Clinical Trials Register

Summary
EudraCT Number:	2019-002101-21
Sponsor's Protocol Code Number:	ARGX-113-1803
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:	2021-06-17
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

2019-002101-21

A.3

Full title of the trial

A Phase 3, Multicenter, Open-Label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia

Sperimentazione di Fase 3, multicentrica, in aperto, a lungo termine per valutare la sicurezza e l’efficacia di Efgartigimod (ARGX-113) 10 mg/kg per via endovenosa in pazienti adulti con trombocitopenia immune primaria

A.3.1

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

A long-term study to assess the safety and efficacy of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder
that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)

Studio a lungo termine per valutare la sicurezza e l’efficacia di efgartigimod in pazienti adulti con trombocitopenia immune primaria (una malattia autoimmune che distrugge le piastrine, le cellule del sangue che favoriscono la coagulazione, e che può portare a facilità o eccessiva contusione o sanguinamento)

A.3.2

Name or abbreviated title of the trial where available

ADVANCE+

A.4.1

Sponsor's protocol code number

ARGX-113-1803

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	ARGENX BV
B.1.3.4	Country	Belgium
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	argenx BVBA
B.4.2	Country	Belgium
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	argenx BVBA
B.5.2	Functional name of contact point	Regulatory
B.5.3	Address:
B.5.3.1	Street Address	Industriepark Zwijnaarde 7
B.5.3.2	Town/ city	Zwijnaarde
B.5.3.3	Post code	B-9052
B.5.3.4	Country	Belgium
B.5.4	Telephone number	003293103400
B.5.5	Fax number	003293103499
B.5.6	E-mail	regulatory@argenx.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/3/19/2230
D.3 Description of the IMP
D.3.1	Product name	Efgartigimod
D.3.2	Product code	[ARGX-113]
D.3.4	Pharmaceutical form	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	Efgartigimod
D.3.9.1	CAS number	1821402-21-4
D.3.9.2	Current sponsor code	ARGX-113
D.3.9.3	Other descriptive name	ARGX-113
D.3.9.4	EV Substance Code	SUB180001
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	20
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.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

Primary immune thrombocytopenia

Trombocitopenia immune primaria

E.1.1.1

Medical condition in easily understood language

Disorder that can lead to easy or excessive bruising and bleeding due to low levels of the cells that help blood clot

Malattia che può portare a facile o eccessiva contusione e sanguinamento a causa dei bassi livelli di cellule che favoriscono la coagulazione del sangue

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	23.0
E.1.2	Level	LLT
E.1.2	Classification code	10050245
E.1.2	Term	Autoimmune thrombocytopenia
E.1.2	System Organ Class	100000004851

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 evaluate the long-term safety of efgartigimod in adult patients with primary immune thrombocytopenia (ITP).

Valutare la sicurezza a lungo termine di efgartigimod in pazienti adulti con trombocitopenia immune (ITP) primaria.

E.2.2

Secondary objectives of the trial

• To evaluate the long-term efficacy of efgartigimod on overall platelet count response.
• To explore the potential for reduction in concurrent ITP therapy.
• To evaluate the effects of efgartigimod treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO).
• To assess the pharmacodynamic (PD) effects of efgartigimod.
• To evaluate the pharmacokinetics (PK) of efgartigimod.
• To assess the immunogenicity of efgartigimod.

