Clinical Trials Register

Summary
EudraCT Number:	2020-003447-28
Sponsor's Protocol Code Number:	XPF-009-302
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-09-01
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

2020-003447-28

A.3

Full title of the trial

An Open-Label Extension Study of XEN496 in Pediatric Subjects with KCNQ2 Developmental and Epileptic Encephalopathy

Estudio de prolongación sin enmascaramiento con XEN496 en niños que padecen encefalopatía epiléptica y del desarrollo por KCNQ2

A.3.1

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

Extension study in pediatric subjects with KCNQ2 Developmental and Epileptic Encephalopathy

Estudio de prolongación en niños que padecen encefalopatía epiléptica y del desarrollo por KCNQ2

A.3.2

Name or abbreviated title of the trial where available

EPIK OLE

A.4.1

Sponsor's protocol code number

XPF-009-302

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04912856

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	Xenon Pharmaceuticals Inc.
B.1.3.4	Country	Canada
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	Xenon Pharmaceuticals Inc.
B.4.2	Country	Canada
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Xenon Pharmaceuticals Inc.
B.5.2	Functional name of contact point	Medical Affairs
B.5.3	Address:
B.5.3.1	Street Address	200-3650 Gilmore Way
B.5.3.2	Town/ city	Burnaby, BC
B.5.3.3	Post code	V5G 4W8
B.5.3.4	Country	Canada
B.5.4	Telephone number	+1-604-484-3300
B.5.5	Fax number	+1-604-484-1336
B.5.6	E-mail	XenonCares@xenon-pharma.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/3/20/2332
D.3 Description of the IMP
D.3.1	Product name	Retigabine
D.3.2	Product code	XEN496
D.3.4	Pharmaceutical form	Capsule
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	RETIGABINE
D.3.9.1	CAS number	150812-12-7
D.3.9.2	Current sponsor code	XEN496
D.3.9.3	Other descriptive name	ezogabine
D.3.9.4	EV Substance Code	SUB10291MIG
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	3
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/3/20/2332
D.3 Description of the IMP
D.3.1	Product name	Retigabine
D.3.2	Product code	XEN496
D.3.4	Pharmaceutical form	Capsule
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	RETIGABINE
D.3.9.1	CAS number	150812-12-7
D.3.9.2	Current sponsor code	XEN496
D.3.9.3	Other descriptive name	ezogabine
D.3.9.4	EV Substance Code	SUB10291MIG
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	12
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/20/2332
D.3 Description of the IMP
D.3.1	Product name	Retigabine
D.3.2	Product code	XEN496
D.3.4	Pharmaceutical form	Capsule
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	RETIGABINE
D.3.9.1	CAS number	150812-12-7
D.3.9.2	Current sponsor code	XEN496
D.3.9.3	Other descriptive name	ezogabine
D.3.9.4	EV Substance Code	SUB10291MIG
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	32
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	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

KCNQ2 Developmental and Epileptic Encephalopathy

Encefalopatía epiléptica y del desarrollo por KCNQ2

E.1.1.1

Medical condition in easily understood language

KCNQ2 Developmental and Epileptic Encephalopathy

Encefalopatía epiléptica y del desarrollo por KCNQ2

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10077380
E.1.2	Term	Epileptic encephalopathy
E.1.2	System Organ Class	10029205 - Nervous system 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

To assess the long-term safety and tolerability of XEN496 in pediatric subjects with KCNQ2-DEE who had participated in the primary study (XPF-009-301)

Evaluar la seguridad y la tolerabilidad a largo plazo del XEN496 en niños con EED-KCNQ2 que participaron en el estudio principal (XPF-009-301).

E.2.2

Secondary objectives of the trial

• To evaluate the intra-subject efficacy of XEN496 in reducing seizure frequency compared to prior placebo treatment in pediatric subjects with KCNQ2-DEE who were previously randomized to receive placebo in the primary double-blind Phase 3 study (XPF-009-301)
• To assess the long-term effect of XEN496 on seizure reduction in pediatric subjects treated with XEN496
• To evaluate CaGI-S and CaGI-C scores in pediatric subjects with KCNQ2-DEE
• To assess neurocognitive development and behavior after dosing with XEN496 in pediatric subjects with KCNQ2-DEE
• To evaluate the investigator’s global impression of the change in the subject’s overall condition, assessed using the CGI-C
• To evaluate the use of rescue medication and change in background antiseizure medications
• To assess the impact of XEN496 on the quality of life of caregivers and subjects with KCNQ2-DEE
• To evaluate the plasma concentrations of ezogabine and N-acetyl metabolite of ezogabine (NAMR)

