A Study to Understand How Nerandomilast Works in People With Active Idiopathic Inflammatory Myopathies
Idiopathic inflammatory myopathies, often simply called myositis, are long-term conditions where the immune system attacks healthy muscle, causing swelling, pain, and weakness. While treatments exist, they do not work well for everyone and can cause unwanted side effects. This Phase 3 clinical trial is testing a new oral medicine called nerandomilast to see if it safely improves myositis symptoms and daily quality of life. Participants will be put into one of two groups by chance: one group takes nerandomilast tablets twice daily, and the other takes matching dummy tablets (a placebo), both added to their standard myositis medication. The trial lasts around 14 months and involves about 16 clinic visits. Doctors will closely track muscle strength, symptom changes, and general health to see if the new medicine makes a meaningful difference.
Överblick
Vad handlar denna studie om?
Idiopathic inflammatory myopathies (IIM), commonly known as myositis, are a group of rare autoimmune conditions. In people with myositis, the body's natural defence system mistakenly attacks its own muscles. This leads to swelling, tenderness, and muscle weakness that can make everyday tasks—such as climbing stairs, lifting objects, or even getting out of a chair—very difficult.
Currently, people with myositis usually take medicines like steroids or other drugs that calm the immune system. While these can help, they do not always work well enough and long-term use can lead to troublesome side effects. Because of this, researchers are working to find new, more targeted treatments.
This study is testing an investigational tablet called nerandomilast. Researchers want to see if adding nerandomilast to a person's regular treatment helps reduce muscle inflammation, restore physical strength, and improve overall quality of life. Because it is a Phase 3 trial, it is one of the final stages of testing before a medicine can be approved for wider NHS use.
Viktiga punkter
- Tests whether a new tablet, nerandomilast, helps reduce active muscle inflammation in myositis.
- Open to adults aged 18 and older who are already receiving treatment for myositis.
- Participants take either nerandomilast or a dummy tablet (placebo) twice daily for one year.
- Involves around 16 clinic visits over roughly 14 months to monitor health and strength.
- Taking part is voluntary and will not replace your usual background myositis care.
Vem kan vara berättigad?
This trial is for adults aged 18 and over who have been diagnosed with an active form of inflammatory myopathy (myositis). Your muscle condition must be causing moderate to severe symptoms right now, and you should already be taking a standard background treatment for it. People with overlap myositis can take part if myositis is their main condition. If you had juvenile dermatomyositis, your symptoms must have started when you were at least 15 years old.
You will not be able to join if you have recently had major surgery (within the past six weeks) or have an operation planned during the study. People who have had cancer within the last five years (apart from certain successfully treated skin or cervical cancers) are also excluded to protect their health.
Because the effects on unborn babies are not fully known, women who could become pregnant must agree to use reliable forms of contraception throughout the study and for a short time afterwards. The study doctor will check all your regular medicines to make sure none of them clash with the trial drug.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- I am aged 18 or older.
- I have been diagnosed with an active inflammatory muscle condition (myositis).
- I am currently experiencing moderate to severe muscle weakness or symptoms.
- I am already taking a standard, regular medicine for my condition.
- I have not had major surgery in the last 6 weeks, and none is planned.
- I have been free of cancer for at least the past 5 years (except certain skin or cervical cancers).
Vad innebär deltagande?
If you decide to take part, you will be in the study for about one year and two months (14 months). You will be randomly assigned to take either nerandomilast or a placebo (a dummy pill with no active medicine) twice every day for one year. Neither you nor your doctor can choose which group you are in, and neither will know which pill you are taking until the study ends. You will continue taking your standard prescribed myositis medicines alongside the trial tablets.
During the trial, you will visit the hospital clinic at least 16 times. At these appointments, the medical team will carry out physical checks, test your muscle strength, take blood and urine samples, and monitor your general well-being. You will also be asked to complete simple questionnaires about your daily symptoms and how you are feeling. After you finish the 12 months of treatment, there is a short follow-up period to ensure you remain well.
Potentiella risker och fördelar
Locations (158)
- Arizona Neuromuscular Research CenterVerifierat postnummerPhoenix, USA
- Mayo ClinicVerifierat postnummerScottsdale, USA
- University of California IrvineVerifierat postnummerIrvine, USA
- University of California Los AngelesVerifierat postnummerLos Angeles, USA
- Universitetet i Kalifornien San FranciscoVerifierat postnummerSan Francisco, USA
- University of Colorado DenverVerifierat postnummerAurora, USA
- Kansas UniversitetssjukhusVerifierat postnummerFairway, USA
- Johns Hopkins sjukhusVerifierat postnummerBaltimore, USA
- Brigham och Women's HospitalVerifierat postnummerBoston, USA
- University of MichiganVerifierat postnummerAnn Arbor, USA
- Mayo Clinic, RochesterVerifierat postnummerRochester, USA
- Washington University School of MedicineVerifierat postnummerSt Louis, USA
Vanliga frågor
What is a placebo and why is it used?
A placebo is a dummy tablet that looks identical to the real medicine but contains no active ingredients. It helps researchers prove whether any health improvements are truly caused by the trial drug rather than other factors.
Will I have to stop my current myositis medication?
No. Participants are required to remain on their standard background treatment for myositis throughout the trial, so you will not be left without care.
Do I have to pay to take part in this study?
No. The study medication and all trial-related tests are provided free of charge, and trial sites often help cover reasonable travel expenses.
Kan jag lämna studien om jag ändrar mig?
Yes. Participation is completely voluntary. You can withdraw your consent at any time without giving an explanation, and your normal healthcare will continue as usual.
How often will I need to take the study tablets?
You will need to take your assigned tablets (either nerandomilast or placebo) by mouth twice a day for approximately one year.
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