Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

Study Overview
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Brief Summary
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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.

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

Conditions
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Pulmonary Sarcoidosis

Intervention / Treatment
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* Drug: Efzofitimod 3 mg/kg
* Drug: Efzofitimod 5 mg/kg
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Efzofitimod 3 mg/kg<br> | Drug: Efzofitimod 3 mg/kg<br>* EfzofitimodIV infusion every 4 weeks for a total of 12 doses<br>* Other names: KRP-R120;|
| Experimental: Efzofitimod 5 mg/kg<br> | Drug: Efzofitimod 5 mg/kg<br>* EfzofitimodIV infusion every 4 weeks for a total of 12 doses<br>* Other names: KRP-R120;|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* Placebo IV infusion every 4 weeks for a total of 12 doses<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in mean daily oral corticosteroid (OCS) dose post-taper |  | Baseline to Week 48 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annual rate of change in absolute value of Forced vital capacity (FVC) |  | Baseline to Week 48 | 
| Percent change from baseline in mean daily OCS dose post-taper |  | Baseline to Week 48 | 
| Change from baseline in King's Sarcoidosis Questionnaire (KSQ)-Lung score |  | Baseline to Week 48 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pulmonary Sarcoidosis, Sarcoidosis, Granuloma, Inflammation, Lymphoproliferative Disorders, Interstitial Lung Disease, Neuropilin-2, Steroids, Oral corticosteroids, Immunomodulatory, tRNA Synthetase, ATYR1923, KRP-R120, Efzofitimod, Fibrosis