Clinical trial NCT02188017: Acthar Gel for Chronic Pulmonary Sarcoidosis (ACPS)
Official title: ACTHAR Gel for Chronic Pulmonary Sarcoidosis (ACPS)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Sarcoidosis
Pulmonary Sarcoidosis
Keywords: prednisone, methotrexate, sarcoidosis, azathioprine
Lead sponsor: University of Cincinnati
Sponsor class: OTHER
Collaborators: name: Mallinckrodt
First posted: 2014-07-11
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Steroid toxicity
timeFrame: 24 weeks
Secondary outcomes:
measure: Forced Vital Capacity (FVC)
timeFrame: 24 weeks
measure: Chest x-ray
timeFrame: 24 weeks
measure: PET scan
timeFrame: 24 weeks
measure: Sarcoidosis Health Questionnaire
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
Study officials:
affiliation: University of Cincinnati
name: Robert P Baughman, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Baughman RP, Sweiss N, Keijsers R, Birring SS, Shipley R, Saketkoo LA, Lower EE. Repository corticotropin for Chronic Pulmonary Sarcoidosis. Lung. 2017 Jun;195(3):313-322. doi: 10.1007/s00408-017-9994-4. Epub 2017 Mar 28.