Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

June 2, 2026 updated by: aTyr Pharma, Inc.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

This is a multicenter, randomized, double-blind, placebo-controlled, study comparing the efficacy and safety of intravenous (IV) efzofitimod 3 mg/kg and 5 mg/kg versus placebo after 48 weeks of treatment. This study will enroll adults with histologically confirmed pulmonary sarcoidosis receiving stable treatment with oral corticosteroid (OCS), with or without immunosuppressant therapy.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
268

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Brazil
      • São Bernardo do Campo, Brazil, 09780-000
        • aTyr Investigative Site
      • São Paulo, Brazil, 04039-901
        • aTyr Investigative Site
      • São Paulo, Brazil, 05403-900
        • aTyr Investigative Site
    • Estado de Bahia
      • Salvador, Estado de Bahia, Brazil, 40220-005
        • aTyr Investigative Site
    • Rio Grande do Sul
      • Porto Alegre, Rio Grande do Sul, Brazil, 90020-090
        • aTyr Investigative Site
  • France
      • Bobigny, France, 93000
        • aTyr Investigative Site
      • La Tronche, France, 38700
        • aTyr Investigative Site
      • Montpellier, France, 34295
        • aTyr Investigative Site
      • Paris, France, 75015
        • aTyr Investigative Site
      • Toulouse, France, 31000
        • aTyr Investigative Site
  • Germany
      • Essen, Germany, 45122
        • aTyr Investigative Site
      • Freiburg im Breisgau, Germany, 79106
        • aTyr Investigative Site
      • Hanover, Germany, 30625
        • aTyr Investigative Site
      • Heidelberg, Germany, 69126
        • aTyr Investigative Site
  • Italy
      • Catania, Italy, 95124
        • aTyr Investigative Site
      • Florence, Italy, 50134
        • aTyr Investigative Site
      • Forlì, Italy, 47121
        • aTyr Investigative Site
      • Milan, Italy, 20123
        • aTyr Investigative Site
      • Naples, Italy, 80131
        • aTyr Investigative Site
      • Padua, Italy, 35128
        • aTyr Investigative Site
      • Rome, Italy, 00168
        • aTyr Investigative Site
      • Siena, Italy, 53100
        • aTyr Investigative Site
      • Trieste, Italy, 34149
        • aTyr Investigative Site
  • Japan
      • Aomori, Japan, 036-8563
        • Kyorin Investigative Site
      • Fukuoka, Japan, 814-0180
        • Kyorin Investigative Site
      • Fukushima, Japan, 960-1295
        • Kyorin Investigative Site
      • Hokkaido, Japan, 060-0033
        • Kyorin Investigative Site
      • Hokkaido, Japan, 060-8648
        • Kyorin Investigative Site
      • Hyōgo, Japan, 653-0013
        • Kyorin Investigative Site
      • Kanagawa, Japan, 236-0004
        • Kyorin Investigative Site
      • Kanagawa, Japan, 236-0051
        • Kyorin Investigative Site
      • Kumamoto, Japan, 860-8556
        • aTyr Investigative Site
      • Miyagi, Japan, 980-8574
        • Kyorin Investigative Site
      • Okayama, Japan, 700-8558
        • Kyorin Investigative Site
      • Osaka, Japan, 591-8025
        • Kyorin Investigative Site
      • Shimane, Japan, 693-8501
        • Kyorin Investigative Site
      • Shizuoka, Japan, 431-3192
        • Kyorin Investigative Site
      • Tokyo, Japan, 150-8935
        • Kyorin Investigative Site
      • Tokyo, Japan, 151-8528
        • Kyorin Investigative Site
  • Netherlands
      • Nieuwegein, Netherlands, 3435CM
        • aTyr Investigative Site
  • Spain
      • Barcelona, Spain, 08036
        • aTyr Investigative Site
      • Barcelona, Spain, 08907
        • aTyr Investigative Site
      • Madrid, Spain, 28006
        • aTyr Investigative Site
      • Madrid, Spain, 28031
        • aTyr Investigative Site
      • Santander, Spain, 39008
        • aTyr Investigative Site
      • Valencia, Spain, 46010
        • aTyr Investigative Site
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TH
        • aTyr Investigative Site
      • Cambridge, United Kingdom, CB233RE
        • aTyr Investigative Site
      • Cottingham, United Kingdom, HU16 5JQ
        • aTyr Investigative Site
      • Coventry, United Kingdom, CV2 2DX
        • aTyr Investigative Site
      • London, United Kingdom, SE59RS
        • aTyr Investigative Site
      • London, United Kingdom, SW3 6NP
        • aTyr Investigative Site
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • aTyr Investigative Site
    • Arizona
      • Phoenix, Arizona, United States, 85027
        • aTyr Investigative Site
    • California
      • Los Angeles, California, United States, 90033
        • aTyr Investigative Site
      • Sacramento, California, United States, 95817
        • aTyr Investigative Site
    • Colorado
      • Denver, Colorado, United States, 80206
        • aTyr Investigative Site
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20060
        • aTyr Investigative Site
    • Florida
      • Gainesville, Florida, United States, 32610
        • aTyr Investigative Site
      • Tampa, Florida, United States, 33606
        • aTyr Investigative Site
      • Weston, Florida, United States, 33331
        • aTyr Investigative Site
    • Georgia
      • Atlanta, Georgia, United States, 30308
        • aTyr Investigative Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • aTyr Investigative Site
      • Chicago, Illinois, United States, 60611
        • aTyr Investigative Site
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • aTyr Investigative Site
    • Louisiana
      • New Orleans, Louisiana, United States, 70115
        • aTyr Investigative Site
    • Maryland
      • Baltimore, Maryland, United States, 21224
        • aTyr Investigative Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • aTyr Investigative Site
    • Michigan
      • Ada, Michigan, United States, 49301
        • aTyr Investigative Site
      • Detroit, Michigan, United States, 48202
        • aTyr Investigative Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • aTyr Investigative Site
      • Rochester, Minnesota, United States, 55905
        • aTyr Investigative Site
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • aTyr Investigative Site
    • Missouri
      • St Louis, Missouri, United States, 63110
        • aTyr Investigative Site
    • New York
      • Albany, New York, United States, 12208
        • aTyr Investigative Site
      • Middletown, New York, United States, 10941
        • aTyr Investigative Site
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • aTyr Investigative Site
      • Greenville, North Carolina, United States, 27834
        • aTyr Investigative Site
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • aTyr Investigative Site
      • Cleveland, Ohio, United States, 44195
        • aTyr Investigative Site
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • aTyr Investigative Site
    • Oregon
      • Portland, Oregon, United States, 97220
        • aTyr Investigative Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19140
        • aTyr Investigative Site
    • South Carolina
      • Charleston, South Carolina, United States, 29425-0630
        • aTyr Investigative Site
    • Tennessee
      • Dickson, Tennessee, United States, 37055
        • aTyr Investigative Site
    • Texas
      • Dallas, Texas, United States, 75246
        • aTyr Investigative Site
      • Dallas, Texas, United States, 75390
        • aTyr Investigative Site
      • Houston, Texas, United States, 77030
        • aTyr Investigative Site
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • aTyr Investigative Site
    • Virginia
      • Falls Church, Virginia, United States, 22042
        • aTyr Investigative Site
      • Richmond, Virginia, United States, 23298
        • aTyr Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Documented history of pulmonary sarcoidosis for at least 6 months, supported by the following evidence: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence
  • Evidence of symptomatic pulmonary sarcoidosis, as demonstrated by the following criteria: Modified Medical Research Council (MRC) dyspnea scale grade of at least 1 and KSQ-Lung score ≤70
  • Patients must be receiving treatment with OCS of ≥ 3 months at Day 1 with a starting dose between ≥ 7.5 and ≤ 25 mg/day ≥ 4 weeks prior to Day 1.
  • Body weight ≥ 40 kg and < 160 kg

