CClinicalTrials.gg
CompletedNCT03320070PULSARUpdated Feb 27, 2023Results posted

Acthar Gel in Participants With Pulmonary Sarcoidosis

A Phase 4 interventional study of Acthar Gel and Placebo in Sarcoidosis, Pulmonary, sponsored by Mallinckrodt. Completed at 29 sites in United States. Open to participants aged 18 Years to 90 Years. Per ClinicalTrials.gov, last updated 2023-02-27.

Sponsored by Mallinckrodt · Phase 4, Interventional, and Treatment

Phase
Phase 4
Study type
Interventional
Enrollment
55
Allocation
Randomized
Ages
18 Years to 90 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Sarcoidosis, Pulmonary
03

Who can participate

Ages eligible
18 Years to 90 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
55 participants (actual)

Study arms

  • 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

  • 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 · Drug: Placebo

Interventions

  • DrugActhar Gel

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

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

  • DrugPlacebo

    Placebo gel for SC injection

    Also known as: Matching Placebo

05

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Week 24

  2. 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.

    Time frame: Week 48

  3. 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.

    Time frame: Week 24

  4. 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.

    Time frame: Week 48

  5. 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.

    Time frame: Week 24

  6. 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.

    Time frame: Week 48

  7. 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.

    Time frame: Week 24

  8. 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.

    Time frame: Week 48

  9. 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.

    Time frame: Week 24

  10. 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.

    Time frame: Week 48

  11. 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.

    Time frame: Week 24

  12. 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.

    Time frame: Week 48

06

Results

Posted Feb 27, 2023

Participant flow

Participants took part in the study at 35 investigative sites in the United States from 21 February 2018 to 15 November 2021.

DBT Phase (Week 1 to Week 24)
Participant flow — DBT Phase (Week 1 to Week 24)
MilestoneActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Started2728
Modified intent-to-treat population (mitt)2728
Safety population2728
Completed2425
Not completed33
Withdrew: Adverse event11
Withdrew: Withdrawal by subject22
OLE Phase (Week 25 to Week 48)
Participant flow — OLE Phase (Week 25 to Week 48)
MilestoneActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Started2225
Open-label safety population2225
Completed1723
Not completed52
Withdrew: Adverse event30
Withdrew: Lost to follow-up01
Withdrew: Non-compliance with study drug10
Withdrew: Withdrawal by subject11

Outcome measures

PrimaryDBT: 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.

Time frame:
Week 24
Reported as:
Count of participants · Participants
DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved107
Unchanged1212
Deteriorate13
Missing Assessment46
Statistical analysis
  • Acthar Gel in DBT Then Acthar Gel in OLE vs Placebo in DBT Then Acthar Gel in OLE · Chi-squared · p = 0.5076
PrimaryOLE: 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.

Time frame:
Week 48
Reported as:
Count of participants · Participants
OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved26
Unchanged1311
Deteriorate13
Missing Assessment65
PrimaryDBT: 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.

Time frame:
Week 24
Reported as:
Count of participants · Participants
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
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved54
Unchanged1111
Deteriorate77
Missing Assessment46
Statistical analysis
  • Acthar Gel in DBT Then Acthar Gel in OLE vs Placebo in DBT Then Acthar Gel in OLE · Chi-squared · p = 1.0000
PrimaryOLE: 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.

Time frame:
Week 48
Reported as:
Count of participants · Participants
OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved96
Unchanged36
Deteriorate48
Missing Assessment65
PrimaryDBT: 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.

Time frame:
Week 24
Reported as:
Count of participants · Participants
DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved21
Unchanged2218
Deteriorate04
Missing Assessment35
Statistical analysis
  • Acthar Gel in DBT Then Acthar Gel in OLE vs Placebo in DBT Then Acthar Gel in OLE · Chi-squared · p = 0.0848
PrimaryOLE: 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.

Time frame:
Week 48
Reported as:
Count of participants · Participants
OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved16
Unchanged1313
Deteriorate12
Missing Assessment74
PrimaryDBT: 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.

