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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 : December 5, 2022
Sponsor:
Collaborator:
Kyorin Pharmaceutical Co.,Ltd
Information provided by (Responsible Party):
aTyr Pharma, Inc.

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

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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

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




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 ]


Information from the National Library of Medicine

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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% at Screening; FVC % predicted < 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

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 Not yet recruiting
Birmingham, Alabama, United States, 35294
Contact: efzofit@cssienroll.com         
United States, California
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, Florida
aTyr Investigative Site Recruiting
Doral, Florida, United States, 33166
Contact: 877-596-6699       efzofit@cssienroll.com   
aTyr Investigative Site Recruiting
Gainesville, Florida, United States, 32610
Contact: efzofit@cssienroll.com         
aTyr Investigative Site Recruiting
Miami Lakes, Florida, United States, 33014
Contact: 877-596-6699       efzofit@cssienroll.com   
United States, Illinois
aTyr Investigative Site Not yet recruiting
Chicago, Illinois, United States, 60611
Contact: 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 Not yet recruiting
Baltimore, Maryland, United States, 21224
Contact: efzofit@cssienroll.com         
United States, Minnesota
aTyr Investigative Site Not yet recruiting
Minneapolis, Minnesota, United States, 55445
Contact: efzofit@cssienroll.com         
aTyr Investigative Site Not yet recruiting
Rochester, Minnesota, United States, 55905
Contact: efzofit@cssienroll.com         
United States, New York
aTyr Investigative Site Recruiting
Brooklyn, New York, United States, 11236
Contact: 877-596-6699       efzofit@cssienroll.com   
United States, North Carolina
aTyr Investigative Site Recruiting
Greenville, North Carolina, United States, 27834
Contact: efzofit@cssienroll.com         
United States, Ohio
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: efzofit@cssienroll.com         
United States, Oregon
aTyr Investigative Site Recruiting
Portland, Oregon, United States, 97220
Contact: efzofit@cssienroll.com         
United States, South Carolina
aTyr Investigative Site Recruiting
Charleston, South Carolina, United States, 29425-0630
Contact: efzofit@cssienroll.com         
United States, Tennessee
aTyr Investigative Site Recruiting
Dickson, Tennessee, United States, 37055
Contact: efzofit@cssienroll.com         
United States, Texas
aTyr Investigative Site Not yet recruiting
Houston, Texas, United States, 77030
Contact: efzofit@cssienroll.com         
United States, Utah
aTyr Investigative Site Not yet recruiting
Salt Lake City, Utah, United States, 84108
Contact: efzofit@cssienroll.com         
United States, Virginia
aTyr Investigative Site Not yet recruiting
Richmond, Virginia, United States, 23298
Contact: efzofit@cssienroll.com         
Netherlands
aTyr Investigative Site Not yet 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   
Sponsors and Collaborators
aTyr Pharma, Inc.
Kyorin Pharmaceutical Co.,Ltd
Investigators
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Study Director: Lisa Carey aTyr Pharma, Inc.
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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: December 5, 2022
Last Verified: December 2022
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
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases