A Multicenter, Randomized, Double Blind, Placebo Controlled Exploratory Study to Assess the Efficacy and Safety of Acthar Gel in Subjects With Pulmonary Sarcoidosis
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Mallinckrodt
- 入组人数
- 55
- 试验地点
- 29
- 主要终点
- OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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.
干预措施: Acthar Gel (Drug)
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.
干预措施: Acthar Gel (Drug)
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.
干预措施: Placebo (Drug)
结局指标
主要结局
OLE: Number of Participants in Each Category of Assessment Based on FVC, a Pulmonary Function Test Parameter at Week 48
时间窗: 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.
OLE: Number of Participants in Each Category of Assessment Based on the DLCO, a Pulmonary Function Test Parameter at Week 48
时间窗: 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.
OLE: Number of Participants in Each Category of Assessment Based on HRCT at Week 48
时间窗: 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 Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO), a Pulmonary Function Test Parameter at Week 24
时间窗: 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.
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
时间窗: 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 FAS, a Quality of Life Parameter at Week 48
时间窗: 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 Forced Vital Capacity (FVC), a Pulmonary Function Test Parameter at Week 24
时间窗: 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 the KSQ (General Health), a Quality of Life Parameter at Week 48
时间窗: 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
时间窗: 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.
DBT: Number of Participants in Each Category of Assessment Based on High Resolution Computer Tomography (HRCT) at Week 24
时间窗: 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.
DBT: Number of Participants in Each Category of Assessment Based on Corticosteroid Taper Score in Participants Receiving Each Dose of Prednisone at Week 24
时间窗: 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
时间窗: 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.
次要结局
未报告次要终点