#1 Clinical Trials Search in Europe

Empasiprubart

Empasiprubart, also known as ARGX-117, is an investigational drug being studied in clinical trials for the treatment of two rare neurological conditions: Multifocal Motor Neuropathy (MMN) and Dermatomyositis (DM). These trials aim to evaluate the safety, tolerability, and effectiveness of Empasiprubart in adult patients with these conditions. The studies involve intravenous administration of the drug and assess various aspects of patient health and disease progression.

Table of Contents

What is EMPASIPRUBART?

EMPASIPRUBART, also known as ARGX-117, is a new medication being developed to treat certain autoimmune disorders[1]. It is a type of drug called a monoclonal antibody, which means it’s a laboratory-made protein that mimics the immune system’s ability to fight off harmful antigens such as viruses[2]. Specifically, EMPASIPRUBART is described as an anti-complement 2 IgG humanized monoclonal antibody.

How does EMPASIPRUBART work?

EMPASIPRUBART works by targeting a part of the immune system called the complement system. Specifically, it binds to and inhibits a protein called complement 2 (C2)[1]. In some autoimmune disorders, the complement system can become overactive and contribute to inflammation and tissue damage. By blocking C2, EMPASIPRUBART aims to reduce this harmful immune activity and alleviate symptoms of certain autoimmune conditions.

What conditions does EMPASIPRUBART treat?

Based on current clinical trials, EMPASIPRUBART is being studied for the treatment of two specific autoimmune conditions:

  1. Multifocal Motor Neuropathy (MMN): This is a rare condition that affects the nerves controlling muscle movement, leading to muscle weakness and atrophy, primarily in the arms and hands[1].
  2. Dermatomyositis (DM): This is an inflammatory disease characterized by muscle weakness and a distinctive skin rash[2].

These conditions can significantly impact a person’s quality of life, and current treatments may not be effective for all patients. EMPASIPRUBART represents a potential new option for these hard-to-treat disorders.

How is EMPASIPRUBART administered?

EMPASIPRUBART is given as an intravenous infusion, which means it’s delivered directly into the bloodstream through a vein[1][2]. The medication comes in the form of a concentrate that is diluted into a solution for infusion. The exact dosage and frequency of administration may vary depending on the specific condition being treated and the individual patient’s needs.

Current Clinical Trials

EMPASIPRUBART is currently being studied in two main clinical trials:

  1. Multifocal Motor Neuropathy (MMN) Trial: This is a long-term extension study evaluating the safety, tolerability, and effectiveness of EMPASIPRUBART in adults with MMN[1]. The study is measuring various aspects of muscle strength, motor function, quality of life, and fatigue in participants.
  2. Dermatomyositis (DM) Trial: This is a Phase 2 study comparing EMPASIPRUBART to a placebo in adults with dermatomyositis[2]. The main goals are to assess the safety and effectiveness of the drug in treating this condition.

Both trials are currently ongoing, and results are not yet available. These studies will help determine if EMPASIPRUBART is safe and effective for treating these conditions.

Safety and Side Effects

As EMPASIPRUBART is still in clinical trials, comprehensive information about its safety profile and potential side effects is not yet available. The ongoing studies are closely monitoring participants for any adverse events[1][2].

It’s important to note that patients receiving EMPASIPRUBART in clinical trials are required to be vaccinated against certain bacterial infections (meningococcal, pneumococcal, and Haemophilus influenzae type B) before starting treatment[2]. This suggests that the medication may affect the immune system’s ability to fight off certain types of infections.

Conclusion

EMPASIPRUBART (ARGX-117) represents a promising new approach to treating certain autoimmune disorders, particularly Multifocal Motor Neuropathy and Dermatomyositis. By targeting the complement system, it aims to reduce harmful immune activity and alleviate symptoms of these conditions. While the results of ongoing clinical trials are eagerly awaited, EMPASIPRUBART offers hope for patients who may not have responded well to existing treatments. As with any new medication, it’s important to discuss the potential benefits and risks with a healthcare provider.

