Clinical trial NCT05405361: 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
Official title: A Long-term Extension of the ARGX-117-2002 Trial to Evaluate the Long-term Safety and Tolerability, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults With Multifocal Motor Neuropathy
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Multifocal Motor Neuropathy (MMN)
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2022-06-06
Enrollment: 51
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Safety outcomes based on adverse event (AE) monitoring.
timeFrame: Until marketing authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
Secondary outcomes:
measure: Change from baseline in the mMRC-10 sum score
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Proportion of participants showing a deterioration of at least 2 points in mMRC-10 sum score
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in the mMRC-14 sum score
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Proportion of participants showing a deterioration of at least 2 points in the mMRC-14 sum score
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC-14 sum score
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in GS
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Proportion of participants with a decline of >30% in GS
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in the MMN-RODS
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in the average time for the upper extremity (arm and hand) function (9-HPT, or timed pegboard test)
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Proportion of participants by level of severity on each dimension of the EQ-5D-5L
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in EQ-5D-5L visual analog scale (VAS)
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in the CAP-PRI
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in the 9-item FSS
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Values of the PGIC scale
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Proportion of participants by level of severity of MMN as assessed by PGIS
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Values for work-related and household chore activities of the HRPQ
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Serum concentrations for ARGX-117
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Change from baseline in free C2, total C2, functional complement activity (CH50).
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
measure: Incidence of antidrug antibodies (ADA) against ARGX-117
timeFrame: Until market authorization of ARGX-117, assessed up to 70 months or treatment discontinuation, whichever comes first
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: HonorHealth Neurology
state: Arizona
city: Glenview
country: United States
facility: NorthShore University HealthSystem
state: Illinois
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania - Perelman Center for Advanced Medicine
state: Pennsylvania
city: Austin
country: United States
facility: Austin Neuromuscular Center
state: Texas
city: Vienna
country: Austria
facility: Medizinische Universitat Wien (Medical University of Vienna)
city: Gent
country: Belgium
facility: AZ Sint-Lucas
city: Toronto
country: Canada
facility: University Health Network
city: Bordeaux
country: France
facility: CHU Bordeaux - Groupe Hospitalier Pellegrin
city: Lille
country: France
facility: Hôpital Roger Salengro (CHU de Lille)
city: Nice
country: France
facility: CHU de Nice
city: Paris
country: France
facility: Hôpital de la Pitié Salpétrière
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Göttingen
country: Germany
facility: Universitatsmedizin Gottingen (UMG) Georg-August-Universitaet
city: Milano
country: Italy
facility: IRCCS Ospedale San Raffaele S.r.l.
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana
city: Rome
country: Italy
facility: Azienda Ospedaliera Sant' Andrea
city: Rozzano
country: Italy
facility: IRCCS Humanitas Research Hospital
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
city: Kraków
country: Poland
facility: Michalski i Partnerzy Lekarze Spolka Partnerska
city: Warsaw
country: Poland
facility: Warszawski Uniwersytet Medyczny
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital de La Santa Creu i Sant Pau
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Trust
Study officials:
References: