Title

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
  • Phase

    Phase 1/Phase 2
  • Study Type

    Interventional
  • Status

    Completed No Results Posted
  • Study Participants

    37
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).
Study Started
Jan 29
2019
Primary Completion
Jun 29
2021
Study Completion
Jun 29
2021
Last Update
Jul 22
2021

Biological ATYR1923 1.0 mg/kg or placebo

ATYR1923 participants to receive ATYR1923 1.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks

Biological ATYR1923 3.0 mg/kg or placebo

ATYR1923 participants to receive ATYR1923 3.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks

Biological ATYR1923 5.0 mg/kg or placebo

ATYR1923 participants to receive ATYR1923 5.0 mg/kg IV every 4 weeks; Placebo participants to receive placebo IV every 4 weeks

Cohort 1 Experimental

ATYR1923 1.0 mg/kg or placebo

Cohort 2 Experimental

ATYR1923 3.0 mg/kg or placebo

Cohort 3 Experimental

ATYR1923 5.0 mg/kg or placebo

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 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.
No Results Posted