A Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Intravenous ATYR1923 in Patients With Pulmonary Sarcoidosis

ATYR1923-C-002

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).

2019-01-29

2021-06-29

2021-06-29

Completed

Early phase I

37

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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': '36 participants with pulmonary sarcoidosis will be randomized into one of 3 sequential cohorts, each comprising 12 participants allocated 2:1 to efzofitimod:placebo matched to efzofitimod', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 37, 'type': 'ACTUAL'}}

2023-07-18