Acthar Gel in Participants With Pulmonary Sarcoidosis (PULSAR)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03320070 |
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Recruitment Status :
Active, not recruiting
First Posted : October 25, 2017
Last Update Posted : May 1, 2020
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Sarcoidosis, Pulmonary | Drug: Acthar Gel Drug: Placebo | Phase 4 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 100 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Double (Participant, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 4, Multicenter, Randomized, Double Blind, Placebo Controlled Pilot Study to Assess the Efficacy and Safety of Acthar Gel in Subjects With Pulmonary Sarcoidosis |
| Actual Study Start Date : | January 24, 2018 |
| Estimated Primary Completion Date : | November 2021 |
| Estimated Study Completion Date : | November 2021 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Acthar Gel
Participants receive Acthar Gel as a 1 mL injection under the skin twice weekly
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Drug: Acthar Gel
Acthar Gel for subcutaneous (SC) injection (80 units per 1 mL)
Other Names:
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Placebo Comparator: Placebo
Participants receive Placebo as a 1 mL injection under the skin twice weekly
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Drug: Placebo
Placebo gel for SC injection
Other Name: Matching Placebo |
- Number of Participants in each Category of Assessment based on Forced Vital Capacity, a Pulmonary Function Test Parameter [ Time Frame: 24 weeks ]
Based on absolute change of percent predicted, FVC is 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]
- Number of Participants in each Category of Assessment based on Forced Vital Capacity, a Pulmonary Function Test Parameter [ Time Frame: 48 weeks ]
Based on absolute change of percent predicted, FVC is 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]
- 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 [ Time Frame: 24 weeks ]
Based on absolute change of percent predicted, DLCO is evaluated to determine if the condition is:
- Improved (+1) [≥ 5% absolute change]
- Unchanged (0) [>- 5% to < 5% absolute change],
- Worse (-1) [≤ -5% absolute change]
- 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 [ Time Frame: 48 weeks ]
Based on absolute change of percent predicted, DLCO is evaluated to determine if the condition is:
- Improved (+1) [≥ 5% absolute change]
- Unchanged (0) [>- 5% to < 5% absolute change],
- Worse (-1) [≤ -5% absolute change]
- Number of Participants in each Category of Assessment based on High Resolution Computer Tomography (HRCT) [ Time Frame: 24 weeks ]HRCT imaging will be evaluated by the investigator/radiology and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1).
- Number of Participants in each Category of Assessment based on High Resolution Computer Tomography (HRCT) [ Time Frame: 48 weeks ]HRCT imaging will be evaluated by the investigator/radiology and the central reader to determine if the condition is improved (+1), unchanged (0), or worse (-1).
- Number of participants in each Category of Assessment based on the King's Sarcoidosis Questionnaire (General Health), a Quality of Life Parameter [ Time Frame: 24 weeks ]
King's Sarcoidosis Questionnaire (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale is 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
- Number of participants in each Category of Assessment based on the King's Sarcoidosis Questionnaire (General Health), a Quality of Life Parameter [ Time Frame: 48 weeks ]
King's Sarcoidosis Questionnaire (General Health) is a 28-item questionnaire for participants to indicate the status of their sarcoidosis and treatment. Higher scores indicate improvement, and a change of 4 points is considered clinically meaningful. The score on the scale is 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
- Number of participants in each Category of Assessment based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter [ Time Frame: 24 weeks ]
The FAS is a 10-item checklist for participants to indicate the level of their fatigue. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale is 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
- Number of participants in each Category of Assessment based on the Fatigue Assessment Score (FAS), a Quality of Life Parameter [ Time Frame: 48 weeks ]
The FAS is a 10-item checklist for participants to indicate the level of their fatigue. Lower scores indicate improvement (less fatigue) and a change of 4 points is considered clinically meaningful. The score on the scale is 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
- Number of Participants Receiving each Dose of Prednisone [ Time Frame: 24 weeks ]
Corticosteroids are typically the first-line when treatment of sarcoidosis is required. Concerns of significant corticosteroid toxicity results in efforts to taper as early as possible. Participants are evaluated at each visit following randomization, and an algorithm is used to taper them off prednisone using incremental doses of 40, 30, 20, 10, 7.5, 5, 2.5 and 0 mg.
When the clinical status is:
- Improvement, they go down by one level
- First stable visit without toxicity, they continue the same dose
- Second stable visit without toxicity, the go down by one level
- Stable visit with toxicity, their toxicity is treated and they may go down by one level
- Worsening, they go up by one or two levels, but do not exceed 40 mg/day
- Number of Participants Receiving each Dose of Prednisone [ Time Frame: 48 weeks ]
Corticosteroids are typically the first-line when treatment of sarcoidosis is required. Concerns of significant corticosteroid toxicity results in efforts to taper as early as possible. Participants are evaluated at each visit following randomization, and an algorithm is used to taper them off prednisone using incremental doses of 40, 30, 20, 10, 7.5, 5, 2.5 and 0 mg.
When the clinical status is:
- Improvement, they go down by one level
- First stable visit without toxicity, they continue the same dose
- Second stable visit without toxicity, the go down by one level
- Stable visit with toxicity, their toxicity is treated and they may go down by one level
- Worsening, they go up by one or two levels, but do not exceed 40 mg/day
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 90 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | 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:
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03320070
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| Study Director: | Clinical Team Leader | Mallinckrodt |
| Responsible Party: | Mallinckrodt |
| ClinicalTrials.gov Identifier: | NCT03320070 |
| Other Study ID Numbers: |
MNK14344100 |
| First Posted: | October 25, 2017 Key Record Dates |
| Last Update Posted: | May 1, 2020 |
| Last Verified: | April 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
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Sarcoidosis, Pulmonary Sarcoidosis Lymphoproliferative Disorders Lymphatic Diseases Lung Diseases, Interstitial Lung Diseases |
Respiratory Tract Diseases Adrenocorticotropic Hormone Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Physiological Effects of Drugs |