Clinical Trials Register

Summary
EudraCT Number:	2019-002101-21
Sponsor's Protocol Code Number:	ARGX-113-1803
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:	2020-02-20
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

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

Étude de phase III, multicentrique, en ouvert, à long terme visant à évaluer la sécurité et l’efficacité de l’efgartigimod (ARGX-113) à 10 mg/kg par voie intraveineuse chez des patients adultes atteints de purpura thrombopénique immunologique primaire

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)

Étude de phase III, à long terme visant à évaluer la sécurité et l’efficacité de l’efgartigimod chez les patients adultes atteints de thrombocytopénie immune primaire (maladie auto-immune qui détruit les plaquettes, cellules sanguines qui contribuent à la coagulation, et susceptible d'entraîner l'apparition rapide ou excessive d'ecchymoses et de saignements)

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 BVBA
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	+32 9 310 3400
B.5.5	Fax number	+32 9 310 3499
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	No
D.2.5.1	Orphan drug designation number
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 alfa
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	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	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

Purpura thrombopénique immunologique primaire

E.1.1.1

Medical condition in easily understood language

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

Maladie susceptible d'entraîner l'apparition rapide ou excessive d'ecchymoses et de saignements en raison du faible taux de cellules qui contribuent à la coagulation sanguine

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	20.1
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).

Évaluer la sécurité à long terme de l’efgartigimod chez des patients adultes atteints de purpura thrombopénique immunologique primaire (PTI).

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.

•Évaluer l’efficacité à long terme de l’efgartigimod sur la réponse globale du taux de plaquettes.
•Explorer les diminutions potentielles des traitements concomitants du PTI.
•Évaluer les effets du traitement par efgartigimod sur les mesures de la qualité de vie (QdV) et sur les résultats rapportés par le/la patient(e).
•Mesurer les effets pharmacodynamiques (PD) de l’efgartigimod.
•Évaluer la pharmacocinétique (PK) de l’efgartigimod.
•Évaluer l’immunogénicité de l’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 treatment 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 (i.e. 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 post-menopausal status in amenorrheic patients not on hormonal replacement therapy.
4. Women of childbearing potential should use a highly effective method of contraception (i.e. 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, post-ovulation 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.Capacité à comprendre les exigences de l’étude, à fournir un consentement éclairé écrit (y compris un consentement à l’utilisation et au partage des informations médicales liées à l’étude) et à se conformer aux procédures du protocole de l’étude (notamment les visites d’étude).
2.Les patients inclus dans l’étude ARGX-113-1801 et qui ont terminé les 24 semaines de la période de traitment de l’étude.
3.Les femmes aptes à procréer doivent présenter un test de grossesse urinaire négatif à l’inclusion avant toute administration (perfusion) du médicament de l’étude. Les femmes sont considérées aptes à procréer sauf si elles sont ménopausées (c’est-à-dire en aménorrhée continue) depuis au moins un an avec un taux d’hormones folliculostimulantes (FSH) supérieur à 40 UI/l, ou si elles ont eu recours à une stérilisation chirurgicale (par exemple, hystérectomie, double ovariectomie ou procédure de stérilisation permanente documentée comme la ligature des trompes). L’hormone folliculostimulante peut servir à confirmer la ménopause chez des patientes en aménorrhée qui ne suivent pas de traitement hormonal de substitution.
4.Les femmes aptes à procréer doivent utiliser une méthode de contraception hautement efficace (c’est-à-dire avec un taux de grossesse inférieur à 1 % par an) pendant l’étude et pendant 90 jours après la dernière administration du ME. Elles doivent être sous un traitement stable pendant au moins un mois de :
•contraception hormonale combinée (contenant œstrogènes et progestatifs) associée à une inhibition de l’ovulation :
-par voie orale,
-intravaginale,
-transdermique ;
•contraception hormonale par progestatifs seuls associée à une inhibition de l’ovulation :
-par voie orale,
-injectable,
-implantable ;
•dispositif intra-utérin (DIU) ;
•système intra-utérin à libération d’hormone ;
•ligature bilatérale des trompes ;
•vasectomie du partenaire (à condition que le partenaire soit le seul partenaire sexuel de la participante à l’étude et que l’aspermie ait été documentée après la procédure) ;
•abstinence continue de tout rapport hétérosexuel. L’abstinence n’est acceptable que si elle correspond au mode de vie de la patiente. L’abstinence périodique (calendrier, méthode sympto-thermique, méthodes post-ovulation) n’est pas acceptable.
5.Les patients masculins qui sont sexuellement actifs avec une partenaire féminine apte à procréer doivent utiliser deux moyens de contraception hautement efficaces, par exemple, un préservatif masculin et une méthode de contraception hautement efficace pour la partenaire féminine apte à procréer (voir les méthodes de contraception décrites au critère d’inclusion no 4). Les patients masculins pratiquant l’abstinence totale (si cela correspond au mode de vie de prédilection du patient) peuvent être inclus. Les patients masculins stérilisés qui ont eu recours à une vasectomie avec aspermie documentée après la procédure peuvent être inclus. De plus, les patients masculins ne sont pas autorisés à donner leur sperme à partir de la signature du formulaire de consentement éclairé, tout au long de l’étude et jusqu’à 90 jours après la dernière administration du ME.

