Clinical Trials Register

Summary
EudraCT Number:	2021-002504-12
Sponsor's Protocol Code Number:	ARGX-113-2003
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-12-02
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Spain - AEMPS

A.2

EudraCT number

2021-002504-12

A.3

Full title of the trial

A Phase 3b, Randomized, Open-label, Parallel-Group Study to Evaluate Different Dosing Regimens of Intravenous Efgartigimod to Maximize and Maintain Clinical Benefit in Patients With Generalized Myasthenia Gravis (gMG)

Estudio en fase IIIb, aleatorizado, abierto, de grupos paralelos para evaluar diferentes pautas posológicas de efgartigimod intravenoso para maximizar y mantener el beneficio clínico en pacientes con miastenia grave generalizada (MGg)

A.3.1

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

An open-label study to investigate the clinical efficacy of different dosing regimens of efgartigimod IV in patients with generalized myasthenia gravis

Estudio abierto para investigar la eficacia clínica de diferentes pautas posológicas de efgartigimod i.v. en pacientes con miastenia grave generalizada

A.3.2

Name or abbreviated title of the trial where available

ADAPT NXT

A.4.1

Sponsor's protocol code number

ARGX-113-2003

A.5.4

Other Identifiers

Name:

IND

Number:

132953

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	Antonio Guglietta
B.5.3	Address:
B.5.3.1	Street Address	Industriepark Zwijnaarde 7
B.5.3.2	Town/ city	Ghent
B.5.3.3	Post code	9052
B.5.3.4	Country	Belgium
B.5.4	Telephone number	+329310 3400
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.3.2	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/kg milligram(s)/kilogram
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

Generalized Myasthenia Gravis (gMG)

Miastenia Grave Generalizada (MGg)

E.1.1.1

Medical condition in easily understood language

Generalized Myasthenia Gravis (gMG)

Miastenia Grave Generalizada (MGg)

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 assess the clinical efficacy of efgartigimod IV 10 mg/kg administered in a q2w continuous regimen compared to that administered in a cyclic regimen

Evaluar la eficacia clínica de efgartigimod i.v. 10 mg/kg administrado en una pauta continua c2s en comparación con la administrada en una
pauta cíclica

E.2.2

Secondary objectives of the trial

• To evaluate the safety and tolerability of both treatment regimens used throughout the study
• To assess the clinical efficacy of efgartigimod IV in both treatment regimens over time
• To compare the number of participants who achieve maximal clinical effect during different treatment regimens

• Evaluar la seguridad y tolerabilidad de ambas pautas de tratamiento
utilizadas a lo largo del estudio.
• Evaluar la eficacia clínica de efgartigimod i.v. en ambas pautas de
tratamiento a lo largo del tiempo.
• Comparar el número de participantes que alcanzan el efecto clínico
máximo durante diferentes pautas de tratamiento.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Capable of giving signed informed consent as described in Section 10.1.3 of the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
2. At least 18 years of age, at the time of signing the informed consent
3. Diagnosed with gMG with confirmed documentation and supported by a physical exam and confirmed seropositivity for AChR-Abs
4. Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MFGA) class II, III, or IV
5. Has an MG-ADL total score ≥5 at screening and the day 1 visit, with more than 50% of the score due to nonocular symptoms
6. Concomitant gMG treatment is permitted. Permitted concomitant gMG treatment includes nonsteroidal immunosuppressive drugs (NSIDs), steroids, and/or acetylcholinesterase (AChE) inhibitors. If receiving corticosteroids and/or NSIDs, must be on a stable dose for at least 1 month before screening.
7. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
a. Male participants:
− Male participants are not allowed to donate sperm from signing the ICF until the end of the study.
b. Female participants:
− WOCBP (defined in Section 10.4.1 of protocol) must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before IMP can be administered.
− The contraception requirements for WOCBP are described in Section 10.4.2.1 of the protocol.

