Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Efficacyand Safety Study Of Daxdilimab Subcutaneous Injection In Adult Participants With Inadequately Controlled Dermatomyositis Or Anti-Synthetase Inflammatory Myositis

This research study is investigating a new medication called Daxdilimab for adults who have dermatomyositis or anti-synthetase inflammatory myositis that hasn't been well controlled by their current treatments. It's a 'Phase 2' study, meaning it's an early step to see if Daxdilimab is safe and if it can help improve symptoms. Participants will be randomly assigned to receive either Daxdilimab or a dummy treatment (placebo), and neither they nor their doctors will know which one they are getting. The main goal is to measure changes in a 'Total Improvement Score' after 24 weeks to understand how well the new medicine works, and also to keep a close eye on any side effects.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Horizon Therapeutics Ireland Designated Activity Company

Enrolment target

Start

24 Jul 2023

Dermatomyositis (DM) or anti-synthetase inflammatory myositis (ASIM)

What is this study about?

This study is for adults living with specific types of inflammatory muscle conditions called dermatomyositis (DM) or anti-synthetase inflammatory myositis (ASIM). These conditions can cause muscle weakness, pain, and sometimes skin rashes. Often, current treatments don't fully control the symptoms, which can make daily life challenging. The new medicine being tested in this study is called Daxdilimab. Researchers want to find out if Daxdilimab could be a new and effective way to manage these conditions.

This is a 'Phase 2' study. This means it's an important early step in developing new medicines. In this phase, researchers are mainly focused on two things: making sure the medicine is safe for people to take, and getting a clearer idea of how well it might work to improve symptoms. It's not the final stage of testing, but it helps decide if the medicine should move forward to larger studies.

Participants will be placed into different groups by chance (randomly). Some will receive the new medicine, Daxdilimab, while others will receive a 'placebo' – which looks like the medicine but contains no active drug. This is done so researchers can fairly compare the effects of Daxdilimab against not taking the active medicine. Neither you nor your doctor will know which treatment you are receiving, which helps ensure the results are unbiased.

Key takeaways

This study is testing a new medicine, Daxdilimab, for specific inflammatory muscle conditions (dermatomyositis or anti-synthetase inflammatory myositis).
It's a 'Phase 2' trial, focused on safety and early signs of effectiveness.
Participants will receive either Daxdilimab or a dummy treatment (placebo) by chance.
The study lasts for 24 weeks, with regular clinic visits and monitoring.
Potential benefits include access to a new treatment, but risks like side effects also exist.
Your participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you need to be an adult, 18 years old or older. You also must have a confirmed diagnosis of either dermatomyositis or anti-synthetase inflammatory myositis, and your condition should not be fully controlled by your current medications.

The study is open to both men and women. The researchers want to include a wide range of people to understand how the new medicine might affect different patients.

There might be other specific health requirements or conditions that would mean you couldn't join the study, but these would be discussed in detail by the study team if you express interest.

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 or anti-synthetase inflammatory myositis?
Are your current treatments not fully controlling your condition?
Are you able to attend regular clinic visits for 24 weeks?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be randomly assigned to one of two groups: either receiving the new medicine, Daxdilimab, or a placebo (a dummy treatment). You would receive this medicine as an injection under your skin. The study would involve regular visits to the clinic for check-ups and assessments over a period of 24 weeks. During these visits, doctors and nurses will monitor your symptoms, perform physical exams, and take blood samples to see how you are responding and to check for any side effects. They will specifically look at a 'Total Improvement Score' to track how your symptoms are changing. After the 24-week treatment period, there will be further follow-up to monitor your health and any effects of the study medication.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit: you could receive a new medicine that could help improve your condition, which is not yet widely available. However, there's also a chance you could receive the placebo and not the active drug. As with all medicines, Daxdilimab could have side effects, which might include allergic reactions, infections like shingles, or other serious health issues. The study team will carefully monitor you for any known and unknown side effects. It's important to remember that participating in a clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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

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Unverified
Spain
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Unverified
Italy
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Unverified
Germany
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Unverified
Czechia
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Unverified
France

Common questions

What is dermatomyositis or anti-synthetase inflammatory myositis?

These are types of muscle diseases where your body's immune system mistakenly attacks your own muscles, causing weakness and other symptoms like skin rashes (in dermatomyositis).

What does 'Phase 2' mean for a study?

Phase 2 is an early stage of testing a new medicine. Researchers are mainly looking at whether the medicine is safe and gives early signs that it might help improve symptoms.

What does 'randomized, double-blind, placebo-controlled' mean?

It means you'll be put into a treatment group by chance (randomized), neither you nor your doctor will know if you're getting the active medicine or a dummy treatment (double-blind), and some people will get a dummy treatment (placebo-controlled) to compare against the real medicine.

What is Daxdilimab?

Daxdilimab is the new investigational medicine being tested in this study. It's given as an injection under the skin.

How long would I be in the study?

The main treatment period for the study is 24 weeks (about 6 months), with further follow-up after that.