Evidence map›Paper›PMID 36356657›Full record

Trial reportChest2023

Efzofitimod for the Treatment of Pulmonary Sarcoidosis.

Daniel A Culver, Shambhu Aryal, Joseph Barney, Connie C W Hsia, W Ennis James, Lisa A Maier, Lucian T Marts, Ogugua Ndili Obi, Peter H S Sporn, Nadera J Sweiss and 4 more

Registry-linked trialOpen access · hybridAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Chest, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03824392 (A Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study of Intravenous ATYR1923 in Patients With Pulmonary Sarcoidosis), which is not on this map. Cited by 23 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed, 2 pooled it
5.5field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03824392 phase1 / phase2completednot on this map

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

TypeinterventionalSponsoraTyr Pharma, Inc.Ran2019 to 2021Enrolled37ConditionsPulmonary SarcoidosisArmsEfzofitimod 1.0 mg/kg or Placebo, Efzofitimod 3.0 mg/kg or Placebo, Efzofitimod 5.0 mg/kg or Placebo
3 · Its place in the literature

Who cites it

23 citing papers in PubMed, 2 syntheses or guidelines pooled it, 50 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Review
  4. Review
  5. Quality of Life in Sarcoidosis.Diagnostics (Basel, Switzerland) · 2026
    Review
  6. Advances in management of sarcoidosis-associated fatigue: A narrative review.Sarcoidosis, vasculitis, and diffuse lung diseases : official journal of WASOG · 2026
    Article
  7. New Therapies for Sarcoidosis: Molecular and Pathophysiological Basis.International journal of molecular sciences · 2026
    Review
  8. Contemporary management of sarcoidosis.Frontiers in medicine · 2026
    Review
  9. Article
  10. Advanced Pulmonary Sarcoidosis: Treatment and Monitoring.Diagnostics (Basel, Switzerland) · 2025
    Review
  11. Article
  12. WASOG Clinical Trial Endpoint Task Force: Executive Summary.Sarcoidosis, vasculitis, and diffuse lung diseases : official journal of WASOG · 2025
    Article
  13. Article
  14. Autoantibodies in sarcoidosis: Innocent bystander or promising biomarker for organ involvement?Sarcoidosis, vasculitis, and diffuse lung diseases : official journal of WASOG · 2024
    Article
  15. Article
  16. Review
  17. Review
  18. D. Geraint James Lecture: The sarcoidosis saga: what insights from the past will guide us in the future.Sarcoidosis, vasculitis, and diffuse lung diseases : official journal of WASOG · 2023
    Article
  19. Review
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors at 12 institutions in 1 country.

Daniel A CulverCleveland Clinic, Cleveland, OH. Electronic address: culverd@ccf.org.
Shambhu AryalAdvanced Lung Disease and Lung Transplant Program, Inova Fairfax Hospital, Falls Church, VA.
Joseph BarneyDepartment of Pulmonary and Critical Care Medicine, University of Alabama, Birmingham, AL.
Connie C W HsiaDivision of Pulmonary and Critical Care Medicine, Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX.
W Ennis JamesSusan Pearlstine Sarcoidosis Center of Excellence, Pulmonary and Critical Care Medicine, Medical University of South Carolina, Charleston, SC.
Lisa A MaierDivision of Environmental and Occupational Health Sciences, National Jewish Health; Division of Pulmonary Sciences and Critical Care, Department of Medicine, School of Medicine, University of Colorado, Denver, CO.
Lucian T MartsDivision of Pulmonary, Allergy, Critical Care, and Sleep Medicine, Department of Medicine, Emory University School of Medicine, Atlanta, GA.
Ogugua Ndili ObiDivision of Pulmonary, Critical Care and Sleep Medicine, Department of Internal Medicine, Brody School of Medicine East Carolina University, Greenville, NC.
Peter H S SpornDivision of Pulmonary and Critical Care Medicine, Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL.
Nadera J SweissDivision of Rheumatology and Medical Director of the Arthritis Clinic, Chicago, IL; Bernie Mac Sarcoidosis Translational Advanced Research Center, University of Illinois College of Medicine, Chicago, IL.
Sanjay ShuklaaTyr Pharma, Inc., San Diego, CA.
Nelson KinnersleyOcta Consulting Services, Ltd., Harpenden, England.
Gennyne WalkeraTyr Pharma, Inc., San Diego, CA.
Robert BaughmanDepartment of Medicine, University of Cincinnati Medical Center, Cincinnati, OH.
aTyr Pharma (United States) · USCleveland Clinic · USEast Carolina University · USEmory University · USInova Fairfax Hospital · USMedical University of South Carolina · USNational Jewish Health · USNorthwestern University · USThe University of Texas Southwestern Medical Center · USUniversity of Alabama at Birmingham · USUniversity of Cincinnati · USUniversity of Illinois Urbana-Champaign · US

