Active not recruitingPHASE2, PHASE3INTERVENTIONAL

A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy.

This study is investigating a new medication called efgartigimod PH20 SC for adults living with certain types of active idiopathic inflammatory myopathy (IIM). IIM is a condition that causes muscle inflammation and weakness. The study aims to find out if this new drug is effective and safe compared to a placebo, which is a dummy treatment with no active medicine. Researchers will be looking at how participants respond to the treatment, specifically checking for improvements in their overall condition. The trial includes specific types of IIM, such as dermatomyositis, immune-mediated necrotizing myopathy, and certain forms of polymyositis. If you have one of these conditions and meet certain criteria, you might be able to take part. More information is available on the study website.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

argenx

Enrolment target

265

Start

12 Oct 2022

Estimated completion

01 Feb 2027

Active Idiopathic Inflammatory Myopathy Myositis Dermatomyositis Polymyositis Immune-Mediated Necrotizing Myopathy Antisynthetase Syndrome

What is this study about?

This study is about a new treatment for a group of conditions known as Idiopathic Inflammatory Myopathies (IIM). These conditions cause your immune system to mistakenly attack your muscles, leading to inflammation, weakness, and sometimes skin problems. This study is specifically looking at adults who have active forms of conditions like dermatomyositis, polymyositis (including antisynthetase syndrome), and immune-mediated necrotizing myopathy.

The main goal is to see if a new drug, called efgartigimod PH20 SC, can help improve symptoms and overall well-being compared to a placebo. A placebo looks like the real treatment but contains no active medication. This comparison helps researchers understand the true effects of the new drug. By carefully studying how people respond, doctors hope to find better ways to manage these challenging conditions and improve the lives of those affected.

Finding new, effective treatments for IIM is really important. Current treatments can have side effects and don't work for everyone. This study could lead to a new option that helps more people feel better, regain muscle strength, and reduce inflammation, ultimately improving their quality of life.

Key takeaways

A new drug for muscle inflammation (IIM) is being tested.
The study compares the new drug to a dummy treatment (placebo).
It's for adults with active forms of dermatomyositis, polymyositis, or IMNM.
The goal is to find out if the drug improves muscle strength and overall well-being.
Participation involves regular clinic visits and health checks.

Who may be eligible?

To join this study, you need to be an adult (18 years or older) with a confirmed diagnosis of idiopathic inflammatory myopathy (IIM). This includes specific types like dermatomyositis, polymyositis (including antisynthetase syndrome), or immune-mediated necrotizing myopathy.

It's also important that your IIM is currently active. This might mean you have certain abnormal blood test results, recent signs of muscle disease on an electromyogram or MRI scan, an active skin rash from dermatomyositis, or changes seen in a recent muscle biopsy. You should also be experiencing muscle weakness and be on a stable background treatment for your IIM.

There are also some reasons why you might not be able to join. For example, if you have an active infection, have recently tested positive for COVID-19, or have another serious autoimmune disease that might complicate the study. You also cannot have a history of cancer unless it's completely cured.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of dermatomyositis, immune-mediated necrotizing myopathy, or polymyositis (including antisynthetase syndrome)?
Is your muscle condition currently active (e.g., recent abnormal blood tests, muscle weakness)?
Are you currently receiving a stable treatment for your muscle condition?
Do you not have an active infection or certain other serious health conditions?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be assigned to either receive the new drug, efgartigimod PH20 SC, or a placebo (a dummy treatment). You won't know which one you're getting, and neither will your study doctor, to ensure the results are unbiased. The new drug is given as an injection under the skin.

Your participation will involve regular visits to the study clinic. During these visits, you'll have various assessments to check your progress and safety. These might include physical examinations, blood tests, and questionnaires about your symptoms and how you're feeling. The study will carefully monitor how your body reacts to the treatment and if your IIM symptoms are improving. The total duration of the study will be explained in detail by the study team, along with specific timelines for follow-up appointments.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might benefit if the new drug helps improve your IIM symptoms, even if it's a placebo effect in the control group. However, there's also a chance the treatment won't help, or you might experience side effects from the drug. Possible risks will be fully explained by the study team. You always have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (202)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Neuromuscular Research Center
Verified postcode
Phoenix, United States
HonorHealth Neurology - Bob Bove Neuroscience Institute - Neurology
Verified postcode
Scottsdale, United States
Attune Health Research, Inc
Verified postcode
Beverly Hills, United States
University of Southern California Norris Comprehensive Cancer Center
Verified postcode
Los Angeles, United States
Profound Research LLC - Oceanside
Verified postcode
Oceanside, United States
University of California, Irvine
Verified postcode
Orange, United States
Eisenhower Medical Center
Verified postcode
Rancho Mirage, United States
Stanford Medicine Outpatient Center - Stanford Dermatology Clinic-Stanford University School of Medicine
Verified postcode
Redwood City, United States
California Pacific Medical Center - Sutter Health
Verified postcode
San Francisco, United States
Amyotrophic Lateral Sclerosis (ALS) Treatment Center,University of California San Francisco (UCSF)
Verified postcode
San Francisco, United States
Denver Arthritis Clinic
Verified postcode
Denver, United States
Yale Cancer Center-Yale University School Of Medicine
Verified postcode
New Haven, United States

Common questions

What is Idiopathic Inflammatory Myopathy (IIM)?

IIM is a group of conditions where your body's immune system mistakenly attacks your muscles, causing inflammation and weakness.

What is the new drug being tested?

The new drug is called efgartigimod PH20 SC. It's hoped to help reduce muscle inflammation and weakness.

What is a placebo?

A placebo is a 'dummy' treatment that looks like the actual medicine but contains no active drug. It helps researchers compare the real drug's effects.

Will I know if I'm getting the drug or the placebo?

No, you won't know if you're receiving the active drug or the placebo. This helps ensure the study results are fair and accurate.

How will the study measure if the treatment works?

The study will measure changes in your overall condition, including muscle strength and how you feel, using something called a 'Total Improvement Score'.