, ZX008-1502 o ZX008-1504 (cohorte 1/pautas posológicas 1 y 2): pacientes que estén recibiendo o que hayan recibido estiripentol en los 21 días anteriores a la visita 1 del ensayo principal.
11.Pacientes que no estén dispuestos a renunciar a raciones grandes o diarias de pomelos o naranjas amargas, así como a sus zumos, desde la visita 1 y a lo largo del ensayo.
12.Pacientes con resultados positivos en las pruebas de tetrahidrocanabinol (THC) en orina o de canabidiol (CBD) en sangre completa en la visita 1.
13.Pacientes que no estén dispuestos o no sean capaces de cumplir con las visitas programadas, el plan de administración del fármaco, las pruebas analíticas, otros procedimientos del ensayo y las restricciones del ensayo.
14.Pacientes con alguna afección de importancia clínica o que hayan sufrido síntomas clínicamente relevantes o enfermedades de importancia clínica en las 4 semanas previas a la visita 1, distintas de la epilepsia, que puedan afectar negativamente a la participación en el ensayo, la recogida de datos del ensayo o suponer un riesgo para el paciente.

E.5 End points

E.5.1

Primary end point(s)

The effectiveness endpoints of the study are:
•Number of seizures by type
•Convulsive seizure-free interval
•Clinical Global Impression – Improvement as assessed by parent/caregiver
•Clinical Global Impression – Improvement as assessed by principal investigator
•QOLCE to measure changes in quality of life of the subject
•PedsQL to measure changes in quality of life of the subject
•QoL of parent/caregiver using the EQ-5D-5L scale
•Affective symptoms of parent/caregiver using the HADS (in parents/caregivers from core studies ZX008-1501 and ZX008-1502 only)
•Duration of prolonged seizures (seizure type that, during pre-Z008 baseline, had duration >2 minutes)
•Number of episodes of status epilepticus
•Number of instances of rescue medication use and number of doses
•Number of inpatient hospital admissions due to seizures

Los criterios de valoración de la efectividad del ensayo son:
•Número de crisis por tipo
•Intervalo sin crisis convulsivas
•Impresión Clínica Global: Mejoría, determinada por el progenitor o el cuidador
•Impresión Clínica Global: Mejoría, determinada por el investigador principal
•QOLCE para medir los cambios producidos en la calidad de vida del paciente
•PedsQL para medir los cambios producidos en la calidad de vida del paciente
•CdV del progenitor o cuidador mediante la escala EQ-5D-5L
•Síntomas afectivos del progenitor o cuidador mediante la HADS (solamente en progenitores o cuidadores de los ensayos principales ZX008-1501 y ZX008-1502)
•Duración de las crisis prolongadas (tipo de convulsiones que, durante el período inicial previo a ZX008, haya tenido una duración de > 2 minutos)
•Número de episodios de estatus epiléptico
•Número de ocasiones en que se ha usado algún medicamento de rescate y número de dosis
•Número de ingresos hospitalarios por crisis.

E.5.1.1

Timepoint(s) of evaluation of this end point

Please refer to study protocol

Por favor refieranse al protocolo

E.5.2

Secondary end point(s)

The safety endpoints of the study are:
•AEs
•Laboratory safety (hematology, chemistry, urinalysis)
•Vital signs (blood pressure, heart rate, temperature, and respiratory rate)
•Physical examination
•Neurological examination
•12-lead ECGs
•Doppler ECHOs
•Body weight
•BRIEF to measure cognition.

For subjects from core study ZX008-1504 only, the exploratory endpoints of this study are:
•Health and social care resource use, including GP visits, speech and language, occupational and physical therapy, in addition to acute hospital and institutional length of stay, loss of work, etc
•Sleep quality
•Mealtime behavior
•Karolinska Sleepiness Scale to measure the effect of study medication on sleepiness

Los criterios de valoración de la seguridad del ensayo son:
•AA
•Seguridad de laboratorio (hematología, bioquímica, análisis de orina)
•Constantes vitales (tensión arterial, frecuencia cardíaca, temperatura y frecuencia respiratoria)
•Exploración física
•Exploración neurológica
•ECG de 12 derivaciones
•ECO Doppler
•Peso corporal
•BRIEF para medir la cognición.

Para los sujetos del ensayo principal ZX008- 1504 exclusivamente, los criterios de valoración exploratorios de este ensayo son:
•Uso de recursos de asistencia sanitaria y seguridad social, incluidas las visitas al médico de atención primaria, la logopedia, fisioterapia y terapia física y ocupacional, además de la duración del ingreso hospitalario o institucional agudo, pérdida de trabajo, etc.
•Calidad del sueño
•Conducta durante las comidas
•Escala de Somnolencia de Karolinska para medir el efecto de la medicación del ensayo en la somnolencia.

E.5.2.1

Timepoint(s) of evaluation of this end point

Please refer to study protocol

Por favor refieranse al protocolo

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

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

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

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

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

Belgium

Canada

Denmark

France

Germany

Italy

Norway

Spain

Sweden

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

Última visita del último paciente

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:

310

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:

217

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)

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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2016-10-26.

Yes

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Children and adolescents

Niños y adolescentes

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

193

F.4.2.2

In the whole clinical trial

310

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)

standard care

Tratamiento convencional

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

2016-12-23

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-12-01

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

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