ICH GCP - EU Clinical trials Registry - 2021-003063-10 (IT)

Prove cliniche Nct

Summary
EudraCT Number:	2021-003063-10
Sponsor's Protocol Code Number:	ARGX-113-2010
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:	2023-01-04
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

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-003063-10

A.3

Full title of the trial

An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

Studio di estensione del ARGX-113-2009, in aperto, volto a valutare la sicurezza, la tollerabilità e l’efficacia a lungo termine di efgartigimod PH20 SC in pazienti adulti affetti da pemfigoide bolloso.

A.3.1

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

A phase 3 study to evaluate the long-term safety, tolerability and efficacy of efgartigimod PH20 SC in adult participants with bullous pemphigoid

Studio di fase 3 volto a valutare la sicurezza, la tollerabilità e l’efficacia a lungo termine di efgartigimod PH20 SC in pazienti adulti affetti da pemfigoide bolloso.

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

ARGX-113-2010

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 (Ghent)
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	Yes
D.2.1.1.1	Trade name	Galen
D.2.1.1.2	Name of the Marketing Authorisation holder	Galen Pharma 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	Galen
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.3.9.2	Current sponsor code	na
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: 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	Galen
D.2.1.1.2	Name of the Marketing Authorisation holder	Galen Pharma 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	Galen
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.3.9.2	Current sponsor code	na
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: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	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	Efgartigimod alfa
D.3.9.4	EV Substance Code	SUB198780
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	180
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	Yes
D.2.1.1.1	Trade name	Galen
D.2.1.1.2	Name of the Marketing Authorisation holder	Galen Pharma 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	Galen
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.3.9.2	Current sponsor code	na
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	Dacortin
D.2.1.1.2	Name of the Marketing Authorisation holder	Laboratorios ERN
D.2.1.2	Country which granted the Marketing Authorisation	Spain
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	Dacortin
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.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB10020MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2500
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Bullous Pemphigoid

Pemfigoide bolloso

E.1.1.1

Medical condition in easily understood language

Autoimmune disease that causes painful or itchy blisters on the skin

Malattia autoimmune che provoca vesciche dolorose o pruriginose sulla pelle.

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	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10006567
E.1.2	Term	Bullous pemphigoid
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 long-term safety and tolerability of treatment with efgartigimod PH20 SC in participants with BP

Valutare la sicurezza e la tollerabilità a lungo termine del trattamento con efgartigimod PH20 SC in pazienti affetti da pemfigoide bolloso (PB)

E.2.2

Secondary objectives of the trial

To assess the long-term efficacy and durability of response with efgartigimod PH20 SC treatment in participants with BP
To evaluate the impact of efgartigimod PH20 SC treatment in reducing long-term glucocorticoid-associated toxicity in participants with BP
To evaluate the impact of efgartigimod PH20 SC treatment on QoL in participants with BP
To evaluate the PD and immunogenicity of efgartigimod PH20 SC in participants with BP

Valutare l’efficacia a lungo termine e la durabilità della risposta con il trattamento con efgartigimod PH20 SC in partecipanti affetti da PB.
Valutare l’impatto del trattamento con efgartigimod PH20 SC nel ridurre la tossicità associata ai glucocorticoidi a lungo termine nei partecipanti con PB.
Valutare l’impatto del trattamento con efgartigimod PH20 SC sulla qualità della vita nei partecipanti con PB.
Valutare la farmacodinamica (Pharmacodynamics, [PK]) e l’immunogenicità di efgartigimod PH20 SC nei partecipanti affetti da PB.

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: Substudy – Vaccination Response
Sites may collect additional blood samples for additional/optional/future vaccination research. Such research may include (but not limited to) the following:
1. Humoral (serum) responses to vaccinations received during the study
2. Cellular (peripheral blood mononuclear cells [PBMCs]) responses to vaccinations received during the study
Postvaccination samples will be collected at the visits identified in the Summary of Activities only from participants who took part in the vaccination substudy during ARGX-113-2009.
Data obtained from this substudy will not be included in the clinical database; the results may be described in a separate report.

