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Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05415137
Recruitment Status : Recruiting
First Posted : June 10, 2022
Last Update Posted : April 13, 2023
Sponsor:
Collaborator:
Kyorin Pharmaceutical Co.,Ltd
Information provided by (Responsible Party):
aTyr Pharma, Inc.

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Pulmonary Sarcoidosis Drug: Efzofitimod 3 mg/kg Drug: Efzofitimod 5 mg/kg Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 264 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis
Actual Study Start Date : September 15, 2022
Estimated Primary Completion Date : December 15, 2024
Estimated Study Completion Date : January 15, 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Sarcoidosis

Arms and Interventions
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Arm Intervention/treatment
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
Placebo Comparator: Placebo Drug: Placebo
Placebo IV infusion every 4 weeks for a total of 12 doses


Outcome Measures
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Primary Outcome Measures :
  1. Change from baseline in mean daily oral corticosteroid (OCS) dose post-taper [ Time Frame: Baseline to Week 48 ]

Secondary Outcome Measures :
  1. Annual rate of change in absolute value of Forced vital capacity (FVC) [ Time Frame: Baseline to Week 48 ]
  2. Percent change from baseline in mean daily OCS dose post-taper [ Time Frame: Baseline to Week 48 ]
  3. Change from baseline in King's Sarcoidosis Questionnaire (KSQ)-Lung score [ Time Frame: Baseline to Week 48 ]

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed diagnosis of pulmonary sarcoidosis for at least 6 months, defined by the following criteria: 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 with a starting dose between ≥ 7.5 and ≤ 25 mg/day.
  • Body weight ≥ 40 kg and < 160 kg

Exclusion Criteria:

  • Treatment with > 1 oral 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
  • Clinically significant pulmonary hypertension requiring treatment with vasodilators
  • Patients with cardiac sarcoidosis, neurosarcoidosis, or renal sarcoidosis
  • Clinically significant cutaneous and ocular sarcoidosis
  • 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 baseline
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05415137


Contacts
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Contact: aTyr Pharma Clinical Research 877-215-5731 clinicaltrials@atyrpharma.com

Locations
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United States, Alabama
aTyr Investigative Site Recruiting
Birmingham, Alabama, United States, 35294
Contact    877-596-6699    efzofit@cssienroll.com   
United States, California
aTyr Investigative Site Recruiting
Los Angeles, California, United States, 90033
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Newport Beach, California, United States, 92663
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Valencia, California, United States, 23823
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Colorado
aTyr Investigative Site Recruiting
Denver, Colorado, United States, 80206
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Florida
aTyr Investigative Site Recruiting
Gainesville, Florida, United States, 32610
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Georgia
aTyr Investigative Site Recruiting
Atlanta, Georgia, United States, 30322
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Illinois
aTyr Investigative Site Recruiting
Chicago, Illinois, United States, 60611
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Chicago, Illinois, United States, 60612
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Kentucky
aTyr Investigative Site Not yet recruiting
Louisville, Kentucky, United States, 40202
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Louisiana
aTyr Investigative Site Recruiting
New Orleans, Louisiana, United States, 70115
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Maryland
aTyr Investigative Site Recruiting
Baltimore, Maryland, United States, 21224
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Minnesota
aTyr Investigative Site Recruiting
Minneapolis, Minnesota, United States, 55445
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Rochester, Minnesota, United States, 55905
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Missouri
aTyr Investigative Site Recruiting
Saint Louis, Missouri, United States, 63110
Contact    877-596-6699    efzofit@cssienroll.com   
United States, New York
aTyr Investigative Site Recruiting
Albany, New York, United States, 12208
Contact    877-596-6699    efzofit@cssienroll.com   
United States, North Carolina
aTyr Investigative Site Recruiting
Durham, North Carolina, United States, 27710
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Greenville, North Carolina, United States, 27834
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Ohio
aTyr Investigative Site Recruiting
Cincinnati, Ohio, United States, 45267
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Cleveland, Ohio, United States, 44195
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Oklahoma
aTyr Investigative Site Recruiting
Oklahoma City, Oklahoma, United States, 73104
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Oregon
aTyr Investigative Site Recruiting
Portland, Oregon, United States, 97220
Contact    877-596-6699    efzofit@cssienroll.com   
United States, South Carolina
aTyr Investigative Site Recruiting
Charleston, South Carolina, United States, 29425-0630
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Tennessee
aTyr Investigative Site Recruiting
Dickson, Tennessee, United States, 37055
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Texas
aTyr Investigative Site Recruiting
Dallas, Texas, United States, 75246
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Houston, Texas, United States, 77030
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Utah
aTyr Investigative Site Recruiting
Salt Lake City, Utah, United States, 84108
Contact    877-596-6699    efzofit@cssienroll.com   
United States, Virginia
aTyr Investigative Site Recruiting
Falls Church, Virginia, United States, 22042
Contact    877-596-6699    efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Richmond, Virginia, United States, 23298
Contact    877-596-6699    efzofit@cssienroll.com   
Japan
Kyorin Investigative Site Recruiting
Hokkaido, Japan, 060-0033
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Kyorin Investigative Site Recruiting
Hokkaido, Japan, 060-8648
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Kyorin Investigative Site Not yet recruiting
Hyōgo, Japan, 653-0013
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Kyorin Investigative Site Recruiting
Kanagawa, Japan, 236-0051
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharm.co.jp   
Kyorin Investigative Site Recruiting
Miyagi, Japan, 980-8574
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Kyorin Investigative Site Recruiting
Osaka, Japan, 591-8025
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Kyorin Investigative Site Recruiting
Shizuoka, Japan, 431-3192
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Kyorin Investigative Site Recruiting
Tokyo, Japan, 150-8935
Contact    +81-3-3525-4788    ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Kyorin Investigative Site Recruiting
Tokyo, Japan, 151-8528
Contact: +81-3-3525-4788       ml-kyorin_clinicaltrials.gov@mb.kyorin-pharma.co.jp   
Netherlands
aTyr Investigative Site Recruiting
Nieuwegein, Netherlands, 3435CM
Contact       efzofit@cssienroll.com   
Puerto Rico
aTyr Investigative Site Recruiting
Guaynabo, Puerto Rico, 00968
Contact    887-596-6699    efzofit@cssienroll.com   
Spain
aTyr Investigative Site Recruiting
Barcelona, Spain, 08036
Contact       efzofit@cssienroll.com   
United Kingdom
aTyr Investigative Site Recruiting
Cottingham, United Kingdom, HU16 5JQ
Contact       efzofit@cssienroll.com   
Sponsors and Collaborators
aTyr Pharma, Inc.
Kyorin Pharmaceutical Co.,Ltd
Investigators
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Study Director: Lisa Carey aTyr Pharma, Inc.
More Information
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Responsible Party: aTyr Pharma, Inc.
ClinicalTrials.gov Identifier: NCT05415137    
Other Study ID Numbers: ATYR1923-C-004
First Posted: June 10, 2022    Key Record Dates
Last Update Posted: April 13, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by aTyr Pharma, Inc.:
Pulmonary Sarcoidosis
Sarcoidosis
Granuloma
Inflammation
Lymphoproliferative Disorders
Interstitial Lung Disease
Neuropilin-2
Steroids
 Oral corticosteroids
Immunomodulatory
tRNA Synthetase
ATYR1923
KRP-R120
Efzofitimod
Fibrosis
Additional relevant MeSH terms:
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Sarcoidosis, Pulmonary
Sarcoidosis
Lymphoproliferative Disorders
Lymphatic Diseases
Hypersensitivity, Delayed
 Hypersensitivity
Immune System Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases