ARGX-119 for ALS

(ReALiSe Trial)

Not currently recruiting at 9 trial locations

Overseen By Sabine s Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: argenx

Disqualifiers: Ventilation, Gene therapies, Pregnancy, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ARGX-119 for people with ALS, a disease that affects muscle function. Researchers aim to determine if ARGX-119 is safe and how it affects muscle strength and other ALS symptoms. Participants will receive one of three doses of ARGX-119 or a placebo (a substance with no active drug) for a period, after which everyone will switch to ARGX-119. This trial may suit adults diagnosed with ALS who can breathe without assistance most of the day. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important ALS research.

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 study team or your doctor.

Is there any evidence suggesting that ARGX-119 is likely to be safe for humans?

Research shows that ARGX-119 has been well tolerated at the doses tested so far. In earlier studies with healthy participants, ARGX-119 was generally safe, with most people experiencing no severe or unexpected side effects. The treatment has been tested in both single and multiple doses, and the safety results were mostly positive.

Ongoing research examines how well patients with amyotrophic lateral sclerosis (ALS) tolerate the treatment. Since this study is in Phase 2, earlier tests showed acceptable safety. However, each new study aims to gather more detailed safety information. Prospective participants should discuss potential risks and benefits with a healthcare provider. ^{1 2 3 4 5}

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

Researchers are excited about ARGX-119 for ALS because it uses a novel approach to target the disease. Unlike current treatments such as riluzole and edaravone, which aim to slow disease progression or manage symptoms, ARGX-119 is designed to modulate the immune system, potentially addressing underlying causes of ALS. The treatment is administered intravenously, allowing for precise dosing, and it has multiple dosage levels under investigation, which could optimize its effectiveness and safety. This unique mechanism and delivery method offer hope for potentially altering the course of ALS, rather than just managing its symptoms.

What evidence suggests that ARGX-119 might be an effective treatment for ALS?

Research shows that ARGX-119, a specially designed antibody, has shown promising results in treating ALS (amyotrophic lateral sclerosis). Early findings from previous studies suggest that ARGX-119 is generally safe and causes few side effects in patients. Importantly, these studies also indicate that the drug might help improve certain ALS symptoms, like muscle function. However, these findings are early, and more research is needed to confirm the drug's effectiveness. Overall, the initial data offers hope for ARGX-119 as a treatment option for ALS. Participants in this trial will receive different dosage levels of ARGX-119 or a placebo during the double-blinded treatment period, followed by ARGX-119 in an active treatment extension period. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for adults aged 18 to 80 with ALS, who have a specific risk profile and at least 60% normal lung function. It's open to those with familial or sporadic ALS as per Gold Coast criteria.

Inclusion Criteria

My ALS risk profile score is between -6.0 and -2.0.

I have been diagnosed with ALS.

My lung function test shows at least 60% of what is expected for someone my age and size.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive one of three ARGX-119 doses or placebo intravenously during the double-blinded treatment period

Approximately 48 weeks

Extension

All participants receive the same dose of ARGX-119 in the active treatment extension period

Approximately 52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ARGX-119

Trial Overview The study tests ARGX-119's safety and its effect on muscle function in ALS patients. Participants will receive either ARGX-119 or a placebo during the treatment period, followed by an extension where all get ARGX-119.

How Is the Trial Designed?

Group I: ARGX-119 - Dose 3 Experimental Treatment 1 Intervention

Participants will receive third dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period

Group II: ARGX-119 - Dose 2 Experimental Treatment 1 Intervention

Participants will receive second dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period

Group III: ARGX-119 - Dose 1 Experimental Treatment 1 Intervention

Participants will receive first dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period

Group IV: Placebo Placebo Group 2 Interventions

Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period

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. clinicaltrials.gov clinicaltrials.gov/study/NCT06441682

NCT06441682 | A Safety and Efficacy Study of ARGX-119 ... This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including ...

2. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/aanem-2024/Safety,%20Tolerability,%20Efficacy,%20Pharmacokinetics,%20and%20Immunogenicity%20of%20ARGX-119%20in%20Patients%20With%20Amyotrophic%20Lateral%20Sclerosis%20A%20Phase%202a%20Study.pdf

A Phase 2a Study in Progress *Blinding to treatment assignment in DBTP will remain throughout the study. Interim results from the ongoing, phase 1,. FIH study suggest that ARGX-119 has a.

3. argenx.com argenx.com/news/2025/press-release-3107132

argenx Advances Clinical Development of ARGX-119 in ... ARGX-119 demonstrated a favorable safety and tolerability profile, which was the primary endpoint. Efficacy was evaluated across multiple ...

4. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-and-efficacy-of-argx-119-for-adults-with-amyotrophic-lateral-sclerosis-als/

Study on the Safety and Efficacy of ARGX-119 for Adults ... This study evaluates the safety and efficacy of ARGX-119, a humanized IgG1 monoclonal antibody, as a potential treatment for adults suffering from ...

5. neurology.org neurology.org/doi/10.1212/WNL.0000000000212107

First-in-Human Dose Selection and Pharmacokinetics ... Preliminary results from the FIH study suggest that ARGX-119 has a favorable safety profile in healthy participants at the doses investigated.