Acthar Gel for Sarcoidosis

(Acthar Trial)

Recruiting in Palo Alto (17 mi)

Overseen by Laura Koth, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Recruiting

Sponsor: University of California, San Francisco

Disqualifiers: Smoking, Cancer, Chronic infections, others

No Placebo Group

Prior Safety Data

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, it mentions that participants should be intolerant of or not responding to certain immunosuppressive drugs like prednisone or methotrexate.

What data supports the effectiveness of the drug Acthar Gel for treating sarcoidosis?

Research suggests that Acthar Gel can reduce the need for corticosteroids (a type of medication often used to reduce inflammation) in sarcoidosis patients, which may help avoid the side effects associated with long-term steroid use. Additionally, Acthar Gel has been studied for its effectiveness in treating various inflammatory diseases, showing promise when other treatments were not effective. ^{1 2 3 4 5}

Is Acthar Gel safe for humans?

Acthar Gel has been studied for safety in various inflammatory diseases, including sarcoidosis, and many studies have shown it to be safe for patients who cannot tolerate standard treatments. However, like any medication, it may have side effects, and its safety profile has been evaluated in over 500 publications. ^{1 2 3 5 6}

How is Acthar Gel different from other drugs for sarcoidosis?

Acthar Gel is unique because it acts as a steroid-sparing therapy, meaning it can reduce the need for long-term use of corticosteroids, which often have significant side effects. It works by activating specific receptors in the body to help manage inflammation, making it a novel option for patients who cannot tolerate standard treatments. ^{1 3 4 5 7}

Research Team

Laura Koth, MD

Principal Investigator

University of California, San Francisco

Eligibility Criteria

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.

Inclusion Criteria

I have sarcoidosis confirmed by biopsy with lung involvement.

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

Exclusion Criteria

I have been diagnosed with osteoporosis.

Smoking

I have a history of chronic infections.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Monitoring

Participants undergo a 4-week period of baseline monitoring before treatment

4 weeks

Treatment

Participants receive Acthar gel with initial dosing of 40 U every 72 hours for 4 weeks, followed by 80 U for up to 16 weeks

20 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Acthar gel (Corticosteroid)

Trial Overview 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.

Participant Groups

Group I: Acthar gel Experimental Treatment 1 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.

Acthar gel is already approved in Japan for the following indications:

Approved in Japan as Synthetic ACTH (e.g., tetracosactide) for:

• Similar to natural ACTH derivatives, but specific indications may vary

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

University of California, San Francisco San Francisco, CA

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2636

Patients Recruited

19,080,000+

Mallinckrodt

Industry Sponsor

Trials

202

Patients Recruited

15,700+

Findings from Research

Acthar Gel significantly reduces corticosteroid use in patients with sarcoidosis compared to other treatments, with a notable decrease in both overall and extended corticosteroid use after treatment.

Patients who adhered better to Acthar Gel treatment experienced even greater reductions in corticosteroid exposure, highlighting the importance of adherence for maximizing treatment benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Corticosteroid Use and Adherence in Patients Treated with Acthar Gel for Advanced Sarcoidosis. Hayes, K., Niewoehner, J., Rice, JB., et al.[2023]

In a multicenter, randomized, placebo-controlled trial involving 55 subjects, repository corticotropin injection (RCI) showed greater improvement in a novel sarcoidosis treatment score (STS) compared to placebo, suggesting its potential efficacy in treating pulmonary sarcoidosis.

RCI was found to be safe and well tolerated, with more patients in the RCI group able to discontinue glucocorticoids compared to those receiving placebo, indicating a promising alternative therapy for patients with pulmonary sarcoidosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Results From a Phase 4, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Repository Corticotropin Injection for the Treatment of Pulmonary Sarcoidosis. Mirsaeidi, M., Baughman, RP., Sahoo, D., et al.[2023]

References

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

Corticosteroid Use and Adherence in Patients Treated with Acthar Gel for Advanced Sarcoidosis. [2023]

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

Results From a Phase 4, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Repository Corticotropin Injection for the Treatment of Pulmonary Sarcoidosis. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Outcomes of Repository Corticotropin Gel for Ocular Sarcoidosis. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Response to Acthar Gel in sarcoidosis uveitis: A prospective open label study. [2023]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Acthar® Gel Treatment for Patients with Autoimmune and Inflammatory Diseases: An Historical Perspective and Characterization of Clinical Evidence. [2023]

6. England pubmed.ncbi.nlm.nih.gov

Repository corticotropin injection in patients with advanced symptomatic sarcoidosis: retrospective analysis of medical records. [2020]

7. New Zealand pubmed.ncbi.nlm.nih.gov

Cost-Effectiveness of Acthar Gel versus Standard of Care for the Treatment of Advanced Symptomatic Sarcoidosis. [2023]