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

aTyr Pharma, Inc.2 sites in 2 countries268 target enrollmentSeptember 15, 2022

ConditionsPulmonary Sarcoidosis

InterventionsPlacebo Efzofitimod 3 mg/kg Efzofitimod 5 mg/kg

Overview

Phase: Phase 3
Intervention: Placebo
Conditions: Pulmonary Sarcoidosis
Sponsor: aTyr Pharma, Inc.
Enrollment: 268
Locations: 2
Primary Endpoint: Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48
Status: Active, not recruiting
Last Updated: 11 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

September 15, 2022

End Date

August 31, 2025

Last Updated

11 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

aTyr Pharma, Inc.

Responsible Party

Sponsor

Eligibility 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
•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