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
- Conditions
- Multifocal Motor Neuropathy (MMN)
- Interventions
- Other: Placebo
- Registration Number
- NCT05405361
- Lead Sponsor
- argenx
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 51
- Capable of providing signed informed consent and complying with protocol requirements. Participants must be able to read and write.
- Must have completed the double-blinded treatment period of the ARGX-117-2002 trial and considered to be eligible for treatment with ARGX-117.
- Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection.
- 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.
- Currently participating in another interventional clinical study.
- Pregnant or lactating or intend to become pregnant during the trial or within 15 months after last dose of the study drug.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Participants receiving placebo during the double-blinded treatment period (DTP) and receiving ARGX-117 during the open-label treatment period (OLTP) ARGX-117 ARGX-117 Participants receiving ARGX-117 during the double-blinded treatment period (DTP) and the open-label treatment period (OLTP) Placebo ARGX-117 Participants receiving placebo during the double-blinded treatment period (DTP) and receiving ARGX-117 during the open-label treatment period (OLTP)
- Primary Outcome Measures
Name Time Method 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
- Secondary Outcome Measures
Name Time Method Values of the PGIC scale Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Proportion of participants by level of severity of MMN as assessed by PGIS Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Change from baseline in the CAP-PRI Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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)
Change from baseline in the 9-item FSS Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Change from baseline in the mMRC-10 sum score Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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
Proportion of participants showing a deterioration of at least 2 points in mMRC-10 sum score Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Change from baseline in the mMRC-14 sum score Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Proportion of participants showing a deterioration of at least 2 points in the mMRC-14 sum score Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Change from baseline in GS Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Proportion of participants with a decline of >30% in GS Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Change from baseline in the MMN-RODS Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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).
Change from baseline in the average time for the upper extremity (arm and hand) function (9-HPT, or timed pegboard test) Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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).
Proportion of participants by level of severity on each dimension of the EQ-5D-5L Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
Change from baseline in EQ-5D-5L visual analog scale (VAS) Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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).
Values for work-related and household chore activities of the HRPQ Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first 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.
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
Trial Locations
- Locations (27)
HonorHealth Neurology
🇺🇸Scottsdale, Arizona, United States
Austin Neuromuscular Center
🇺🇸Austin, Texas, United States
Medizinische Universitat Wien (Medical University of Vienna)
🇦🇹Vienna, Austria
University Health Network
🇨🇦Toronto, Canada
Hôpital Roger Salengro (CHU de Lille)
🇫🇷Lille, France
CHU Bordeaux - Groupe Hospitalier Pellegrin
🇫🇷Bordeaux, France
Hôpital de la Pitié Salpétrière
🇫🇷Paris, France
CHU de Nice
🇫🇷Nice, France
Universitätsklinikum Essen
🇩🇪Essen, Germany
Universitatsmedizin Gottingen (UMG) Georg-August-Universitaet
🇩🇪Göttingen, Germany
IRCCS Ospedale San Raffaele S.r.l.
🇮🇹Milano, Italy
Azienda Ospedaliera Sant' Andrea
🇮🇹Rome, Italy
Michalski i Partnerzy Lekarze Spolka Partnerska
🇵🇱Kraków, Poland
Warszawski Uniwersytet Medyczny
🇵🇱Warsaw, Poland
Hospital Universitario Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital de La Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital Universitari i Politecnic La Fe de Valencia
🇪🇸Valencia, Spain
Queen Elizabeth University Hospital
🇬🇧Glasgow, United Kingdom
Oxford University Hospitals NHS Trust
🇬🇧Oxford, United Kingdom
NorthShore University HealthSystem
🇺🇸Glenview, Illinois, United States
The Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
University of Pennsylvania - Perelman Center for Advanced Medicine
🇺🇸Philadelphia, Pennsylvania, United States
Universitair Medisch Centrum Utrecht
🇳🇱Utrecht, Netherlands
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
IRCCS Humanitas Research Hospital
🇮🇹Rozzano, Italy
AZ Sint-Lucas
🇧🇪Gent, Belgium
Azienda Ospedaliero Universitaria Pisana
🇮🇹Pisa, Italy