Clinical Trials Register

Summary
EudraCT Number:	2020-002917-16
Sponsor's Protocol Code Number:	ARGX-113-1905
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:	2021-05-24
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

2020-002917-16

A.3

Full title of the trial

An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients with Pemphigus

Studio di follow-up, in aperto, multicentrico su ARGX-113-1904 per valutare la sicurezza, la tollerabilità e l’efficacia di efgartigimod PH20 SC in pazienti affetti da pemfigo (ADDRESS+)

A.3.1

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

A study to assess the safety and efficacy of a subcutaneous formulation of efgartigimod PH20 SC in adults with Pemphigus (Vulgaris or Foliaceus)

Studio per valutare la sicurezza e l’efficacia di una formulazione sottocutanea di efgartigimod PH20 SC negli adulti affetti da pemfigo (volgare o foliaceo)

A.3.2

Name or abbreviated title of the trial where available

ADDRESS+

A.4.1

Sponsor's protocol code number

ARGX-113-1905

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 BV
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 BV
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	000000
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 PH20 SC
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous 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 milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	165
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	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	Yes
D.2.1.1.1	Trade name	Prednison 5 mg GALEN
D.2.1.1.2	Name of the Marketing Authorisation holder	GALENpharma GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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	na
D.3.2	Product code	[na]
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	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	PREDNISONE
D.3.9.4	EV Substance Code	SUB10020MIG
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	5
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	Yes
D.2.1.1.1	Trade name	Prednison 10 mg GALEN
D.2.1.1.2	Name of the Marketing Authorisation holder	GALENpharma GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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	na
D.3.2	Product code	[na]
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	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	PREDNISONE
D.3.9.4	EV Substance Code	SUB10020MIG
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	10
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: 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	Yes
D.2.1.1.1	Trade name	Prednison20 mg GALEN
D.2.1.1.2	Name of the Marketing Authorisation holder	GALENpharma GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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	na
D.3.2	Product code	[na]
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	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	PREDNISONE
D.3.9.4	EV Substance Code	SUB10020MIG
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	20
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: 5
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	Yes
D.2.1.1.1	Trade name	Prednison 50 mg GALEN
D.2.1.1.2	Name of the Marketing Authorisation holder	GALENpharma GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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	na
D.3.2	Product code	[na]
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	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	PREDNISONE
D.3.9.4	EV Substance Code	SUB10020MIG
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	50
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

Pemphigus Vulgaris or Pemphigus Foliaceus

Pemfigo volgare o pemfigo foliaceo

E.1.1.1

Medical condition in easily understood language

Blistering autoimmune diseases that affect the skin and mucous membranes

Malattie autoimmuni con formazione di vesciche che interessano la pelle e le membrane mucose

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	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10057069
E.1.2	Term	Pemphigus foliaceus
E.1.2	System Organ Class	100000004858

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10052802
E.1.2	Term	Pemphigus vulgaris
E.1.2	System Organ Class	100000004858

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 safety of extended treatment and re-treatment with efgartigimod PH20 SC in patients with PV or PF

Valutare la sicurezza di un trattamento prolungato e del ri-trattamento con efgartigimod PH20 SC in pazienti affetti da PV o PF

E.2.2

Secondary objectives of the trial

To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PF
To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in patients with PV or PF
To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in patients with PV or PF
To evaluate the PD of efgartigimod PH20 SC in patients with PV or PF
To evaluate the immunogenicity of efgartigimod PH20 SC in patients with PV or PF

• Valutare l’efficacia del trattamento con efgartigimod PH20 SC in PV e PF
• Valutare gli effetti di efgartigimod PH20 SC sulla qualità della vita (QoL) nei pazienti affetti da PV o PF
• Valutare la PK di efgartigimod PH20 SC nei pazienti affetti da PV o PF
• Valutare la PD di efgartigimod PH20 SC nei pazienti affetti da PV o PF
• Valutare l’immunogenicità di efgartigimod PH20 SC nei pazienti affetti da PV o PF

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

Other types of substudies
Specify title, date and version of each substudy with relative objectives: Evaluation of biomarkers of pemphigus pathology from skin biopsis

