Efzofitimod for Scleroderma-Related Lung Disease

Recruiting at 12 trial locations

Overseen By aTyr Pharma Clinical Research

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: aTyr Pharma, Inc.

Must be taking: Mycophenolate, Immunosuppressants

Must not be taking: Corticosteroids, Rituximab

Disqualifiers: Pulmonary hypertension, Heavy smoker, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 5

Duration: 20 weeks

25 Participants Needed

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. ^{1 2 3 4 5}

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. ^{1 2 4 6 7}

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

I have been diagnosed with systemic sclerosis according to the 2013 criteria.

My condition is either limited or diffuse systemic sclerosis.

My scleroderma symptoms started less than 4 years ago, not counting Raynaud's.

Exclusion Criteria

I have an autoimmune disease that is not systemic sclerosis.

My lung function is significantly reduced.

I have high blood pressure in the lungs and am on IV medication or have signs of heart failure.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Treatment

Participants receive either efzofitimod or placebo every 4 weeks up to and including Week 20

20 weeks

5 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment with 450 mg efzofitimod every 4 weeks for 6 doses

24 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efzofitimod

Trial Overview

The study tests efzofitimod at two different doses (450 mg and 270 mg) against a placebo in people with SSc-ILD. It's double-blind meaning neither doctors nor participants know who gets what treatment. The main goal is to see if it improves lung disease and skin changes over time.

How Is the Trial Designed?

Group I: efzofitimod 450 mg Experimental Treatment 1 Intervention

Administered IV infusion

Group II: efzofitimod 270 mg Experimental Treatment 1 Intervention

Administered IV infusion

Group III: Placebo Placebo Group 1 Intervention

Administered IV infusion

Who Is Running the Clinical Trial?

aTyr Pharma, Inc.

Lead Sponsor

Trials

10

Recruited

480+

Published Research Related to This Trial

Cyclophosphamide is the primary treatment for progressive skin involvement and active interstitial lung disease in scleroderma, while mycophenolate mofetil shows promise as an alternative.

Newer therapies targeting T-cells (like sirolimus and alefacept) and B-cells (like rituximab) have shown encouraging results in small studies, but the safety and efficacy of antifibrotic treatments like imatinib still need further confirmation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunotherapy of systemic sclerosis. Manno, R., Boin, F.[2022]

Citations

1.

investors.atyrpharma.com

investors.atyrpharma.com/news-releases/news-release-details/atyr-pharma-announces-findings-interim-analysis-ongoing-phase-2

aTyr Pharma Announces Findings from Interim Analysis of ...

Three out of four efzofitimod-treated diffuse SSc-ILD patients showed clinically important improvement based on the modified Rodnan Skin Score (mRSS) ...

2.

investors.atyrpharma.com

investors.atyrpharma.com/news-releases/news-release-details/atyr-pharma-announces-topline-results-phase-3-efzo-fittm-study

aTyr Pharma Announces Topline Results from Phase 3 ...

52.6% of patients treated with 5.0 mg/kg efzofitimod achieved complete steroid withdrawal at week 48 vs 40.2% on placebo (p=0.0919). Clinical ...

3.

scleroderma.org.au

scleroderma.org.au/news/efzofitimod-found-safe-well-tolerated-in-study-of-ssc-ild

Efzofitimod found safe, well tolerated in study of SSc-ILD

Recent interim results from the first eight participants showed efzofitimod was generally safe and well tolerated, with no treatment-related ...

4.

hcplive.com

hcplive.com/view/efzofitimod-shows-promising-early-efficacy-in-systemic-sclerosis-related-interstitial-lung-disease

Efzofitimod Shows Promising Early Efficacy in Systemic ...

Efzofitimod has demonstrated early signs of clinical efficacy in people with diffuse and limited systemic sclerosis-related interstitial lung disease (SSc-ILD).

5.

investors.atyrpharma.com

investors.atyrpharma.com/news-releases/news-release-details/atyr-pharma-presents-additional-findings-phase-3-efzo-fittm

News Releases

Despite missing the primary endpoint of the study, efzofitimod has clearly demonstrated the potential to durably improve multiple disease- ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05892614

NCT05892614 | Study to Evaluate the Efficacy, Safety, and ...

This is a 2-Part study with Part A, a double-blind, randomized, placebo-controlled, PoC study to evaluate the efficacy, safety, and tolerability of efzofitimod ...

7.

investors.atyrpharma.com

investors.atyrpharma.com/node/16496/pdf

aTyr Pharma Announces First Quarter 2025 Results and ...

Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group ...