ARGX-117 for Multifocal Motor Neuropathy

(ARDA Trial)

No longer recruiting at 83 trial locations

Overseen By Sabine s Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: argenx

Must be taking: IVIg

Must not be taking: Cyclophosphamide, Rituximab, Eculizumab, others

Disqualifiers: Severe psychiatric disorder, Autoimmune disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called ARGX-117 for individuals with multifocal motor neuropathy (MMN), a condition affecting nerve function and muscle strength. The study aims to determine if ARGX-117 is safe and effective compared to a placebo for those who rely on regular intravenous immunoglobulin (IVIg) treatments. Participants must have been on a stable IVIg regimen for at least three months. Those diagnosed with MMN and dependent on IVIg may be suitable candidates for this trial. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important findings.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must have been on a stable IVIg regimen for at least 3 months before screening. Some medications like cyclophosphamide, rituximab, eculizumab, and mycophenolate mofetil should not have been used within 3 months prior to screening.

Is there any evidence suggesting that ARGX-117 is likely to be safe for humans?

Research has shown that ARGX-117 has been tested for safety in people with multifocal motor neuropathy (MMN). Earlier studies found that most participants tolerated the treatment well. Although there were small differences in age and strength among participants, these did not appear to affect safety.

No major safety issues were reported, but like any treatment, some side effects may occur. This ongoing research aims to confirm that ARGX-117 is safe and effective. As a phase 2 study, it indicates that the treatment has already passed initial safety tests in earlier studies. However, more information is needed to ensure its safety for a broader population. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for MMN?

Unlike the standard treatments for multifocal motor neuropathy, which often include intravenous immunoglobulin (IVIG) and immunosuppressants, ARGX-117 stands out because it targets a specific complement protein involved in the immune system. This investigational drug is designed to be administered intravenously and works by inhibiting the classical complement pathway, potentially reducing inflammation and nerve damage more directly than current options. Researchers are particularly excited about ARGX-117 because it could offer a more targeted approach with fewer side effects, providing patients with a more effective and precise treatment option.

What evidence suggests that ARGX-117 might be an effective treatment for MMN?

In this trial, participants will receive either ARGX-117, also known as empasiprubart, or a placebo. Research has shown that ARGX-117 may help treat multifocal motor neuropathy (MMN). Studies have found that this treatment can reduce the need for repeat IVIg (intravenous immunoglobulin) treatments. Patients in these studies experienced better grip strength, which is important for everyday tasks. ARGX-117 blocks certain harmful actions of the immune system on nerve cells. Early results suggest it could improve patients' perceptions of their condition, offering hope for those with MMN. ^{2 3 4 5 6}

Are You a Good Fit for This Trial?

Adults with Multifocal Motor Neuropathy (MMN) who are stable on IVIg treatment can join this trial. They must be at least 18, have been vaccinated against certain infections, and use approved contraception methods. People with severe psychiatric issues, other serious diseases, recent major surgery, drug abuse history or certain medication use in the last 3 months cannot participate.

Inclusion Criteria

My need for IVIg treatment has been confirmed by the MMN Committee.

I have been on a stable IVIg treatment for at least 3 months.

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

Known hypersensitivity reaction to 1 of the components of the IMP or any of its excipients

Any coexisting condition which may interfere with the outcome assessments

I do not have any serious ongoing infections.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravenous administration of ARGX-117 or placebo

16 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ARGX-117
• Placebo

Trial Overview The trial is testing two different doses of a new drug called ARGX-117 compared to a placebo in people with MMN. It's designed to see if ARGX-117 is safe and works well when added to their regular IVIg therapy. Participants will be randomly assigned to one of the groups without knowing which one they're in.

How Is the Trial Designed?

Group I: ARGX-117 Experimental Treatment 1 Intervention

Intravenous administration of ARGX-117

Group II: Placebo Placebo Group 1 Intervention

Intravenous administration of placebo

Who Is Running the Clinical Trial?

argenx

Lead Sponsor

Trials

76

Recruited

11,500+

Tim Van Hauwermeiren

argenx

Chief Executive Officer since 2008

B.Sc. and M.Sc. in Bioengineering from Ghent University, Executive MBA from The Vlerick School of Management

Dr. Peter Ulrichts

argenx

Chief Medical Officer since 2023

MD from Maastricht University, PhD in Molecular Immunology from Maastricht University

Citations

1. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/aanem-2024/Empasiprubart%20(ARGX-117)%20in%20Multifocal%20Motor%20Neuropathy%20Initial%20Safety%20and%20Efficacy%20Data%20of%20the%20Phase%202%20ARDA%20Study.pdf

Empasiprubart (ARGX-117) in Multifocal Motor Neuropathy Slight imbalances were observed in median age, grip strength, MMN-RODS score, and CAP-PRI score between treatment arms, with lower disease-specific QoL and ...

2. neurologylive.com neurologylive.com/view/phase-2-arda-findings-support-empasiprubart-multifocal-motor-neuropathy

Phase 2 ARDA Findings Support Development ... Empasiprubart showed safety and reduced IVIg retreatment need in MMN patients, with improved grip strength and condition perception. The agent ...

3. argenx.com argenx.com/news/2024/argenx-highlights-breadth-autoimmune-pipeline-new-multifocal-motor-neuropathy-data-2024

argenx Highlights Breadth of Autoimmune Pipeline with ... argenx Highlights Breadth of Autoimmune Pipeline with New Multifocal Motor Neuropathy Data at 2024 Peripheral Nerve Society Annual Meeting.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05225675

NCT05225675 | A Clinical Trial to Investigate the Safety ... A Clinical Trial to Investigate the Safety and Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics and Immunogenicity of 2 Dose Regimens of ARGX-117 in ...

5. neurology.org neurology.org/doi/10.1212/01.wnl.0000903320.23411.f9

Safety, Efficacy, And Pharmacokinetics of Argx-117 in ... Using an in vitro model for MMN, ARGX-117 was shown to block IgM-mediated classical pathway complement activation on both motor neurons and ...

6. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/nmsg2024/Empasiprubart%20(ARGX-117)%20in%20Multifocal%20Motor%20Neuropathy_Initial%20Safety%20and%20Efficacy%20Data%20of%20the%20Phase%202%20ARDA%20Study.pdf

Initial Safety and Efficacy Data of the Phase 2 ARDA Study Slight imbalances were observed in median age, grip strength, MMN-RODS score, and CAP-PRI score between treatment arms, with lower disease-specific QoL and ...