RecruitingPHASE3INTERVENTIONAL

Cardiac Sarcoidosis Randomized Trial

This research study is looking into new ways to treat Cardiac Sarcoidosis, a condition where tiny clumps of cells grow in the heart. The study compares a standard treatment, which involves a steroid medication called Prednisone (or Prednisolone), with a new approach. The new approach uses a lower dose of Prednisone/Prednisolone alongside another medication called Methotrexate. Researchers want to see if this combination treatment works just as well as the standard high-dose steroid, but with the added benefits of fewer side effects and a better quality of life for patients. Participants will be randomly assigned to one of the two treatment groups and closely monitored over six months.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Ottawa Heart Institute Research Corporation

Enrolment target

194

Start

15 Jan 2019

Estimated completion

01 Dec 2026

Cardiac Sarcoidosis Sarcoidosis

What is this study about?

This research study, called a clinical trial, is focusing on a condition called Cardiac Sarcoidosis. This is when a disease called sarcoidosis affects the heart, causing small clumps of cells (granulomas) to form. These clumps can interfere with how your heart works.

Currently, the main treatment often involves a type of steroid medication called Prednisone or Prednisolone. While effective, higher doses of these steroids can sometimes cause unwanted side effects or impact a person's quality of life. This study is exploring if a different treatment combination could be just as good at treating the condition, but with fewer downsides.

The main idea is to compare the standard treatment – a regular dose of Prednisone/Prednisolone – with a new approach. The new approach uses a lower dose of Prednisone/Prednisolone combined with another medication called Methotrexate. Researchers hope that this combination might be equally effective in controlling the sarcoidosis in the heart, but lead to fewer side effects and help people feel better overall. Participants will be carefully monitored throughout the study to see how the treatments compare.

Key takeaways

Compares two treatment approaches for Cardiac Sarcoidosis.
Aims for effective treatment with fewer side effects and better quality of life.
Involves regular medical check-ups and heart scans.
Participation lasts for six months of treatment and monitoring.
You have the right to withdraw from the study at any time.

Who may be eligible?

To be considered for this study, your doctor would need to confirm that your Cardiac Sarcoidosis is currently active and hasn't yet been treated. You would also need to show specific signs of heart problems related to sarcoidosis, such as issues with your heart's electrical system, an irregular heartbeat, or your heart muscle not pumping as strongly as it should.

It's also important that your symptoms can't be explained by another medical condition. You would need to have had specific heart scans (like an FDG-PET scan) within the last six months that suggest active sarcoidosis in your heart. Additionally, you would need to have either a confirmed biopsy of sarcoidosis (from your heart or another part of your body) OR evidence of sarcoidosis in your lungs or lymph nodes on a CT scan.

There are also reasons why you might not be able to join. For instance, if you've recently received treatment for sarcoidosis, if you're already taking Methotrexate or Prednisone for another health condition, or if you're unable to take these medications due to other health issues. This study is not for pregnant or breastfeeding women, or women of childbearing age who are not willing to use effective contraception.

Quick self-check

Do you have confirmed active Cardiac Sarcoidosis that hasn't been treated yet?
Do you have specific heart problems linked to sarcoidosis (e.g., heart rhythm issues, weak heart muscle)?
Have you had recent heart scans (like an FDG-PET) showing active sarcoidosis in your heart?
Are you able to take Prednisone/Prednisolone and Methotrexate?
Are you 18 years old or older?
Are you not pregnant or breastfeeding, and if female, willing to use effective contraception?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you would first have a series of initial tests. These include blood tests, special heart scans (like FDG-PET with perfusion imaging, ECG, and echocardiogram), and possibly an MRI scan of your heart. You would also complete some questionnaires about your quality of life.

After these initial checks, you would be randomly assigned to one of two treatment groups for six months. One group will receive a standard daily dose of Prednisone. The other group will receive a weekly dose of Methotrexate (taken as a pill, injection under the skin, or into a muscle) along with a daily folic acid supplement (to help reduce Methotrexate side effects), and a gradually decreasing dose of Prednisone over three months.

Throughout the six months, you would have several follow-up appointments: at 4 weeks, 8 weeks (only if you're in the Methotrexate group), and 12 weeks, with a final visit at 6 months. At each visit, you'll have blood tests for safety, and your doctor will ask about any side effects you might be experiencing. You'll complete the quality of life questionnaires again at the 12-week mark. The total duration of active treatment and follow-up in the study is six months.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving close medical monitoring for your Cardiac Sarcoidosis and potentially a treatment that leads to fewer side effects and a better quality of life. However, like all medications, Prednisone, Prednisolone, and Methotrexate can have side effects, and some people may not respond well to specific treatments. Detailed information about these potential risks will be discussed with you before you decide to join. It is very important to remember that joining a clinical trial is always voluntary, and you have the right to withdraw from the study at any time without it affecting your usual medical care.

Locations (30)

Yale-New Haven Hospital
New Haven, United States· Recruiting
Tufts Medical Center
Boston, United States· Recruiting
University of Michigan-Michigan Medicine Cardiovascular Center
Ann Arbor, United States· Recruiting
University of Minnesota
Minneota, United States· Not yet recruiting
Montefiore Medical Center
New York, United States· Recruiting
The Ohio State University Wexner Medical Center
Columbus, United States· Recruiting
Allegheny General Hospital
Pittsburgh, United States· Recruiting
University of Utah
Salt Lake City, United States· Recruiting
Virginia Commonwealth University
Richmond, United States· Recruiting
Libin Cardiovascular Institute of Alberta
Calgary, Canada· Recruiting
St. Paul's Hospital
Vancouver, Canada· Recruiting
Eastern Health Health Sciences Centre
St. John's, Canada· Recruiting

+18 more sites — see the official record for the full list.

Common questions

What is Cardiac Sarcoidosis?

It's a condition where inflammatory cells form tiny clumps (granulomas) in your heart, which can affect its normal function.

What are Prednisone and Methotrexate?

Prednisone (or Prednisolone) is a steroid medication that reduces inflammation. Methotrexate is a medication that also helps control your immune system.

Why is this study important?

This study aims to find a treatment for Cardiac Sarcoidosis that is just as effective as current options but causes fewer side effects and improves your quality of life.

Will I know which treatment I'm receiving?

No, this is a 'randomised' study, meaning you'll be assigned to one treatment group by chance, and neither you nor your doctor will know which treatment you are on (this is called 'blinding').

Can I leave the study at any time?

Yes, your participation is completely voluntary, and you can withdraw from the study at any point without it affecting your ongoing medical care.

How to find out more

David H Birnie, MD

dbirnie@ottawaheart.ca 613-696-7269 Official trial record

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