Efzofitimod for Scleroderma-Related Lung Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the drug efzofitimod to determine its effectiveness for lung disease caused by scleroderma, a condition that hardens the skin and organs. Researchers aim to discover if this treatment can improve lung health and potentially alleviate skin symptoms. Participants will receive either efzofitimod or a placebo through an IV, with the option to continue with the drug if desired. Individuals who have had scleroderma with lung involvement for less than seven years and are experiencing related lung issues might be suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Do I need to stop my current medications for the trial?
The trial requires that you have been on a stable dose of an immunosuppressant for at least 4 weeks before starting. You cannot be on more than one immunosuppressant or on high-dose corticosteroids. Some specific treatments are not allowed if taken in the past year.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that efzofitimod is generally safe and well-tolerated by patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). In studies, most participants did not experience serious side effects, indicating that efzofitimod did not cause major health issues during the trials. Most patients reported no severe reactions to the drug. The treatment has shown promise in improving symptoms while keeping side effects minimal. Overall, evidence suggests that efzofitimod is safe for human use, based on current data from these studies.12345
Why do researchers think this study treatment might be promising for scleroderma-related lung disease?
Unlike the standard treatments for scleroderma-related lung disease, which often include immunosuppressants and antifibrotics, efzofitimod represents a novel approach by targeting specific immune responses. Efzofitimod is unique because it is designed to modulate the immune system to reduce inflammation and fibrosis in the lungs. Researchers are particularly excited about its potential to offer a more targeted action with fewer side effects compared to conventional therapies. Additionally, its delivery as an intravenous infusion allows for precise dosing and monitoring, which could lead to better outcomes for patients.
What evidence suggests that efzofitimod might be an effective treatment for scleroderma-related lung disease?
Research has shown that efzofitimod may help treat lung disease related to systemic sclerosis (SSc-ILD). In an earlier study, three out of four patients using efzofitimod showed noticeable improvements in their skin symptoms, measured by a specific skin score. Early research also suggests that efzofitimod is generally safe and well tolerated. Although some studies did not achieve all their goals, efzofitimod still showed potential to improve several symptoms of the disease. Therefore, this treatment seems to positively affect both lung and skin issues linked to SSc-ILD.12467
Who Is on the Research Team?
Lisa Carey
Principal Investigator
aTyr Pharma, Inc.
Are You a Good Fit for This Trial?
This trial is for patients with a specific lung condition called SSc-ILD, linked to systemic sclerosis. Participants must have been diagnosed within the last 4 years and show significant lung involvement. They should be on a stable dose of mycophenolate and can have limited or diffuse skin disease. Smokers, those with severe lung function decline, other rheumatic diseases, or recent use of certain medications are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either efzofitimod or placebo every 4 weeks up to and including Week 20
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of treatment with 450 mg efzofitimod every 4 weeks for 6 doses
What Are the Treatments Tested in This Trial?
Interventions
- Efzofitimod
Find a Clinic Near You
Who Is Running the Clinical Trial?
aTyr Pharma, Inc.
Lead Sponsor