Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

• Status: Not yet recruiting
• Trial end date: 2025-01-15
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: aTyr Pharma, Inc.
• Collaborator: Kyorin Pharmaceutical Co.,Ltd

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