Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF INTRAVENOUS EFZOFITIMOD IN PATIENTS WITH PULMONARY SARCOIDOSIS

This research study is looking at a new medicine called Efzofitimod for individuals who have pulmonary sarcoidosis – a condition that mainly affects the lungs. The main goal is to find out if this new medicine can help people reduce their daily dose of steroid medication over 48 weeks. We also want to understand if Efzofitimod is safe and if it can help improve how well people breathe and how they feel overall. Participants will receive either the new medicine or a dummy medicine, without knowing which one, and will be carefully monitored throughout the study.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

aTYR Pharma Inc.

Enrolment target

101

Start

10 Jul 2023

pulmonary sarcoidosis

What is this study about?

This study is a very important step in finding new treatments for pulmonary sarcoidosis, a condition where tiny clumps of inflammatory cells, called granulomas, can form in your lungs. These granulomas can cause various symptoms and sometimes lead to breathing difficulties. Often, powerful steroid medications are used to help control this inflammation, but these can have side effects when used for a long time.

That's why this study is looking at a new investigational medicine called Efzofitimod. The main idea is to see if Efzofitimod can help people with pulmonary sarcoidosis manage their condition, specifically if it can reduce the amount of steroid medication they need to take each day. Reducing steroids could potentially lead to fewer side effects and a better quality of life.

Throughout the 48-week study, researchers will be carefully observing participants. They will check how much steroid medication people are taking, measure their lung function (how well they breathe), and ask questions about how they are feeling. This will help them understand if Efzofitimod is an effective and safe treatment option for this condition.

Key takeaways

This study is testing a new medicine (Efzofitimod) for lung sarcoidosis.
The main goal is to see if it can help reduce the daily steroid dose.
It also checks how safe the new medicine is and if it improves lung health.
Participants will receive either the new medicine or a dummy pill.
The study lasts for 48 weeks with regular check-ups.
It's for adults aged 18 and over, both men and women.

Who may be eligible?

To be able to take part in this study, you would need to be at least 18 years old. There is no upper age limit, meaning older adults can also participate.

Both men and women are welcome to join this study, as the researchers want to understand how the new medicine works in all adults with pulmonary sarcoidosis.

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 pulmonary sarcoidosis (sarcoidosis affecting your lungs)?
Are you willing to attend regular clinic visits for about a year?
Are you able to take study medication as instructed?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be involved for about 48 weeks, which is nearly a year. During this time, you would have regular visits to the clinic for check-ups. You would receive either the new medicine, Efzofitimod, or a dummy medicine (a placebo). Neither you nor your study doctor would know which one you are getting, to make sure the results are fair. At these visits, doctors would monitor your health, check your lung function, and carefully track any changes in your steroid medication dose. There would also be follow-up appointments after the main treatment period.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit could be that you receive a new medicine that might help improve your condition, possibly reducing your need for steroids. However, there's also a chance you might receive the dummy medicine, or that the new medicine doesn't help. Potential risks could include side effects from the new medicine, or from the procedures involved in the study (like blood tests). All known risks will be fully explained to you before you agree to participate. You are completely free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France
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Unverified
Netherlands
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Unverified
Spain
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Unverified
Italy
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Unverified
Germany

Common questions

What is pulmonary sarcoidosis?

Pulmonary sarcoidosis is a lung condition where tiny clumps of cells (called granulomas) form, causing inflammation and sometimes breathing problems.

What are 'steroids' in this context?

In this study, 'steroids' refer to corticosteroid medications, which are commonly used to reduce inflammation in conditions like sarcoidosis.

What is a 'placebo'?

A placebo is a dummy treatment that looks just like the real medicine but contains no active drug. It helps researchers compare the effects of the new medicine accurately.

Why is it 'double-blind'?

Double-blind means neither you nor your study doctor will know if you're receiving the new medicine or the placebo. This helps prevent bias and ensures the results are as objective as possible.

How long will the study last?

The main part of the study where you receive treatment will last for 48 weeks, which is almost a year. There might also be follow-up appointments after that.