Acthar Gel in Participants With Pulmonary Sarcoidosis

NCT03320070 | Completed Phase 4 Results FDA Drug

• 1 other identifier: MNK14344100
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 29

Brief 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.

Conditions

Sarcoidosis, Pulmonary

Outcome Measures

Primary Outcomes (12)

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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

• 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: 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

  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

Study Arms (2)

Acthar Gel in DBT Then Acthar Gel in OLE

EXPERIMENTAL

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

Placebo in DBT Then Acthar Gel in OLE

EXPERIMENTAL

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; Drug: Placebo

Interventions

Acthar Gel DRUG

Acthar Gel for subcutaneous (SC) injection (80 units per 1 mL)

Also known as: H.P. Acthar Gel, Repository Corticotropin Injection

Acthar Gel in DBT Then Acthar Gel in OLE; Placebo in DBT Then Acthar Gel in OLE

Placebo DRUG

Placebo gel for SC injection

Also known as: Matching Placebo

Placebo in DBT Then Acthar Gel in OLE

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mallinckrodt: lead

Study Sites (29)

UAB Lung Health Center

Birmingham, Alabama, 35294, United States

Location

David Geffen School of Medicine

Los Angeles, California, 90095, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

University of Florida Division of Pulmonary, Critical Care, and Sleep Medicine

Gainesville, Florida, 32610, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33125, United States

Location

Central Florida Pulmonary Group PA

Orlando, Florida, 32803, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Laporte County Institute For Clinical Research

Michigan City, Indiana, 46360, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Howard County Center for Lung and Sleep Medicine, LLC

Columbia, Maryland, 21044, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Valley Medical Group

Ridgewood, New Jersey, 07450, United States

Location

Albany Medical Center

Albany, New York, 12208, United States

Location

American Health Research Inc

Charlotte, North Carolina, 28207, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Clinical Research of Gastonia

Gastonia, North Carolina, 28054, United States

Location

University of Cincinnati Medical Center

Cincinnati, Ohio, 45267, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Penn State Milton S Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Temple Lung Center

Philadelphia, Pennsylvania, 19140, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Berks Schuylkill Respiratory Specialists, Ltd

Wyomissing, Pennsylvania, 19610, United States

Location

VitaLink Research - Anderson

Anderson, South Carolina, 29621, United States

Location

Medical University of South Carolina - PPDS

Charleston, South Carolina, 29425, United States

Location

Clinical Research of Charleston

Mt. Pleasant, South Carolina, 29464, United States

Location

Clinical Research of Rock Hill

Rock Hill, South Carolina, 29732, United States

Location

VitaLink Research - Spartanburg

Spartanburg, South Carolina, 29303, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

University of Texas Health Science Center at Tyler

Tyler, Texas, 75708, United States

Location

Related Publications (1)

• Mirsaeidi M, Baughman RP, Sahoo D, Tarau E. Results From a Phase 4, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Repository Corticotropin Injection for the Treatment of Pulmonary Sarcoidosis. Pulm Ther. 2023 Jun;9(2):237-253. doi: 10.1007/s41030-023-00222-2. Epub 2023 Apr 19.

  PMID: 37072607 DERIVED

MeSH Terms

Conditions

Sarcoidosis, Pulmonary

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Sarcoidosis; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases

Results Point of Contact

• Title: Medical Information Call Center
• Organization: Mallinckrodt

medInfo@mnk.com

1-800-844-2830

Study Officials

• Clinical Team Leader

  Mallinckrodt

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2017
• First Posted: October 25, 2017
• Study Start: February 21, 2018
• Primary Completion: November 15, 2021
• Study Completion: November 15, 2021
• Last Updated: February 27, 2023
• Results First Posted: February 27, 2023

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (29)