Acthar Gel for Chronic Pulmonary Sarcoidosis (ACPS)

Study Overview
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Brief Summary
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An randomized trial of two maintenance doses of Acthar Gel for patients with chronic pulmonary sarcoidosis. Patients will be observed for 24 weeks of treatment.

Detailed Description
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This is a multi-center double-blind trial to determine dose and effect size. A total of 20 chronic pulmonary sarcoidosis patients will be recruited at eight clinical sites across the United States. After initial evaluation, including pulmonary function, CT and PET scanning, patients will be randomized to receive 80 Units if Acthar gel daily for 10days, followed by either 40 or 80 units of Acthar gel twice a week for an additional 22 weeks. At the end of total 24 weeks of treatment, they will undergo repeat evaluation including pulmonary function, CT, and PET scanning.

Official Title
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ACTHAR Gel for Chronic Pulmonary Sarcoidosis (ACPS)

Conditions
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Sarcoidosis, Pulmonary Sarcoidosis

Intervention / Treatment
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* Drug: Acthar gel


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient with biopsy confirmed sarcoidosis meeting American Thoracic Society criteria 23 Patient on >5 mg prednisone for pulmonary indications FVC <85% predicted Prednisone dose not reduced in prior 3 months Deterioration of pulmonary disease over the past year Decrease in FVC >5% Age: 18 through 90 (i.e., candidates must have had their 18th birthday, but not had their 91st birthday). Exclusion Criteria: adrenal insufficiency (Addison's disease) Scleroderma a fungal infection herpes infection of the eyes osteoporosis a stomach ulcer congestive heart failure high blood pressure recent surgery if you are allergic to pork proteins Do not receive a smallpox vaccine or any live vaccine while you are using corticotropin. Patients receiving anti-Tumor Necrosis Factor antibody (e.g. infliximab, adalimumab) in prior six months Uncontrolled diabetes, hypertension, or other contra-indication to increased dosage of glucocorticoids Patients requiring therapy for pulmonary hypertension Females of childbearing potential who are known to be pregnant and/or lactating or who have a positive urine pregnancy test on screening. Current participation in another research drug treatment protocol (patient cannot start another experimental agent until after 90 days) Any other condition that the investigator feels would pose a significant hazard to the patient if Acthar Gel therapy is initiated.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 90 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 80 units<br>80 units ACTHAR gel will be given twice a week for 22 weeks after initial loading | Drug: Acthar gel<br>* Given 80 units daily for 10 days, then randomized to receive either 40 or 80 units twice a week<br>* Other names: Cortysin;|
| Active Comparator: 40 units<br>40 units of ACTHAR gel will be given twice a week for 22 weeks after loading | Drug: Acthar gel<br>* Given 80 units daily for 10 days, then randomized to receive either 40 or 80 units twice a week<br>* Other names: Cortysin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Steroid toxicity | Cumulative toxicity between two arms of study over 24 weeks of study This will be assessed using a steroid toxicity questionnaire | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Forced Vital Capacity (FVC) | Change in FVC over course of study | 24 weeks | 
| Chest x-ray | Change in chest x-ray | 24 weeks | 
| PET scan | Change in lung uptake of PET scan during course of study | 24 weeks | 
| Sarcoidosis Health Questionnaire | Change in quality of life as assessed by King's Sarcoidosis Health Questionnaire | 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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prednisone, methotrexate, sarcoidosis, azathioprine