Altre tipologie di sottostudi
specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Valutazione dei biomarcatori della patologia del pemfigo tramite biopsie cutanee

E.3

Principal inclusion criteria

Participants must meet all of the following 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), willingness and ability to comply with the trial protocol procedures (including required trial visits).
2. The patient participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.
3. Women of childbearing potential:
a. Must have a negative urine pregnancy test at baseline before trial medication can be administered.
b. Must be on a stable regimen for at least 1 month of at least 1 highly effective method of contraception (ie, failure rate of less than 1% per year) during the trial and for 90 days after the last administration of IMP.
4. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective contraception from first administration of IMP through 90 days after the last administration of the IMP. Male patients practicing true sexual abstinence (as consistent with preferred and usual life style) can be included. Sterilized male patients who have had a vasectomy and with documented absence of sperm post-procedure can be included. Male patients are not allowed to donate sperm from first administration of IMP through 90 days after the last dose of IMP.

I partecipanti devono soddisfare i seguenti criteri di inclusione:
1. Capacità di comprendere i requisiti della sperimentazione, fornire un consenso informato scritto (compreso il consenso per l’utilizzo e la divulgazione di informazioni sanitarie correlate alla ricerca), volontà e capacità di attenersi alle procedure del protocollo di sperimentazione (comprese le visite previste della sperimentazione).
2. Partecipazione del/la paziente alla sperimentazione ARGX-113-1904 e aver completato lo studio o possedere i criteri prestabiliti per il rollover.
3. Donne in età fertile:
a. Devono presentare un test di gravidanza sulle urine negativo alla visita basale prima che il farmaco sperimentale possa essere somministrato.
b. Devono essere in un regime stabile da almeno 1 mese con minimo 1 metodo contraccettivo altamente efficace (ossia, tasso di fallimento inferiore all’1% annuo) durante la sperimentazione e per 90 giorni dopo l’ultima somministrazione dell’IMP.
4. I pazienti di sesso maschile non sterilizzati, che sono sessualmente attivi con una partner di sesso femminile in età fertile, devono usare un metodo contraccettivo efficace dalla prima somministrazione dell’IMP fino a 90 giorni dopo l’ultima somministrazione dell’IMP. Potranno essere inclusi i pazienti di sesso maschile che pratichino realmente l’astinenza sessuale (se ciò è in linea con lo stile di vita preferito e abituale del partecipante). Potranno essere inclusi i pazienti di sesso maschile sterilizzati sottoposti a vasectomia e con aspermia post-procedurale documentata. Inoltre, ai pazienti di sesso maschile non sarà consentito donare lo sperma a partire dalla prima somministrazione dell’IMP e per 90 giorni dopo l’ultima dose dell’IMP.

E.4

Principal exclusion criteria

Participants are excluded from the trial if any of the following criteria apply:
1. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after the last administration of IMP.
2. Patients with clinical evidence of other significant serious disease or patients who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the patient at undue risk.
3. Known hypersensitivity to any of the components of the administered treatments.

I partecipanti saranno esclusi dalla sperimentazione se rientreranno in uno qualsiasi dei seguenti criteri:
1. Le donne incinte e in fase di allattamento e quelle che intendono rimanere incinte durante la sperimentazione o entro i 90 giorni successivi all’ultima somministrazione dell’IMP.
2. I pazienti con evidenza clinica di altra malattia significativa grave o i pazienti sottoposti di recente, o candidati, a intervento di chirurgia maggiore durante il periodo della sperimentazione o qualsiasi altra condizione che a giudizio dello sperimentatore potrebbe confondere i risultati della sperimentazione o comporterebbe un rischio eccessivo per il paziente.
3. Ipersensibilità nota a uno qualsiasi dei componenti dei trattamenti somministrati.

