Clinical Trials Register

Summary
EudraCT Number:	2020-003447-28
Sponsor's Protocol Code Number:	XPF-009-302
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-09-08
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

Italy - Italian Medicines Agency

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

Studio di estensione in aperto di XEN496 in soggetti pediatrici con
encefalopatia epilettica e dello sviluppo correlata a 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

Studio di Estensione in soggetti pediatrici con
encefalopatia epilettica e dello sviluppo correlata a 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	+16044843300
B.5.5	Fax number	+1044841336
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	retigabina
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	RETIGABINA
D.3.9.1	CAS number	150812-12-7
D.3.9.2	Current sponsor code	XEN496
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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	Retigabina
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	RETIGABINA
D.3.9.1	CAS number	150812-12-7
D.3.9.2	Current sponsor code	XEN496
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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	retigabina
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	RETIGABINA
D.3.9.1	CAS number	150812-12-7
D.3.9.2	Current sponsor code	XEN496
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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

encefalopatia epilettica e dello sviluppo correlata a KCNQ2

E.1.1.1

Medical condition in easily understood language

KCNQ2 Developmental and Epileptic Encephalopathy

encefalopatia epilettica e dello sviluppo correlata a 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)

Valutare la sicurezza a lungo termine e la tollerabilità di XEN496 in soggetti pediatrici con KCNQ2-DEE che hanno partecipato allo studio primario (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)

Valutare:
- efficacia nello stesso sogg di XEN496 nel ridurre la frequenza di convulsioni rispetto al precedente trattamento con placebo, in sogg pediatrici con KCNQ2-DEE che erano stati precedentemente randomizzati a ricevere placebo nello studio primario di Fase 3 in doppio cieco(XPF-009-301).
-effetto a lungo termine di XEN496 sulla riduzione delle convulsioni in sogg pediatrici trattati con XEN496.
-i punteggi di CaGI-S e CaGI-C in sogg pediatrici con KCNQ2-DEE.
-lo sviluppo e il comportamento neurocognitivo dopo il dosaggio con XEN496 in sogg pediatrici con KCNQ2-DEE.
-l'Impressione globale di cambiamento da parte dello sperimentatore nella condizione complessiva del sogg, valutata usando la scala CGI-C.
-l'uso del medicinale di salvataggio e la variazione dei medicinali anticonvulsivi di base.
-impatto di XEN496 sulla qualità della vita dei caregiver e dei sogg con KCNQ2-DEE.
-le concentrazioni plasmatiche di ezogabina e del metabolita N-acetile di ezogabina (NAMR).

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 protocolspecified 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. Il soggetto ha completato la partecipazione come definito nello studio primario, XPF 009-301. Un soggetto che si ritira dallo studio primario perché soddisfa i criteri di peggioramento specificati nel protocollo, sarà considerato come soggetto che ha completato la partecipazione allo studio primario.
2. Il caregiver accetta ed è in grado di aderire ai requisiti riguardanti il completamento del diario, i programmi delle visite e la somministrazione del farmaco in studio.
3. Il caregiver del soggetto ha raggiunto un minimo dell’85% di aderenza con il completamento del diario sia durante il periodo basale che quello in doppio cieco dello studio primario.

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. Qualsiasi evento avverso (AE) o evento avverso grave (SAE) durante lo studio XPF-009-301 che, a parere dello sperimentatore e del monitor clinico dello sponsor, precluderebbe l’ingresso del soggetto nello studio OLE.
2. Una condizione o malattia clinicamente significativa, o sintomi diversi da quelli derivanti da KCNQ2-DEE, presenti allo screening/basale che, a parere dello sperimentatore, metterebbero a rischio il soggetto se dovesse entrare nello studio.
3. Qualsiasi condizione che era stata descritta come criterio di esclusione nello studio primario (XPF 009-301).
4. Si prevede che il soggetto necessiterà di trattamento con almeno uno dei medicinali non concessi durante lo studio.
5. Qualsiasi cambiamento nel ritmo cardiaco o condizione atrioventircolare nello studio primario che, a parere dello sperimentatore, sia un rischio significativo per la sicurezza del soggetto.

