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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-507052-69-00 | EU Trial (CTIS) Number |
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This trial is an extension of the antecedent trial ARGX-117-2002. It is a multicenter trial that has been designed to evaluate the long-term safety and tolerability, efficacy, immunogenicity, Pharmacokinetics (PK), and Pharmacodynamics (PD) of ARGX-117 Intravenously (IV) in adults with Multifocal Motor Neuropathy (MMN). The trial will include a double-blinded rollover treatment period (DTP), an open-label treatment period (OTP), and a safety follow-up period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARGX-117 | Experimental | Participants receiving ARGX-117 during the double-blinded treatment period (DTP) and the open-label treatment period (OLTP) |
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| Placebo | Experimental | Participants receiving placebo during the double-blinded treatment period (DTP) and receiving ARGX-117 during the open-label treatment period (OLTP) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ARGX-117 | Biological | Intravenous administration of ARGX-117 |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Safety outcomes based on adverse event (AE) monitoring. | Until marketing authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in the mMRC-10 sum score | The modified Medical Research Council (mMRC) sum score evaluates motor strength/weakness from predetermined muscle groups (upper and lower limbs). The higher the score, the better the muscle strength | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HonorHealth Neurology | Scottsdale | Arizona | 85251 | United States | ||
| NorthShore University HealthSystem |
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During the double-blinded rollover treatment period (DTP),the investigator, trial nurse/coordinator, participant, and the sponsor's designated contract research organization (CRO), and sponsor trial team (except the sponsor's clinical trial supplies team) are blinded to IMP. The interactive response technology (IRT) system is used for blinding.
| Placebo |
| Other |
Intravenous administration of placebo |
|
| Proportion of participants showing a deterioration of at least 2 points in mMRC-10 sum score | The modified Medical Research Council (mMRC) sum score evaluates motor strength/weakness from predetermined muscle groups (upper and lower limbs). The higher the score, the better the muscle strength. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in the mMRC-14 sum score | The modified Medical Research Council (mMRC) sum score evaluates motor strength/weakness from predetermined muscle groups (upper and lower limbs). The higher the score, the better the muscle strength. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Proportion of participants showing a deterioration of at least 2 points in the mMRC-14 sum score | The modified Medical Research Council (mMRC) sum score evaluates motor strength/weakness from predetermined muscle groups (upper and lower limbs). The higher the score, the better the muscle strength. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score | The modified Medical Research Council (mMRC) sum score evaluates motor strength/weakness from predetermined muscle groups (upper and lower limbs). The higher the score, the better the muscle strength. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in GS | The grip strength (GS) will be measured by using a Martin-Vigorimeter. Each GS measurement will consist of 3 repeated contractions with the participant's maximal effort. The duration of each contraction will be 3 seconds. 3 repetitions will be executed consecutively by the right hand followed by 3 repetitions of the left hand. There will be a 30-second rest period between each of the 3 repetitions and a 2-minute rest period between each hand. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Proportion of participants with a decline of >30% in GS | The grip strength (GS) will be measured by using a Martin-Vigorimeter. Each GS measurement will consist of 3 repeated contractions with the participant's maximal effort. The duration of each contraction will be 3 seconds. 3 repetitions will be executed consecutively by the right hand followed by 3 repetitions of the left hand. There will be a 30-second rest period between each of the 3 repetitions and a 2-minute rest period between each hand. