Clinical Trials Register

Summary
EudraCT Number:	2021-002320-20
Sponsor's Protocol Code Number:	EFC16158
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-10-22
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

2021-002320-20

A.3

Full title of the trial

A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy

Studio di fase 3 randomizzato, in aperto, a gruppi paralleli, della durata di 18 mesi per valutare l’effetto di venglustat rispetto alla terapia standard abituale sull’indice di massa del ventricolo sinistro in
partecipanti con malattia di Fabry e ipertrofia ventricolare sinistra

A.3.1

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

A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease

Uno studio per valutare l’effetto di venglustat in compresse sull’indice di massa del ventricolo sinistro in partecipanti adulti di sesso maschile e femminile con malattia di Fabry

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

EFC16158

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1266-5068

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	SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
B.1.3.4	Country	France
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	sanofi-aventis recherche et développement
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Sanofi S.r.l
B.5.2	Functional name of contact point	Contact point
B.5.3	Address:
B.5.3.1	Street Address	Viale Luigi Bodio 37/A
B.5.3.2	Town/ city	Milano
B.5.3.3	Post code	20158
B.5.3.4	Country	Italy
B.5.4	Telephone number	800536389
B.5.5	Fax number	000000
B.5.6	E-mail	informazioni.medicoscientifiche@sanofi.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Fabrazyme
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe B.V - numero MA EU/1/01/188/001
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	.
D.3.2	Product code	[.]
D.3.4	Pharmaceutical form	Powder for concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	AGALSIDASI BETA
D.3.9.1	CAS number	104138-64-9
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB12457MIG
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	35
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Galafold
D.2.1.1.2	Name of the Marketing Authorisation holder	Amicus Therapeutics Europe Limited - Numero MA: EU/1/15/1082/001
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/06/368
D.3 Description of the IMP
D.3.1	Product name	.
D.3.2	Product code	[.]
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Migalastat
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB177094
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	123
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	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Replagal
D.2.1.1.2	Name of the Marketing Authorisation holder	Shire Human Genetic Therapies AB - numero MA: EU/1/01/189/001
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	.
D.3.2	Product code	[.]
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	AGALSIDASI ALFA
D.3.9.1	CAS number	104138-64-9
D.3.9.2	Current sponsor code	NA
D.3.9.4	EV Substance Code	SUB12456MIG
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	1
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 4
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/3/14/1310
D.3 Description of the IMP
D.3.1	Product name	Venglustat
D.3.2	Product code	[SAR402671, GZ402671 o GZ/SAR402671]
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	venglustat malato
D.3.9.1	CAS number	1629063-78-0
D.3.9.2	Current sponsor code	GZ402671
D.3.9.3	Other descriptive name	GZ/SAR402671
D.3.9.4	EV Substance Code	SUB166602
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	15
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Fabry's disease

malattia di Fabry

E.1.1.1

Medical condition in easily understood language

Fabry's disease

malattia di Fabry

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	24.1
E.1.2	Level	PT
E.1.2	Classification code	10016016
E.1.2	Term	Fabry's disease
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic 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 compare the effect of venglustat with standard of care Fabry therapies on left ventricular mass index over 18 months in participants with Fabry disease and left ventricular hypertrophy

Valutare l’effetto di venglustat con le terapie standard di cura per la malattia di Fabry sull’indice di massa del ventricolo sinistro nell’arco di 18 mesi nei/nelle partecipanti con malattia di Fabry e ipertrofia ventricolare sinistra

E.2.2

Secondary objectives of the trial

- To evaluate the effect of venglustat on renal function
- To evaluate the effect of venglustat versus standard therapy on measures of cardiac function and cardiac lipid storage
- To assess the safety and tolerability of venglustat in participants with Fabry disease
- To evaluate the PK of venglustat in participants with Fabry disease

- Valutare l’effetto di venglustat sulla funzionalità renale
- Valutare l’effetto di venglustat rispetto alla terapia standard sulle misure della funzione cardiaca e dell’accumulo di lipidi cardiaci
- Valutare la sicurezza e la tollerabilità di venglustat in partecipanti affetti da malattia di Fabry
- Valutare la PK di venglustat in partecipanti affetti da malattia di Fabry

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease.
- Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated.
- Left ventricular hypertrophy.
- Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant.
- A signed informed consent must be provided prior to any study-related procedures.

- Partecipanti adulti di sesso maschile e femminile di età compresa tra 18 e 65 anni con una diagnosi precedentemente confermata di malattia di Fabry e un’anamnesi di sintomi clinici della malattia di Fabry.
- I partecipanti potrebbero ricevere una terapia con agalsidasi alfa, agalsidasi beta o migalastat, oppure potrebbero essere non trattati
- Ipertrofia ventricolare sinistra
- L'uso di contraccettivi da parte di uomini e donne: non in gravidanza o in allattamento; non donatori di sperma per i partecipanti di sesso maschile
- Un consenso informato scritto sarà ottenuto prima della conduzione di qualsiasi procedura correlata allo studio.

