Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

May 15, 2025 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

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

268

Phase

  • 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 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
    • BA
      • Salvador, BA, Brazil, 40220-005
        • aTyr Investigative Site
    • RS
      • Porto Alegre, RS, 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, Germany, 79106
        • aTyr Investigative Site
      • Hannover, 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
      • Napoli, 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, 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
      • Saint 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

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
Intervention / Treatment
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
EfzofitimodIV 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
EfzofitimodIV infusion every 4 weeks for a total of 12 doses
Other Names:
  • ATYR1923
  • KRP-R120

What is the study measuring?

Primary Outcome Measures

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

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

aTyr Pharma, Inc.

Collaborators

Kyorin Pharmaceutical Co.,Ltd

Investigators

  • Study Director: Lisa Carey, aTyr Pharma, Inc.

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)

September 15, 2022

Primary Completion (Estimated)

July 31, 2025

Study Completion (Estimated)

August 31, 2025

Study Registration Dates

First Submitted

June 8, 2022

First Submitted That Met QC Criteria

June 8, 2022

First Posted (Actual)

June 10, 2022

Study Record Updates

Last Update Posted (Estimated)

May 20, 2025

Last Update Submitted That Met QC Criteria

May 15, 2025

Last Verified

May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID 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.

Clinical Trials on Pulmonary Sarcoidosis

Clinical Trials on Efzofitimod 3 mg/kg

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Philippines

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe