A Clinical Trial to Investigate the Safety and Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics and Immunogenicity of 2 Dose Regimens of ARGX-117 in Adults with Multifocal Motor Neuropathy

NCT ID: NCT05225675

Last Updated: 2024-10-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-31

Study Completion Date

2024-06-04

Brief Summary

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This is a phase 2, randomized, double-blinded, placebo-controlled, parallel-group, multicenter trial to evaluate the safety and efficacy of 2 dose regimens of ARGX-117 versus placebo, in participants with MMN previously stabilized with IVIg (intravenous immunoglobulin).

Detailed Description

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Conditions

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Multifocal Motor Neuropathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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ARGX-117

Intravenous administration of ARGX-117

Group Type EXPERIMENTAL

ARGX-117

Intervention Type BIOLOGICAL

Intravenous administration of ARGX-117

Placebo

Intravenous administration of placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Intravenous administration of placebo

Interventions

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ARGX-117

Intravenous administration of ARGX-117

Intervention Type BIOLOGICAL

Placebo

Intravenous administration of placebo

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Capable of giving signed informed consent form (ICF)
2. Male/female at least 18 years of age at the time the informed consent form (ICF) is signed
3. Probable or definite MMN according to the European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) (EFNS/PNS) 2010 guidelines at screening confirmed by the MMN Confirmation Committee (MCC)
4. Receiving a stable IVIg regimen for at least 3 months before screening or recently initiated IVIg treatment
5. IVIg treatment dependency confirmation by the MMN Confirmation Committee (MCC)
6. Immunization with the first meningococcal vaccine and pneumococcal vaccine, and the single Haemophilus influenza type B vaccine must be performed at least 14 days before IMP administration at V1 according to local country-specific immunization schedules. A documented history of vaccination against Neisseria meningitides, Haemophilus influenza type B, and streptococcus pneumonia will be permitted
7. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

Exclusion Criteria

1. Any coexisting condition which may interfere with the outcome assessments
2. Clinical signs or symptoms suggestive for neuropathies other than MMN such as motor neuron disease or other inflammatory neuropathies
3. Severe psychiatric disorder, history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the participant or could affect adherence with the trial protocol.
4. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection during the screening and/or IVIg monitoring period (IVMP).
5. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of MMN or put the participant at undue risk (eg, SLE).
6. History of malignancy unless resolved by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following carcinomas will be eligible:

1. Adequately treated basal cell or squamous cell skin cancer
2. Carcinoma in situ of the cervix
3. Carcinoma in situ of the breast
4. Incidental histological finding of prostate cancer
7. Clinical evidence of other significant serious diseases, have had a recent major surgery (including a splenectomy at any time), 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
8. Prior/concomitant therapy

1. Cyclophosphamide and/or rituximab and/or eculizumab and/or mycophenolate mofetil within 3 months prior to screening
2. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
9. Positive serum test at screening for an active viral infection with any of the following conditions:

1. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection
2. Hepatitis C virus (HCV) based on HCV antibody assay
3. HIV based on test results that are associated with an AIDS-defining condition
10. Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse
11. Known hypersensitivity reaction to 1 of the components of the IMP or any of its excipients
12. Female participants with a positive serum or urine pregnancy test, lactating females, and those who intend to become pregnant during the trial or within 15 months after last dose of the IMP
13. ALT or AST ≥2 × upper limit of normal and total bilirubin ≥1.5 × upper limit of normal of the central laboratory reference range
14. An estimated glomerular filtration rate of ≤60 mL/min/1.73m2
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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argenx

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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HonorHealth Research Institute-Neuroscience Research

Scottsdate, Arizona, United States

Site Status

California Pacific Medical Center-Forbes Norris MDA/ALS Research Center

San Francisco, California, United States

Site Status

George Washington Medical Faculty Associates

Washington D.C., District of Columbia, United States

Site Status

HonorHealth Research Institute-Neuroscience Research

Maitland, Florida, United States

Site Status

University of South Florida Carol and Frank Morsani Center for Advanced Healthcare

Tampa, Florida, United States

Site Status

NorthShore University HealthSystem

Glenview, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

University of Minnesota Delware Clinic Research Unit

Minneapolis, Minnesota, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Perelman Center for Advanced Medicine-University of Penssylvania

Philadelphia, Pennsylvania, United States

Site Status

Austin Neuromuscular Center

Austin, Texas, United States

Site Status

West Virginia University Medicine

Morgantown, West Virginia, United States

Site Status

Medizinische Universitat Wien Universitatsklienik fur Neurologie

Vienna, , Austria

Site Status

AZ Sint-Lucas

Ghent, , Belgium

Site Status

Genge Partners Montreal

Québec, , Canada

Site Status

Toronto General Hospital

Toronto, , Canada

Site Status

CHU de Bordeaux-Hopital Pellegrin

Bordeaux, , France

Site Status

CHRU de Lille-Hopital Roger Salengro

Lille, , France

Site Status

CHU de Nice-Hopital Pasteur 2

Nice, , France

Site Status

Hopital Pitie Salpetriere

Paris, , France

Site Status

Katholisches Klinikum Bochum

Bochum, , Germany

Site Status

Universitatsklinikum Essen

Essen, , Germany

Site Status

Universitatsmedzin Gottingen, Klinik fur Neurologie

Göttingen, , Germany

Site Status

Medizinische Hochschule Hannover Klinik Fur Neurologie

Hanover, , Germany

Site Status

Universitatsklinikum Munster

Münster, , Germany

Site Status

IRCCS Ospedale San Raffaele

Milan, , Italy

Site Status

Azienda Ospedaliero Univeritaria Pisana-UOS Neurologia

Pisa, , Italy

Site Status

Azienda Ospedaliera Sant'Andrea-UOS Malattie Neuromuscolari

Rome, , Italy

Site Status

Instituto Clinico Humanitas (IRCCS)

Rozzano, , Italy

Site Status

Amsterdam UMC location AMC, Dep of Neurology

Amsterdam, , Netherlands

Site Status

University Medical Centre Utrecht

Utrecht, , Netherlands

Site Status

Michalscy I Partnerzy Lekarze Spolka Partnerska

Krakow, , Poland

Site Status

Uniwersyteckie centrum kliniczne Warszawskiego

Warsaw, , Poland

Site Status

Hospital Universitario Vall d'Herbon

Barcelona, , Spain

Site Status

Hospital de la Santa Creu I Santa Pau -Sevicio Neurologia

Barcelona, , Spain

Site Status

Hospital Universitari I Politecnic La Fe de Valencia-Servicio Neurologia

Valencia, , Spain

Site Status

Queen Elisabeth University Hospital

Glasgow, , United Kingdom

Site Status

University College London Hospital

London, , United Kingdom

Site Status

Oxford University Hospitals NHS Trust-Jonh Radcliffe Hospital

Oxford, , United Kingdom

Site Status

Countries

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United States Austria Belgium Canada France Germany Italy Netherlands Poland Spain United Kingdom

Other Identifiers

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ARGX-117-2002

Identifier Type: -

Identifier Source: org_study_id

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