Exclusion Criteria:

  • Treatment with > 1 immunosuppressant therapy
  • Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-α) inhibitors or antifibrotics or interleukin inhibitors
  • Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: High resolution CT fibrosis > 20% within the last 12 months; FVC percent predicted (FVCPP) < 50% and KSQ-Lung score < 30
  • In the opinion of the investigator, clinically significant pulmonary hypertension
  • Patients with active cardiac, neuro, or renal sarcoidosis requiring organ-specific therapy in the past 2 years
  • Patients with cutaneous or ocular sarcoidosis, which in the opinion of the Investigator, are at risk for exacerbation, necessitating OCS rescue or other systemic therapy
  • History of Addisonian symptoms that precluded previous OCS taper attempts
  • Is an active, heavy smoker of tobacco/nicotine-containing products
  • History of anti-synthetase syndrome or Jo-1 positive at Screening
  • Patients with active tuberculosis or those currently undergoing treatment for tuberculosis

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Drug: Placebo
Placebo IV infusion every 4 weeks for a total of 12 doses
Experimental: Efzofitimod 3 mg/kg
Drug: Efzofitimod 3 mg/kg
Efzofitimod IV infusion every 4 weeks for a total of 12 doses
Other Names:
  • ATYR1923
  • KRP-R120
Experimental: Efzofitimod 5 mg/kg
Drug: Efzofitimod 5 mg/kg
Efzofitimod IV infusion every 4 weeks for a total of 12 doses
Other Names:
  • ATYR1923
  • KRP-R120

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48
Time Frame: Baseline to Week 48
Baseline to Week 48

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Change from baseline in KSQ-Lung score at Week 48
Time Frame: Baseline to Week 48
Baseline to Week 48
Steroid withdrawal rate
Time Frame: Baseline to Week 48
Baseline to Week 48
Change from baseline in absolute value of FVC at Week 48
Time Frame: Baseline to Week 48
Baseline to Week 48

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
aTyr Pharma, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Kyorin Pharmaceutical Co.,Ltd

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Lisa Carey, aTyr Pharma, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 15, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 25, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 14, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 8, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 8, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 10, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 4, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 2, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ATYR1923-C-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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