Phase 2
N=37
Study of Intravenous ATYR1923 (Efzofitimod) for Pulmonary Sarcoidosis
Pulmonary Sarcoidosis
Bottom Line
View on ClinicalTrials.gov: NCT03824392 ↗Enrolled (actual)
37
Serious AEs
5.4%
Results posted
Jul 2023
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 10; 8; 7; 8 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Efzofitimod 1.0 mg/kg or Placebo (Biological); Efzofitimod 3.0 mg/kg or Placebo (Biological); Efzofitimod 5.0 mg/kg or Placebo (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- aTyr Pharma, Inc.
- Primary completion
- Jun 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
10; 8; 7; 8; 1; 1 | — |
| SECONDARY Time-adjusted Area Under the Curve (AUC) of Background Oral Corticosteroid (OCS) Usage Over Study Period |
8.64; 6.83; 8.36; 7.43 | — |
| SECONDARY Number of Participants Who Achieved and Maintained The Targeted Tapered Dose of Prednisone 5 mg/Day (or Equivalent) |
7; 1; 4; 5 | — |
| SECONDARY Number of Participants With Positive Anti-Drug Antibodies (Anti-Efzofitimod) |
1; 0; 1; 0 | — |
| SECONDARY Number of Participants With at Least One Positive Anti-Jo-1 Antibodies Titers |
0; 0; 0; 0 | — |
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 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.
Data sourced from ClinicalTrials.gov (NCT03824392). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.