Study of Intravenous ATYR1923 for 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).
- Pulmonary Sarcoidosis
- Eligible Ages
- Between 18 Years and 70 Years
- Eligible Genders
- Accepts Healthy Volunteers
- 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 percent predicted; and
- Positive 18F-fluorodeoxyglucose positron-emission tomography/computed tomography (18F-FDG-PET/CT) scan showing increased metabolic activity in lung parenchyma within 4 weeks prior to Day 1.
- 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.
- 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.
- Has been an active smoker (including e-cigarettes or e-vaporizers) within 3 months prior to Screening.
- 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.
- Phase 1/Phase 2
- Study Type
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- 36 participants with pulmonary sarcoidosis will be randomized into one of 3 sequential cohorts, each comprising 12 participants allocated 2:1 to ATYR1923:Placebo
- Primary Purpose
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
|ATYR1923 1.0 mg/kg or placebo||
|ATYR1923 3.0 mg/kg or placebo||
|ATYR1923 5.0 mg/kg or placebo||
- aTyr Pharma, Inc.
Study ContactaTyr Pharma Clinical Research