Clinical Trials Register

Summary
EudraCT Number:	2018-002132-25
Sponsor's Protocol Code Number:	ARGX-113-1704
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-10-29
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

France - ANSM

A.2

EudraCT number

2018-002132-25

A.3

Full title of the trial

A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients with Myasthenia Gravis Having Generalized Muscle Weakness

Essai multicentrique, randomisé, en double aveugle, contrôlé par placebo, de phase 3 visant à évaluer l’efficacité, la sécurité et la tolérance de l’ARGX-113 chez des patients atteints de myasthénie grave présentant une faiblesse musculaire généralisée

A.3.1

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

An efficacy and safety study of ARGX-113 in patients with myasthenia gravis who have generalized muscle weakness.

Une étude visant à évaluer l'efficacité et la sécurité d'ARGX-113 chez des patients atteints de myasthénie grave présentant une faiblesse musculaire généralisée.

A.4.1

Sponsor's protocol code number

ARGX-113-1704

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	argenx BVBA
B.1.3.4	Country	Belgium
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	argenx BVBA
B.4.2	Country	Belgium
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	argenx BVBA
B.5.2	Functional name of contact point	Regulatory affairs
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.5.6	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
D.3.9.1	CAS number	1821402-21-4
D.3.9.2	Current sponsor code	ARGX-113
D.3.9.3	Other descriptive name	ARGX-113
D.3.9.4	EV Substance Code	SUB180001
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Myasthenia Gravis

myasthénie grave (MG)

E.1.1.1

Medical condition in easily understood language

Myasthenia Gravis in patients who have generalized Muscle Weakness

Patients atteints de myasthénie grave présentant une faiblesse musculaire généralisée

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

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10028423
E.1.2	Term	Myasthenia gravis-like syndrome
E.1.2	System Organ Class	10029205 - Nervous system disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	HLT
E.1.2	Classification code	10071942
E.1.2	Term	Myasthenia gravis and related conditions
E.1.2	System Organ Class	100000004859

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

Efficacy of ARGX-113 as assessed by the percentage of “Myasthenia Gravis Activities of Daily Living (MG-ADL) responders” in the acetylcholine receptor (AChR)- antibody (Ab) seropositive population.

Évaluer l’efficacité d’ARGX-113 mesurée par le pourcentage de « Répondants au questionnaire d’activités de la vie quotidienne des personnes atteintes de myasthénie grave (MG-ADL) » après le premier cycle de traitement dans la population séropositive pour l’anticorps (Ac) du récepteur de l’acétylcholine (RACh).

E.2.2

Secondary objectives of the trial

1. Efficacy of ARGX-113 as assessed by the percentage of “QMG responders” in the AChR-Ab seropositive population.
2. Efficacy of ARGX-113 as assessed by the percentage of “MG-ADL responders” in the overall population (AChR-Ab seropositive and AChR-Ab seronegative patients).
3. Efficacy of ARGX-113 as assessed by the percentage of time that patients show a “clinically meaningful improvement” in total MG-ADL score during the trial in the AChR-Ab seropositive population.
4. Duration of response.
5. Onset of efficacy of ARGX-113 as assessed by the percentage of “early MG-ADL responders” in the AChR-Ab seropositive population.
6. Safety and tolerability of ARGX-113 in the overall population and in subgroups.

Évaluer l’efficacité d’ARGX-113 mesurée par :
1. Le pourcentage de « Répondants QMG » dans la population séropositive pour l’Ac-RACh.
2. Le pourcentage de « Répondants MG-ADL » dans la population générale (patients séropositifs pour l’Ac-RACh et patients séronégatifs pour l’Ac-RACh).
3. Le pourcentage de fois ou les patients présentent une « amélioration cliniquement significative » du score total MG-ADL pendant l’essai dans la population séropositive pour l’Ac-RACh.
4. La durée de réponse.
5. Évaluer l’apparition de l’efficacité de l’ARGX-113 mesurée par le pourcentage de « répondants MG-ADL précoces » dans la population séropositive pour l’Ac-RACh.
6. Évaluer la sécurité et la tolérance de l’ARGX-113 dans la population générale et les sous-groupes.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.
2. Male or female patients aged ≥ 18 years.
3. Diagnosis of MG with generalized muscle weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa and IVb.

