study of intravenous atyr1923 (efzofitimod) for pulmonary sarcoidosis

Conditions: Pulmonary Sarcoidosis

Summary: this randomized, double-blind, placebo matched to efzofitimod-controlled, study will evaluate the safety, tolerability, immunogenicity, pharmacokinetic (pk), and preliminary efficacy of multiple ascending doses of iv efzofitimod in participants 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 efzofitimod (n=8) or placebo matched to efzofitimod (n=4).


Eligibility criteria:
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 evidenced 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 efzofitimod 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 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. * participant 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.

Keywords: pulmonary sarcoidosis resokine sarcoidosis granulomas inflammation lymphoproliferative disorders interstitial lung disease neuropilin-2 steroids oral corticosteroids immunomodulatory trna synthetase atyr1923 (efzofitimod)