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in the MMN-RODS | The Rasch-Built Overall Disability Scale for MMN (MMN-RODS) is a disease specific patient-reported outcome instrument constructed specifically to capture activity limitations in patients with MMN. The total score varies between 0 (worst outcome) and 50 (best outcome). | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in the average time for the upper extremity (arm and hand) function (9-HPT, or timed pegboard test) | The 9-Hole Peg Test (9-HPT) is a quantitative measure of upper extremity (arm and hand) function. Both the dominant and nondominant hands will be tested twice (2 consecutive trials of the dominant hand, followed immediately by 2 consecutive trials of the nondominant hand). | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Proportion of participants by level of severity on each dimension of the EQ-5D-5L | The Euro-Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Scores for each dimension include 5 levels: no problem, slight problem, moderate problem, severe problem, and extreme problem. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in EQ-5D-5L visual analog scale (VAS) | A vertical visual analog scale (VAS) is included in the EQ-5D-5L. Participants mark their health status from 0 (the worst health you can imagine) to 100 (the best health you can imagine). | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in the CAP-PRI | The Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI) includes the assessment of 15 items. The total score varies between 0 (best outcome) and 30 (worst outcome) | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in the 9-item FSS | In the 9-item Fatigue Severity Scale (FSS) fatigue levels are rated from 1 to 7. A low value indicates that the statement is not very appropriate whereas a high value indicates agreement. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Values of the PGIC scale | The Patient Global Impression of Change (PGIC) is a 7-point scale depicting a participant's rating of overall improvement. The lower the score, the better the improvement. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Proportion of participants by level of severity of MMN as assessed by PGIS | The Patient Global Impression of Severity (PGIS) is a 7-point scale depicting a participant's rating of overall illness severity. Higher scores mean a higher severity. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Values for work-related and household chore activities of the HRPQ | The Health-Related Productivity Questionnaire (HRPQ) provides data related to missed hours at work or educational activities and reduced effectiveness during any attempted work. These criteria form an important portion of work-related productivity and will be used to assess health-related and work-related productivity in the trial. | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Serum concentrations for ARGX-117 | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Change from baseline in free C2, total C2, functional complement activity (CH50). | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Incidence of antidrug antibodies (ADA) against ARGX-117 | Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first |
| Glenview |
| Illinois |
| 60026 |
| United States |
| University of Minnesota | Minneapolis | Minnesota | 55414 | United States |
| The Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| University of Pennsylvania - Perelman Center for Advanced Medicine | Philadelphia | Pennsylvania | 19104 | United States |
| Austin Neuromuscular Center | Austin | Texas | 78759 | United States |
| Medizinische Universitat Wien (Medical University of Vienna) | Vienna | 1090 | Austria |
| AZ Sint-Lucas | Ghent | 9000 | Belgium |
| University Health Network | Toronto | Canada |
| CHU Bordeaux - Groupe Hospitalier Pellegrin | Bordeaux | 33076 | France |
| Hôpital Roger Salengro (CHU de Lille) | Lille | 59037 | France |
| CHU de Nice | Nice | 06001 | France |
| Hôpital de la Pitié Salpétrière | Paris | 75651 | France |
| Universitätsklinikum Essen | Essen | 45147 | Germany |
| Universitatsmedizin Gottingen (UMG) Georg-August-Universitaet | Göttingen | 37075 | Germany |
| IRCCS Ospedale San Raffaele S.r.l. | Milan | 20132 | Italy |
| Azienda Ospedaliero Universitaria Pisana | Pisa | 56126 | Italy |
| Azienda Ospedaliera Sant' Andrea | Rome | 00189 | Italy |
| IRCCS Humanitas Research Hospital | Rozzano | 20089 | Italy |
| Universitair Medisch Centrum Utrecht | Utrecht | 3584 CX | Netherlands |
| Michalski i Partnerzy Lekarze Spolka Partnerska | Krakow | 31-426 | Poland |
| Warszawski Uniwersytet Medyczny | Warsaw | 02-097 | Poland |
| Hospital Universitario Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital de La Santa Creu i Sant Pau | Barcelona | 08041 | Spain |
| Hospital Universitari i Politecnic La Fe de Valencia | Valencia | 46026 | Spain |
| Queen Elizabeth University Hospital | Glasgow | G51 4TF | United Kingdom |
| Oxford University Hospitals NHS Trust | Oxford | OX3 9DU | United Kingdom |