Aspect Multifocal Motor Neuropathy (MMN) Trial Dermatomyositis (DM) Trial
Trial Phase Phase 2 Phase 2
Study Design Long-term extension, double-blinded rollover, and open-label treatment periods Randomized, double-blinded, placebo-controlled
Primary Objective Evaluate long-term safety and tolerability of ARGX-117 Evaluate safety and tolerability compared to placebo
Key Secondary Objectives Assess long-term efficacy on muscle strength, functional ability, and quality of life Evaluate clinical efficacy compared to placebo
Main Inclusion Criteria Completed previous ARGX-117-2002 trial, eligible for ARGX-117 treatment Adults with active DM or juvenile DM, mild skin disease
Key Assessments mMRC scale, grip strength, disability scales, quality of life measures Adverse events, Total Improvement Score (TIS)
Treatment Duration Up to 4 weeks, with long-term follow-up Up to 25 weeks

FAQ

  • What is Empasiprubart? Empasiprubart, also known as ARGX-117, is an investigational drug being studied for the treatment of Multifocal Motor Neuropathy (MMN) and Dermatomyositis (DM). It is a humanized monoclonal antibody that targets complement component 2 (C2) in the body's immune system.
  • What conditions are being studied in these clinical trials? The clinical trials are studying Empasiprubart in two rare conditions: Multifocal Motor Neuropathy (MMN), a neurological disorder affecting motor nerves, and Dermatomyositis (DM), an inflammatory muscle disease that also affects the skin.
  • How is Empasiprubart administered in these trials? Empasiprubart is administered intravenously (through a vein) during scheduled visits to the clinical trial sites. The exact dosing and frequency may vary depending on the specific trial protocol.
  • What are the main goals of these clinical trials? The main goals of these trials are to evaluate the long-term safety, tolerability, and effectiveness of Empasiprubart in adult patients with MMN and DM. The studies also aim to assess how the drug affects muscle strength, functional ability, quality of life, and various disease-specific measures.
  • Who can participate in these clinical trials? Eligibility criteria vary between the trials, but generally, adult patients diagnosed with either MMN or DM who meet specific disease activity criteria may be eligible. Participants must be able to provide informed consent and comply with the study requirements. Certain medical conditions or previous treatments may exclude some individuals from participating.

Ongoing Clinical Trials on Empasiprubart

  • A study to test if empasiprubart can improve function and is safe for adults with chronic inflammatory demyelinating polyneuropathy

    Recruiting

    3 1
    Investigated drugs:
    Austria Bulgaria Czechia Denmark Estonia Finland +9

  • A study to evaluate the safety and effectiveness of empasiprubart and efgartigimod alfa in patients with generalized myasthenia gravis.

    Recruiting

    2 1 1
    Investigated drugs:
    Belgium Germany Greece Italy The Netherlands Poland +1

  • A Study Comparing Empasiprubart Versus Intravenous Immunoglobulin in Adults With Chronic Inflammatory Demyelinating Polyneuropathy

    Recruiting

    3 1 1
    Investigated drugs:
    Austria Bulgaria Czechia Denmark Estonia France +13

  • Study comparing empasiprubart versus intravenous immunoglobulin for treating adults with multifocal motor neuropathy

    Not recruiting

    3 1 1
    Investigated drugs:
    Austria Belgium Czechia Denmark Estonia France +13

  • Study on the Safety and Efficacy of Empasiprubart for Adults with Dermatomyositis

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Greece Italy Poland Spain

  • Study on Long-term Safety and Effects of ARGX-117 for Adults with Multifocal Motor Neuropathy

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium France Germany Italy The Netherlands +2

  • Study on ARGX-117 for Improving Kidney Function in Patients with Delayed Graft Function After Deceased Donor Transplant

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium France Italy Portugal Spain +1

Glossary

  • Multifocal Motor Neuropathy (MMN): A rare neurological disorder that causes progressive muscle weakness and affects motor nerves, typically in the arms and legs.
  • Dermatomyositis (DM): An inflammatory muscle disease characterized by muscle weakness and skin rashes. It can affect both adults and children.
  • Monoclonal antibody: A type of protein made in the laboratory that can bind to specific targets in the body. In this case, Empasiprubart is designed to target complement component 2 (C2) in the immune system.
  • Intravenous (IV): A method of administering medication directly into a vein using a needle or tube.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Pharmacodynamics (PD): The study of how a drug affects the body, including its mechanism of action and relationship between drug concentration and effect.
  • Immunogenicity: The ability of a substance to provoke an immune response in the body. In clinical trials, this is often monitored to check if the body develops antibodies against the study drug.
  • Modified Medical Research Council (mMRC) scale: A standardized scale used to measure muscle strength in various muscle groups.
  • Quality of Life (QOL): A measure of an individual's overall well-being and ability to function in daily life, often assessed using specific questionnaires in clinical trials.
  • Antidrug antibodies (ADA): Antibodies produced by the immune system against a therapeutic drug, which can potentially affect its efficacy or safety.

References

  1. http://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-argx-117-for-adults-with-multifocal-motor-neuropathy/
  2. http://clinicaltrials.eu/trial/study-on-the-safety-and-efficacy-of-empasiprubart-for-adults-with-dermatomyositis/