Clinical Trials Register

Summary
EudraCT Number:	2021-004998-32
Sponsor's Protocol Code Number:	ARGX-117-2003
National Competent Authority:	Belgium - FPS Health-DGM
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2022-08-09
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

Belgium - FPS Health-DGM

A.2

EudraCT number

2021-004998-32

A.3

Full title of the trial

A Long-Term Extension of the ARGX-117-2002 Trial to Evaluate the Long-term Safety and Tolerabillity, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults with Multifocal Motor Neuropathy

A.3.1

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

A clinical trial to investigate the Long-term Safety and Tolerability, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults With Multifocal Motor Neuropathy

A.3.2

Name or abbreviated title of the trial where available

ARDA+

A.4.1

Sponsor's protocol code number

ARGX-117-2003

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	+32(0)9310 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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	ARGX-117 IV
D.3.2	Product code	ARGX-117 IV
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.3.9.3	Other descriptive name	ARGX-117
D.3.9.4	EV Substance Code	SUB206651
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	120
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	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	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	ARGX-117 IV
D.3.2	Product code	ARGX-117 IV
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.3.9.3	Other descriptive name	ARGX-117
D.3.9.4	EV Substance Code	SUB206651
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	165
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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	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	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

Multifocal Motor Neuropathy

E.1.1.1

Medical condition in easily understood language

Multifocal Motor Neuropathy

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

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	10065579
E.1.2	Term	Multifocal motor neuropathy
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 evaluate the long-term safety and tolerability of ARGX-117 in adult participants with MMN

E.2.2

Secondary objectives of the trial

• To evaluate the long-term efficacy of ARGX-117 on muscle strength and/or motor function in adult participants with MMN
• To evaluate the long-term efficacy of ARGX-117 on functional ability, arm and hand function, quality of life (QOL), and fatigue in adult participants with MMN
• To evaluate the long-term effect of ARGX-117 on health-related productivity and work productivity
• To assess the PK, PD, and immunogenicity of ARGX-117

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. Capable of providing signed informed consent, and complying with protocol requirements. Participants must be able to read and write.
2. Must have completed the double-blinded treatment period of the ARGX-117-2002 trial and considered to be eligible for treatment with ARGX-117
3. Agrees to use contraceptive measures consistent with local regulations and the following:
a. Male participants: must use an acceptable contraceptive method that should be maintained at minimum until 15 months after last dose of Investigational Medicinal Product (IMP).
b. Female participants (women) of childbearing potential must have a negative urine pregnancy test at baseline before IMP can be administered.

E.4

Principal exclusion criteria

Participants will be excluded from the trial if any of the following criteria apply:
1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection
2. Clinical evidence of other significant serious diseases, have had a recent major surgery, or who have any other condition, in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk.
a. Germany only: A SARS-CoV-2 rapid antigen test must be completed or a negative rapid antigen test not older than 48 hours (depending on local requirements) must be presented upon site entry during DV1. Rapid antigen testing will only be mandatory if required locally.
3. Currently participating in another interventional clinical study
4. Pregnant or lactating or intend to become pregnant during the trial or within 15 months after last dose of the IMP.

E.5 End points

E.5.1

Primary end point(s)

• Safety outcomes based on AE monitoring and other safety assessments

E.5.1.1

Timepoint(s) of evaluation of this end point

continuously

E.5.2

Secondary end point(s)

• Modified Medical Research Council (mMRC)
− Value and change from baseline in the mMRC-10 sum score
− Proportion of participants showing a deterioration of at least 2 points in mMRC-10 sum score
− Value and change from baseline in the mMRC-14 sum score
− Proportion of participants showing a deterioration of at least 2 points in the mMRC-14 sum score
− Proportion of participants showing a deterioration of 1 or more points in at least 2 muscle groups as assessed by the mMRC-14 sum score
− Proportion of participants with no deterioration in 2 or more muscle groups as assessed by mMRC-14 sum score
− Value and change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score
• Grip strength (GS)
− Values, change, and percent change from baseline in GS
− Proportion of participants with a decline of >30% in GS
− Proportion of participants with a GS decrease of 8 kilopascals (kPa) or more
• Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN‐RODS)
• Values and change from baseline in the average time for the upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed pegboard test)
• Proportion of participants by level of severity on each dimension of EQ-5D-5L
• Value and change from baseline in EQ-5D-5L visual analog scale (VAS)
• Values and change from baseline in the Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI)
• Values and change from baseline in the 9-item Fatigue Severity Scale (FSS)
• Values of the Patient Global Impression of Change (PGIC) scale
• Proportion of participants by level of severity of MMN as assessed by the Patient Global Impression of Severity (PGIS)
• Values for work-related and household chore activities of the Health-Related Productivity Questionnaire (HRPQ)
• Serum concentrations and PK parameters for ARGX-117
• Values and change from baseline in free C2, total C2, and functional complement activity (CH50) over time
• Incidence and prevalence of antidrug antibodies (ADA) against ARGX-117

E.5.2.1

Timepoint(s) of evaluation of this end point

Please refer to section 1.3 of protocol

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

tolerability, immunogenicity

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)

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

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Double - blinded rollover treatment (DTP) followed by Open - label treatment period (OTP)

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

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

Yes

E.8.4

The trial involves multiple sites in the 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

Canada

United States

Austria

France

Poland

Netherlands

Spain

Germany

Italy

Belgium

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 trial is defined as the date of the last participant’s last visit.

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)

The trial will continue until marketing authorisation of ARGX-117 or until the trial is discontinued by the sponsor.

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

2022-09-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-08-30

P. End of Trial
P.	End of Trial Status	Trial now transitioned

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials