RecruitingPHASE2INTERVENTIONAL

Mycophenolate Mofetil in Systemic Sclerosis With Subclinical Interstitial Lung Disease

This study aims to discover if an existing medicine, mycophenolate mofetil, could help people with systemic sclerosis who have early signs of lung damage. Systemic sclerosis can cause the immune system to wrongly attack the body, often leading to lung issues which can be very serious. While this medicine is already used for more advanced lung problems caused by systemic sclerosis, researchers believe treating it earlier, when lung function is still good, might lead to better results. This initial study will help determine if a bigger study is practical. Participants will be randomly assigned to receive either the medicine or a dummy pill for about 96 weeks, helping researchers understand if early treatment with this drug could be beneficial.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Centre hospitalier de l'Université de Montréal (CHUM)

Enrolment target

Start

29 Aug 2024

Estimated completion

01 Dec 2028

Systemic Sclerosis With Lung Involvement Systemic Sclerosis Interstitial Lung Disease

What is this study about?

Systemic sclerosis is a rare condition where your body's immune system, which normally protects you from illness, starts to attack healthy tissues instead. This can cause various problems, including changes to your skin and internal organs. A significant concern for people with systemic sclerosis is when the condition affects their lungs, leading to something called interstitial lung disease (ILD). This can cause scarring and make it harder to breathe, and it's a major reason why the illness can become very serious.

Currently, medicines like mycophenolate mofetil are used to treat lung problems when they are already more advanced. However, doctors are now wondering if starting treatment earlier, when signs of lung damage are just beginning to appear on scans but before breathing is significantly affected, could be more effective. This study is designed to explore this idea. It's a 'pilot' study, which means it's a smaller, early-stage investigation to help researchers figure out if a much larger study on this topic would be possible and useful.

The main goal of this particular study isn't to prove that the medicine works for sure, but rather to see if it's practical to conduct a bigger study later on. They'll be looking at things like how easy it is to find people for the study, whether people stick with the treatment, and if they can collect all the necessary information. Any early results about how people respond to the medicine will also help guide the planning for the future, larger study.

Key takeaways

This study explores early treatment for lung problems in systemic sclerosis.
It uses an existing medicine, mycophenolate mofetil, or a dummy pill.
The main aim is to see if a bigger study is possible, not to prove the medicine works yet.
Participants will be monitored for about 96 weeks.
It focuses on patients with early signs of lung damage but good lung function.

Who may be eligible?

To join this study, you need to be an adult aged 18 or older with systemic sclerosis and have been diagnosed with early interstitial lung disease (ILD) within the last three years. This ILD should show up on a special type of scan called an HRCT, but affect less than 20% of your lungs. Most importantly, your breathing tests must show that your lung function is still mostly normal (80% or better).

There are also some reasons why you might not be able to join. For example, if your lung disease is already rapidly progressing, or if you're currently taking certain medications that could interfere with the study's results (like high doses of steroids or other strong immune-suppressing drugs). Also, if you're pregnant, breastfeeding, or a woman of childbearing age not using reliable contraception, you wouldn't be able to participate. Certain blood test results, like very low white blood cell counts, platelet counts, or haemoglobin levels, would also prevent you from joining.

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?
Have you been diagnosed with systemic sclerosis?
Do you have early lung changes (ILD) on a scan, affecting less than 20% of your lungs?
Are your breathing tests showing mostly normal lung function (80% or better)?
Are you not currently pregnant or breastfeeding, and if you can get pregnant, are you using reliable contraception?
Are you not taking certain specific medications for your immune system or lung disease?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be assigned by chance (like flipping a coin) to receive either the study medicine, mycophenolate mofetil, or a 'placebo'. A placebo is a dummy pill that looks just like the real medicine but contains no active drug. This helps researchers compare the effects accurately. You'll take this assigned treatment for about 96 weeks (just under two years).

Throughout this time, you'll need to visit one of the three study centres in Canada regularly for check-ups and tests. These visits will involve blood tests, breathing tests, and possibly scans to monitor your lung health and overall condition. The researchers will be collecting information over a 12-month period to see how well people are recruited and how consistently they receive their treatment and complete all the required assessments.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit if you receive the active medicine and it helps slow down or prevent your lung condition from worsening earlier than current treatments. However, there's no guarantee of direct personal benefit, as this is a pilot study. As with all medicines, mycophenolate mofetil has potential side effects, and some people may experience them. These potential risks will be thoroughly explained to you, including any risks associated with the assessments and procedures. You'll be closely monitored by the study team, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Centre hospitalier de l'Université de Montréal (CHUM)
Verified postcode
Montreal, Canada· Recruiting
Jewish General Hospital - CIUSSS-COMTL
Verified postcode
Montreal, Canada· Not yet recruiting
Institut Universitaire de Cardiologie et Pneumologie de Québec
Verified postcode
Québec, Canada· Not yet recruiting

Common questions

What is systemic sclerosis?

It's an autoimmune disease where your body's immune system mistakenly attacks healthy tissues, which can affect your skin and internal organs like the lungs.

What is interstitial lung disease (ILD)?

ILD means your lungs are getting scarred, making it harder to breathe. For people with systemic sclerosis, it's a common and serious complication.

What is mycophenolate mofetil?

It's a medicine that works by calming down your immune system. It's already used to treat more advanced lung problems in systemic sclerosis.

What is a 'placebo' in a study?

A placebo is a fake pill that looks like the real medicine but doesn't have any active drug. It helps researchers compare how well the actual medicine works.

Why is this a 'pilot' study?

It's a smaller, early study to check if a larger study about the medicine's effectiveness for early lung problems is practical and worth doing.

How to find out more

Sabrina Hoa, MD MSc

sabrina.anh-tu.hoa.med@ssss.gouv.qc.ca 514-890-8000 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.