Acthar Gel in Participants With Pulmonary Sarcoidosis (PULSAR)

February 24, 2023 updated by: Mallinckrodt

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

55

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • UAB Lung Health Center
    • California
      • Los Angeles, California, United States, 90095
        • David Geffen School of Medicine
    • Colorado
      • Denver, Colorado, United States, 80206
        • National Jewish Health
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida Division of Pulmonary, Critical Care, and Sleep Medicine
      • Miami, Florida, United States, 33125
        • University of Miami Miller School of Medicine
      • Orlando, Florida, United States, 32803
        • Central Florida Pulmonary Group PA
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center
    • Indiana
      • Michigan City, Indiana, United States, 46360
        • LaPorte County Institute for Clinical Research
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Columbia, Maryland, United States, 21044
        • Howard County Center for Lung and Sleep Medicine, LLC
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New Jersey
      • Ridgewood, New Jersey, United States, 07450
        • Valley Medical Group
    • New York
      • Albany, New York, United States, 12208
        • Albany Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28207
        • American Health Research Inc
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
      • Gastonia, North Carolina, United States, 28054
        • Clinical Research of Gastonia
    • Ohio
      • Cincinnati, Ohio, United States, 45267
        • University of Cincinnati Medical Center
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Milton S Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19140
        • Temple Lung Center
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center
      • Wyomissing, Pennsylvania, United States, 19610
        • Berks Schuylkill Respiratory Specialists, Ltd
    • South Carolina
      • Anderson, South Carolina, United States, 29621
        • Vitalink Research - Anderson
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina - PPDS
      • Mount Pleasant, South Carolina, United States, 29464
        • Clinical Research of Charleston
      • Rock Hill, South Carolina, United States, 29732
        • Clinical Research of Rock Hill
      • Spartanburg, South Carolina, United States, 29303
        • VitaLink Research - Spartanburg
    • Texas
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern Medical Center
      • Tyler, Texas, United States, 75708
        • University of Texas Health Science Center at Tyler

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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:

  • 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-α antibody within the past 3 months
  • Has any pulmonary condition that requires treatment, therefore impeding corticosteroid tapering

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Acthar Gel in DBT Then Acthar Gel in OLE
Participants received Acthar Gel as a 1 milliliter (mL) injection under the skin, twice weekly, for 24 weeks in the double-blind treatment (DBT) phase. Participants, who chose to continue into the optional OLE phase, then received Acthar Gel as a 1 mL injection under the skin, twice weekly, for another 24 weeks in the optional open-label extension (OLE) phase.
Drug: Acthar Gel
Acthar Gel for subcutaneous (SC) injection (80 units per 1 mL)
Other Names:
  • Repository Corticotropin Injection
  • H.P. Acthar Gel
Experimental: Placebo in DBT Then Acthar Gel in OLE
Participants received Acthar Gel matching placebo as a 1 mL injection under the skin, twice weekly, for 24 weeks in the DBT phase. Participants, who chose to continue into the optional OLE phase, then received Acthar Gel as a 1 mL injection under the skin, twice weekly, for another 24 weeks in the optional OLE phase.
Drug: Acthar Gel
Acthar Gel for subcutaneous (SC) injection (80 units per 1 mL)
Other Names:
  • Repository Corticotropin Injection
  • H.P. Acthar Gel
Drug: Placebo
Placebo gel for SC injection
Other Names:
  • Matching Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24
Time Frame: 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
OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48
Time Frame: 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
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
Time Frame: 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
OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48
Time Frame: 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
DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24
Time Frame: 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
OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48
Time Frame: 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
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
Time Frame: 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
OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48
Time Frame: 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
DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24
Time Frame: 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
OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48
Time Frame: 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
DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24
Time Frame: 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:

    1. When stable condition without toxicity: At first stable visit they continue same dose; at second stable visit, reduce dose by 1 level
    2. 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
OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48
Time Frame: 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:

    1. When stable condition without toxicity: At first stable visit they continue same dose; at second stable visit, reduce dose by 1 level
    2. 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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mallinckrodt

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 21, 2018

Primary Completion (Actual)

November 15, 2021

Study Completion (Actual)

November 15, 2021

Study Registration Dates

First Submitted

October 20, 2017

First Submitted That Met QC Criteria

October 20, 2017

First Posted (Actual)

October 25, 2017

Study Record Updates

Last Update Posted (Estimate)

February 27, 2023

Last Update Submitted That Met QC Criteria

February 24, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MNK14344100

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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