ARGX-117 for Multifocal Motor Neuropathy

(ARDA+ Trial)

Not currently recruiting at 52 trial locations

Overseen By Sabine s Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: argenx

Disqualifiers: Infections, Serious diseases, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: 9

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term safety and effectiveness of ARGX-117 (also known as Empasiprubart) for individuals with Multifocal Motor Neuropathy (MMN), a condition affecting muscle strength. Participants will receive either ARGX-117 or a placebo (a look-alike substance with no active drug) during the initial phase, followed by ARGX-117 in the later phase. The trial targets those who previously participated in an ARGX-117 trial and qualify for continued treatment. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to significant findings.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

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

Research has shown that ARGX-117 has been tested in adults with Multifocal Motor Neuropathy (MMN) to assess its safety. In these studies, participants generally tolerated the treatment well. Although there were small differences in age and grip strength between groups, these did not appear to affect overall safety. No major safety issues emerged, suggesting ARGX-117 as a promising option for potential clinical trial participants. As a Phase 2 trial, the treatment has already demonstrated some safety in earlier trials, but additional data will help confirm this. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Multifocal Motor Neuropathy?

Most treatments for multifocal motor neuropathy, like intravenous immunoglobulin (IVIG), work by modulating the immune system. But ARGX-117 works differently, targeting a protein in the immune system called C2, which is part of the complement system. This new mechanism of action aims to reduce the immune attack on nerves more directly. Researchers are excited about ARGX-117 because it could offer a more efficient and targeted approach, potentially leading to better outcomes with fewer side effects compared to existing therapies.

What evidence suggests that ARGX-117 might be an effective treatment for Multifocal Motor Neuropathy?

Research has shown that ARGX-117, also known as empasiprubart, may help treat multifocal motor neuropathy (MMN). In this trial, participants will receive either ARGX-117 or a placebo during the double-blinded treatment period. Patients using ARGX-117 reported stronger grip and improved feelings about their condition. They also required fewer repeat treatments of IVIg, a common therapy for MMN. Lab studies suggest that ARGX-117 works by stopping harmful effects of the immune system on nerve cells that control movement. These findings indicate that ARGX-117 could be a helpful option for people with MMN. ^{2 3 4 5 6}

Are You a Good Fit for This Trial?

Adults with Multifocal Motor Neuropathy who completed the ARGX-117-2002 trial can join. They must be able to consent, follow the study plan, and use effective contraception. Excluded are those with serious diseases, recent major surgery, other clinical study participation, pregnancy or lactation.

Inclusion Criteria

I will use birth control until 15 months after my last treatment dose.

Capable of providing signed informed consent and complying with protocol requirements. Participants must be able to read and write.

I finished the ARGX-117-2002 trial and can be treated with ARGX-117.

Exclusion Criteria

I do not have any serious ongoing infections.

I do not have any serious health conditions that could interfere with the trial.

Pregnant or lactating or intend to become pregnant during the trial or within 15 months after last dose of the Investigational Medicinal Product

Timeline for a Trial Participant

Double-blinded Rollover Treatment Period (DTP)

Participants receive ARGX-117 or placebo intravenously in a double-blinded manner

12 weeks

Visits on day 1, 8, 15, 22 followed by every 4 weeks

Open-label Treatment Period (OTP)

Participants receive ARGX-117 intravenously in an open-label manner

Until market authorization or treatment discontinuation, assessed up to 70 months

Every 8 weeks

Safety Follow-up Period

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ARGX-117

Trial Overview The trial tests long-term safety and effectiveness of ARGX-117 for MMN patients. It includes a double-blinded period where some get ARGX-117 and others a placebo, followed by an open-label period where all receive ARGX-117.

How Is the Trial Designed?

Group I: Placebo Experimental Treatment 2 Interventions

Participants receiving placebo during the double-blinded treatment period (DTP) and receiving ARGX-117 during the open-label treatment period (OLTP)

Group II: ARGX-117 Experimental Treatment 1 Intervention

Participants receiving ARGX-117 during the double-blinded treatment period (DTP) and the open-label treatment period (OLTP)

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 ...