What side effects are associated with this type of intervention?

Common treatments for sarcoidosis, such as glucocorticoids and other immunosuppressive agents, can have a range of side effects. Glucocorticoids, like prednisone, may cause weight gain, hypertension, diabetes, osteoporosis, and increased risk of infections. Other immunosuppressive agents, such as methotrexate and azathioprine, can lead to liver toxicity, bone marrow suppression, and gastrointestinal disturbances. These side effects necessitate careful monitoring and individualized treatment plans.

What prior FDA approvals exist?

The FDA has approved several treatments for sarcoidosis, particularly those with anti-inflammatory and immunomodulatory properties. Acthar Gel (repository corticotropin injection) is one such treatment, known for its ability to modulate the immune system and reduce inflammation. Other treatments include corticosteroids like prednisone, which are commonly used to manage inflammation in sarcoidosis. Immunosuppressive agents such as methotrexate, azathioprine, and mycophenolate mofetil are also used off-label to treat sarcoidosis by suppressing the immune response. These treatments aim to control symptoms and prevent disease progression.

What other types of research exist?

Promising novel alternatives to Acthar Gel for sarcoidosis patients include: 1) Biologic agents targeting specific inflammatory pathways, such as TNF inhibitors (e.g., infliximab) and IL-17 inhibitors (e.g., secukinumab). 2) JAK inhibitors, which modulate the immune response by inhibiting Janus kinase enzymes. 3) Anti-fibrotic agents like pamrevlumab, which have shown potential in treating fibrotic lung diseases. Patients should discuss these options with their healthcare provider to determine the best course of treatment.

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Corticosteroid

Acthar Gel for Sarcoidosis (Acthar Trial)

Phase 4

Recruiting

Led By Laura Koth, MD

Research Sponsored by University of California, San Francisco

Eligibility Criteria Checklist

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

Must have

Biopsy-proven diagnosis of sarcoidosis with demonstrated pulmonary involvement

Refractoriness to or intolerance of immunosuppressive agents like prednisone or methotrexate

Must not have

History of osteoporosis

History of chronic infections (e.g. tuberculosis, viral, fungal, bacterial)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Drug Has Already Been Approved

Summary

This trial is testing Acthar gel, a medication that reduces inflammation, in patients with pulmonary sarcoidosis. The goal is to see if it improves lung function and symptoms by calming the immune system. Researchers will also check if certain blood markers decrease as symptoms improve.

Who is the study for?

This trial is for individuals with biopsy-proven sarcoidosis affecting the lungs, who haven't responded well to or can't tolerate standard treatments like prednisone. It's not open to recent surgery patients, smokers, those with osteoporosis, cancer, heart failure, high blood pressure that isn't managed, chronic infections (like TB), stomach ulcers or other lung diseases.

What is being tested?

The study is examining if Acthar gel improves symptoms and lung function in sarcoidosis patients. It also looks at whether levels of a blood marker called CXCL9 change with treatment. The goal is to see if this marker can predict how well the drug works.

What are the potential side effects?

Acthar gel may cause side effects such as increased blood pressure and sugar levels, mood swings, weight gain due to fluid retention, digestive issues and risk of infection due to its immune system effects.

Eligibility Criteria

Inclusion Criteria

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

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I have sarcoidosis confirmed by biopsy with lung involvement.

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My body did not respond well to treatments like prednisone or methotrexate.

Exclusion Criteria

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

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I have been diagnosed with osteoporosis.

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I have a history of chronic infections.

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I have active stomach ulcers.

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I have had cancer in the past.

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I have a lung condition besides cancer.

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I have been diagnosed with congestive heart failure.

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My high blood pressure is not under control.

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

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

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Acthar gelExperimental Treatment1 Intervention

After a 4-week period of baseline monitoring, Acthar gel will be administered by intramuscular or subcutaneous injection. Initial dosing will be 40 U every 72 hours (or twice per week) for 4 weeks. Dosage will then be increased to 80 U with similar frequency for 8 weeks and up to 16 weeks.

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

Sarcoidosis treatments often target inflammation and immune system modulation. Glucocorticoids, like prednisone, reduce inflammation by suppressing the immune response, which helps alleviate symptoms and prevent organ damage. TNF inhibitors, such as infliximab, block tumor necrosis factor-alpha, a cytokine involved in granuloma formation, thus reducing inflammation and granuloma development. Immunosuppressive agents like methotrexate inhibit immune cell proliferation, further controlling the immune response. These treatments are crucial for Sarcoidosis patients as they help manage symptoms, prevent disease progression, and improve quality of life by targeting the underlying inflammatory and immune processes.