A Multicenter, Open-label Extension Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis
This research is testing a new drug, Fipaxalparant, for adults with a condition called diffuse cutaneous systemic sclerosis, which affects the skin and sometimes internal organs like the lungs. This trial is an extension, meaning some people might have already taken part in an earlier study. We want to see how effective and safe Fipaxalparant is over 52 weeks (about a year). Doctors will be paying close attention to changes in lung function, specifically how much air people can breathe out, and also looking at overall well-being and daily activities. This is an important step in understanding if this new medicine could help people manage their condition better.
At a glance
What is this study about?
This study is designed for people who have diffuse cutaneous systemic sclerosis. This is a condition where the immune system mistakenly attacks healthy tissues, causing the skin to thicken and tighten across larger areas of the body, and it can also affect internal organs like the lungs. The study is particularly interested in a new medicine called Fipaxalparant, and it's an 'open-label extension' trial. This means that everyone taking part will receive the study medication, and this trial is designed to gather more information over a longer period, building on what was learned in earlier stages.
The main goal of this study is to see how Fipaxalparant affects lung function over 52 weeks (roughly one year). Doctors will measure how much air people can breathe out forcefully after taking a deep breath – this is called Forced Vital Capacity or FVC. They want to see if the medicine can help improve this measure or prevent it from getting worse. This is really important because lung problems can be a serious part of diffuse cutaneous systemic sclerosis.
Beyond lung function, the study will also look at several other aspects of health. This includes how the condition affects daily activities, overall well-being, and other physical symptoms. By looking at all these different areas, doctors hope to get a comprehensive picture of how Fipaxalparant might help people with this challenging condition.
Key takeaways
- This study is for adults with diffuse cutaneous systemic sclerosis.
- It's testing a new medicine called Fipaxalparant over 52 weeks.
- The main focus is on lung health (FVC) and overall well-being.
- You must have been in a previous study with this medication to join this one.
- Participation involves regular clinic visits and health checks.
Who may be eligible?
To be considered for this study, you need to be an adult, aged 18 or older. Your gender does not matter, as the study is open to everyone.
Crucially, this particular study is an 'extension' trial. This means it's for people who have already taken part in a previous clinical trial involving the study medicine, HZN-825 (which is the same as Fipaxalparant). If you haven't been part of that earlier trial, this specific study would not be the right one for you.
There will be other detailed health requirements that the study doctors will go through with you. These are to make sure the study medicine is safe for you and that the results of the study are clear and accurate. They will check your medical history and current health carefully to see if you are a good fit.
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 diffuse cutaneous systemic sclerosis?
- Have you previously taken part in a study involving the medication HZN-825 (Fipaxalparant)?
- Are you able to attend regular clinic appointments?
What does participation involve?
If you decide to take part in this study, you would receive the study medicine, Fipaxalparant, for a period of 52 weeks (about a year). You'd have regular visits to the clinic where the study team would monitor your health closely. These visits would involve various assessments, such as breathing tests to check your lung function (FVC), detailed questionnaires about how you're feeling and how the condition affects your daily life, and physical examinations. They would also take blood and urine samples to check for safety and how the medicine is working. The study team will explain the exact schedule of visits and what each visit will involve. After the 52 weeks of taking the medication, there might be a follow-up period to see how you are doing without the study drug.
Potential risks and benefits
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Common questions
What is diffuse cutaneous systemic sclerosis?
It's a condition where your skin becomes thick and tight, often on larger parts of your body. It can also affect internal organs like your lungs and heart.
What is Fipaxalparant?
It's a new medicine being tested in this study to see if it can help people with diffuse cutaneous systemic sclerosis.
How long will the study last if I join?
The main treatment period for the study is 52 weeks, which is about one year. There might be follow-up appointments after that.
Will I know if I'm getting the actual medicine?
Yes, this is an 'open-label' study, which means everyone taking part will receive the study medicine, Fipaxalparant.
Can I leave the study if I want to?
Yes, you have the right to leave the study at any time, for any reason, and it won't affect your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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