Time frame:
Week 24
Reported as:
Count of participants · Participants
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
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved117
Unchanged38
Deteriorate109
Missing Assessment34
Statistical analysis
  • Acthar Gel in DBT Then Acthar Gel in OLE vs Placebo in DBT Then Acthar Gel in OLE · Chi-squared · p = 0.2005
PrimaryOLE: 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.

Time frame:
Week 48
Reported as:
Count of participants · Participants
OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved88
Unchanged25
Deteriorate69
Missing Assessment63
PrimaryDBT: 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.

Time frame:
Week 24
Reported as:
Count of participants · Participants
DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved66
Unchanged1211
Deteriorate67
Missing Assessment34
Statistical analysis
  • Acthar Gel in DBT Then Acthar Gel in OLE vs Placebo in DBT Then Acthar Gel in OLE · Chi-squared · p = 0.9416
PrimaryOLE: 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.

Time frame:
Week 48
Reported as:
Count of participants · Participants
OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved74
Unchanged513
Deteriorate45
Missing Assessment63
PrimaryDBT: 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.

Time frame:
Week 24
Reported as:
Count of participants · Participants
DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved1721
Unchanged84
Deteriorate00
Missing Assessment23
Statistical analysis
  • Acthar Gel in DBT Then Acthar Gel in OLE vs Placebo in DBT Then Acthar Gel in OLE · Chi-squared · p = 0.1853
PrimaryOLE: 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.

Time frame:
Week 48
Reported as:
Count of participants · Participants
OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48
ParticipantsActhar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLE
Improved1519
Unchanged55
Deteriorate00
Missing Assessment21

Adverse events

Collected over From the signing of Informed Consent Form (ICF) to follow up visit, 4 weeks after the last dose of study drug (approximately 52 weeks for a participant). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
DBT: Acthar Gel 80 U (1 mL)0/27 (0%)4/27 (14.8%)22/27 (81.5%)
DBT: Placebo0/28 (0%)3/28 (10.7%)25/28 (89.3%)
OLE: Acthar Gel 80 U (1 mL) [Received Acthar in DBT]0/22 (0%)6/22 (27.3%)14/22 (63.6%)
OLE: Acthar Gel 80 U (1 mL) [Received Placebo in DBT]0/25 (0%)0/25 (0%)14/25 (56%)
Most frequent serious events
Showing 10 of 15
Most frequent serious events
EventDBT: Acthar Gel 80 U (1 mL)DBT: PlaceboOLE: Acthar Gel 80 U (1 mL) [Received Acthar in DBT]OLE: Acthar Gel 80 U (1 mL) [Received Placebo in DBT]
PneumoniaInfections and infestations0/271/281/220/25
Upper gastrointestinal haemorrhageGastrointestinal disorders0/270/281/220/25
Haemoglobin decreasedInvestigations0/270/281/220/25
Metastatic renal cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/270/281/220/25
Idiopathic pulmonary fibrosisRespiratory, thoracic and mediastinal disorders0/270/281/220/25
Pulmonary embolismRespiratory, thoracic and mediastinal disorders0/270/281/220/25
Pulmonary hypertensionRespiratory, thoracic and mediastinal disorders0/270/281/220/25
Respiratory failureRespiratory, thoracic and mediastinal disorders0/270/281/220/25
UveitisEye disorders1/270/280/220/25
CholelithiasisHepatobiliary disorders1/270/280/220/25
Most frequent other events
Showing 10 of 46
Most frequent other events
EventDBT: Acthar Gel 80 U (1 mL)DBT: PlaceboOLE: Acthar Gel 80 U (1 mL) [Received Acthar in DBT]OLE: Acthar Gel 80 U (1 mL) [Received Placebo in DBT]
ArthralgiaMusculoskeletal and connective tissue disorders2/278/282/221/25
HeadacheNervous system disorders4/277/281/220/25
Diabetes mellitusMetabolism and nutrition disorders6/271/281/221/25
Upper respiratory tract infectionInfections and infestations5/272/281/220/25
Injection site bruisingGeneral disorders2/275/281/222/25
CoughRespiratory, thoracic and mediastinal disorders2/275/283/222/25
FatigueGeneral disorders4/274/282/222/25
NauseaGastrointestinal disorders2/272/283/221/25
BronchitisInfections and infestations1/272/283/220/25
Gastrooesophageal reflux diseaseGastrointestinal disorders1/271/281/223/25

Baseline characteristics

The safety population included all randomized participants who received at least 1 dose of IMP.