Altre tipologie di sottostudi
specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Sottostudio - Risposta alla vaccinazione
I centri possono raccogliere ulteriori campioni di sangue per la ricerca aggiuntiva/facoltativa/futura sulle vaccinazioni. Tale ricerca può includere (a titolo esemplificativo ma non esaustivo) quanto segue:
1.. Risposte umorali (su siero) alle vaccinazioni ricevute durante lo studio.
2. Risposte cellulari (cellule mononucleate del sangue periferico [Peripheral Blood Mononuclear Cells, PBMC]) alle vaccinazioni ricevute durante lo studio.
I campioni post-vaccinazione saranno raccolti alle visite identificate nella Sintesi delle attività solo dai partecipanti che hanno partecipato al sottostudio sulla vaccinazione durante lo studio ARGX-113-2009.
I dati ottenuti da questo sottostudio non saranno inclusi nel database clinico; i risultati possono essere descritti in una relazione separata.

E.3

Principal inclusion criteria

1. Has completed the week 36 visit of ARGX-113-2009
2. Is capable of providing signed informed consent and complying with protocol requirements
3. Agrees to use contraceptive measures consistent with local regulations and the following:
a. Male participants: An acceptable method of contraception is a condom. All nonsterilized male participants must use this method from signing of the ICF until the date of the last dose of IMP.
b. Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. WOCBP must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP

1. Ha completato la visita della settimana 36 di ARGX-113-2009.
2. È in grado di fornire il consenso informato firmato e di attenersi ai requisiti del protocollo.
3. Accetta di utilizzare misure contraccettive conformi alle normative locali e a quanto segue:
a. Partecipanti di sesso maschile: un metodo contraccettivo accettabile è il preservativo.
Tutti i partecipanti di sesso maschile non sterilizzati devono utilizzare questo metodo dalla firma del Modulo di consenso informato (Informed Consent Form, [ICF]) fino alla data dell’ultima dose di IMP.
b. Le donne in età fertile devono risultare negative al test
di gravidanza sulle urine al basale prima di ricevere l’IMP. Le donne in età fertile (Women of Childbearing Potential, [WOCBP]) devono utilizzare uno dei metodi contraccettivi descritti nel protocollo dalla firma dell’ICF fino all’ultima dose dell’IMP.

E.4

Principal exclusion criteria

1. Clinically significant disease, recent major surgery (within 3 months of baseline), or intention to have surgery during the study; or any other medical condition that, in the investigator’s opinion would confound the results of the study or put the participant at undue risk
2. Known hypersensitivity to IMP or 1 of its excipients

1. Malattia clinicamente significativa, intervento di chirurgia maggiore recente (entro 3 mesi dal basale) o intenzione di sottoporsi a un intervento chirurgico durante lo studio; o qualsiasi altra condizione medica che, a giudizio dello sperimentatore, potrebbe confondere i risultati dello studio o esporre il partecipante a un rischio ingiustificato 2. Ipersensibilità nota all’IMP o a uno dei suoi eccipienti

E.5 End points

E.5.1

Primary end point(s)

Incidence and severity of treatment-emergent adverse events (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)
Rate of treatment discontinuation because of safety concerns

Incidenza e gravità degli eventi avversi emergenti dal trattamento (Treatment-Emergent Adverse Event, [TEAE]), degli eventi avversi seri (Serious Adverse Event, [SAE]) e del tasso di interruzione del trattamento per motivi di sicurezza

E.5.1.1

Timepoint(s) of evaluation of this end point

Up to wk 56

Fino alla sett. 56

E.5.2

Secondary end point(s)

1. Proportions of participants achieving:
- complete remission (CR) while off oral corticosteroids (OCS) for =8 weeks
- CR or partial remission (PR) while off OCS for =8 weeks
- CR while on minimal OCS therapy for =8 weeks. (Minimal OCS therapy is defined as =0.10 mg/kg/day of prednisone [or an equivalent dose of another OCS])
- CR while off both OCS and efgartigimod PH20 SC for =8 weeks
- CR or PR while off both OCS and efgartigimod PH20 SC for =8 weeks
2. Duration of sustained remission
3. Proportion of participants who relapse
4. Time to relapse
5. Incidence and severity of relapse
6. Bullous Pemphigoid Disease Area Index (BPDAI) activity scores, Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores, and itch numerical rating scale (NRS) over time
7. Rate of treatment failure
8. GTI-related scores, including the GTI-AIS, GTI CWS, and GTI SL over time
9. EQ-5D-5L, DLQI, and ABQoL scores over time
10. Percent change from baseline over time for anti BP180 and anti BP230 antibody levels
11. Incidence and prevalence of ADA against efgartigimod (serum levels)