•Evaluar la eficacia intraindividual del XEN496 en la disminución de la frecuencia de las convulsiones en comparación con el tratamiento anterior de placebo en niños con EED-KCNQ2 que fueron aleatorizados anteriormente para recibir placebo en el estudio principal en fase III con enmascaramiento doble (XPF 009-301)•Evaluar el efecto a largo plazo del XEN496 en la disminución de las convulsiones en niños tratados con XEN496•Evaluar las puntuaciones de la Impresión global de la intensidad por parte del cuidador (CaGI-S) y la Impresión global del cambio por parte del cuidador (CaGI-C) en niños que padecen EED-KCNQ2•Evaluar el comportamiento y el desarrollo neurocognitivo tras administrar XEN496 en niños que padecen EED-KCNQ2•Evaluar la impresión global del cambio por parte del investigador en el estado global del paciente, determinada mediante la escala Impresión global clínica del cambio (CGI-C). Ver objetivos en inglés.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject completed participation in the primary study, XPF-009-301. A subject who withdraws from the primary study due to meeting protocol-specified worsening criteria will be considered as having completed participation in the primary study.
2. The caregiver is willing and able to be compliant with diary completion, visit schedule, and study drug administration.
3. Subject’s caregiver achieved a minimum of 85% compliance with daily diary completion during both baseline and the double-blind period of the primary study.

1. El paciente completó la participación según lo definido en el estudio principal, XPF 009-301. Se considerará que los pacientes que se retiren del estudio principal por cumplir los criterios de empeoramiento especificados en el protocolo han completado la participación en el estudio principal.
2. El cuidador está dispuesto y puede cumplir con los requisitos de cumplimentación del diario, del calendario de visitas y de la administración del fármaco del estudio.
3. El cuidador del paciente alcanzó un cumplimiento mínimo del 85 % en la cumplimentación del diario durante los periodos inicial y con enmascaramiento doble del estudio principal.

E.4

Principal exclusion criteria

1. Any AE or SAE during Study XPF-009-301, which in the opinion of the investigator and sponsor’s medical monitor, would preclude the subject’s entry into the OLE.
2. A clinically significant condition or illness, or symptoms other than those resulting from KCNQ2-DEE, present at screening/baseline that, in the opinion of the investigator, would pose a risk to the subject if s/he were to enter the study.
3. Any conditions that were specified as exclusion criteria in the primary study (XPF 009-301).
4. It is anticipated that the subject will require treatment with at least 1 of the disallowed medications during the study.
5. Any change in cardiac rhythm or atrioventricular conduction in the primary study that, in the investigator’s opinion, is a significant risk to subject safety.

1. Cualquier acontecimiento adverso (AA) o acontecimiento adverso grave (AAG) durante el estudio XPF-009-301 que, en opinión del investigador y del monitor médico del promotor, impediría que el paciente comenzara la PSE.
2. Una enfermedad o afección clínicamente significativa, o síntomas diferentes a los causados por la EED-KCNQ2, presentes en la selección o el inicio que, en opinión del investigador, supondría un riesgo para el paciente si participara en el estudio.
3. Cualquier afección especificada como criterio de exclusión en el estudio principal (XPF 009-301).
4. Previsión de que el paciente requerirá tratamiento con al menos uno de los medicamentos prohibidos durante el estudio.
5. Cualquier cambio en el ritmo cardíaco o la conducción auriculoventricular en el estudio principal que, en opinión del investigador, supone un riesgo significativo para la seguridad del paciente.

E.5 End points

E.5.1

Primary end point(s)

Severity and frequency of AEs and serious adverse events (SAEs); clinically significant changes in laboratory tests, vital signs, ECGs, physical and neurologic examinations, urological examinations, and ophthalmology examinations.

• Intensidad y frecuencia de los AA y los AAG; cambios clínicamente significativos en las pruebas analíticas, constantes vitales, electrocardiogramas (ECG), exploraciones físicas y neurológicas, exploraciones urológicas y exploraciones oftalmológicas

E.5.1.1

Timepoint(s) of evaluation of this end point

From Screening/Baseline to the End of the Study

Desde la selección/basal hasta el final del estudio.

E.5.2

Secondary end point(s)

1. Change in monthly countable motor seizure frequency, comparing the first 15 weeks of XEN496 treatment in the OLE study to the seizure frequency reported during treatment in the preceding primary study (XPF-009-301), among only those subjects who were randomized to the placebo arm in the primary study
2. Change from pre-randomization baseline in the previous study over time based on response categories (<25%, 25 to <50%, 50 to <75%, 75 to <100%, 100%), based on estimated seizure frequency every 3 months during the OLE period.
3. Percent change from baseline in countable motor seizure frequency, relative to prerandomization baseline of the previous study (XPF-009-301), assessed over time every 3 months during the OLE.
4. Percent change from baseline in countable motor seizure frequency, relative to prerandomization baseline of the previous study (XPF-009-301), for every 3 months based on combined data from both XPF-009-301 and the OLE, by the treatment group in the previous study.
5. Change over time in CaGI-S scores for the subject’s seizures and overall condition.
6. Change over time in CaGI-C scores for the subject in the following domains: overall condition, seizures, behavior, alertness, motor skills, visual function, and communication.
7. Change over time in neurocognitive development based on the Bayley Scales of Infant and Toddler Development III.
8. Change over time in adaptive behavior based on the Adaptive Behavior Assessment System, Third Edition (ABAS-3)
9. Change over time in CGI-C scores for the subject’s seizures and overall condition
10. Use of rescue medication.
11. Use of concomitant medications and treatments used for seizure control
12.Change in quality of life of subjects with KCNQ2-DEE, based on the Pediatric Quality of Life Inventory.
13. Change in quality of life of subjects with KCNQ2-DEE, based on the Pediatric Quality of Life Inventory, Family Impact scale
14. Plasma concentrations of ezogabine and NAMR (for subjects treated in the OLE study only)

