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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 : February 2, 2024
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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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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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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: February 2, 2024
Last Verified: January 2024
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