The purpose of this study is to compare the effectiveness and the safety of sarilumab in patients with glucocorticoid-dependent sarcoidosis.
The purpose of this study is to compare the effectivness and the safety of sarilumab in patients with glucocorticoid-dependent sarcoidosis. To demonstrate that sarilumab treatment will be effective for inducing and maintaining glucocorticoid-free remission in male or female patients with biopsy proven active, glucocorticoid-dependent sarcoidosis affecting the lungs, lymph nodes, liver, kidneys, spleen, bone, soft tissues, skin, and/or eyes.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
16
Stanford University
Palo Alto, California, United States
Number of Participants Without Sarcoidosis Flare (Flare-Free Survival)
The primary outcome was flare-free survival of sarilumab-treated patients compared to placebo-treated controls. Patients will be considered to have flared if they receive rescue medication including increased glucocorticoids, or if they discontinue the study treatment in order to start a different therapy.
Time frame: Week 16 to Week 28
Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted
FVC is a pulmonary function test, and is defined as the volume of air that can forcibly be blown out after taking a full breath. FVC% predicted is defined as FVC% of the patient divided by the average FVC% in the population for any person of similar age, sex and body composition.
Time frame: Baseline and week 16
Change From Baseline in Hemoglobin-corrected Diffusing Capacity for Carbon Monoxide (DLCO) Percent Predicted
DLCO is a pulmonary function test, and measures the partial pressure difference between inspired and expired carbon monoxide. DLCO% predicted is defined as DLCO% of the patient divided by the average DLCO% in the population for any person of similar age, sex and body composition.
Time frame: Baseline, and week 16
Change in Pulmonary Function (FEV1) Percent Predicted
FEV1 percent predicted is a normalized value of FEV1 calculated using the Knudson equation and based upon participant age, gender, and height.
Time frame: Baseline and week 16
Change From Baseline in Extrapulmonary Physician Organ Severity Tool (ePOST) Scale Score
Physician and patient assessments assessed using the extrapulmonary physician organ severity tool (ePOST). Score Description: 0: Not affected 1. Slight 2. Mild 3. Moderate 4. Moderate to severe 5. Severe 6. Very Severe 17 organ domains were rated and summed to create a total score (range 0-102, higher scores correspond with more severity).
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Time frame: Baseline, week 16, and week 28
Change From Baseline in Physician Disease Activity Visual Analogue Scale (VAS)
Physician rates patient's disease on a 100 mm VAS. Score range: 0 to 100, higher scores correspond to worse disease state.
Time frame: Baseline, week 16, and week 28
Change From Baseline in Patient Disease Activity Visual Analogue Scale (VAS)
Patient rates their disease on a 100 mm VAS. Score range: 0 to 100, higher scores correspond to worse disease state.
Time frame: Baseline, week 16, and week 28
Change From Baseline in FACIT-F Score (Fatigue Scale)
FACIT-F score Total score range: 0-52, lower scores correspond with more fatigue.
Time frame: Baseline, week 16, and week 28
Number of Tender and Swollen Joints Per 68/66 Joint Evaluation
The 66/68 Joint Count evaluates 68 joints for tenderness and pain with movement and 66 joints for swelling (hip joints can be evaluated for tenderness only, not for swelling. Joint evaluation score: 0: Absent 1: Present 9: Not applicable
Time frame: Baseline, week 16, and week 28
Sarcoidosis Activity and Severity Index for Cutaneous Sarcoidosis
Sarcoidosis Activity and Severity Index evaluates 7 parameters on a 0 to 4 scale, summed for an overall scale score of 0 to 28 (higher values indicate higher activity/severity).
Time frame: Baseline, week 16, and week 28
Change in Size of Sarcoidosis Lesions
Time frame: Baseline, week 16, and week 28
Change From Baseline in Serum Angiotensin Converting Enzyme
ACE is a serum marker that is increased in sarcoidosis. ACE is produced by epithelioid cells that are derived from recently-activated macrophages in granulomas; thus, ACE is an appropriate representative of whole-body granuloma.
Time frame: Baseline, week 16, and week 28
Change From Baseline in Serum C-Reactive Protein (CRP)
CRP is a protein made by the liver. The level of CRP increases when there's inflammation in the body.
Time frame: Baseline, week 16, and week 28
Change From Baseline in Erythrocyte Sedimentation Rate (ESR)
ESR is the rate at which red blood cells in anticoagulated whole blood descend in a standardized tube over a period of one hour.
Time frame: Baseline, week 16, and week 28
Change in Prednisone Dose
Time frame: Baseline, week 16, and week 28
Number of Participants With Alanine Aminotransferase (ALT) Outside Normal Range
Normal range as calculated by the local laboratory.
Time frame: Baseline, week 16, and week 28
Number of Participants With Aspartate Aminotransferase (AST) Outside Normal Range
Normal range as calculated by the local laboratory.
Time frame: Baseline, week 16, and week 28
Number of Participants With Serum Creatinine Outside Normal Range
Normal range as calculated by the local laboratory.
Time frame: Baseline, week 16, and week 28
Number of Participants With Urine Protein Outside Normal Range
Normal range as calculated by the local laboratory.
Time frame: Baseline, week 16, and week 28