Other more specific inclusion criteria are further defined in the protocol.

1. Patients capables de comprendre les exigences de l’étude, de fournir un consentement éclairé écrit et de suivre les procédures du protocole de l’essai.
2. Hommes ou femmes de ≥ 18 ans.
3. Diagnostic de MG avec faiblesse musculaire généralisée remplissant les critères cliniques du diagnostic de MG tels que définis par la Fondation américaine pour la myasthénie grave (Myasthenia Gravis Foundation of America [MGFA]) de classe II, II, IVa et IVb.

D'autres critères d'inclusion plus spécifiques sont définis dans le protocole.

E.4

Principal exclusion criteria

1.Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
2. Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 90 days after the last dosing or male patients who plan to donate
sperm during the trial or within 90 days after the last dosing.
3. MGFA Class I and V patients
4. Patients with worsening muscle weakness secondary to concurrent infections or medications.
5.Patients with known seropositivity or who test positive for an active viral infection at Screening with: o Hepatitis B Virus (HBV) (except patients who are seropositive because of HBV vaccination) o Hepatitis C Virus (HCV) o Human Immunodeficiency Virus (HIV)

Other more specific exclusion criteria are further defined in the protocol.

1. Femmes enceintes et allaitantes et femmes prévoyant de devenir enceintes pendant l’étude ou dans les 90 jours après la dernière administration.
2. Les patients masculins qui ont une activité sexuelle et qui n’ont pas l’intention d’utiliser les méthodes efficaces de contraception (indiquées ci-dessus) pendant l’étude ou pendant 90 jours après la dernière administration ou les patients masculins qui prévoient de donner du sperme pendant l’étude ou pendant 90 jours après la dernière administration.
3. Patients MGFA de classe I et V.
4. Patients présentant une aggravation de la faiblesse musculaire suite à des infections ou des traitements médicamenteux concomitants
5. Patients dont la séropositivité est connue ou ayant obtenu un résultat positif lors d’un test de détection d’une infection virale active à la Sélection par : o Virus de l’hépatite B (à l’exception des patients qui sont séropositifs en raison d’une vaccination contre le VHB) o Virus de l’hépatite C (VHC) o Virus de l’immunodéficience humaine (VIH)

D'autres critères d'exclusion plus spécifiques sont définis dans le protocole.

E.5 End points

E.5.1

Primary end point(s)

Efficacy of ARGX-113 as assessed by the percentage of “Myasthenia Gravis Activities of Daily Living (MG-ADL) responders” (acetylcholine receptor (AChR)- antibody (Ab) seropositive population)

Évaluer l’efficacité d’ARGX-113 mesurée par le pourcentage de « Répondants au questionnaire d’activités de la vie quotidienne des personnes atteintes de myasthénie grave (MG-ADL) » dans la population séropositive pour l’anticorps (Ac) du récepteur de l’acétylcholine (RACh).

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 8

E.5.2

Secondary end point(s)

E.5.2.1

Timepoint(s) of evaluation of this end point

Sec EP 1: Week 8
Sec EP 2: Week 8
Sec EP 3: Duration of the trial
Sec EP 4: Duration of the trial
Sec EP 5: Week 8

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

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

Tolerability

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

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

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

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Belgium

Canada

Czech Republic

Denmark

France

Germany

Hungary

Italy

Japan

Netherlands

Poland

Russian Federation

Sweden

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

The protocol allows for legal representative or impartial witness to sign ICF for patients who are unable to read and write

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

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)

At the EoS visit, patients will be offered the option to roll over into a long-term, single-arm, open-label follow-on trial (ARGX-113-1705)

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

2018-12-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-01-15

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2020-04-06

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