1. Percentuali di partecipanti che ottengono:
- remissione completa (Complete Remission, [CR]) senza assunzione di corticosteroidi orali (Oral Corticosteroids, [OCS]) per =8 settimane;
- CR o remissione parziale (Partial Remission, [PR]) senza assunzione di OCS per =8 settimane;
- CR durante la terapia minima con OCS per =8 settimane. (La terapia minima con OCS è definita come =0,10 mg/kg/die di prednisone [o una dose equivalente di un altro OCS]);
- CR in un periodo senza assunzione di OCS né di efgartigimod PH20 SC per =8 settimane;
- CR o PR in un periodo senza assunzione di OCS né di efgartigimod PH20 SC per =8 settimane.
2. Durata della remissione sostenuta
3. Percentuale di partecipanti che manifestano recidiva
4. Tempo alla recidiva
5. Incidenza e gravità della recidiva
6. Punteggi di attività dell’Indice di area della malattia del pemfigoide bolloso (Bullous Pemphigoid Disease Area Index, [BPDAI]), punteggi della Valutazione globale dello sperimentatore del pemfigoide bolloso (Investigator Global Assessment of Bullous Pemphigoid, [IGA-BP]) e scala di valutazione numerica del prurito (Itch Numerical Rating Scale, [NRS]) nel tempo
7. Tasso di fallimento del trattamento
8. Punteggi correlati all’indice di tossicità dei glucocorticoidi (Glucocorticoid Toxicity Index, [GTI]), inclusi il punteggio di miglioramento aggregato (Aggregate Improvement Score, [AIS]) del GTI (GTI-AIS), il punteggio cumulativo di peggioramento (Cumulative Worsening Score, CWS) del GTI (GTI CWS) e l’elenco specifico (Specific List, [SL]) del GTI (GTI SL) nel tempo
9. Punteggi del questionario del gruppo EuroQoL, versione a 5 dimensioni e 5 livelli (EQ-5D-5L), dell’indice dermatologico della qualità della vita (Dermatology Life Quality Index, [DLQI] e del questionario sulla qualità della vita con malattia autoimmune bollosa (Autoimmune Bullous Quality of Life, [ABQoL] nel tempo
10. Variazione percentuale rispetto al basale nel tempo per i livelli degli anticorpi anti BP180 e anti BP230
11. Incidenza e prevalenza di anticorpi anti-farmaco (Anti-Drug Antibodies, [ADA] contro efgartigimod (livelli sierici)

E.5.2.1

Timepoint(s) of evaluation of this end point

1-5 & 7: Up to wk 56
6: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after & at wks 48, 52 & 56
8 & 9: At wks 0, 24 & 48 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 8, every 16 wks up to & after EFG stop, then at wk 48
10: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56
11: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 4, 8, then every 8 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56

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

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

Yes

E.6.13.1

Other scope of the trial description

Tolerability

Tollerabilità

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

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

Studio in aperto

open study

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

China

Israel

Japan

United States

France

Latvia

Poland

Bulgaria

Netherlands

Romania

Spain

Czechia

Germany

Greece

Italy

Croatia

Hungary

Russian Federation

Slovakia

Ukraine

United Kingdom

Serbia

E.8.7

Trial has a data monitoring committee

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

Ultima visita dell'utlimo paziente

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:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

120

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

160

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)

Participants will receive standard of care as directed by their doctor

I partecipanti riceveranno lo standard di cura come da istruzioni del proprio medico

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

2023-04-05

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-12-14

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

Prove cliniche Nct

Sponsor e collaboratori

Condizioni mediche

Interventi farmacologici