Clinical Trials Register

Summary
EudraCT Number:	2020-003447-28
Sponsor's Protocol Code Number:	XPF-009-302
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-08-27
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

France - ANSM

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

Étude d’extension en ouvert évaluant le XEN496 chez des patients pédiatriques présentant une encéphalopathie épileptique et développementale associée à 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

Étude d’extension chez des sujets pédiatriques présentant une encéphalopathie épileptique et développementale associée à 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

Encéphalopathie épileptique et troubles du développement liés à KCNQ2

E.1.1.1

Medical condition in easily understood language

KCNQ2 Developmental and Epileptic Encephalopathy

Encéphalopathie épileptique et troubles du développement liés à 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)

Évaluer la sécurité et la tolérance à long terme du XEN496 chez des patients pédiatriques souffrant d’EED-KCNQ2 ayant participé à l’étude principale (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)

• Évaluer l’efficacité intra-patient du XEN496 en termes de réduction de la fréquence des crises épileptiques en comparaison à un traitement antérieur par placebo chez des patients pédiatriques atteints d’EED-KCNQ2 précédemment randomisés dans le groupe placebo de l’étude principale de phase III en double aveugle (XPF 009-301).
• Évaluer l’effet à long terme du XEN496 sur la réduction des crises épileptiques chez des patients pédiatriques traités par XEN496.
• Évaluer les scores à l’échelle d’impression globale de la sévérité rapportée par l’aidant (CaGI-S) et à l’échelle d’impression globale du changement rapportée par l’aidant (CaGI-C) chez des patients pédiatriques atteints d’EED-KCNQ2.
• Évaluer le développement neurocognitif et le comportement après l’administration de XEN496 chez des patients pédiatriques atteints d’EED-KCNQ2.

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. Le patient doit avoir terminé sa participation l’étude principale XPF 009-301 comme défini dans le protocole. Si un patient est retiré de l’étude principale car il répond aux critères d’aggravation définis dans le protocole, il sera considéré comme ayant terminé sa participation à l’étude principale.
2. L’aidant du patient doit accepter et être en mesure de respecter les exigences en termes de consignation des données dans le journal, de visites et d’administration du médicament de l’étude.
3. L’aidant du patient doit avoir obtenu un score d’observance de 85 % minimum concernant la consignation quotidienne des données dans le journal lors des périodes d’inclusion et de traitement en double aveugle de l’étude principale.

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. Le patient a présenté un événement indésirable (EI) ou un événement indésirable grave (EIG) durant l’étude XPF-009-301 qui, de l’avis de l’investigateur et du moniteur médical du promoteur, contre-indique la participation du patient à l’étude d’extension en ouvert.
2. Une pathologie ou une affection cliniquement significative ou des symptômes autres que ceux associés à l’EED-KCNQ2 sont observés à la sélection/référence et, de l’avis de l’investigateur, compromettraient la sécurité du patient en cas de participation à l’étude.
3. Le patient présente une pathologie constituant un critère d’exclusion d’après le protocole de l’étude principale (XPF 009-301).
4. L’état du patient nécessitera probablement l’administration d’un ou plusieurs médicaments interdits pendant l’étude.
5. Une modification du rythme cardiaque ou de la conduction auriculoventriculaire a été observée pendant l’étude principale et, de l’avis de l’investigateur, présente un risque significatif pour la sécurité du patient.

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.

Gravité et fréquence des EI et des événements indésirables graves (EIG) ; changements cliniquement significatifs au niveau des analyses de laboratoire, constantes vitales, ECG, examens physique et neurologique, examen urologique et examen ophtalmologique.

E.5.1.1

Timepoint(s) of evaluation of this end point

From Screening/Baseline to the End of the Study

De la sélection/référence à la fin de l’étude

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)

1. Modification de la fréquence mensuelle des crises motrices pouvant être recensées, comparaison des 15 premières semaines du traitement par XEN496 dans l’étude OLE à la fréquence des crises rapportée pendant le traitement dans l’étude primaire précédente (XPF-009-301), uniquement parmi les sujets qui ont été randomisés dans le bras placebo dans l’étude principale
2. Modification au cours du temps par rapport à la référence pré-randomisation dans l’étude précédente, sur base des catégories de réponse (<25 %, 25 à <50 %, 50 à <75 %, 75 à <100 %, 100 %), sur base de la fréquence estimée des crises tous les 3 mois pendant la période OLE.
3. Pourcentage de modification par rapport à la référence, de la fréquence des crises motrices pouvant être recensées, par rapport à la référence pré-randomisation de l’étude précédente (XPF-009-301), évalué au cours du temps tous les 3 mois pendant l’OLE.
4. Pourcentage de modification par rapport à la référence, de la fréquence des crises motrices pouvant être recensées, par rapport à la référence pré-randomisation de l’étude précédente (XPF-009-301), tous les 3 mois sur la base des données combinées de XPF-009-301 et de l’OLE, par groupe de traitement dans l’étude précédente.
5. Modification au cours du temps des scores CaGI-S pour les crises et l’état général du sujet.
6. Modification au cours du temps des scores CaGI-S du sujet dans les domaines suivants : état général, crises, comportement, vigilance, capacités motrices, fonction visuelle et communication.
7. Modification au cours du temps du développement neurocognitif sur base des échelles de développement de Bayley du nourrisson et du jeune enfant III (Bayley Scales of Infant and Toddler Development III).
8. Modification au cours du temps du comportement adaptatif sur base du système d’évaluation du comportement adaptatif (Adaptive Behavior Assessment System), troisième édition (ABAS-3)
9. Modification au cours du temps des scores CGI-S pour les crises et l’état général du sujet.

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

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

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.

La fin de l’étude est atteinte une fois réalisée la dernière visite du dernier sujet de l’étude.

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

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

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

2021-10-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-09-21

P. End of Trial
P.	End of Trial Status	Completed

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