ARGX-117 for Multifocal Motor Neuropathy

(ARDA+ Trial)

Recruiting at 49 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

51 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the drug ARGX-117 (Empasiprubart) for treating multifocal motor neuropathy?

The research articles provided focus on the effectiveness of intravenous immunoglobulin (IVIg) for treating multifocal motor neuropathy, which suggests that similar treatments targeting the immune system may be effective. While ARGX-117 (Empasiprubart) is not directly mentioned, its potential effectiveness could be inferred from the success of IVIg in treating this condition. ^{1 2 3 4 5}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

References

1. United States pubmed.ncbi.nlm.nih.gov

Correlates of outcome and response to IVIg in 88 patients with multifocal motor neuropathy. [2014]

2. England pubmed.ncbi.nlm.nih.gov

Intravenous immunoglobulin as short- and long-term therapy of multifocal motor neuropathy: a retrospective study of response to IVIg and of its predictive criteria in 40 patients. [2017]

3. England pubmed.ncbi.nlm.nih.gov

Use of human intravenous immunoglobulin in lower motor neuron syndromes. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Multifocal motor neuropathy: the diagnostic spectrum and response to treatment. [2007]

5. Japan pubmed.ncbi.nlm.nih.gov

[Clinical features of 8 patients with multifocal motor neuropathy in the long-term follow-up]. [2023]