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

NCT05225675 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: ARGX-117-2002
• Study Type: interventional
• Target: 54
• Locations: 11 countries
• Sites: 40

Brief Summary

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).

Conditions

Multifocal Motor Neuropathy

Outcome Measures

Primary Outcomes (1)

• Safety outcomes based on adverse event (AE) monitoring and other safety assessments

  16 weeks

Secondary Outcomes (24)

• Time to the first retreatment with IVIg since the final IVIg treatment of the IVIg monitoring period

  16 weeks

• AUC (area under curve) of the change from baseline in mMRC (modified Medical Research Council)-10 sum score

  16 weeks

• Change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC (modified Medical Research Council)-14 sum score

  16 weeks

• Value baseline in the mMRC (modified Medical Research Council)-14 sum score

  16 weeks

• Change from baseline in the mMRC (modified Medical Research Council)-14 sum score

  16 weeks

Study Arms (2)

ARGX-117

EXPERIMENTAL

Intravenous administration of ARGX-117

Biological: ARGX-117

Placebo

PLACEBO COMPARATOR

Intravenous administration of placebo

Other: Placebo

Interventions

ARGX-117 BIOLOGICAL

Intravenous administration of ARGX-117

ARGX-117

Placebo OTHER

Intravenous administration of placebo

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable of giving signed informed consent form (ICF)
• Male/female at least 18 years of age at the time the informed consent form (ICF) is signed
• 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)
• Receiving a stable IVIg regimen for at least 3 months before screening or recently initiated IVIg treatment
• IVIg treatment dependency confirmation by the MMN Confirmation Committee (MCC)
• 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
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

You may not qualify if:

• Any coexisting condition which may interfere with the outcome assessments
• Clinical signs or symptoms suggestive for neuropathies other than MMN such as motor neuron disease or other inflammatory neuropathies
• 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.
• Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection during the screening and/or IVIg monitoring period (IVMP).
• 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).
• 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:
• Adequately treated basal cell or squamous cell skin cancer
• Carcinoma in situ of the cervix
• Carcinoma in situ of the breast
• Incidental histological finding of prostate cancer
• 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
• Prior/concomitant therapy
• Cyclophosphamide and/or rituximab and/or eculizumab and/or mycophenolate mofetil within 3 months prior to screening
• Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
• Positive serum test at screening for an active viral infection with any of the following conditions:

Sponsors & Collaborators

• argenx: lead

Study Sites (40)

HonorHealth Research Institute-Neuroscience Research

Scottsdate, Arizona, 85251, United States

Location

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

San Francisco, California, 94109, United States

Location

George Washington Medical Faculty Associates

Washington D.C., District of Columbia, 20037, United States

Location

HonorHealth Research Institute-Neuroscience Research

Maitland, Florida, 32751, United States

Location

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

Tampa, Florida, 33612, United States

Location

NorthShore University HealthSystem

Glenview, Illinois, 60026, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Minnesota Delware Clinic Research Unit

Minneapolis, Minnesota, 55414, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Perelman Center for Advanced Medicine-University of Penssylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Austin Neuromuscular Center

Austin, Texas, 78756, United States

Location

West Virginia University Medicine

Morgantown, West Virginia, 26506, United States

Location

Medizinische Universitat Wien Universitatsklienik fur Neurologie

Vienna, 1090, Austria

Location

AZ Sint-Lucas

Ghent, 9000, Belgium

Location

Genge Partners Montreal

Québec, H4A 3TA, Canada

Location

Toronto General Hospital

Toronto, M5G 2C4, Canada

Location

CHU de Bordeaux-Hopital Pellegrin

Bordeaux, 33076, France

Location

CHRU de Lille-Hopital Roger Salengro

Lille, 59037, France

Location

CHU de Nice-Hopital Pasteur 2

Nice, 06001, France

Location

Hopital Pitie Salpetriere

Paris, 75651, France

Location

Katholisches Klinikum Bochum

Bochum, 44791, Germany

Location

Universitatsklinikum Essen

Essen, 45147, Germany

Location

Universitatsmedzin Gottingen, Klinik fur Neurologie

Göttingen, 37075, Germany

Location

Medizinische Hochschule Hannover Klinik Fur Neurologie

Hanover, 30625, Germany

Location

Universitatsklinikum Munster

Münster, 48419, Germany

Location

IRCCS Ospedale San Raffaele

Milan, 20132, Italy

Location

Azienda Ospedaliero Univeritaria Pisana-UOS Neurologia

Pisa, 56126, Italy

Location

Azienda Ospedaliera Sant'Andrea-UOS Malattie Neuromuscolari

Rome, 00189, Italy

Location

Instituto Clinico Humanitas (IRCCS)

Rozzano, 20089, Italy

Location

Amsterdam UMC location AMC, Dep of Neurology

Amsterdam, 1105 AZ, Netherlands

Location

University Medical Centre Utrecht

Utrecht, 3584 CX, Netherlands

Location

Michalscy I Partnerzy Lekarze Spolka Partnerska

Krakow, 31-426, Poland

Location

Uniwersyteckie centrum kliniczne Warszawskiego

Warsaw, 02-097, Poland

Location

Hospital Universitario Vall d'Herbon

Barcelona, 08035, Spain

Location

Hospital de la Santa Creu I Santa Pau -Sevicio Neurologia

Barcelona, 08041, Spain

Location

Hospital Universitari I Politecnic La Fe de Valencia-Servicio Neurologia

Valencia, 46026, Spain

Location

Queen Elisabeth University Hospital

Glasgow, G51 4TF, United Kingdom

Location

University College London Hospital

London, ZC1N 3BG, United Kingdom

Location

Oxford University Hospitals NHS Trust-Jonh Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom

Location

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2021
• First Posted: February 4, 2022
• Study Start: March 31, 2022
• Primary Completion: June 4, 2024
• Study Completion: June 4, 2024
• Last Updated: October 18, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); BE (1); PL (2); CA (2); FR (4); AU (1); IT (4); DE (5); NL (2); US (13); GB (3)