What side effects are associated with this type of intervention?

Common treatments for sarcoidosis, such as glucocorticoids, methotrexate, and TNF antagonists, have various side effects. Glucocorticoids can cause weight gain, osteoporosis, hypertension, and diabetes. Methotrexate may lead to liver toxicity, bone marrow suppression, and gastrointestinal issues. TNF antagonists, like infliximab and adalimumab, can increase the risk of infections, including tuberculosis, and may cause injection site reactions and autoimmune conditions. These side effects highlight the need for careful monitoring and consideration of glucocorticoid-sparing agents like efzofitimod.

What prior FDA approvals exist?

The provided context does not mention specific FDA approvals for the treatment of Sarcoidosis. However, it does describe a study on the efficacy and safety of intravenous Efzofitimod, an immune-modulating agent, in patients with pulmonary Sarcoidosis. This suggests ongoing research into immune modulation as a therapeutic approach for Sarcoidosis. For a comprehensive understanding of FDA-approved treatments, consulting additional sources or discussing with a healthcare provider is recommended.

What other types of research exist?

Promising, novel alternatives to Efzofitimod for sarcoidosis patients in 2024 may include other immune modulation therapies such as anti-PD-1 or anti-PD-L1 therapies, which have shown efficacy in other inflammatory conditions. Additionally, treatments like JAK inhibitors, which are used in myelofibrosis and other autoimmune diseases, could be considered. It is essential to consult with a healthcare provider to evaluate the suitability and potential benefits of these alternatives based on individual patient conditions.

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Immunomodulator

Efzofitimod for Sarcoidosis

Phase 3

Waitlist Available

Research Sponsored by aTyr Pharma, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Evidence of symptomatic pulmonary sarcoidosis with specific dyspnea scale grade and KSQ-Lung score criteria

Patients receiving treatment with OCS for at least 3 months prior to Day 1 with specified dose range

Must not have

Treatment with more than 1 immunosuppressant therapy

Active tuberculosis or undergoing treatment for tuberculosis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for Efzofitimod aren't listed here, common side effects from similar treatments may include infusion reactions, fatigue, possible immune system changes leading to increased risk of infection or inflammation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have breathing problems due to sarcoidosis.

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

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I have been diagnosed with pulmonary sarcoidosis for over 6 months, confirmed by tissue sample and lung scans.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been treated with more than one immunosuppressant.

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I do not have active tuberculosis nor am I being treated for it.

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

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I am currently on or have been treated with drugs that affect my immune system.

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My skin or eye condition could get worse.

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I have experienced symptoms like those of Addison's disease when trying to reduce corticosteroids.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2020 Phase 2 trial • 36 Patients • NCT04412668

20%

Tachycardia

10%

Acute respiratory distress syndrome

10%

Hypoxia

10%

Pulmonary hypertension

10%

Respiratory failure

10%

Septic shock

10%

Urosepsis

10%

Acute myocardial infarction

10%

Carbon dioxide increased

10%

Hepatitis B virus test positive

10%

N-terminal prohormone brain natriuretic peptide increased

10%

Pneumonia streptococcal

10%

Pruritus

10%

Dry eye

10%

Contusion

10%

Acute kidney injury

10%

Pneumomediastinum

10%

Pneumonitis

10%

Pulmonary embolism

10%

Hypotension

10%

Epistaxis

10%

Nasal dryness

10%

Oropharyngeal pain

10%

Pneumothorax

10%

Pulmonary fibrosis

10%

Blood urea increased

10%

Hypercoagulation

10%

Blood potassium decreased

10%

Leukocytosis

10%

Dyspnoea exertional

100%

80%

60%

40%

20%

Study treatment Arm

Efzofitimod 1 mg/kg

Efzofitimod 3 mg/kg

Placebo

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Efzofitimod 5 mg/kgExperimental Treatment1 Intervention

Group II: Efzofitimod 3 mg/kgExperimental Treatment1 Intervention

Group III: PlaceboPlacebo Group1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Efzofitimod 3 mg/kg

2020

Completed Phase 2

~40

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for sarcoidosis include glucocorticoids, immunosuppressive agents, and biologics that modulate the immune system. Glucocorticoids, such as prednisone, reduce inflammation by suppressing the immune response, which helps prevent tissue damage. Immunosuppressive agents like methotrexate and mycophenolate mofetil further dampen the immune system to reduce inflammation and allow for lower doses of glucocorticoids, minimizing their side effects. Biologics, including TNF inhibitors like infliximab and adalimumab, target specific components of the immune system to reduce inflammation more precisely. Efzofitimod, an immune modulation therapy, aims to modulate the immune response to reduce inflammation and fibrosis in the lungs. These treatments are crucial for sarcoidosis patients as they help manage symptoms, prevent organ damage, and improve quality of life by controlling the overactive immune response characteristic of the disease.