Sarcoidosis, Pulmonary
Conditions
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.
Interventions
DRUGActhar Gel
Acthar Gel for subcutaneous (SC) injection (80 units per 1 mL)
DRUGPlacebo
Placebo gel for SC injection
Sponsors
Mallinckrodt
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
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
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | 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. |
| OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | 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. |
| 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 | 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. |
| OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | 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. |
| DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | 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. |
| OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | 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. |
| 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 | 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. |
| OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | 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. |
| DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | 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. |
| OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | 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. |
| DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | 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. |
| OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | 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. |
Countries
United States
Participant flow
Recruitment details
Participants took part in the study at 35 investigative sites in the United States from 21 February 2018 to 15 November 2021.
Pre-assignment details
The study consisted of 2 phases: a double-blind treatment (DBT) phase and an optional open-label extension (OLE) phase. Out of the 49 participants who completed the DBT phase, 47 participants chose to continue the treatment in the optional OLE phase.
Participants by arm
| Arm | Count |
|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE Participants received Acthar Gel 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 OLE phase. | 27 |
| 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 OLE phase. | 28 |
| Total | 55 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| DBT Phase (Week 1 to Week 24) | Adverse Event | 1 | 1 |
| DBT Phase (Week 1 to Week 24) | Withdrawal by Subject | 2 | 2 |
| OLE Phase (Week 25 to Week 48) | Adverse Event | 3 | 0 |
| OLE Phase (Week 25 to Week 48) | Lost to Follow-up | 0 | 1 |
| OLE Phase (Week 25 to Week 48) | Non-compliance with Study Drug | 1 | 0 |
| OLE Phase (Week 25 to Week 48) | Withdrawal by Subject | 1 | 1 |
Baseline characteristics
| Characteristic | Acthar Gel in DBT Then Acthar Gel in OLE | Total | Placebo in DBT Then Acthar Gel in OLE |
|---|---|---|---|
| Age, Continuous | 53.6 years STANDARD_DEVIATION 9.07 | 54.2 years STANDARD_DEVIATION 10.21 | 54.8 years STANDARD_DEVIATION 11.33 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants | 8 Participants | 4 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 23 Participants | 47 Participants | 24 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 6 Participants | 19 Participants | 13 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 21 Participants | 36 Participants | 15 Participants |
| Region of Enrollment United States | 27 participants | 55 participants | 28 participants |
| Sex: Female, Male Female | 11 Participants | 26 Participants | 15 Participants |
| Sex: Female, Male Male | 16 Participants | 29 Participants | 13 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 27 | 0 / 28 | 0 / 22 | 0 / 25 |
| other Total, other adverse events | 22 / 27 | 25 / 28 | 14 / 22 | 14 / 25 |
| serious Total, serious adverse events | 4 / 27 | 3 / 28 | 6 / 22 | 0 / 25 |
Outcome results
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: 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
Population: The mITT population included all randomized participants who received at least 1 dose of the IMP and who contributed any efficacy data to the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Improved | 17 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Unchanged | 8 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Deteriorate | 0 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Missing Assessment | 2 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Missing Assessment | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Improved | 21 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Deteriorate | 0 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24 | Unchanged | 4 Participants |
p-value: 0.1853Chi-squared
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.
Time frame: Week 24
Population: The mITT population included all randomized participants who received at least 1 dose of the IMP and who contributed any efficacy data to the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Improved | 10 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Unchanged | 12 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Deteriorate | 1 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Missing Assessment | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Missing Assessment | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Improved | 7 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Deteriorate | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24 | Unchanged | 12 Participants |
p-value: 0.5076Chi-squared
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.
Time frame: Week 24
Population: The mITT population included all randomized participants who received at least 1 dose of the IMP and who contributed any efficacy data to the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Improved | 2 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Unchanged | 22 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Deteriorate | 0 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Missing Assessment | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Missing Assessment | 5 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Improved | 1 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Deteriorate | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24 | Unchanged | 18 Participants |
p-value: 0.0848Chi-squared
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.
Time frame: Week 24
Population: The mITT population included all randomized participants who received at least 1 dose of the IMP and who contributed any efficacy data to the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Improved | 5 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Unchanged | 11 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Deteriorate | 7 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Missing Assessment | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Missing Assessment | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Improved | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Deteriorate | 7 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Unchanged | 11 Participants |
p-value: 1Chi-squared
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.
Time frame: Week 24
Population: The mITT population included all randomized participants who received at least 1 dose of the IMP and who contributed any efficacy data to the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Improved | 6 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Unchanged | 12 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Deteriorate | 6 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Missing Assessment | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Missing Assessment | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Improved | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Deteriorate | 7 Participants |
| Placebo in DBT Then Acthar Gel in OLE | DBT: Number of Participants in Each Category of Assessment Based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter at Week 24 | Unchanged | 11 Participants |
p-value: 0.9416Chi-squared
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.
Time frame: Week 24
Population: The mITT population included all randomized participants who received at least 1 dose of the IMP and who contributed any efficacy data to the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Improved | 11 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Unchanged | 3 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Deteriorate | 10 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | 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 | Missing Assessment | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Missing Assessment | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Improved | 7 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Deteriorate | 9 Participants |
| Placebo in DBT Then Acthar Gel in OLE | 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 | Unchanged | 8 Participants |
p-value: 0.2005Chi-squared
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: 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
Population: Open-label safety population included participants who continued into the OLE phase and received at least one dose of IMP during the OLE phase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Improved | 15 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Unchanged | 5 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Deteriorate | 0 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Missing Assessment | 2 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Missing Assessment | 1 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Improved | 19 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Deteriorate | 0 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 48 | Unchanged | 5 Participants |
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.
Time frame: Week 48
Population: Open-label safety population included participants who continued into the OLE phase and received at least one dose of IMP during the OLE phase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Improved | 7 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Unchanged | 5 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Deteriorate | 4 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Missing Assessment | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Missing Assessment | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Improved | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Deteriorate | 5 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FAS, a Quality of Life Parameter at Week 48 | Unchanged | 13 Participants |
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.
Time frame: Week 48
Population: Open-label safety population included participants who continued into the OLE phase and received at least one dose of IMP during the OLE phase
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Improved | 2 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Unchanged | 13 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Deteriorate | 1 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Missing Assessment | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Missing Assessment | 5 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Improved | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Deteriorate | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48 | Unchanged | 11 Participants |
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.
Time frame: Week 48
Population: Open-label safety population included participants who continued into the OLE phase and received at least one dose of IMP during the OLE phase
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Deteriorate | 1 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Improved | 1 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Missing Assessment | 7 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Unchanged | 13 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Missing Assessment | 4 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Improved | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Deteriorate | 2 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48 | Unchanged | 13 Participants |
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.
Time frame: Week 48
Population: Open-label safety population included participants who continued into the OLE phase and received at least one dose of IMP during the OLE phase
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Improved | 9 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Unchanged | 3 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Deteriorate | 4 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Missing Assessment | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Missing Assessment | 5 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Improved | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Deteriorate | 8 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48 | Unchanged | 6 Participants |
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.
Time frame: Week 48
Population: Open-label safety population included participants who continued into the OLE phase and received at least one dose of IMP during the OLE phase.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Improved | 8 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Unchanged | 2 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Deteriorate | 6 Participants |
| Acthar Gel in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Missing Assessment | 6 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Missing Assessment | 3 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Improved | 8 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Deteriorate | 9 Participants |
| Placebo in DBT Then Acthar Gel in OLE | OLE: Number of Participants in Each Category of Assessment Based on the KSQ (General Health), a Quality of Life Parameter at Week 48 | Unchanged | 5 Participants |