Active not recruitingPHASE3INTERVENTIONAL

Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

This research study is looking into a new medication called efzofitimod for adults who have sarcoidosis affecting their lungs. We want to find out if this drug is safe and effective in improving symptoms, especially compared to a placebo (a dummy treatment). Participants will be randomly assigned to receive one of two doses of efzofitimod or the placebo, given directly into a vein. The study will last for 48 weeks and is for people who are already on a stable dose of steroids for their lung sarcoidosis, with or without other immune-suppressing medicines. The aim is to see if efzofitimod can help manage the condition and potentially reduce the need for long-term steroid use.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

aTyr Pharma, Inc.

Enrolment target

268

Start

15 Sep 2022

Estimated completion

31 Aug 2025

Pulmonary Sarcoidosis

What is this study about?

This study is investigating a new treatment called efzofitimod for people in the UK who have pulmonary sarcoidosis – a condition where tiny clumps of cells, called granulomas, grow in the lungs. These granulomas can cause inflammation and lead to symptoms like shortness of breath and fatigue. The main goal of this research is to see if efzofitimod can help manage lung sarcoidosis effectively and safely.

Researchers are comparing two different doses of efzofitimod given directly into a vein, to a 'placebo'. A placebo looks exactly like the real treatment but contains no active medicine. This helps researchers understand if any improvements are due to the new drug or other factors. Participants won't know if they are receiving efzofitimod or the placebo, and neither will their doctor – this is called a 'double-blind' study and helps make the results as fair and unbiased as possible.

The study will follow participants for 48 weeks (just under a year) to carefully observe how they respond to the treatment. It's for adults who have already been diagnosed with lung sarcoidosis and are currently taking a stable dose of steroid medication, which they might also be taking with other medicines that affect the immune system. The hope is that efzofitimod could offer a new way to help people with lung sarcoidosis, potentially improving their symptoms and their quality of life.

Key takeaways

Tests a new drug (efzofitimod) for lung sarcoidosis.
Compares two doses of the drug to a dummy treatment (placebo).
Study lasts for 48 weeks, with regular hospital visits.
For adults already taking steroids for their lung sarcoidosis.
Participation is voluntary; you can withdraw at any time.
Aims to find out if efzofitimod is safe and effective.

Who may be eligible?

To join this study, you need to be an adult (18 years or older) with a confirmed diagnosis of sarcoidosis in your lungs for at least six months. Your doctor must have confirmed this with a tissue sample, and you need to have signs of lung involvement seen on scans.

You should also be experiencing symptoms related to your lung sarcoidosis. Specifically, you'd need to have at least a mild level of breathlessness and a low score on a special questionnaire that measures how sarcoidosis affects your daily life. You also need to be taking a stable dose of oral steroids (between 7.5 and 25 mg daily) for at least three months, and your weight should be between 40 kg and 160 kg.

There are also some reasons why you might not be able to join. For example, if you're taking more than one immune-suppressing drug, or certain other types of immune treatments. You also wouldn't be able to participate if you have very severe scarring in your lungs, other significant health problems related to sarcoidosis (like in your heart or brain), or if you are a heavy smoker. The research team will review all your medical information carefully to see if this study is suitable for you.

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 or older?
Do you have a confirmed diagnosis of lung sarcoidosis (for at least 6 months)?
Are you currently taking oral steroids (7.5-25 mg/day) for at least 3 months?
Do you have symptoms like breathlessness from your lung sarcoidosis?
Are you not taking more than one other immune suppressant, or certain other specific immune drugs?
Do you weigh between 40 kg and 160 kg?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be assigned by chance (like flipping a coin) to one of three groups: one group will receive 3 mg/kg of efzofitimod, another will receive 5 mg/kg of efzofitimod, and the third group will receive a placebo. Both the efzofitimod and the placebo will be given directly into your vein (intravenously).

The study will last for 48 weeks, which is about 11 months. During this time, you'll have regular hospital or clinic visits where you'll receive your study medication and undergo various tests and assessments. These might include blood tests, lung function tests, questionnaires about your symptoms and quality of life, and possibly imaging scans like CT scans of your lungs. The research team will monitor your health closely throughout the study to ensure your safety and track any changes in your condition. After the 48 weeks, there will likely be a follow-up period to ensure your well-being.

Potential risks and benefits

Participating in research studies has both potential benefits and risks. You might find that taking part gives you access to a new treatment for your sarcoidosis before it's widely available, and you'll receive careful monitoring of your health throughout the study. However, there's no guarantee the treatment will help, and you might receive the placebo. Potential risks could include side effects from the study medication, though these will be closely monitored. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (91)

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

aTyr Investigative Site
Verified postcode
Birmingham, United States
aTyr Investigative Site
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Phoenix, United States
aTyr Investigative Site
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Los Angeles, United States
aTyr Investigative Site
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Sacramento, United States
aTyr Investigative Site
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Denver, United States
aTyr Investigative Site
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Washington D.C., United States
aTyr Investigative Site
Verified postcode
Gainesville, United States
aTyr Investigative Site
Verified postcode
Tampa, United States
aTyr Investigative Site
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Weston, United States
aTyr Investigative Site
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Atlanta, United States
aTyr Investigative Site
Verified postcode
Chicago, United States
aTyr Investigative Site
Verified postcode
Chicago, United States

Common questions

What is pulmonary sarcoidosis?

It's a condition where tiny clumps of cells, called granulomas, grow in your lungs, causing inflammation and affecting how your lungs work.

What is a placebo?

A placebo is a 'dummy' treatment that looks just like the real drug but contains no active medicine. It helps researchers understand if the new drug is truly effective.

Will I know if I'm getting the real drug or the placebo?

No, this is a 'double-blind' study, meaning neither you nor your doctors will know whether you are receiving efzofitimod or the placebo to ensure unbiased results.

How long will the study last?

The treatment part of the study will last for 48 weeks, which is almost a year. There might be follow-up appointments after this.

Can I stop participating in the study at any time?

Yes, you have the right to leave the study at any point, and this will not affect your regular medical care.