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.

Gravità e frequenza degli AE e SAE; variazioni clinicamente significative negli esami di laboratorio, parametri vitali, elettrocardiogrammi (ECG), esame obiettivo e neurologico, esami urologici ed esami oftalmologici.

E.5.1.1

Timepoint(s) of evaluation of this end point

From Screening/Baseline to the End of the Study

Dallo screening/basale alla fine dello studio

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-009301), 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-009301), for every 3 months based on combined data from both XPF-009301 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)

-Variazione nella frequenza mensile delle convulsioni motorie numerabili, confrontando le prime 15 settimane di trattamento con XEN496 nello studio OLE, rispetto alla frequenza di convulsioni riportata durante il trattamento nel precedente studio primario (XPF-009-301), solo tra quei soggetti che erano randomizzati al braccio placebo nello studio primario.
-Variazione dal basale pre-randomizzazione nel tempo nello studio precedente, in base a categorie di risposta (<25%, da 25 a <50%, da 50 a <75%, da 75 a <100%, 100%), basata su frequenza stimata delle convulsioni ogni 3 mesi durante il periodo OLE.
-Percentuale di variazione dal basale nella frequenza delle convulsioni motorie numerabili, relative al basale pre-randomizzazione dello studio precedente (XPF 009 301), valutata nel tempo ogni 3 mesi durante lo studio OLE.
-Percentuale di variazione dal basale nella frequenza delle convulsioni motorie numerabili, relative al basale pre-randomizzazione dello studio precedente (XPF 009 301), ogni 3 mesi basata sui dati combinati di XPF 009-301 e OLE, per gruppo di trattamento nello studio precedente.
-Variazione nel tempo dei punteggi CaGI-S per le convulsioni del soggetto e condizione complessiva.
-Variazione nel tempo dei punteggi CaGI-C per il soggetto nei seguenti domini: condizione complessiva, convulsioni, comportamento, allerta, capacità motorie, funzione visiva e comunicazione.
-Variazione nel tempo dello sviluppo neurocognitivo basato sulle Scale Bayley III dello Sviluppo del neonato e del bambino.
-Variazione nel tempo del comportamento adattativo basato sul Sistema di valutazione del comportamento adattativo, terza edizione.
-Variazione nel tempo dei punteggi CGI-C per le convulsioni del soggetto e la condizione generale.
-Uso del medicinale di salvataggio.
-Uso di tutti i medicinali concomitanti compresi i trattamenti usati per il controllo delle convulsioni.
-Variazione della qualità della vita di soggetti con KCNQ2-DEE, basato sull’Inventario della qualità della vita pediatrica.
-Variazione della qualità della vita di soggetti con KCNQ2-DEE, basato sull’Inventario della qualità della vita pediatrica, Scala dell’impatto sulla famiglia.
-Concentrazioni plasmatiche di ezogabine e NAMR (per soggetti trattati solo nello studio OLE).

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. Screening alle prime 15 settimane (fino alla visita 10)
2. Dallo screening fino alla fine dello studio
3. Dallo screening fino alla fine dello studio
4. Dallo screening fino

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

lungo termine, tollerabilità

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

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

Yes

E.8.1.7.1

Other trial design description

estensione in aperto

open label extension

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

Information not present in EudraCT

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 fine dello studio è definita come completamento dell'ultima visita per l'ultimo soggetto nello studio.

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.1.1.1

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

è richiesto l'accordo del genitore(i)/tutore(i) per la partecipazione del proprio figlio al
lo studio

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

nessuno

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

2021-11-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-01-24

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2023-05-15

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IMP with orphan designation in the indication
Orphan Designation Number:
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