Acthar Gel in Participants With Pulmonary Sarcoidosis

Sarcoidosis, Pulmonary Clinical Trial

— PULSAR  

Official title:

A Multicenter, Randomized, Double Blind, Placebo Controlled Exploratory Study to Assess the Efficacy and Safety of Acthar Gel in Subjects With Pulmonary Sarcoidosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03320070
• Other study ID #: MNK14344100
• Status: Completed
• Phase: Phase 4
• Start date: February 21, 2018
• Est. completion date: November 15, 2021

Study information

• Verified date: February 2023
• Source: Mallinckrodt
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to find out if Acthar Gel is safe and effective to treat pulmonary sarcoidosis. Participants will be randomly assigned (like flipping a coin) to receive a shot under their skin of Acthar Gel or a matching placebo gel that has no drug in it. They will receive their assigned shot twice a week for 24 weeks. All participants who complete the 24-week treatment period will be eligible to receive Acthar Gel for 24 more weeks, even if they were originally in the placebo group.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 55
• Est. completion date: November 15, 2021
• Est. primary completion date: November 15, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 90 Years

Eligibility

Inclusion Criteria:

• Has biopsy-confirmed sarcoidosis meeting American Thoracic Society criteria = 1 year at screening (Visit 1)
• Has protocol-defined symptomatic pulmonary disease
• Has been receiving a stable prednisone dose between 5 mg and 40 mg (or equivalent) for pulmonary sarcoidosis, for at least 4 weeks before screening, or a stable dose of another disease-modifying anti-sarcoidosis drug for at least 3 months before screening
• Has lung function within protocol-defined parameters

Exclusion Criteria:

• Has at least a 10% change in forced vital capacity (FVC) on spirometry between Visits 1 and 2
• Has pulmonary arterial hypertension requiring treatment
• Has been treated with antitumor necrosis factor-a antibody within the past 3 months
• Has any pulmonary condition that requires treatment, therefore impeding corticosteroid tapering

Related Conditions & MeSH terms

• Sarcoidosis
• Sarcoidosis, Pulmonary

Intervention

Drug:
• Acthar Gel
Acthar Gel for subcutaneous (SC) injection (80 units per 1 mL)
• Placebo
Placebo gel for SC injection

Locations

Country	Name	City	State
United States	Albany Medical Center	Albany	New York
United States	VitaLink Research - Anderson	Anderson	South Carolina
United States	UAB Lung Health Center	Birmingham	Alabama
United States	Medical University of South Carolina - PPDS	Charleston	South Carolina
United States	American Health Research Inc	Charlotte	North Carolina
United States	Rush University Medical Center	Chicago	Illinois
United States	University of Cincinnati Medical Center	Cincinnati	Ohio
United States	Cleveland Clinic	Cleveland	Ohio
United States	Howard County Center for Lung and Sleep Medicine, LLC	Columbia	Maryland
United States	University of Texas Southwestern Medical Center	Dallas	Texas
United States	National Jewish Health	Denver	Colorado
United States	Duke University Medical Center	Durham	North Carolina
United States	University of Florida Division of Pulmonary, Critical Care, and Sleep Medicine	Gainesville	Florida
United States	Clinical Research of Gastonia	Gastonia	North Carolina
United States	Penn State Milton S Hershey Medical Center	Hershey	Pennsylvania
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	David Geffen School of Medicine	Los Angeles	California
United States	University of Miami Miller School of Medicine	Miami	Florida
United States	Laporte County Institute For Clinical Research	Michigan City	Indiana
United States	Clinical Research of Charleston	Mount Pleasant	South Carolina
United States	Central Florida Pulmonary Group PA	Orlando	Florida
United States	Temple Lung Center	Philadelphia	Pennsylvania
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	Valley Medical Group	Ridgewood	New Jersey
United States	Clinical Research of Rock Hill	Rock Hill	South Carolina
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	VitaLink Research - Spartanburg	Spartanburg	South Carolina
United States	University of Texas Health Science Center at Tyler	Tyler	Texas
United States	Berks Schuylkill Respiratory Specialists, Ltd	Wyomissing	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Mallinckrodt