• Valutare l’efficacia a lungo termine di efgartigimod sulla risposta complessiva in termini di conta piastrinica.
• Esplorare la potenziale riduzione della terapia concomitante per l’ITP.
• Valutare gli effetti del trattamento con efgartigimod sulle misurazioni della qualità della vita (QoL) e sugli esiti segnalati dal/la paziente (PRO).
• Valutare gli effetti farmacodinamici (PD) di efgartigimod.
• Valutare la farmacocinetica (PK) di efgartigimod.
• Valutare l’immunogenicità di efgartigimod.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
2. Patients enrolled in the ARGX-113-1801 trial who completed the 24- week trial period.
3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. Women are considered of childbearing potential unless they are post-menopausal (defined by continuous amenorrhea) for at least 1 year with a FSH of >40 IU/L or are surgically sterilized (ie, women who had a hysterectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm postmenopausal status in amenorrheic patients not on hormonal replacement therapy.
4. Women of childbearing potential should use a highly effective method of contraception (ie, pregnancy rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month:
• combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
- oral
- intravaginal
- transdermal
• progestogen-only hormonal contraception associated with inhibition of ovulation:
- oral
- injectable
- implantable
• intrauterine device (IUD)
• intrauterine hormone-releasing system
• bilateral tubal occlusion
• vasectomized partner(provided that the partner is the sole sexual partner of the trial participant and that aspermia was documented post procedure)
• continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, postovulation methods) is not acceptable.
5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception, being a condom for male patients and a highly effective form of contraception for the female partner of childbearing potential (same as for female patients described in inclusion criterion 4). Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant) can be included. Sterilized male patients who have had vasectomy with documented aspermia post procedure can be included. In addition, male patients are not allowed to donate sperm during this period from signing of informed consent form, throughout the duration of the trial, and for 90 days after the last administration of IMP.

1. Capacità di comprendere i requisiti della sperimentazione, di fornire il consenso informato scritto (compreso il consenso ad usare e divulgare le informazioni sanitarie correlate alla ricerca), e a seguire le procedure del protocollo della sperimentazione (comprese le visite della sperimentazione richieste).
2. Pazienti arruolati nello studio ARGX-113-1801 che hanno completato il periodo di sperimentazione di 24 settimane.
3. Le donne potenzialmente fertili devono avere un test di gravidanza delle urine negativo al basale prima che possa essere somministrato il medicinale della sperimentazione (infusione). Le donne sono considerate potenzialmente fertile salvo che siano in menopausa (definita da amenorrea continua) da almeno 1 anno con un ormone follicolo-stimolante (FSH) >40 IU/L o che siano sterilizzate chirurgicamente (cioè donne che sono state sottoposte a isterectomia, rimozione chirurgica di entrambe le ovaie o che sono state sottoposte ad una procedura di sterilizzazione femminile permanente documentata, compresa la legatura delle tube). L’ormone follicolo-stimolante può essere usato per confermare lo stato post-menopausa in pazienti amenorreiche che non assumono la terapia ormonale sostitutiva.
4. Le donne potenzialmente fertili devono usare un metodo contraccettivo altamente efficace (cioè con tasso di gravidanza di meno dell’1% all’anno) durante la sperimentazione e per 90 giorni dopo l’ultima somministrazione dell’IMP. Devono essere in regime stabile da almeno 1 mese:
• contraccezione ormonale combinata (contenente estrogeno e progestinico) associata all’inibizione dell’ovulazione:
o orale
o intravaginale
o transdermica
• contraccezione ormonale a base di solo progestinico associata all’inibizione dell’ovulazione:
o orale
o iniettabile
o impiantabile
• dispositivo intrauterino (IUD)
• sistema intrauterino a rilascio ormonale
• occlusione bilaterale delle tube
• partner vasectomizzato (purché il partner sia l’unico partner sessuale della partecipante alla sperimentazione e purché l’aspermia sia stata documentata dopo l’intervento)
• astinenza continua dal contatto sessuale eterosessuale. L’astinenza sessuale è accettabile solo se corrisponde allo stile di vita preferito e abituale del/della paziente. L’astinenza periodica (metodi basati sul calendario, sintotermici, post-ovulazione) non è accettabile.
5. I pazienti di sesso maschile non sterilizzati che sono sessualmente attivi con una partner potenzialmente fertile, devono usare un doppio metodo contraccettivo efficace, cioè un preservativo per il paziente di sesso maschile e un metodo contraccettivo altamente efficace per la partner potenzialmente fertile (stessi metodi descritti per le pazienti di sesso femminile nel criterio di inclusione 4). I soggetti di sesso maschile che praticano l’astinenza totale (quando ciò è conforme allo stile di vita preferito e abituale del partecipante) possono essere inclusi. Pazienti di sesso maschile che hanno avuto una vasectomia con aspermia documentata post-procedura, possono essere inclusi. Inoltre i pazienti di sesso maschile non devono donare sperma durante questo periodo, dalla firma del consenso informato, per tutta la durata della sperimentazione e per 90 giorni dopo l’ultima somministrazione di IMP.