1.Capaz de otorgar su consentimiento informado firmado tal como se describe en la Sección10.1.3, que incluye el cumplimiento de los requisitos y las restricciones enumeradas en el formulario de consentimiento informado (FCI) y en este protocolo.
2.Tener al menos 18 años de edad en el momento de firmar el consentimiento informado
3.Con diagnóstico de MGg con documentación confirmada y con respaldo de una exploración física y seropositividad confirmada para Ac anti-AChR
4.Cumplir los criterios clínicos definidos por las clases II, III o IV de la Fundación de Miastenia Gravis de América (Myasthenia Gravis Foundation of America, MFGA)
5.Tiene una puntuación total de MG-ADL (miastenia grave-actividades de la vida diaria) ≥5 en la selección y en la visita del día 1, con más del 50 % de la puntuación debida a síntomas no oculares
6.Se permite el tratamiento concomitante de MGg. El tratamiento concomitante de la MGg permitido incluye fármacos inmunodepresores no esteroideos (nonsteroidal immunosuppressive drugs, NSID), corticoesteroides y/o inhibidores de la acetilcolinesterasa (AChE). Si recibe corticosteroides y/o NSID, debe estar recibiendo una dosis estable durante al menos 1 mes antes de la selección.
7.El uso de anticonceptivos por parte de hombres y mujeres debe ser coherente con las normativas locales en relación con los métodos anticonceptivos para los participantes en estudios clínicos y:
a.Varones participantes:
Los participantes varones no pueden donar esperma desde la firma del FCI hasta el final del estudio.
b.Mujeres participantes:
Las MEF (definidas en la sección 10.4.1) deben tener una prueba de embarazo en suero negativa en la selección y una prueba de embarazo en orina negativa en el inicio antes de poder administrar el PEI.
Los requisitos anticonceptivos para las MEF se describen en la sección 10.4.2.1.

E.4

Principal exclusion criteria

1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
2. A positive test for SARS-CoV-2 at screening
3. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of the clinical symptoms of gMG and/or put the participant at undue risk
4. History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated at screening:
a. Basal cell or squamous cell skin cancer
b. Carcinoma in situ of the cervix
c. Carcinoma in situ of the breast
d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
5. Clinical evidence of other significant serious diseases, a recent (<3 months) major surgery, or any other condition that, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk
6. A thymectomy within 3 months of screening
7. Pregnant or lactating females and those who intend to become pregnant during the study or within 90 days of the last dose of IMP
8. Use of the following prior or concomitant therapies:
a. IVIg or subcutaneous (SC)Ig within 14 days of day 1
b. Rituximab within 6 months of day 1
c. Eculizumab within 1 month of day 1
d. Other monoclonal antibodies (eg, adalimumab, tocilizumab, ixekizumab) within 5 half-lives of the monoclonal antibodies before day 1
e. Use of any other investigational product within 3 months or 5 half-lives, whichever is longer, before day 1
f. Receipt of a live or live-attenuated vaccine within 4 weeks of screening. The receipt of any inactivated, subunit, polysaccharide, conjugate vaccine at any time before screening is not considered exclusionary.
9. Previous participation in a clinical study or patient access program during which they were treated with efgartigimod
10. Positive serum test at screening for an active viral infection with any of the following conditions:
a. Hepatitis B virus (HBV) that is indictive of an acute or chronic infection
b. Hepatitis C virus (HCV) based on HCV antibody assay (unless associated with a negative HCV RNA test)
c. HIV based on test results that are associated with an AIDS-defining condition or a CD4 count ≤200 cells/mm3
11. Total IgG <6 g/L at screening
12. Known hypersensitivity reaction to efgartigimod or any of its excipients
13. The participant stands in any relationship of dependency with the sponsor.
14. The participant has been institutionalized due to an official or judicial order.