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, fostamatinib, monoclonal antibodies, or Fc fusion proteins).
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.Introduction ou poursuite de traitements non autorisés pendant l’étude ARGX-113-1801 (tels que traitement anti-CD20, romiplostime, fostamatinib, anticorps monoclonaux ou protéines de fusion Fc).
2.Femmes enceintes ou qui allaitent, ou qui souhaitent tomber enceintes pendant l’étude ou dans les 90 jours suivant la dernière administration.
3.Patients avec antécédents d’hypersensibilité à l’un des ingrédients de l’efgartigimod.
4.Utilisation d’un autre médicament expérimental ou participation à une autre étude clinique.

E.5 End points

E.5.1

Primary end point(s)

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

Fréquence et gravité des EI, constantes vitales et analyses biologiques.

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

Fréquence et gravité des EI - à chaque visite
Signes vitaux - lors de la visite de référence (semaine 1), suivie de chaque semaine paire (semaine 2 à semaine 52) et Arrêt précoce, suivi 1 et 2, visites non prévues.
Évaluations en laboratoire - veuillez vous reporter au tableau 1 du protocole des évaluations pour les diverses évaluations en laboratoire

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 ≥50×10^9/L.
2. Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥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 ≥50×10^9/L.
5. The number of cumulative weeks over the planned 52-week treatment period with platelet counts of ≥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 ≥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 visit 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 visits 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. Pharmacodynamics markers: total IgG, IgG isotypes (IgG1, IgG2, IgG3, IgG4).

1.Contrôle de l’étendue de la maladie, défini comme le nombre de semaines cumulées sur les 52 semaines prévues de la période de traitement où le taux de plaquettes est supérieur ou égal à 50 × 109/l.
2.Pourcentage de patients avec réponse globale concernant le taux de plaquettes défini comme un taux de plaquettes supérieur ou égal à 50 × 109/l à au moins quatre occasions à tout moment pendant les 52 semaines de la période de traitement.
3.Variation moyenne du taux de plaquettes par rapport à l’inclusion à chaque visite.
4.Pour les patients participant à nouveau après l’étude ARGX-113-1801 avec un taux de plaquettes inférieur à 30 x 109/l : le temps de réponse se définit comme le temps nécessaire pour obtenir deux fois de suite un taux de plaquettes supérieur ou égal à 50 x 109/l.
5.Nombre de semaines cumulées sur les 52 semaines prévues de la période de traitement où le taux de plaquettes est supérieur ou égal à 30×109/l et au moins à 20 x 109/l au-dessus du taux de plaquettes à l’inclusion.
6.Pour les patients avec un taux de plaquettes à l’inclusion inférieur à 15 x 109/l dans l’étude en cours (ARGX-113-1803), le nombre de semaines cumulées sur les 52 semaines prévues de la période de traitement où le taux de plaquettes est supérieur ou égal à 30×109/l et au moins à 20 x 109/l au-dessus du taux de plaquettes à l’inclusion.
7.Pour les patients exposés pour la première fois à l’efgartigimod : la proportion de patients dont la réponse du taux de plaquettes est maintenue, à savoir au moins 50 x 109/l pendant au moins quatre des six visites entre les visites 19 et 24 de l’étude.
8.Pour les patients exposés pour la première fois à l’efgartigimod : la proportion de patients parmi la population totale de l’étude qui obtiennent un taux de plaquettes d’au moins 50 x 109/l pendant au moins six des huit visites entre les visites 17 et 24 de l’étude.
9.Fréquence de traitement de secours administré (par patient[e] et par mois).
10.Diminution des traitements concomitants pour le PTI.
11.Incidence et gravité des événements hémorragiques d’après la classification de l’OMS.
12.Variations depuis l’inclusion des résultats rapportés par les patients (FACIT-Fatigue, Fact-Th6) et de la QdV (SF-36) lors de visites programmées.
13.Incidence des anticorps antimédicament (AAM) sur l’efgartigimod.
14.Marqueurs pharmacodynamiques : IgG totale, isotypes de l’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 visit 19 and 24
8. Between visits 17 and 24
9. At each visit
10. At each visit
11. At each visit
12. PRO: Weeks 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 47, 49, 52 of Treatment period, Early Discontinuation
QoL: Weeks 1, 9, 17, 25, 33, 41, 49, 52 of Treatment period, Early Discontinuation
13. Weeks 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. Weeks 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.Sur la période de traitement de 52 semaines 2.Sur la période de traitement de 52 semaines 3.À chaque visite 4.À chaque visite 5.Sur la période de traitement de 52 semaines 6.Sur la période de traitement de 52 semaines 7.Entre la visite 19 et 24 8.Entre la visite 17 et 24 9.À chaque visite 10.À chaque visite 11.À chaque visite 12.PRO: Semaine 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 47, 49, 52 de la période de traitement, arrêt précoce QdV: Semaine 1, 9, 17, 25, 33, 41, 49, 52 de la période de traitement, arrêt précoce 13.Semaine 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 de la période de traitement, arrêt précoce, suivi 2, visite non prévue 14.Semaine 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 de la période de traitement, arrêt précoce, suivi 1/2, visite non prévue

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

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

Austria

Belgium

Bulgaria

Canada

Czech Republic

France

Germany

Hungary

Italy

Japan

Netherlands

Poland

Russian Federation

Spain

Ukraine

United Kingdom

United States

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.

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

2020-04-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-04-22

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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