E.4

Principal exclusion criteria

1. Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).
2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
4. Use of any other investigational drug or participation in any other investigational trial.

1. Introduzione o continuazione di medicinali non permessi durante la sperimentazione ARGX 113 1801 (come la terapia anti-CD20, romiplostim, anticorpi monoclonali, proteine di fusione Fc o vaccini vivi/vivi-attenuati).
2. Donne in gravidanza o che allattano con latte materno e quelle che programmano di iniziare una gravidanza durante la sperimentazione o entro 90 giorni dopo l’ultima somministrazione della dose.
3. Pazienti con anamnesi clinica nota di ipersensibilità ad uno qualsiasi dei componenti di efgartigimod.
4. Uso di un qualsiasi altro medicinale sperimentale o partecipazione a qualsiasi altra sperimentazione.

E.5 End points

E.5.1

Primary end point(s)

Frequency and severity of AEs, vital signs, and laboratory assessments.

Frequenza e gravità degli eventi avversi, parametri vitali, valutazioni di laboratorio.

E.5.1.1

Timepoint(s) of evaluation of this end point

Frequency and severity of AEs - at each visit
Vital signs - at baseline visit (week 1), followed by every even week (week 2 to week 52) and Early discontinuation, follow-up 1 and 2,
unscheduled visits.
Laboratory assessments - please refer to protocol Table 1 Schedule of Assessments for various laboratory assessments

Frequenza e gravità degli EA - a ciascuna visita
Parametri vitali - alla visita basale (settimana 1) e, in seguito, ad ogni visita pari (dalla settimana 2 alla settimana 52) e alle visite di Interruzione anticipata, follow-up 1 e 2 e non programmate.
Valutazioni di laboratorio- vedere Tabella 1 del protocollo, Programma delle valutazioni per le varie valutazioni di laboratorio

E.5.2

Secondary end point(s)

1. Extent of disease control defined as the number of cumulative weeks over the planned 52-week treatment period with platelet counts of > and =
50×10^9/L.
2. Percentage of patients with overall platelet count response defined as achieving a platelet count of >and =50×10^9/L on at least 4 occasions at any time during the 52-week treatment period.
3. Mean change from baseline in platelet count at each visit.
4. For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of > and =50×10^9/L.
5. The number of cumulative weeks over the planned 52-week treatment period with platelet counts of > and =30×10^9/L and at least 20×10^9/L
above baseline.
6. In patients with baseline platelet count of <15×10^9/L in the current trial (ARGX-113-1803), the number of cumulative weeks over the
planned 52-week treatment period with platelet counts of > and =30×10^9/L and at least 20×10^9/L above baseline.
7. In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial.
8. In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.
9. Rate of receipt of rescue therapy (rescue per patient per month).
10. Reduction in concurrent ITP therapy.
11. Incidence and severity of the WHO-classified bleeding events.
12. Change from baseline in PRO (FACIT-Fatigue, Fact-Th6) and QoL (SF-36) at planned visits.
13. Incidence of anti-drug antibodies (ADA) to efgartigimod.
14.Pharmacokinetic parameters of efgartigimod:maximum observed serum concentration (C max) and serum concentration observed predose (Ctrough)
15. Pharmacodynamics markers: total IgG, IgG isotypes (IgG1, IgG2, IgG3, IgG4).; 1. Grado di controllo della malattia definito come il numero cumulativo di settimane con conta piastrinica > e=50×109/L nel periodo di trattamento previsto di 52 settimane.
2. Percentuale di pazienti con risposta complessiva in termini di conta piastrinica definita come il raggiungimento di una conta piastrinica > e =50×109/L in almeno 4 occasioni in qualsiasi momento nel periodo di trattamento di 52 settimane.
3. Cambiamento medio dal basale nella conta piastrinica a ciascuna visita.
4. Per i pazienti che passano dallo studio ARGX-113-1801 con una conta piastrinica <30×109/L: il tempo alla risposta è definito come il tempo per raggiungere 2 conte piastriniche consecutive di > e =50×109/L.
5. Numero cumulativo di settimane con conta piastrinica > e =30×109/L e almeno 20×109/L sopra il basale nel periodo di trattamento previsto di 52 settimane.
6. Nei pazienti con conta piastrinica <15×109/L nella sperimentazione attuale (ARGX-113-1803), numero cumulativo di settimane con conta piastrinica > e =30×109/L e almeno 20×109/L sopra il basale nel periodo di trattamento previsto di 52 settimane.
7. In pazienti con prima esposizione a efgartigimod: proporzione di pazienti che raggiungono una risposta piastrinica sostenuta, definita come il raggiungimento di conte piastriniche di almeno 50×109/L per almeno 4 delle 6 visite tra la settimana 19 e la 24 della sperimentazione.
8. In pazienti con prima esposizione a efgartigimod: proporzione di pazienti nella popolazione complessiva che raggiungono conte piastriniche di almeno 50×109/L per almeno 6 delle 8 visite tra la settimana 17 e la 24 della sperimentazione.
9. Tasso di utilizzo della terapia di emergenza (emergenza per paziente per mese).
10. Riduzione della terapia concomitante per l’ITP.
11. Incidenza e gravità degli eventi di sanguinamento classificate in base a WHO.
12. Cambiamento dal basale in PRO (FACIT-Affaticamento, Fact-Th6) e QoL (SF-36) alle visite programmate.
13. Incidenza di anticorpi anti farmaco (ADA) contro efgartigimod.
14.Parametri farmacocinetici di efgartigimod: massima concentrazione sierica osservata (Cmax) e concentrazione sierica osservata pre-dose (Cvalle)
15. Marcatori di farmacodinamica: IgG totale, isotipi IgG (IgG1, IgG2, IgG3, IgG4).