1.Infección bacteriana, vírica o fúngica crónica o activa no controlada clínicamente significativa en la selección
2.Una prueba positiva para SARS-CoV-2 en la selección
3.Cualquier otra enfermedad autoinmunitaria conocida que, en opinión del investigador, pudiera interferir con una evaluación precisa de los síntomas clínicos de la MGg y/o poner al participante en riesgo indebido
4.Antecedentes de neoplasia maligna, a menos que se considere curada con un tratamiento adecuado sin indicios de reaparición durante ≥3 años antes de la primera administración del PEI. Los participantes con los siguientes tipos de cáncer pueden incluirse en cualquier momento, siempre que reciban el tratamiento adecuado en la selección:
a. cáncer de piel de células basales o escamosas
b. carcinoma de cérvix in situ
c. carcinoma de mama in situ
d. hallazgo histológico incidental de cáncer de próstata (estadio T1a o T1b de la clasificación Tumor/Ganglio/Metástasis [Tumour/Node/Metastasis, TNM])
5.Evidencia clínica de otras enfermedades graves significativas, de una cirugía mayor reciente (<3 meses) o de cualquier otra afección que, en opinión del investigador, pueda confundir los resultados del estudio o poner al participante en riesgo indebido
6.Una timectomía en los 3 meses anteriores a la selección
7.Mujeres embarazadas o en periodo de lactancia y aquellas que tengan intención de quedarse embarazadas durante el estudio o en los 90 días posteriores a la última dosis del PEI
8.Uso de los siguientes tratamientos previos o concomitantes:
a. IgIV o subcutánea (s.c.) en los 14 días anteriores al día 1
b. Rituximab en los 6 meses anteriores al día 1
c. Eculizumab en el mes anterior al día 1
d. Otros anticuerpos monoclonales (p. ej., adalimumab, tocilizumab, ixekizumab) en el plazo de 5 semividas de los anticuerpos monoclonales antes del día 1
e. Uso de cualquier otro producto en investigación en los 3 meses o 5 semividas, lo que sea lleve más tiempo, antes del día 1
f. Recepción de una vacuna de microbios vivos o de una vacuna atenuada en las 4 semanas anteriores a la selección. La recepción de cualquier vacuna inactivada, de subunidad, polisacárido o conjugada en cualquier momento antes de la selección no se considera excluyente.
9. Participación previa en un estudio clínico o programa de acceso al paciente durante el cual se haya tratado con efgartigimod
10. Prueba positiva en suero en la selección para una infección vírica activa con cualquiera de las siguientes afecciones (véase la Sección 10.2.1):
a. Virus de la hepatitis B (VHB) que es indicativo de una infección aguda o crónica (Tabla 8) (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
b. Virus de la hepatitis C (VHC) basado en el análisis de anticuerpos del VHC (a menos que esté asociado a una prueba negativa de ARN del VHC) (Tabla 9)
c. El VIH se basa en resultados de pruebas que están asociados a una afección definitoria de SIDA o a un recuento de CD4 ≤200 células/mm3 (Tabla 10)
11. IgG total <6 g/l en la selección
12. Reacción de hipersensibilidad conocida a efgartigimod o a cualquiera de sus excipientes
13. El participante mantiene cualquier relación de dependencia con el promotor.
14. El participante ha sido institucionalizado debido a una orden oficial o judicial.

E.5 End points

E.5.1

Primary end point(s)

Mean of the average MG-ADL total score change from baseline during the visit of week (W)1 through W21 by regimen arm

Cambio medio en el promedio de la puntuación total de la escala de
actividades de la vida diaria en la miastenia grave (Myasthenia Gravis
Activities of Daily Living, MG-ADL) con respecto al inicio durante la visita
de la semana (S)1 hasta la S21 por grupo de pauta

E.5.1.1

Timepoint(s) of evaluation of this end point

21 weeks

21 Semanas

E.5.2

Secondary end point(s)

1: Incidence and severity of adverse events (AEs), serious adverse events (SAEs) and AEs of special interest (AESIs) Time point: 126 weeks
2: Incidence of serious adverse events (SAEs) and AEs of special interest (AESIs) Time point: 126 weeks
3: Change from baseline in the Myasthenia Gravis – Activities of Daily Living (MG-ADL) total score over time. A higher total score indicates more impairment. Time point: 128 weeks
4: Normalized area under the effect curve (AUEC) of MG-ADL total score improvement from baseline during following intervals: Day 1 through Week7, Week 7 through Week 14, Week 14 trough Week 21 and Week 7 through Week 21. Time point: 21 weeks
5: Characterization of MG-ADL total score change from baseline during the following 5 intervals using mean and standard deviation: Week 1 through Week 7, Week 8 through Week 14, Week 15 through Week 21, Week 8 through Week 21 and Week 1 through Week 21. Time point: 21 weeks
6: Number of participants who have a ≥2, 3, 4, or 5 points improvement in MG-ADL total score from baseline. during the following 5 intervals: Week 1 through Week 7, Week 8 through Week 14, Week 15 through Week 21, Week 8 through Week 21 and Week 1 through Week 21. Time point: 21 weeks
7: percentage of participants who have a ≥2, 3, 4, or 5 points improvement in MG-ADL total score from baseline during the following 5 intervals: Week 1 through Week 7, Week 8 through Week 14, Week 15 through Week 21, Week 8 through Week 21 and Week 1 through Week 21. Time point: 21 weeks
8: Percentage of time, participants have a change in MG-ADL total score of at least 2 points from baseline during Week 4 through Week 21. Time point: 21 weeks
9: Number of participants who achieve minimal symptom expression (MSE), defined as a MG-ADL total score of 0 or 1. Time point: 21 weeks
10: Percentage of participants who achieve minimal symptom expression (MSE), defined as a MG-ADL total score of 0 or 1 in the following 5 intervals: Week 1 through Week 7, Week 8 through Week 14, Week 15 through Week 21, Week 8 through Week 21 and Week 1 through Week 21. Time point: 21 weeks

