Study of Intravenous ATYR1923 for Pulmonary Sarcoidosis
A Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Intravenous ATYR1923 in Patients With Pulmonary Sarcoidosis
This randomized, double-blind, placebo-controlled, study will evaluate the safety, tolerability, immunogenicity, pharmacokinetic (PK), and preliminary efficacy of multiple ascending doses of IV ATYR1923 in patients with pulmonary sarcoidosis undergoing a protocol-guided oral corticosteroid (OCS) tapering regimen.This study will consist of 3 staggered multiple dose cohorts. Each eligible participant will participate in only one cohort during the study. Within each cohort, 12 participants will be randomized 2:1 to ATYR1923 (N=8) or placebo (N=4).
ATYR1923 participants to receive ATYR1923 1.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks
ATYR1923 participants to receive ATYR1923 3.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks
ATYR1923 participants to receive ATYR1923 5.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks
Key Inclusion Criteria: Diagnosis of pulmonary sarcoidosis for ≥6 months (cutaneous and ocular involvement allowed), defined as: Histologically proven diagnosis of sarcoidosis by bronchoscopy, biopsy (any organ) or bronchioalveolar lavage Parenchymal lung involvement by historical radiological evidence Must have symptomatic and/or active pulmonary sarcoidosis as evidence by: Modified Medical Research Council Dyspnea Scale grade of >= 1; and Forced vital capacity ≥50%; and Receiving treatment with 10 to 25 mg/day of oral prednisone (or equivalent), at a stable dose for ≥4 weeks prior to Day 1, and capable of undergoing the protocol-specified steroid taper regimen. Body weight ≥45 kg and <160 kg. Key Exclusion Criteria: Current disease presentation consistent with Lofgren's syndrome. History of severe allergic or anaphylactic reactions to therapeutic proteins or known sensitivity to ATYR1923 or to its inactive components (L-histidine, sodium chloride, sucrose, L-methionine, and polysorbate-20). Treatment with biological immunomodulators such as tumor necrosis factor-alpha inhibitors. Current evidence of clinically significant cardiovascular, hepatic, renal, hematological, metabolic, or gastrointestinal disease, or has a condition that requires other treatment. Clinically significant pulmonary hypertension requiring vasodilator treatment. Any history of tuberculosis or evidence of active systemic non-tuberculosis fungal or mycobacterial infection within 1 year of Screening. History of clinically significant cardiac, neurological, gastrointestinal, and/or renal manifestations of their sarcoidosis. Any condition that necessitated hospitalization within the 3 months prior to Day 1 or is likely to require so during the study. Participation in another clinical study of an investigational agent or device within 3 months (small molecules) / 6 months (biologics) or 5 half-lives (if known) of the agent, whichever is longer. History of or positive results of screening for hepatitis B, hepatitis C or human immunodeficiency virus. Is an active, heavy smoker of tobacco/nicotine-containing products (defined as >20 cigarettes/day or e-cigarette equivalent). Active substance abuse or history of substance abuse within the 12 months prior to Screening. Patient has received a live vaccination within 8 weeks before Day 1 or inoculation with a live vaccine is planned during study participation. Positive for Jo-1 antibodies (Ab) at Screening, or past history of Jo-1 Ab positivity. Significant and/or acute illness within 5 days prior to drug administration that may impact safety assessments, in the opinion of the Investigator.