A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared With Placebo Added to Standard of Care in Adult Participants With Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis)
This study is investigating a new medication called anifrolumab for adults who have polymyositis or dermatomyositis, which are types of inflammatory muscle conditions. Participants will receive either anifrolumab or a dummy treatment (placebo), in addition to their current usual care. The main goal is to find out if anifrolumab is effective in reducing the severity of these conditions and if it’s safe to use. This is a Phase 3 study, meaning it’s a later stage of research designed to confirm the drug's benefits and monitor for side effects in a larger group of people before it might become widely available.
At a glance
What is this study about?
This research study is focused on two specific conditions: polymyositis and dermatomyositis. These are types of idiopathic inflammatory myopathies (IIM), which means they are muscle diseases where the body's immune system attacks its own healthy tissues, leading to muscle weakness, pain, and sometimes skin problems. The study aims to see if a new drug, anifrolumab, can help improve the symptoms and overall health of adults living with moderate to severe forms of these conditions.
Participants in the study will be given either anifrolumab or a placebo (an inactive substance that looks like the drug but contains no medicine) through an injection under the skin. Both groups will also continue to receive their standard medical treatments, such as steroids or other medications their doctor has prescribed. This setup helps researchers determine if any improvements seen are truly due to anifrolumab or other factors.
The study is 'double-blind,' which means neither the participants nor the doctors and nurses know who is receiving the actual drug and who is receiving the placebo. This is done to ensure the results are unbiased and accurate. The ultimate goal is to understand if anifrolumab can be a safe and effective new treatment option for people with polymyositis and dermatomyositis.
Key takeaways
- This study is for adults with moderate to severe polymyositis or dermatomyositis.
- It's testing a new drug, anifrolumab, given as an injection, alongside usual treatments.
- You might receive the new drug or a dummy treatment (placebo).
- Neither you nor the doctors will know which treatment you are on initially.
- The study aims to see if anifrolumab is safe and effective in improving symptoms.
- Your decision to participate is voluntary, and you can leave the study at any time.
Who may be eligible?
To join this study, you would generally need to be an adult between 18 and 75 years old and have a confirmed diagnosis of polymyositis or dermatomyositis that is causing moderate to severe symptoms. You should also be currently taking treatments for your condition, like oral steroids, at a stable dose. Your overall health would be assessed to make sure taking part is safe for you, and women of childbearing age would need to agree to use contraception.
There are certain reasons why someone might not be able to join. For example, if you have other specific types of muscle conditions (like inclusion body myositis or muscular dystrophy), or if you've had certain serious infections (like recent severe COVID-19, TB, or certain viral infections that haven't cleared up). A history of most cancers, serious lung disease, or conditions causing severe muscle damage not related to polymyositis or dermatomyositis would also prevent participation.
Additionally, if you are pregnant, breastfeeding, or planning to become pregnant or breastfeed, you would not be able to join. Your doctor would carefully review all your medical history to determine if this study is suitable for you.
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 between 18 and 75 years old?
- Do you have a confirmed diagnosis of polymyositis or dermatomyositis?
- Are your symptoms currently moderate to severe?
- Are you currently taking treatments for your condition at a stable dose?
- Do you have stable, general good health, without recent serious infections or specific other medical conditions?
- If you are a woman of childbearing age, are you willing to use contraception during the study?
What does participation involve?
If you decide to take part in this study, you would first have a full medical check-up to ensure you meet all the criteria. After that, you would be randomly assigned to either receive the study drug (anifrolumab) or a placebo, given as an injection under your skin. You and your study team wouldn't know which one you're receiving at the start.
Throughout the study, you would have regular visits to the clinic for assessments. These visits would involve physical examinations, blood tests, and other procedures to monitor your condition and check for any side effects. You would continue to take your usual prescribed medications for polymyositis or dermatomyositis alongside the study treatment. The total duration of your participation would involve a initial 'blinded' treatment period where you don't know what you're receiving, followed by an 'open-label' period where everyone receives anifrolumab, and then a follow-up period after treatment ends. The exact number of visits and the total length of the study would be fully explained to you.
Potential risks and benefits
Locations (236)
- Research SiteVerified postcodePhoenix, United States· Not yet recruiting
- Research SiteVerified postcodeIrvine, United States· Recruiting
- Research SiteVerified postcodeAurora, United States· Recruiting
- Research SiteVerified postcodeDenver, United States· Not yet recruiting
- Research SiteVerified postcodeNew Haven, United States· Recruiting
- Research SiteVerified postcodeWashington D.C., United States· Withdrawn
- Research SiteVerified postcodeBoca Raton, United States· Recruiting
- Research SiteVerified postcodeBoynton Beach, United States· Withdrawn
- Research SiteVerified postcodeGainesville, United States· Withdrawn
- Research SiteVerified postcodeMargate, United States· Suspended
- Research SiteVerified postcodeMiami, United States· Withdrawn
- Research SiteVerified postcodeMiami, United States· Recruiting
Common questions
What are polymyositis and dermatomyositis?
They are types of long-term muscle conditions where your body's immune system mistakenly attacks muscle tissues, causing weakness and sometimes skin rashes in dermatomyositis.
What is 'standard of care'?
This refers to the common and accepted treatments that doctors currently use for polymyositis and dermatomyositis, such as steroids or other immune-suppressing medications.
What does 'double-blind' mean in a study?
It means that neither you nor your study doctor will know whether you are receiving the active study drug or a placebo, to keep the results fair and unbiased.
What is a 'placebo'?
A placebo is a dummy treatment that looks exactly like the study drug but doesn't contain any active medication. It helps researchers compare the effects of the active drug.
Can I stop participating in the study if I change my mind?
Yes, your participation is completely voluntary, and you can withdraw from the study at any time without it affecting your future medical care.
How to find out more
AstraZeneca Clinical Study Information Center
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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