E.4

Principal exclusion criteria

- History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery or kidney transplantation.
- History of seizures currently requiring treatment.
- Underlying medical condition that may cause or contribute to left ventricular hypertrophy.
- Asymmetric hypertrophy by echocardiography at screening.
- Advanced cardiac fibrosis, defined as significant late gadolinium enhancement beyond the basal inferolateral left ventricular wall on cardiac MRI.
- History of ongoing clinically significant cardiac arrythmia, prior or ongoing treatment for the above.
- Estimated glomerular filtration rate <60 ml/min/1.73m2
- Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II >28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit.
- Patients with hepatitis C, HIV, or hepatitis B infection.
- Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID 19 requiring hospitalization within 6 months of enrollment.
- History of drug and/or alcohol abuse.
- Moderate to severe hepatic impairment.
- History of or active hepatobiliary disease.
- Liver enzymes (alanine aminotransferase/aspartate aminotransferase) or total bilirubin >2 times the upper limit of normal.
- Cortical cataract >/=one-quarter of the lens circumference (Grade cortical cataract [COR-2]) or a posterior subcapsular cataract >/=2 mm (Grade [posterior subcapsular cataract [PSC-2]) at the time of screening.
- Chronic regimen of potentially cataractogenic medications.
- Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A4 per Food and Drug Administration (FDA) classification within 14 days or 5 half lives, whichever is longer, prior to randomization.

- Anamnesi di attacco ischemico transitorio, ictus, infarto del miocardio, insufficienza cardiaca, chirurgia cardiovascolare maggiore o trapianto renale.
- Anamnesi di crisi convulsive che attualmente richiedano un trattamento.
- Condizione medica di base che può causare o contribuire all’ipertrofia ventricolare sinistra.
-Ipertrofia asimmetrica mediante ecocardiografia allo screening.
- Fibrosi cardiaca avanzata, definita come significativo aumento tardivo del gadolinio oltre la porzione basale della parete inferolaterale del ventricolo sinistro alla RM cardiaca allo screening.
-Anamnesi di Aritmia cardiaca clinicamente significativa in corso, oppure trattamento per quanto sopra indicato precedente o in corso.
-Velocità di filtrazione glomerulare stimata <60 ml/min/1,73 m2
- Presenza di depressione grave misurata mediante classificazione della depressione di Beck (BDI)-II >28 e/o anamnesi di disturbo affettivo maggiore instabile non trattato entro 1 anno dalla visita di screening.
- Pazienti con epatite C,HIV, o infezioni di epatite B
- Positività nota al test del virus SARS-CoV-2 entro 2 settimane dall’arruolamento o COVID-19 che richiede il ricovero ospedaliero entro 6 mesi dall’arruolamento.
- Anamnesi di abuso di droghe e/o alcol.
- Insufficienza epatica da moderata a grave.
- Anamnesi di malattia epatobiliare in passato o attiva
- Enzimi epatici (alanina aminotransferasi/aspartato aminotransferasi) o bilirubina totale >2 volte il limite superiore della norma
- Il partecipante presenta una cataratta corticale >/= un quarto della circonferenza del cristallino (grado della cataratta corticale [cortical-2, COR-2]) o una cataratta sottocapsulare posteriore >/=2 mm (grado di cataratta sottocapsulare posteriore [posterior subcapsular-2, PSC-2]) al momento dello screening
- Regime cronico di farmaci potenzialmente catarattogenici
- Induttori o inibitori forti o moderati del citocromo P450 CYP3A secondo la classificazione della Food and Drug Administration (FDA) entro 14 giorni o 5 emivite, a seconda di quale sia il periodo più lungo, prima della randomizzazione.

E.5 End points

E.5.1

Primary end point(s)

Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)

Pendenza dell’indice di massa del ventricolo sinistro misurata mediante RM cardiaca (lettura centralizzata)

E.5.1.1

Timepoint(s) of evaluation of this end point

from baseline to 18 months

dal basale a 18 mesi

E.5.2

Secondary end point(s)

1. Slope of estimated glomerular filtration rate (eGFR) as assessed by the chronic kidney disease epidemiology collaboration (CKD-EPI) creatinine equation
2. Change in T1 relaxation time, measured by cardiac MRI (central reading)
3. Change in global longitudinal strain, measured by echocardiography (central reading)
4. Number of participants with adverse event (AE) and serious adverse event (SAE)
5. Change in the lens clarity by ophthalmological examination
6. Change in Beck Depression Inventory-II (BDI-II) score
7. Plasma venglustat concentrations at prespecified visits over the study duration; 1. Pendenza della velocità di filtrazione glomerulare (eGFR) valutata mediante l’equazione CKD-EPI della creatinina
2. Variazione nel tempo di rilassamento T1, misurato mediante RM cardiaca (lettura centralizzata)
3. Variazione nello sforzo longitudinale globale, misurato mediante ecocardiografia (lettura centralizzata)
4. Numero di partecipanti con eventi avversi (AE) e eventi avversi seri (SAE)
5. Variazione della trasparenza del cristallino attraverso un esame oftalmologico
6. Variazione nel punteggio BDI-II
7. Concentrazioni plasmatiche di venglustat alle visite predefinite per tutta la durata dello studio

E.5.2.1

Timepoint(s) of evaluation of this end point

From 1/ to 7/: from baseline to 18 months

Da 1 a 7 : dal basale a 18 mesi

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

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

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

Canada

China

Japan

Korea, Republic of

Taiwan

Turkey

United States

Austria

Denmark

France

Germany

Italy

Netherlands

Norway

Poland

Spain

United Kingdom

Czechia

Greece

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

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

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)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

110

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

110

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

2022-01-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-01-11

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

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