Clinical Trials Register

Summary
EudraCT Number:	2020-005841-18
Sponsor's Protocol Code Number:	ARGX-113-2006
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:	2021-08-25
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

2020-005841-18

A.3

Full title of the trial

Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

Sperimentazione non controllata in aperto per valutare la farmacocinetica, la farmacodinamica, la sicurezza e l’attività di efgartigimod in pazienti pediatrici di età compresa tra 2 e meno di 18 anni con miastenia gravis generalizzata

A.3.1

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

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

Valutazione di farmacocinetica, farmacodinamica e sicurezza di efgartigimod somministrato per via endovenosa nei pazienti pediatrici con miastenia gravis generalizzata

A.3.2

Name or abbreviated title of the trial where available

ADAPT JR

A.4.1

Sponsor's protocol code number

ARGX-113-2006

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/097/2020

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	9052
B.5.3.4	Country	Belgium
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	EMA/OD/245/17
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.3.9.4	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	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

Generalized Myasthenia Gravis

Miastenia Gravis Generalizzata

E.1.1.1

Medical condition in easily understood language

Myasthenia Gravis in patients aged 2 to 18 years who have generalized
Muscle Weakness

Miastenia gravis in pazienti di età compresa tra 2 e 18 anni con debolezza muscolare generalizzata

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	PT
E.1.2	Classification code	10028417
E.1.2	Term	Myasthenia gravis
E.1.2	System Organ Class	10029205 - Nervous system disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To confirm an age-adjusted optimum dose of efgartigimod IV and provide (model-predicted) evidence for a treatment response

Confermare una dose ottimale corretta per l’età di efgartigimod e.v. e fornire evidenze (previste dal modello) per una risposta al trattamento

E.2.2

Secondary objectives of the trial

-To evaluate the safety and tolerability of efgartigimod IV
-To evaluate the PK and PD of efgartigimod IV
-To evaluate the immunogenicity of efgartigimod IV
-To evaluate the activity and impact on quality of life of efgartigimod IV
-To evaluate the effect of efgartigimod treatment on antibody response to vaccines

-Valutare PK e PD di efgartigimod e.v.
-Valutare l’immunogenicità di efgartigimod e.v.
-Valutare l’attività e l’impatto sulla qualità della vita di efgartigimod e.v.
-Valutare l’effetto del trattamento con efgartigimod sulla risposta anticorpale ai vaccini

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Participants are eligible to be included in the trial only if all of the
following criteria apply:
1. Ability of the participant and/or his/her legally authorized
representative to understand the requirements of the trial and provide
written informed consent/assent, if applicable (including
consent/assent for the use and disclosure of research-related health
information), willingness and ability to comply with the trial protocol
procedures (including attending the required trial visits).
2. Male or female participants between 2 to less than 18 years of age at
the time of providing informed consent/assent. Age groups are enrolled
in a staggered fashion respectively: 6 participants in the 12 to less than
18 years of age group followed by 6 participants in the 2 to less than 12
years of age group at the time of providing informed consent/assent.
3. Diagnosed with gMG with confirmed documentation
4. Meeting the clinical criteria as defined by the Myasthenia Gravis
Foundation of America (MGFA) class II, III, and IVa.
5. Eligible participants should have an unsatisfactory response (efficacy
and/or safety) to immunosuppressants, steroids or AChE inhibitors and should be on stable concomitant gMG therapy of adequate duration
before screening.
6. Positive serologic test for anti-AChR antibodies at screening (for
younger participants (<15kg) historical values can be used).
7. Contraceptive use should be consistent with local regulations
regarding the methods of contraception for those participating in clinical
trials. A subject is of childbearing potential if, in the opinion of the
investigator, he/she is biologically capable of having children and is
sexually active.
a. Male participants: Male participants must agree to not donate sperm
from the time the ICF was signed until the end of the trial.
b. Female participants:
Female adolescents of childbearing potential must have a negative
serum pregnancy test at screening and a negative urine pregnancy test
at baseline before IMP can be administered.