Age, Continuous
Age, Continuous(years)Acthar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLETotal
Mean53.6 ± 9.0754.8 ± 11.3354.2 ± 10.21
Sex: Female, Male
Sex: Female, Male(Participants)Acthar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLETotal
Female111526
Male161329
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Acthar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLETotal
Hispanic or Latino448
Not Hispanic or Latino232447
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Acthar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLETotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American61319
White211536
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)Acthar Gel in DBT Then Acthar Gel in OLEPlacebo in DBT Then Acthar Gel in OLETotal
United States272855
07

Study locations

29 sites
  • UAB Lung Health Center
    Birmingham, Alabama 35294, United States
  • David Geffen School of Medicine
    Los Angeles, California 90095, United States
  • National Jewish Health
    Denver, Colorado 80206, United States
  • University of Florida Division of Pulmonary, Critical Care, and Sleep Medicine
    Gainesville, Florida 32610, United States
  • University of Miami Miller School of Medicine
    Miami, Florida 33125, United States
  • Central Florida Pulmonary Group PA
    Orlando, Florida 32803, United States
  • Rush University Medical Center
    Chicago, Illinois 60612, United States
  • Laporte County Institute For Clinical Research
    Michigan City, Indiana 46360, United States
  • University of Kansas Medical Center
    Kansas City, Kansas 66160, United States
  • Howard County Center for Lung and Sleep Medicine, LLC
    Columbia, Maryland 21044, United States
  • Washington University School of Medicine
    Saint Louis, Missouri 63110, United States
  • Valley Medical Group
    Ridgewood, New Jersey 07450, United States
  • Albany Medical Center
    Albany, New York 12208, United States
  • American Health Research Inc
    Charlotte, North Carolina 28207, United States
  • Duke University Medical Center
    Durham, North Carolina 27710, United States
  • Clinical Research of Gastonia
    Gastonia, North Carolina 28054, United States
  • University of Cincinnati Medical Center
    Cincinnati, Ohio 45267, United States
  • Cleveland Clinic
    Cleveland, Ohio 44195, United States
  • Penn State Milton S Hershey Medical Center
    Hershey, Pennsylvania 17033, United States
  • Temple Lung Center
    Philadelphia, Pennsylvania 19140, United States
  • University of Pittsburgh Medical Center
    Pittsburgh, Pennsylvania 15213, United States
  • Berks Schuylkill Respiratory Specialists, Ltd
    Wyomissing, Pennsylvania 19610, United States
  • VitaLink Research - Anderson
    Anderson, South Carolina 29621, United States
  • Medical University of South Carolina - PPDS
    Charleston, South Carolina 29425, United States
  • Clinical Research of Charleston
    Mount Pleasant, South Carolina 29464, United States
  • Clinical Research of Rock Hill
    Rock Hill, South Carolina 29732, United States
  • VitaLink Research - Spartanburg
    Spartanburg, South Carolina 29303, United States
  • University of Texas Southwestern Medical Center
    Dallas, Texas 75390, United States
  • University of Texas Health Science Center at Tyler
    Tyler, Texas 75708, United States
08

References and documents

Study documents

  • Study protocol · May 18, 2020
  • Statistical analysis plan · Feb 2, 2022

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

09

Registry details

Key details

Study ID
NCT03320070
Lead sponsor
Mallinckrodt
Responsible party
Sponsor
First posted
Oct 25, 2017
Start date
Feb 21, 2018
Primary completion
Nov 15, 2021
Completion
Nov 15, 2021
Results posted
Feb 27, 2023
Last update
Feb 27, 2023

Study contacts

Clinical Team Leader
study director · Mallinckrodt

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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