Efzofitimod for Sarcoidosis

Recruiting at 90 trial locations

Overseen By Melissa Meyer

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: aTyr Pharma, Inc.

Must be taking: Oral corticosteroids

Must not be taking: Biological immunomodulators

Disqualifiers: Pulmonary fibrosis, Hypertension, Cardiac sarcoidosis, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 48 weeks

268 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new treatment to help adults with a lung disease called pulmonary sarcoidosis. These patients are already on other treatments but may need more help. The new treatment aims to improve their condition.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must be on a stable dose of oral corticosteroids for at least 3 months before starting the trial. You cannot be on more than one immunosuppressant or any biological immunomodulators.

What data supports the effectiveness of the drug Efzofitimod for treating sarcoidosis?

Efzofitimod has shown promise in treating sarcoidosis by reducing inflammation in animal models and improving quality of life in patients with pulmonary sarcoidosis. Studies found that as the exposure to Efzofitimod increased, patients needed fewer steroids and showed improved lung function and symptoms. ^{1 2 3 4 5}

What safety data exists for efzofitimod in humans?

Efzofitimod has been tested in a small number of people with sarcoidosis and healthy volunteers, showing a trend towards improved quality of life and stable lung function without specific safety concerns reported. However, detailed safety data is limited, and a larger Phase 3 trial is underway to better understand its safety profile. ^{3 4 6 7 8}

How is the drug Efzofitimod unique in treating sarcoidosis?

Efzofitimod is unique because it is a first-in-class biologic that targets a specific protein called neuropilin-2 (NRP2) to reduce inflammation in sarcoidosis, unlike traditional treatments that often rely on corticosteroids. This novel approach may help improve quality of life and reduce the need for steroids, which can have significant side effects. ^{3 4 7 9 10}

Research Team

Lisa Carey

Principal Investigator

aTyr Pharma, Inc.

Eligibility Criteria

Adults with confirmed pulmonary sarcoidosis, on stable oral corticosteroids for at least 3 months, and experiencing symptoms like breathlessness. They must weigh between 40-160 kg and have a history of lung involvement in sarcoidosis. Excluded are those on multiple immunosuppressants, biologic immunomodulators, with significant lung fibrosis or other severe forms of sarcoidosis.

Inclusion Criteria

I have breathing problems due to sarcoidosis.

I have been on oral corticosteroids for at least 3 months.

Body weight between 40 kg and 160 kg

Exclusion Criteria

I have been treated with more than one immunosuppressant.

I have been treated for sarcoidosis affecting my heart, brain, or kidneys in the last 2 years.

I have been diagnosed with anti-synthetase syndrome or tested positive for Jo-1.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravenous efzofitimod 3 mg/kg or 5 mg/kg, or placebo for 48 weeks

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

Treatment Details

Interventions

• Efzofitimod

Trial Overview The trial is testing the effectiveness and safety of two doses (3 mg/kg and 5 mg/kg) of Efzofitimod compared to a placebo in treating pulmonary sarcoidosis over 48 weeks. Participants will be randomly assigned to receive either the drug or placebo intravenously.

Participant Groups

Group I: Efzofitimod 5 mg/kg Experimental Treatment 1 Intervention

Group II: Efzofitimod 3 mg/kg Experimental Treatment 1 Intervention

Group III: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

aTyr Pharma, Inc.

Lead Sponsor

Trials

10

Recruited

480+

Kyorin Pharmaceutical Co.,Ltd

Industry Sponsor

Trials

9

Recruited

9,600+

Findings from Research

Efzofitimod, a novel biologic targeting neuropilin-2 (NRP2), has shown promise in reducing granulomatous inflammation in sarcoidosis, as demonstrated in preclinical studies with an animal model.

In a dose-escalating trial involving patients with chronic symptomatic pulmonary sarcoidosis, efzofitimod improved quality of life and showed a trend towards reduced use of glucocorticoids while maintaining or improving pulmonary function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efzofitimod: a novel anti-inflammatory agent for sarcoidosis. Baughman, RP., Niranjan, V., Walker, G., et al.[2023]

In a study involving 25 participants with pulmonary sarcoidosis, increased exposure to efzofitimod was associated with a reduction in the mean daily dose of oral corticosteroids and improvements in lung function and quality of life as measured by the ppFVC and KSQ-Lung score.

The analysis suggests a positive exposure-response relationship, indicating that efzofitimod may be an effective treatment option for pulmonary sarcoidosis, although the small sample size means these findings are preliminary and require further validation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Exposure-response analyses of efzofitimod in patients with pulmonary sarcoidosis. Walker, G., Adams, R., Guy, L., et al.[2023]

In a study of 28 patients with chronic steroid-resistant sarcoidosis treated with infliximab over a period from 2003 to 2010, 88% of patients benefited from long-term treatment, showing significant clinical improvement, especially in those with extrapulmonary disease.

The treatment was found to be safe, with only one patient (6%) experiencing adverse effects that required temporary withdrawal, indicating a favorable safety profile for long-term infliximab use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term treatment with infliximab in patients with sarcoidosis. Hostettler, KE., Studler, U., Tamm, M., et al.[2015]

Sarcoidosis can present in various ways, with some patients experiencing spontaneous remission while others face severe, progressive disease that affects their survival and quality of life.

Four high-risk phenotypes of sarcoidosis—treatment-refractory pulmonary disease, cardiac sarcoidosis, neurosarcoidosis, and multiorgan sarcoidosis—have been identified as critical areas for research, highlighting the need for better understanding of their diagnosis, biomarkers, and treatment strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Presentation and Treatment of High-Risk Sarcoidosis. Perlman, DM., Sudheendra, MT., Furuya, Y., et al.[2022]

References

1. Italy pubmed.ncbi.nlm.nih.gov

Factor analysis of sarcoidosis phenotypes at two referral centers in Brazil. [2016]

2. Denmark pubmed.ncbi.nlm.nih.gov

[Pulmonary sarcoidosis]. [2006]

3. Italy pubmed.ncbi.nlm.nih.gov

Efzofitimod: a novel anti-inflammatory agent for sarcoidosis. [2023]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Exposure-response analyses of efzofitimod in patients with pulmonary sarcoidosis. [2023]

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

Efzofitimod for the Treatment of Pulmonary Sarcoidosis. [2023]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Long-term treatment with infliximab in patients with sarcoidosis. [2015]

7. England pubmed.ncbi.nlm.nih.gov

Mycophenolate mofetil as an alternative treatment in sarcoidosis. [2020]

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

Clinical Presentation and Treatment of High-Risk Sarcoidosis. [2022]

9. England pubmed.ncbi.nlm.nih.gov

Mycophenolate mofetil may serve as a steroid-sparing agent for sarcoidosis. [2019]

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

Pentoxifylline in treatment of sarcoidosis. [2013]