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

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called efzofitimod to see if it can help people with a lung disease linked to systemic sclerosis. The goal is to find out if the drug can improve their breathing and skin health.

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.

How is the drug Efzofitimod different from other treatments for scleroderma-related lung disease?

Efzofitimod is unique because it is a novel treatment option for scleroderma-related lung disease, whereas existing treatments like cyclophosphamide and mycophenolate mofetil have shown limited effectiveness and tolerability issues. Unlike traditional immunosuppressants, Efzofitimod may offer a new mechanism of action, potentially improving outcomes for patients with this challenging condition. ^{1 2 3 4 5}

Research Team

Lisa Carey

Principal Investigator

aTyr Pharma, Inc.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Learnings from clinical trials in patients with connective tissue disease-associated interstitial lung disease. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Treating CTDs related fibrotic ILDs by immunosuppressants: "facts and faults". [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Effect of mycophenolate mofetil on pulmonary function in scleroderma-associated interstitial lung disease. [2022]

4. Italy pubmed.ncbi.nlm.nih.gov

Clinical experience with pirfenidone in five patients with scleroderma-related interstitial lung disease. [2016]

5. England pubmed.ncbi.nlm.nih.gov

Immunotherapy of systemic sclerosis. [2022]