Trial Design and Procedures
sarcoidosis, pulmonary sarcoidosis
ADVERSE EVENTS, THORACIC, PULMONARY SARCOIDOSIS, SARCOIDOSIS
Patients were 18 to 75 years of age, had a diagnosis of pulmonary sarcoidosis for ≥ 6 months according to the 1999 American Thoracic Society standards,This was a randomized, double-blind, placebo-controlled multiple ascending dose study with three sequential dose cohorts with a 2:1 randomization (efzofitimod to placebo) in each cohort; the planned study size was 36 patients (Safety and tolerability assessments consisted of evaluation of treatment-emergent adverse events (TEAEs), physical examinations, vital signs and temperature, 12-lead ECGs, pulse oximetry, weight, immunogenicity, and clinical laboratory tests. Assessment of daily corticosteroid dose over the study period (day 1-week 24) and the number of patients who achieved the targeted tapered dose of prednisone 5 mg/day (or equivalent) through week 24 also were recorded. Pulmonary function tests including the FVC % predicted and diffusing capacity of the lungs for carbon monoxide (DStarting on day 15, patients began a taper (reduction) of corticosteroid from the starting dose of 10 to 25 mg/d of prednisone (or equivalent) to a target dose of 5 mg/d, which was to be completed on or before day 50. The corticosteroid dose was to be tapered every 1 to 2 weeks, depending on the starting dose, with smaller incremental titrations allowed per the investigator’s judgment, as long as the patient reached the goal dose by day 50. Patients were maintained at the target corticosteroid dose of 5 mg/d (or equivalent) through week 24. Optional titrations in the corticosteroid dose < 5 mg/d could occur after week 16 if the investigator determined further titration to be feasible. Patients who demonstrated acute worsening of sarcoidosis symptoms or who were unable to tolerate the taper were allowed to receive rescue treatment with higher corticosteroid doses per the site investigators’ clinical judgement; on resolution of symptoms, the taper could be reattempted per the investigators’ judgment.