I partecipanti possono essere inclusi nella sperimentazione solo se soddisfano tutti i seguenti criteri:
1. Capacità del/la partecipante e/o del suo rappresentante legale di comprendere i requisiti della sperimentazione e fornire consenso/assenso informato scritto, se applicabile (compreso il consenso/l’assenso per l’uso e la divulgazione di informazioni sanitarie relative alla ricerca), disponibilità e capacità di rispettare le procedure del protocollo di sperimentazione (inclusa la partecipazione alle visite della sperimentazione richieste).
2. Partecipanti di sesso maschile o femminile di età compresa tra 2 e meno di 18 anni al momento della fornitura del consenso/assenso informato. Le fasce di età sono arruolate in modo scaglionato: 6 partecipanti nella fascia di età compresa tra 12 e meno di 18 anni, seguiti da 6 partecipanti nella fascia di età compresa tra 2 e meno di 12 anni al momento della fornitura del consenso/assenso informato.
3. Diagnosi di gMG con documentazione confermata
4. Soddisfacimento dei criteri clinici definiti dalla classe II, III e IVa della Myasthenia Gravis Foundation of America (MGFA).
5. I partecipanti idonei devono presentare una risposta insoddisfacente (efficacia e/o sicurezza) a immunosoppressori, steroidi o AChE-inibitori e devono stare assumendo una terapia per la gMG concomitante stabile per un periodo di tempo adeguato prima dello screening.
6. Test sierologico positivo per anticorpi anti-AChR allo screening (per i partecipanti più giovani (<15 kg) possono essere utilizzati valori storici).
7. L’uso di contraccettivi deve essere conforme alle normative locali riguardanti i metodi di contraccezione per coloro che partecipano a sperimentazioni cliniche. Un soggetto è in età fertile se, a giudizio del
PI, è biologicamente in grado di avere figli ed è sessualmente attivo.
a. Partecipanti di sesso maschile: I partecipanti di sesso maschile devono accettare di non donare sperma dal momento della firma del modulo di consenso informato fino alla fine della sperimentazione.
b. Partecipanti di sesso femminile: Le adolescenti di sesso femminile in età fertile devono avere un test di gravidanza sul siero negativo allo screening e un test di gravidanza sulle urine negativo al basale prima che possa essere somministrato l’IMP.

E.4

Principal exclusion criteria

Participants are excluded from the trial if any of the following criteria
apply:
1. Participants with MGFA class I, IVb, and V.
2. FAOCBP: Pregnancy or lactation, or the participant intends to become
pregnant during the trial or within 90 days after the last dose of IMP.
3. Has any of the following medical conditions:
a) Clinically significant uncontrolled active or chronic bacterial, viral, or
fungal infection at screening.
b) Any other known autoimmune disease that, in the opinion of the
investigator, would interfere with an accurate assessment of clinical
symptoms of myasthenia gravis or put the participant at undue risk.
c) History of malignancy unless deemed cured by adequate treatment
with no evidence of recurrence for =3 years before the first
administration of the IMP. Participants with the following cancers can be
included at any time:
-Adequately treated basal cell or squamous cell skin cancer
-Carcinoma in situ of the cervix
-Carcinoma in situ of the breast
-Incidental histological findings of prostate cancer (TNM Classification of
Malignant Tumors stage T1a or T1b)
d) Clinical evidence of other significant serious diseases, or have had a
recent major surgery, or who have any other condition that, in the
opinion of the investigator, could confound the results of the trial or put
the participant at undue risk.
4. Worsening muscle weakness secondary to concurrent infections or
medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).
5. A documented lack of clinical response to plasma exchange (PLEX).
6. Received a live or live-attenuated vaccine fewer than 28 days before
screening. Receiving an inactivated, subunit, polysaccharide, or
conjugate vaccine any time before screening is not exclusionary.
7. Received a thymectomy <3 months before screening or 1 is planned to
be performed during the trial period.
8. The following results from these diagnostic assessments will be
considered exclusionary:
a. Positive serum test at screening for an active viral infection with any of the following conditions:
-Hepatitis B virus (HBV) that is indicative of an acute or chronic infection
(https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
-Hepatitis C virus (HCV) based on HCV antibody assay
-Human immunodeficiency virus (HIV) associated with a CD4 count
<200 cells/mm3 with an acquired immunodeficiency syndrome (AIDS)defining condition, such as: Cytomegalovirus retinitis with loss of vision,
Pneumocystis jiroveci pneumonia, chronic intestinal cryptosporidiosis,
HIV-related encephalopathy, Mycobacterium tuberculosis (pulmonary or extrapulmonary), or invasive cervical cancer
b. Positive nasopharyngeal swab PCR test for SARS-CoV-2 at creening .
9. Using the following prior or concomitant therapies:
a. Use of an investigational product within 3 months or 5 half-lives
(whichever is longer) before the first dose of the IMP.
b. Use of any monoclonal antibody within the 6 months before the first
dose of the IMP.
c. Use of IVIg, immunoglobulins administered SC or intramuscularly, or
PLEX within 4 weeks before screening.
10. Total IgG levels levels below the lower limit of normal (LLN) according to
the reference ranges of the central laboratory for participant by sex and age at screening.
11. A known hypersensitivity reaction to efgartigimod or any of its
excipients.