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Over the 52-week treatment period
2. Over the 52-week treatment period
3. At each visit
4. At each visit
5. Over the 52-week treatment period
6. Over the 52-week treatment period
7. Between week 19 and 24
8. Between weeks 17 and 24
9. At each visit
10. At each visit
11. At each visit
12. PRO: Visit 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 47, 49, 52 of Treatment period, Early Discontinuation QoL: Visit 1, 9, 17, 25, 33, 41, 49, 52 of Treatment period, Early Discontinuation
13. Visit 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 of Treatment period, Early Discontinuation, Follow-up 2, Unscheduled Visit
14 and 15. Visit 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 of Treatment period, Early Discontinuation, Follow-up 1/2, Unscheduled Visit

1.Durante trattamento di 52 sett, 2.Durante trattamento di 52 sett, 3.Ad ogni visita, 4.Ad ogni visita, 5.Durante trattamento di 52 settimane, 6.Durante trattamento di 52 sett, 7.Tra la sett.19 e 24, 8.Tra le sett. 17 e 24, 9.Ad ogni visita, 10.Ad ogni visita, 11.Ad ogni visita, 12.PRO: Visita 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 47, 49, 52 del Per. di tratt., Interruz anticipata QoL: Visita 1, 9, 17, 25, 33, 41, 49, 52 del Periodo di trattamento, Interruz anticipata, 13.Visita 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, del Periodo di trattamento, Interruzione anticipata, Follow-up 2, Visita non programmata, 14 e 15.Visita 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, del Periodo di trattamento, Interruzione anticipata, Follow-up 1/2, Visita non programmata

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

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

Immunogenicity of efgartigimod

Immunogenicità di efgartigimod

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

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

Yes

E.8.2.3.1

Comparator description

non applicabile

not applicable

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

Georgia

Japan

Russian Federation

Turkey

United States

Austria

Belgium

Bulgaria

France

Germany

Hungary

Italy

Netherlands

Poland

Spain

United Kingdom

Czechia

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:

140

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

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

140

F.4.2.2

In the whole clinical trial

156

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)

After a patient has completed the trial or has withdrawn/discontinued early, usual treatment will be administered if required, in accordance with the trial sites' standard of care and generally accepted medical practice depending on the patient's individual needs.

Dopo che un paziente ha completato la sperimentazione o si è ritirato/ha interrotto anticipatamente, il trattamento abituale sarà somministrato, se necessario, in conformità allo standard di cura del centro della sperimentazione e alla pratica clinica generalmente accettata, in base alle necessità individuali del/la paziente.

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

2020-05-12

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-05-18

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

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IMP with orphan designation in the indication
Orphan Designation Number:
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