A Randomized, Double-blind, Placebo-controlled, Repeat-dose, Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis [HZNP-HZN-825-301]
This research study is looking into a new medicine, Fipaxalparant, for adults with a condition called diffuse cutaneous systemic sclerosis. This condition can affect the skin and internal organs, like the lungs. The study wants to find out if this new medicine is safe, if it helps improve lung function, and if it makes people feel better overall. Participants will take either the new medicine or a dummy pill (placebo) every day, and doctors will closely monitor their health over a year. The main goal is to see if the medicine helps improve how well the lungs work. The study is in an early stage (Phase II) of development.
At a glance
What is this study about?
This clinical trial is investigating a new medication called Fipaxalparant. This medicine is designed for people living with diffuse cutaneous systemic sclerosis, a long-term condition that can cause hardening and tightening of the skin and can also affect internal organs, including the lungs. The main purpose of this study is to see if Fipaxalparant can help to slow down or even improve some of the changes that happen in the lungs of people with this condition.
Researchers also want to learn if the new medicine is safe to use and if it has unwanted side effects. They will be watching closely to see how participants feel generally and if their daily lives improve. Participants will be taking the medicine (or a dummy version) as a tablet. The study is 'double-blind', meaning neither the patient nor their doctor will know if they are receiving the actual medicine or the dummy pill. This helps to make sure the results are as fair and accurate as possible.
This is an early-stage study (Phase II), which means it’s one of the first times this medicine is being tested in a larger group of people with the condition. The results of this study will help doctors understand if Fipaxalparant could be a helpful treatment option in the future. The study will last for about a year.
Key takeaways
- This study is testing a new medicine called Fipaxalparant for diffuse cutaneous systemic sclerosis.
- It aims to see if the medicine improves lung function and overall well-being.
- Participants will receive either the new drug or a dummy pill daily.
- The study lasts about one year with regular clinic visits.
- It's a 'Phase II' study, meaning it's an early look at the medicine's effects.
- Your health will be closely monitored throughout the study.
Who may be eligible?
This study is looking for adult volunteers, aged 18 years or older, who have been diagnosed with diffuse cutaneous systemic sclerosis. It's important that people who join have this specific type of scleroderma.
Both men and women can take part in this research. The medical team will check specific details of your health and medical history to make sure the study is a good fit for you and that it's safe for you to participate.
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 diffuse cutaneous systemic sclerosis?
- Are you able to take a tablet every day?
- Are you willing and able to attend regular clinic appointments for about a year?
What does participation involve?
If you join this study, you will take a study tablet every day for about a year. You won't know if it's the active medicine or a dummy pill, and neither will your study doctor. You'll have regular visits to the study clinic where the research team will do various checks. These checks might include blood tests, lung function tests to see how well your lungs are working, and physical examinations. You'll also be asked to answer questions about your symptoms and how you're feeling. These visits help the doctors monitor your health, check for any changes, and see if the medicine is helping. The total time you'd be involved in the study is just over a year.
Potential risks and benefits
Locations (9)
- —UnverifiedRomania
- —UnverifiedAustria
- —UnverifiedGermany
- —UnverifiedGreece
- —UnverifiedSpain
- —UnverifiedPoland
- —UnverifiedFrance
- —UnverifiedPortugal
- —UnverifiedItaly
Common questions
What is diffuse cutaneous systemic sclerosis?
It's a rare and serious long-term condition that causes the skin to harden and tighten, and can also affect internal organs like the lungs and heart.
What is Fipaxalparant?
Fipaxalparant is the new medicine being tested in this study to see if it can help people with diffuse cutaneous systemic sclerosis.
What does 'double-blind' mean?
It means that neither you nor your study doctor will know if you are receiving the active medicine or a dummy pill, which helps make the study results fair.
Why is lung function important in this study?
Lung problems are common in diffuse cutaneous systemic sclerosis, so the study wants to see if the new medicine can help improve how well the lungs work.
How long will the study last?
If you join this study, you will be involved for about one year of treatment and follow-up.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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