Funding

A Prospective Study of T cell Immune-phenotype and Disease Outcome in Pulmonary SarcoidosisR01HL136681 · NHLBI · NATIONAL JEWISH HEALTH · PI HAMZEH, NABEEL, KOTH, LAURA L · 2018 to 2021
$4.0M
Epigenetic Regulation of Immune Pathways in SarcoidosisR01HL140357 · NHLBI · NATIONAL JEWISH HEALTH · PI MAIER, LISA A, YANG, IVANA V · 2018 to 2021
$2.8M
Novel integrative approaches for disease phenotyping, utilizing radiomics in SarcoidosisR01HL142049 · NHLBI · NATIONAL JEWISH HEALTH · PI CARLSON, NICHOLE, FINGERLIN, TASHA E. · 2019 to 2022
$2.6M
Hypercapnia and Suppression of Anti-viral Host DefenseR01HL131745 · NHLBI · NORTHWESTERN UNIVERSITY AT CHICAGO · PI SPORN, PETER H · 2017 to 2019
$1.2M
2018 Meeting of the American Association of Sarcoidosis and Other Granulomatous Disorders (AASOG)R13HL142300 · NHLBI · NORTHWESTERN UNIVERSITY AT CHICAGO · PI SPORN, PETER H · 2018 to 2018
$5k
NHLBI NIH HHS R01 HL131745NHLBI NIH HHS R01 HL136681NHLBI NIH HHS R01 HL140357NHLBI NIH HHS R01 HL142049NHLBI NIH HHS R13 HL142300
6 · The paper itself

Abstract

backgroundPulmonary sarcoidosis is characterized by the accumulation of immune cells that form granulomas affecting the lungs. Efzofitimod (ATYR1923), a novel immunomodulator, selectively binds neuropilin 2, which is upregulated on immune cells in response to lung inflammation. RESEARCH QUESTION: What is the tolerability, safety, and effect on outcomes of efzofitimod in pulmonary sarcoidosis? STUDY DESIGN AND

methodsIn this randomized, double-blind, placebo-controlled study evaluating multiple ascending doses of efzofitimod administered intravenously every 4 weeks for 24 weeks, randomized patients (2:1) underwent a steroid taper to 5 mg/d by week 8 or < 5 mg/d after week 16. The primary end point was the incidence of adverse events (AEs); secondary end points included steroid reduction, change in lung function, and patient-reported outcomes on health-related quality-of-life scales.

resultsThirty-seven patients received at least one dose of study medication. Efzofitimod was well tolerated at all doses, with no new or unexpected AEs and no dose-dependent AE incidence. Average daily steroid doses through end of study were 6.8 mg, 6.5 mg, and 5.6 mg for the 1 mg/kg, 3 mg/kg, and 5 mg/kg groups compared with 7.2 mg for placebo, resulting in a baseline-adjusted relative steroid reduction of 5%, 9%, and 22%, respectively. Clinically meaningful improvements were achieved across several patient-reported outcomes, several of which reached statistical significance in the 5 mg/kg dose arm. A dose-dependent but nonsignificant trend toward improved lung function also was observed for 3 and 5 mg/kg.

interpretationEfzofitimod was safe and well tolerated and was associated with dose-dependent improvements of several clinically relevant end points compared with placebo. The results of this study support further evaluation of efzofitimod in pulmonary sarcoidosis. TRIAL REGISTRY: ClinicalTrials.gov; No.: NCT03824392; URL: www. CLINICALTRIALS: gov.

Indexed as

Sarcoidosis, PulmonaryHumansLungATYR1923corticosteroidsefzofitimodfatigue assessment scaleimmunomodulatorlung functionneuropilin 2pulmonary sarcoidosisquality of lifesteroid taper

Identifiers

PMID36356657
PMCPMC10258437
OpenAlexW4308381477

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.