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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests Acthar gel (Repository Corticotropin Injection) to determine its effectiveness for people with sarcoidosis, a condition where clusters of inflamed tissue, called granulomas, affect the lungs. Researchers aim to assess whether Acthar gel can improve symptoms and lung function and reduce certain blood markers linked to the disease. The treatment involves injections twice a week and may suit those with a biopsy-confirmed diagnosis of sarcoidosis in the lungs who have not responded well to other treatments like prednisone. As a Phase 4 trial, this research focuses on understanding how this already FDA-approved and effective treatment can benefit more patients.

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 is the safety track record for Acthar gel?

Research has shown that Acthar Gel has been used safely for many conditions over the years. The FDA has approved it for several diseases, indicating its general safety for most people. However, Acthar Gel should not be administered intravenously and is not intended for infants who might have infections.

For those considering joining a clinical trial for Acthar Gel to treat sarcoidosis, it is reassuring that many patients have used it safely. However, specific safety studies for sarcoidosis are limited. Always consult your doctor about the potential risks and benefits before deciding to participate in a trial. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Acthar gel is unique because it offers a different approach to treating sarcoidosis compared to standard therapies like corticosteroids. Unlike most treatments that primarily suppress inflammation, Acthar gel stimulates the body's own production of hormones that help reduce inflammation and modulate the immune system. It's administered through injections, which allows for a controlled dosage that can be tailored over time. Researchers are excited about Acthar gel because it could offer a viable alternative for patients who either can't tolerate or don't respond well to traditional treatments.

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

Research has shown that Acthar Gel can help treat conditions such as multiple sclerosis. In a large study with 302 patients who had advanced sarcoidosis, 95% showed improvement after using Acthar Gel. This gel reduces inflammation and eases symptoms. The FDA has approved Acthar Gel for other conditions, suggesting it might also be effective for sarcoidosis. Participants in this trial will receive Acthar Gel to evaluate its effectiveness specifically for sarcoidosis. ^{6 7 8 9 10}

Who Is on the Research Team?

Laura Koth, MD

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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 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]

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]

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]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37072607/

Results From a Phase 4, Multicenter, Randomized, Double ... This clinical trial investigated whether repository corticotropin injection (RCI, Acthar ® Gel) was safe and effective in patients who were already taking ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7170160/

Repository corticotropin injection (H.P. Acthar gel) for the ... The FDA subsequently approved hydrocortisone and prednisone for the treatment of sarcoidosis, which resulted in decreased use of corticotrophin treatment ...

3. ir.mallinckrodt.com ir.mallinckrodt.com/news-releases/news-release-details/mallinckrodt-presents-latest-health-economics-data-actharr-gel

Release Details - Investor Relations ... Data on Acthar® Gel (Repository Corticotropin ... Cost-Effectiveness of Acthar Gel for the Treatment of Advanced Symptomatic Sarcoidosis.

4. actharhcp.com actharhcp.com/clinical-experience/sarcoidosis/chopra/

Sarcoidosis retrospective analysis Controlled clinical trials have shown Acthar to be effective in speeding the resolution of acute exacerbations of multiple sclerosis. However, there is no ...

5. dovepress.com dovepress.com/cost-effectiveness-of-acthar-gel-versus-standard-of-care-for-the-treat-peer-reviewed-fulltext-article-CEOR

Cost-effectiveness of Acthar Gel for Sarcoidosis | CEOR A large case series study utilizing medical records for 302 patients with advanced sarcoidosis found overall status improvement in 95% of ...

6. actharhcp.com actharhcp.com/safety-data/

Safety data | Acthar® Gel (repository corticotropin injection) Acthar Gel is supported by decades of safety data across multiple diseases. Please see full Prescribing Information for complete dosing information.

7. acthar.com acthar.com/Static/pdf/Acthar-PI.pdf

Acthar® Gel 4 CONTRAINDICATIONS Acthar Gel is contraindicated: • for intravenous administration. in infants under 2 years of age who have suspected congenital infections.

8. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2024/008372s074lbl.pdf

acthar® gel - accessdata.fda.gov Acthar Gel (repository corticotropin injection) is a clear light amber solution mobile at room temperature. 4. CONTRAINDICATIONS. Acthar Gel is contraindicated:.

9. acthar.com acthar.com/sarcoidosis/

Sarcoidosis | Acthar® Gel (repository corticotropin injection) Three months after treatment with Acthar Gel ended, only 39 patients were being treated with steroids. Safety of Acthar Gel was not studied. Remember that ...

10. aetna.com aetna.com/cpb/medical/data/700_799/0762.html

Repository Corticotropin Injection (Acthar Gel and Purified ... Aetna considers use of Acthar Gel in combination with Purified Cortrophin Gel as experimental, investigational, or unproven because the safety and efficacy for ...