I partecipanti sono esclusi dalla sperimentazione se soddisfano uno qualsiasi dei seguenti criteri:
1. Partecipanti con MGFA di classe I, IVb e V.
2. FAOCBP: gravidanza o allattamento, o la partecipante intende rimanere incinta durante lo studio o entro 90 giorni dall’ultima dose di IMP.
3. Presenta una delle seguenti condizioni mediche:
a) Infezione batterica, virale o fungina attiva o cronica clinicamente significativa allo screening.
b) Qualsiasi altra malattia autoimmune nota che, secondo il parere dello sperimentatore, interferirebbe con una valutazione accurata dei sintomi clinici della miastenia gravis o esporrebbe il/la partecipante a un rischio eccessivo.
c) Anamnesi di tumore maligno a meno che non si ritenga curato da un trattamento adeguato senza evidenza di recidiva per =3 anni prima della prima somministrazione dell’IMP. I partecipanti con i seguenti tumori possono essere inclusi in qualsiasi momento:
-Carcinoma cutaneo a cellule basali
-Carcinoma in situ della cervice
-Carcinoma in situ della mammella
-Reperti istologici accidentali di cancro alla prostata (stadio T1a o T1b della Classificazione TNM dei tumori maligni)
d) Evidenze cliniche di altre malattie gravi significative, o soggetti che hanno subito un intervento chirurgico maggiore recente o che presentano qualsiasi altra condizione che, a giudizio dello sperimentatore, potrebbe alterare i risultati della sperimentazione o esporre il/la partecipante a un rischio eccessivo.
4. Peggioramento della debolezza muscolare secondaria a infezioni o farmaci concomitanti (aminoglicosidi, fluoro-chinoloni, beta-bloccanti, ecc.).
5. Documentata mancanza di risposta clinica alla plasmaferesi (PLEX).
6. Inoculazione di un vaccino vivo o vivo attenuato meno di 28 giorni prima dello screening. L’inoculazione di un vaccino inattivato, subunità, polisaccaride o coniugato in qualsiasi momento prima dello screening non è motivo di esclusione.
7. Timectomia <3 mesi prima dello screening o che si prevede venga eseguita durante il periodo di sperimentazione.
8. I seguenti risultati di queste valutazioni diagnostiche saranno considerati motivo di esclusione:
Test sul siero positivo allo screening per un’infezione virale attiva con una delle seguenti condizioni:
-Virus dell’epatite B (HBV) che è indicativo di un’infezione acuta o cronica (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
-Virus dell’epatite C (HCV) basato sul dosaggio degli anticorpi anti-HCV
-Virus dell’immunodeficienza umana (HIV) associato a una conta CD4 <200 cellule/mm3 con una condizione che definisce la sindrome da immunodeficienza acquisita (AIDS), come: Retinite da citomegalovirus con perdita della vista, polmonite da Pneumocystis jiroveci, criptosporidiosi intestinale cronica, encefalopatia correlata all’HIV, Mycobacterium tuberculosis (polmonare o extrapolmonare) o cancro cervicale invasivo
Test PCR su tampone nasofaringeo positivo per SARS-CoV-2 allo screening
9. Uso delle seguenti terapie precedenti o concomitanti:
a. Uso di un prodotto sperimentale entro 3 mesi o 5 emivite (a seconda di quale sia il periodo più lungo) prima della prima dose di IMP.
b. Uso di qualsiasi anticorpo monoclonale nei 6 mesi precedenti la prima dose di IMP.
c. Uso di IVIg, immunoglobuline somministrate per via s.c. o per via intramuscolare o PLEX nelle 4 settimane precedenti lo screening.
10. Livelli totali di IgG al di sotto del limite inferiore della norma (LLN) secondo
i range di riferimento del laboratorio centrale per i partecipanti per sesso ed età allo screening.
11. Nota reazione di ipersensibilità a efgartigimod o ad uno qualsiasi dei suoi eccipienti.

