Acthar Gel for Chronic Pulmonary Sarcoidosis (ACPS) (ACPS)

ACTHAR Gel for Chronic Pulmonary Sarcoidosis (ACPS)

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.

Study Overview

• Status: Unknown
• Conditions: Sarcoidosis; Pulmonary Sarcoidosis
• Intervention / Treatment: Drug: Acthar gel

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.

• Study Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Robert P Baughman, MD; Phone Number: 513-58405225; Email: bob.baughman@uc.edu

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • Recruiting
      • University of Cincinnati
      • Principal Investigator: Robert P Baughman, MD
      • Contact: Felicia Thompson; Phone Number: 513-584-6252; Email: THOMPSFA@ucmail.uc.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 90 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: University of Cincinnati
• Collaborators: Mallinckrodt
• Investigators: Principal Investigator: Robert P Baughman, MD, University of Cincinnati

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: June 1, 2014
• Primary Completion (Anticipated): December 1, 2016
• Study Completion (Anticipated): September 1, 2017

Study Registration Dates

• First Submitted: April 24, 2014
• First Submitted That Met QC Criteria: July 8, 2014
• First Posted (Estimate): July 11, 2014

Study Record Updates

• Last Update Posted (Estimate): December 8, 2015
• Last Update Submitted That Met QC Criteria: December 4, 2015
• Last Verified: December 1, 2015

More Information

Terms related to this study

• Keywords: prednisone, methotrexate, sarcoidosis, azathioprine
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Lung Diseases; Lung Diseases, Interstitial; Sarcoidosis, Pulmonary; Sarcoidosis
• Other Study ID Numbers: ACPS