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24	Forced vital capacity (FVC) is a pulmonary function test parameter that indicates the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It's measured by spirometry, which is a common breathing test to check lung function. Based on the absolute change of percent predicted, FVC was evaluated to determine if the condition is: Improved (+1) [= 5% absolute change]; Unchanged (0) [>- 5% to < 5% absolute change], or; Worse (-1) [= -5% absolute change]; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 24
Primary	OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48	Forced vital capacity (FVC) is a pulmonary function test parameter that indicates the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It's measured by spirometry, which is a common breathing test to check lung function. Based on the absolute change of percent predicted, FVC was evaluated to determine if the condition is: Improved (+1) [= 5% absolute change]; Unchanged (0) [>- 5% to < 5% absolute change], or; Worse (-1) [= -5% absolute change]; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 48
Primary	DBT: Number of Participants in Each Category of Assessment Based on the Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO), a Pulmonary Function Test Parameter at Week 24	The diffusing capacity of the lungs for carbon monoxide (DLCO) measures the ability of the lungs to transfer gas from inhaled air to the red blood cells in the blood. Participants are asked to fully inhale a low concentration of carbon monoxide and an inert tracer gas. Based on the absolute change of percent predicted, DLCO was evaluated to determine if the condition is: Improved (+1) [= 5% absolute change]; Unchanged (0) [>- 5% to < 5% absolute change],; Worse (-1) [= -5% absolute change]; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 24
Primary	OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48	The diffusing capacity of the lungs for carbon monoxide (DLCO) measures the ability of the lungs to transfer gas from inhaled air to the red blood cells in the blood. Participants are asked to fully inhale a low concentration of carbon monoxide and an inert tracer gas. Based on the absolute change of percent predicted, DLCO was evaluated to determine if the condition is: Improved (+1) [= 5% absolute change]; Unchanged (0) [>- 5% to < 5% absolute change],; Worse (-1) [= -5% absolute change]; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 48
Primary	DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24	High-resolution computer tomography (HRCT) is a type of computed tomography (CT) with specific techniques to enhance image resolution. It is used in the diagnosis of various health problems, most commonly for lung disease. These images show cross sections (slices) through the lungs. HRCT imaging was evaluated by the investigator/radiologist and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1). An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 24
Primary	OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48	High-resolution computer tomography (HRCT) is a type of computed tomography (CT) with specific techniques to enhance image resolution. It is used in the diagnosis of various health problems, most commonly for lung disease. These images show cross sections (slices) through the lungs. HRCT imaging was evaluated by the investigator/radiologist and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1). An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 48
Primary	DBT: Number of Participants in Each Category of Assessment Based on the King's Sarcoidosis Questionnaire (KSQ) (General Health), a Quality of Life Parameter at Week 24	King's sarcoidosis questionnaire (KSQ) (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Each item was answered on a 7-point scale where 1 means the participant experiences the symptom all the time and 7 means the participant does not experience the symptom at all. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: Improved (+1) based on a change of = 4 points; Unchanged (0) based on a change of >- 4 to < 4 points; Worse (-1) based on a change of = -4 points; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 24
Primary	OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48	King's sarcoidosis questionnaire (KSQ) (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Each item was answered on a 7-point scale where 1 means the participant experiences the symptom all the time and 7 means the participant does not experience the symptom at all. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: Improved (+1) based on a change of = 4 points; Unchanged (0) based on a change of >- 4 to < 4 points; Worse (-1) based on a change of = -4 points; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 48
Primary	DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24	The fatigue assessment score (FAS) is a 10-item checklist for participants to indicate the level of their fatigue. Each item was answered on a 5-point scale where 1 means the participant does not experience the symptom all and 5 means the participant experiences the symptom all the time. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: Improved (+1) based on a change of = -4 points; Unchanged (0) based on a change of >- 4 to < 4 points; Worse (-1) based on a change of = 4 points; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 24
Primary	OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48	The fatigue assessment score (FAS) is a 10-item checklist for participants to indicate the level of their fatigue. Each item was answered on a 5-point scale where 1 means the participant does not experience the symptom all and 5 means the participant experiences the symptom all the time. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale was evaluated to determine if the condition is: Improved (+1) based on a change of = -4 points; Unchanged (0) based on a change of >- 4 to < 4 points; Worse (-1) based on a change of = 4 points; An additional category "Missing Assessment" indicates the participants who had a missing assessment for this outcome measure.	Week 48
Primary	DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24	Corticosteroids are first line treatment for sarcoidosis. Concerns of corticosteroid toxicity led to efforts to taper dose sooner. Participants were clinically evaluated at each visit by investigator and categorized by their condition; dose was tapered using algorithm to take them off prednisone using incremental doses of 40,30,20,10,7.5,5,2.5,0 mg, as explained with each category below.; Improved: When condition improved, reduce dose by 1 level; Unchanged: When stable condition without toxicity: At first stable visit they continue same dose; at second stable visit, reduce dose by 1 level; When stable condition with toxicity: toxicity is treated; reduce dose by 1 level; Deteriorate: When worsening condition, increase dose by 1 or 2 levels but not >40mg/day; Dose tapering was done based on the participant's clinical condition.; Category "Missing Assessment" indicates participants who had a missing assessment for this outcome measure.	Week 24
Primary	OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48	Corticosteroids are first line treatment for sarcoidosis. Concerns of corticosteroid toxicity led to efforts to taper dose sooner. Participants were clinically evaluated at each visit by investigator and categorized by their condition; dose was tapered using algorithm to take them off prednisone using incremental doses of 40,30,20,10,7.5,5,2.5,0 mg, as explained with each category below.; Improved: When condition improved, reduce dose by 1 level; Unchanged: When stable condition without toxicity: At first stable visit they continue same dose; at second stable visit, reduce dose by 1 level; When stable condition with toxicity: toxicity is treated; reduce dose by 1 level; Deteriorate: When worsening condition, increase dose by 1 or 2 levels but not >40mg/day; Dose tapering was done based on the participant's clinical condition.; Category "Missing Assessment" indicates participants who had a missing assessment for this outcome measure.	Week 48