Efzofitimod for Sarcoidosis

Not currently recruiting at 95 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests efzofitimod, a new treatment for pulmonary sarcoidosis, a condition that causes lung inflammation. Researchers aim to determine if efzofitimod reduces symptoms and is safe compared to a placebo (a substance with no active medicine). Participants will receive either a low or high dose of efzofitimod or a placebo. Ideal participants have had pulmonary sarcoidosis for at least six months and are already on stable oral corticosteroid treatment. As a Phase 3 trial, this is the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

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.

Is there any evidence suggesting that efzofitimod is likely to be safe for humans?

Research has shown that efzofitimod is generally safe and well tolerated. In a previous study, participants who received higher doses of efzofitimod experienced better outcomes compared to those who took a placebo, with no major safety concerns reported. This indicates that the treatment does not cause significant harmful effects. Some participants experienced side effects, but these were not serious enough to discontinue the treatment. Overall, the evidence suggests that efzofitimod could be a safe option for treating pulmonary sarcoidosis, especially if other treatments are ineffective. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for sarcoidosis?

Unlike the standard treatments for sarcoidosis, which typically involve corticosteroids and immunosuppressants, Efzofitimod offers a novel approach. Efzofitimod works by modulating the immune response, specifically targeting the activity of certain cytokines that play a role in inflammation. This targeted mechanism could potentially reduce side effects associated with broader immune suppression seen in current therapies. Researchers are excited because Efzofitimod might provide more effective control of sarcoidosis symptoms with fewer adverse effects, improving the quality of life for patients.

What evidence suggests that efzofitimod might be an effective treatment for sarcoidosis?

Research has shown that efzofitimod may help treat pulmonary sarcoidosis. In earlier studies, patients taking 5 mg/kg of efzofitimod experienced noticeable improvements in lung health and overall well-being compared to those on a placebo. The King's Sarcoidosis Questionnaire, which assesses lung symptoms and general health, measured this improvement. Patients on higher doses of efzofitimod also reported better outcomes. Although efzofitimod did not achieve all main goals in every study, these results suggest it might improve symptoms and reduce the need for steroids in people with sarcoidosis. ^{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?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1.

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 ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10258437/

Efzofitimod for the Treatment of Pulmonary Sarcoidosis

Patient-reported outcomes improved in the higher-dose arms and positive trends for other end points were found. Interpretation: Efzofitimod may be useful for ...

3.

rheumatologyadvisor.com

rheumatologyadvisor.com/news/efzofitimod-misses-primary-endpoint-in-pulmonary-sarcoidosis-trial/

Efzofitimod Misses Primary Endpoint in Pulmonary ...

Results also showed significant improvement in King's Sarcoidosis Questionnaire (KSQ)-Lung score with efzofitimod 5mg/kg vs placebo (change from ...

4.

investors.atyrpharma.com

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

News Releases

Improvement in King's Sarcoidosis Questionnaire-General Health score at week 48 observed in the 5.0 mg/kg efzofitimod treatment group vs placebo ...

5.

publications.ersnet.org

publications.ersnet.org/content/erjor/11/1/00536-2024

Therapeutic doses of efzofitimod demonstrate efficacy in ...

In the phase 1b/2a trial, efzofitimod was safe and well tolerated, and treatment was associated with statistically significant dose-dependent improvement in ...

6.

healio.com

healio.com/news/pulmonology/20251016/more-patients-with-sarcoidosis-are-steroidfree-with-48week-efzofitimod-vs-placebo

Efzofitimod helps patients with sarcoidosis become steroid- ...

A higher proportion of patients with pulmonary sarcoidosis were steroid-free after 48 weeks of treatment with efzofitimod vs. placebo, according ...

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05415137

Efficacy and Safety of Intravenous Efzofitimod in Patients ...

This is a multicenter, randomized, double-blind, placebo-controlled, study comparing the efficacy and safety of intravenous (IV) efzofitimod 3 mg/kg and 5 mg/ ...