• Cambio en la frecuencia de las convulsiones motrices contadas mensualmente, comparando las 15 primeras semanas de tratamiento con XEN496 en el estudio de PSE con la frecuencia de convulsiones notificada durante el tratamiento en el estudio principal anterior (XPF-009-301), únicamente en aquellos pacientes que fueron aleatorizados al grupo de placebo en el estudio principal (XPF 009-301).
• Cambio a lo largo del tiempo desde el valor inicial previo a la aleatorización del estudio anterior, en función de las categorías de respuesta (<25 %, de 25 a <50 %, de 50 a <75 %, de 75 a <100 % y 100 %), basado en la frecuencia estimada de las convulsiones cada 3 meses durante el periodo de PSE.
• Cambio porcentual desde el valor inicial en la frecuencia de las convulsiones motrices contadas, en relación con el valor inicial previo a la aleatorización del estudio anterior (XPF 009 301), determinado a lo largo del tiempo cada 3 meses durante la PSE.
• Cambio porcentual desde el valor inicial en la frecuencia de las convulsiones motrices contadas, en relación con el valor inicial previo a la aleatorización del estudio anterior (XPF 009 301), para cada 3 meses a partir de los datos combinados tanto del XPF-009-301 como la PSE, por grupo de tratamiento del estudio anterior.
• Cambio a lo largo del tiempo en las puntuaciones de la CaGI-S para las convulsiones y el estado general del paciente.
• Cambio a lo largo del tiempo en las puntuaciones de la CaGI-C para el paciente en los dominios siguientes: estado general, convulsiones, comportamiento, estado de alerta, habilidades motrices, función visual y comunicación.

• Cambio a lo largo del tiempo en el desarrollo neurocognitivo según las escalas de desarrollo infantil de Bayley III (Bayley Scales of Infant and Toddler Development III).
• Cambio a lo largo del tiempo en la conducta adaptativa según el sistema de evaluación de la conducta adaptativa, tercera edición (Adaptive Behavior Assessment System, Third Edition).
• Cambio a lo largo del tiempo en las puntuaciones de la CGI-C para las convulsiones y el estado general del paciente.
• Uso de medicación de rescate.
• Uso de todos los medicamentos concomitantes, incluidos los tratamientos administrados para controlar las convulsiones.
• Cambio en la calidad de vida de los pacientes con EED-KCNQ2, según el inventario de la calidad de vida pediátrica (Pediatric Quality of Life Inventory).
• Cambio en la calidad de vida de los pacientes con EED-KCNQ2, según la escala de repercusión familiar del inventario de la calidad de vida pediátrica (Pediatric Quality of Life Inventory, Family Impact Scale).
• Concentraciones plasmáticas de ezogabina y NAMR (en los pacientes tratados únicamente en el estudio de PSE).

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Screening to first 15 weeks (up to Visit 10)
2. From screening through to the end of the study
3. From screening through to the end of the study
4. From screening through to the end of the study
5. Study days: 1, 24, 67, 109, 182, 273 and 364
6. Study days: 24, 67, 109, 182, 273 and 364
7. Study days: 1, 109 and 364
8. Study days: 1, 109, 182 and 364
9. Study days: 67, 109, 182 and 364
10. From screening through to the end of the study
11. From screening through to the end of the study
12. Study days: 1, 67, 109, 182 and 364
13. Study days: 1, 67, 109, 182 and 364
14. Study days: 1, 16, 32, 67, 109, 182 and 364

1. Selección hasta las primeras 15 semanas (hasta la visita 10)
2. Desde la selección hasta el final del estudio
3. Desde la selección hasta el final del estudio
4. Desde la selección hasta el final del estudio
5. Días de estudio: 1, 24, 67, 109, 182, 273 y 364
6. Días de estudio: 24, 67, 109, 182, 273 y 364
7. Días de estudio: 1, 109 y 364
8. Días de estudio: 1, 109, 182 y 364
9. Días de estudio: 67, 109, 182 y 364
10. Desde la selección hasta el final del estudio
11. Desde la selección hasta el final del estudio
12. Días de estudio: 1, 67, 109, 182 y 364
13. Días de estudio: 1, 67, 109, 182 y 364
14. Días de estudio: 1, 16, 32, 67, 109, 182 y 364

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

Yes

E.6.13.1

Other scope of the trial description

long-term;
tolerability

largo plazo
tolerabilidad

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.8.2.4

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

China

United States

Belgium

France

Germany

Italy

Poland

Spain

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

The end of the study is defined as completion of the last visit for the last subject in this study.

El final del estudio se define como la finalización de la última visita del último sujeto de este estudio.

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

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

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)

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

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

parent(s)/guardian(s) agreement for their child to participate in the study is required

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)

None

Ninguno

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

2022-01-12

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-10-13

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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