E.5 End points

E.5.1

Primary end point(s)

1. Incidence and severity of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) by System Organ Class (SOC) and Preferred Term (PT)
2. Vital signs, physical examination, electrocardiogram (ECG), and clinical laboratory safety evaluations

• Incidenza e gravità di eventi avversi emergenti dal trattamento (TEAE), di eventi avversi di interesse particolare (AESI) e di eventi avversi seri (SAE) in base alla classificazione per sistemi e organi (SOC) e termine preferito (PT)
• Misurazioni dei parametri vitali, esami obiettivi, elettrocardiogrammi (ECG) e valutazioni cliniche di laboratorio per la sicurezza

E.5.1.1

Timepoint(s) of evaluation of this end point

Up to 60 weeks

Fino a 60 settimane

E.5.2

Secondary end point(s)

1. Proportion of PV patients who achieve CR on minimal prednisone therapy
2. Proportion of PV and PF patients who achieve CR on minimal prednisone therapy in patients with PV and PF 3.
3. Time to DC
4. Time to CR
5. Time to CR on minimal prednisone therapy
6. Time to CR off therapy
7. Time to flare
8. Rate of treatment failure
9. Rate of flare
10. Cumulative prednisone dose over the trial
11. Pemphigus Disease Area Index (PDAI) at each visit
12. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score
13. Autoimmune Bullous Disease Quality of Life (ABQOL) score
14. Efgartigimod serum concentrations
15. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
16. Anti-Dsg-1 and -3 autoantibodies serum levels
17. Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and rHuPH20 (plasma levels)

• Percentuale di pazienti che ottengono una CR con terapia minima trai i pazienti affetti da PV
• Percentuale di pazienti che ottengono una CR con terapia minima tra i pazienti affetti da PV e PF
• Tempo al DC
• Tempo alla CR
• Tempo alla CR con terapia minima con prednisone
• Tempo alla CR senza terapia
• Tempo alla riacutizzazione
• Tasso di fallimento del trattamento
• Tasso di riacutizzazione
• Dose cumulativa di prednisone nell’arco della sperimentazione
• Indice dell’area della malattia di pemfigo (PDAI) a ogni visita
• Scala EuroQol a 5 dimensioni e 5 livelli (EQ-5D-5L)
• Punteggio del Questionario sulla qualità della vita con malattia autoimmune bollosa (ABQOL)
• Concentrazioni sieriche di efgartigimod
• Livelli sierici di IgG totale e sottotipo (IgG1, IgG2, IgG3, IgG4)
• Livelli sierici di autoanticorpi anti-Dsg-1 e -3
• Anticorpi anti-farmaco (ADA) per efgartigimod (livelli sierici) e rHuPH20 (livelli plasmatici)

E.5.2.1

Timepoint(s) of evaluation of this end point

1+2: up to 52 weeks treatment period
3,4,5,6,7,8,9: up to 60 weeks
10,11,12,13: up to 52 weeks treatment period
14,15,16, 17: up to 60 weeks

1+2: periodo di trattamento fino a 52 settimane
3, 4, 5, 6, 7, 8, 9: fino a 60 settimane
10, 11, 12, 13: periodo di trattamento fino a 52 settimane
14, 15, 16, 17: fino a 60 settimane

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

immunogenicità

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

in aperto

open

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

Georgia

India

Israel

Japan

Russian Federation

Turkey

Ukraine

United States

Bulgaria

France

Germany

Greece

Hungary

Italy

Poland

Romania

Spain

United Kingdom

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:

105

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

100

F.4.2.2

In the whole clinical trial

150

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)

Under certain conditions described in the protocol, the patients may be eligible to roll over to the open-label extension trial ARGX-113-1905. For patients, randomized to the efgartigimod PH20 SC or placebo treatment arm in ARGX-113-1904, trial ARGX-113-1905 provides extended or first treatment and re-treatment options in the respective treatment arms. argenx is currently assessing the appropriateness and possibility of making efgartigimod PH20 SC available for trial participants post-trial.

In det condizioni descritte nel protocollo, i paz potrebbero essere idonei a passare allo studio di estensione in aperto ARGX-113-1905. Per i paz, randomizzati a efgartigimod PH20 SC o con placebo nello studio ARGX-113-1904, la speri. ARGX-113-1905 fornisce opzioni di trattamento prolungato o primo trattamento e ri-trattamento nei rispettivi bracci. Argenx sta valutando l'appropriatezza e la possibilità di rendere efgartigimod PH20 SC disponibile ai partecipanti alla speri. dopo la stessa.

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

2020-12-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-10-06

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2024-01-15

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