E.5 End points

E.5.1

Primary end point(s)

- Efgartigimod concentrations as input for compartmental, model-driven
analysis to determine (age and size dependency of) clearance (CL) and
volume of distribution (Vd)
- PD parameters: total IgG levels and anti-acetylcholine receptors
antibodies (AChR-Ab) as input for PK/PD modeling analysis

-Concentrazioni di efgartigimod come input per l’analisi compartimentale basata sul modello per determinare (dipendenza dall’età e dalla dimensione di) clearance (CL) e volume di distribuzione (Vd)
-Parametri PD: livelli totali di IgG e anticorpi anti-recettori dell’acetilcolina (AChR-Ab) come input per l’analisi di modellazione PK/PD

E.5.1.1

Timepoint(s) of evaluation of this end point

26 weeks

26 settimane

E.5.2

Secondary end point(s)

-Incidence and severity of adverse events (AEs), incidence of serious
adverse events (SAEs)
- Efgartigimod serum concentrations
-Levels of total IgG and anti-acetylcholine receptor antibodies (AChRAb): absolute values, change from baseline and percent (%) change

from
baseline
-Incidence and prevalence of anti-drug antibodies (ADAs) against

efgartigimod
-MG-ADL: absolute value and change from baseline of total MG-ADL score
-Total QMG score: absolute value and change from baseline of total QMG score
-Total score EQ-5D-Y: absolute value and change from baseline
-Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score: values and change from baseline
-Change in protective antibody titers to vaccines received before or received during the trial

-Incidenza e gravità di eventi avversi (EA), incidenza di eventi avversi gravi (SAE)
-Concentrazioni sieriche di efgartigimod
-Livelli di IgG totali e anticorpi diretti con il recettore dell’acetilcolina (Ab AChR): valori assoluti, variazione rispetto al basale e variazione percentuale (%) rispetto al basale
-Incidenza e prevalenza di anticorpi anti-farmaco (ADA) contro efgartigimod
-MG-ADL: valore assoluto e variazione rispetto al basale del punteggio MG-ADL totale
-Punteggio QMG totale: valore assoluto e variazione rispetto al basale del punteggio QMG totale
-Punteggio totale EQ-5D-Y: valore assoluto e variazione rispetto al basale
-Punteggio Pediatric Fatigue Neurological Quality of Life (Neuro-QoL): valori e variazione rispetto al basale
-Variazione dei titoli anticorpali protettivi ai vaccini ricevuti prima o durante la sperimentazione

E.5.2.1

Timepoint(s) of evaluation of this end point

2nd endpoint 1,4,8 = 28 weeks
endpoint 2,3,5,6,7 = 26 weeks

2o endpoint 1,4,8 = 28 settimane
endpoint 2,3,5,6,7 = 26 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

Yes

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

Yes

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

- Dose confirmation for paediatric patients aged 2 to 18 years old. - Quality of Life - Tolerability - Biomarkers - Immunogenicity - Health Economics

-Conferma della dose per pazienti pediatrici di età compresa tra 2 e 18 anni. -Qualità della vita -Tollerabilità -Biomarcatori -Immunogenicità -Farmacoeconomia

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)

Yes

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

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

Canada

Georgia

United States

Austria

Belgium

France

Germany

Italy

Netherlands

Poland

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

The end of the trial is defined as the date of the last visit of the last
participant in the ARGX-113-2006 trial. A participant is considered to
have completed the trial if he/she completed the end of trial (EoT) visit
at day 182 ±3 days (trial entry in Part A) or day 126 ±3 days (trial
entry in Part B), even if the participant did not complete treatment.

La fine della sperimentazione corrisponde alla data dell’ultima visita dell’ultimo partecipante alla sperimentazione ARGX-113-2006. Si considera che un partecipante abbia completato la sperimentazione se ha completato la visita di fine sperimentazione (EoT) al giorno 182 ±3 giorni (ingresso nella Parte A della sperimentazione) o al giorno 126 ±3 giorni (ingresso nella Parte B della sperimentazione), anche se il partecipante non ha completato il trattamento.

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

Yes

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

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 the end of the trial participants will have the option to roll over
into a long-term safety extension trial, ARGX-113-2008, to assess long term safety and will be provided continued access until efgartigimod IV becomes commercially available or another option to access efgartigimod IV is available. Treatment may be continued based on investigator discretion provided that there is clear evidence of clinical benefit.

Al termine della sperimentazione, i partecipanti avranno possibilità di passare a sperimentazione di estensione sulla sicurezza a lungo termine, ARGX-113-2008, con l’obiettivo di valutare la sicurezza a lungo termine e sarà garantito loro accesso continuo fino a quando efgartigimod e.v. non sarà in commercio o non lo sarà un’altra opzione per accedere a efgartigimod e.v. Il trattamento può essere continuato a discrezione del PI, a condizione che vi sia una chiara evidenza di beneficio clinico.

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

2021-11-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-10-12

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

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IMP with orphan designation in the indication
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