1•Incidencia e intensidad de los acontecimientos adversos (AA),
incidencia de los acontecimientos adversos graves (AAG), incidencia de
los AA de especial interés (AAEI) Periodo de valoración: 126 semanas.
2. Cambio con respecto al inicio de actividades de la vida diaria en la
miastenia grave (MG-ADL) puntuación total a lo largo del tiempo. Una
puntuación total elevada indica mayor deterioro. Periodo de valoración:
128 semanas.
3•Área bajo la curva del efecto (ABCE) normalizada de la mejora de la
puntuación total de la MG-ADL con respecto al inicio durante los
siguientes intervalos:o día 1 hasta semana 7, Semana 7 hasta Semana
14, Semana 14 hasta Semana 21 y Semana 7 hasta Semana 21. Periodo
de valoración:21 semanas.
4.•Caracterización del cambio en la puntuación total de la MG-ADL con
respecto al inicio durante los siguientes 5 intervalos utilizando la media
y la desviación estándar:Semana 1 hasta Semana7, Semana 8 hasta
Semana14, Semana 15 hasta Semana 21, Semana 8 hasta Semana 21 y
Semana 1 hasta Semana 21. Periodo de valoración:21 semanas.
5.•Número y porcentaje de participantes que tienen una mejora de ≥2, 3, 4 o 5 puntos en la puntuación total de la MG-ADL con respecto al inicio
durante los siguientes 5 intervalos: Semana 1 hasta Semana 7, Semana
8 hasta Semana 14, Semana 15 hasta Semana 21, Semana 8 hasta
Semana 21,Semana 1 hasta Semana 21 .Periodo de valoración:21 semanas.
6•Porcentaje de veces que los participantes tienen una reducción en la
puntuación total de la MG-ADL de al menos 2 puntos con respecto al
inicio durante la Semana4 hasta la Semana21.Periodo de valoración:21
semanas.
7•Número de participantes que alcanzan una expresión mínima de los
síntomas (EMS), definida como una puntuación total de 0 o 1 en la MGADL.
Periodo de valoración:21 semanas.
8• Porcentaje de participantes que alcanzan una expresión mínima de
los síntomas (EMS), definida como una puntuación total de 0 o 1 en la
MG-ADL en los siguientes 5 intervalos: Semana1 hasta la Semana7, Semana 8 hasta la Semana14,Semana15 hasta la Semana 21,Semana 8 hasta la Semana21 ySemana1 hasta la Semana21.Periodo de
valoración:21 semanas.

E.5.2.1

Timepoint(s) of evaluation of this end point

Please refer individual end points mentioned in above section.

Por favor consulte los puntos de valoración individuales mencionados en
la sección anterior.

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 and tolerability

Inmunogenicidad y tolerabilidad

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

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

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)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

efgartigimod administrado en un régimen continuo versus efgartigimod en un régimen cíclico

efgartigimod administered in a continuous regimen vs efgartigimod in a cyclic regimen

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

Georgia

Austria

Belgium

Canada

Germany

Italy

Netherlands

Poland

Spain

United Kingdom

United States

France

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

UVUP

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:

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)

At the end of the study, argenx will comply with all local regulations for ensuring continued access to IMP medically identified as essential.

Al final del estudio, argenx cumplirá con todas las regulaciones locales para garantizar el acceso continuo a IMP identificado médicamente como esencial.

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

2021-11-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-10-27

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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