Acthar Gel for Sarcoidosis

(Acthar Trial)

Overseen By Laura Koth, MD

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: University of California, San Francisco

Disqualifiers: Smoking, Cancer, Chronic infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 20 weeks

14 Participants Needed

Trial Summary

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}

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.

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.

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

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 United States, European Union, Japan for the following indications:

Approved in United States as Acthar Gel for:

• Infantile spasms
• Exacerbations of multiple sclerosis
• Rheumatic disorders
• Collagen diseases
• Dermatologic diseases
• Allergic states
• Ophthalmic diseases
• Respiratory diseases
• Edematous states

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

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

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

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

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2,636

Recruited

19,080,000+

Mallinckrodt

Industry Sponsor

Trials

202

Recruited

15,700+

Mario Saltarelli

Mallinckrodt

Chief Medical Officer

MD

Siggi Olafsson

Mallinckrodt

Chief Executive Officer

PhD in Pharmacy

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]

In a retrospective review of 6 patients with ocular sarcoidosis treated with subcutaneous repository corticotropin gel (RCI), most did not achieve sufficient control of ocular inflammation and had to continue using corticosteroids.

Two-thirds of the patients experienced significant side effects, including hyperpigmentation, alopecia, and severe hypertension, leading to the discontinuation of RCI therapy in 5 out of 6 patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Outcomes of Repository Corticotropin Gel for Ocular Sarcoidosis. Oh, DJ., Singh, A., Kanu, LN., et al.[2022]

In a study of nine patients with moderate to severe sarcoidosis uveitis, Acthar Gel showed some efficacy, with three out of four patients completing the treatment achieving the primary endpoint of improved visual acuity or reduced inflammation.

However, the treatment was associated with significant adverse effects, including fluid retention, insomnia, hypertension, and hyperglycemia, leading to a high dropout rate, as four patients discontinued due to worsening symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Response to Acthar Gel in sarcoidosis uveitis: A prospective open label study. Ungprasert, P., Lowder, C., Sharma, S., et al.[2023]

Acthar® Gel has been shown to have both steroidogenic and nonsteroidogenic immunomodulatory effects, making it a versatile treatment option for various autoimmune and inflammatory diseases.

Over 500 studies have demonstrated the safety and efficacy of Acthar in patients who did not respond well to standard treatments, highlighting its importance in managing conditions like rheumatoid arthritis and multiple sclerosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acthar® Gel Treatment for Patients with Autoimmune and Inflammatory Diseases: An Historical Perspective and Characterization of Clinical Evidence. Kaplan, J., Askanase, A., Chu, D., et al.[2023]

Repository corticotropin injection (RCI) is an effective treatment for advanced symptomatic sarcoidosis, with 95% of patients showing overall health improvement after therapy, based on a study of 302 patients.

The use of RCI led to a significant reduction in corticosteroid use, with the percentage of patients on corticosteroids dropping from 61.3% to 12.9% after 3 months of RCI treatment, indicating its potential to reduce reliance on traditional steroid therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Repository corticotropin injection in patients with advanced symptomatic sarcoidosis: retrospective analysis of medical records. Chopra, I., Qin, Y., Kranyak, J., et al.[2020]

Acthar® Gel is shown to be a cost-effective treatment option for advanced symptomatic sarcoidosis, with an incremental cost-effectiveness ratio (ICER) of $134,796 per quality-adjusted life-year (QALY) from a payer perspective over 2 years.

The analysis indicates that Acthar Gel provides similar or better value compared to standard of care (SoC), with a lower ICER of $21,967 per QALY from a societal perspective over 3 years, suggesting it is a viable treatment option for patients with this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cost-Effectiveness of Acthar Gel versus Standard of Care for the Treatment of Advanced Symptomatic Sarcoidosis. Bindra, J., Chopra, I., Hayes, K., 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]