Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis

aTyr Pharma

Status and phase: Active, not recruiting; Phase 3

Conditions: Pulmonary Sarcoidosis

Treatments: Drug: Efzofitimod 5 mg/kg; Drug: Placebo; Drug: Efzofitimod 3 mg/kg

Study type: Interventional

Funder types: Industry

Identifiers

NCT05415137

ATYR1923-C-004

Details and patient eligibility

About

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.

Enrollment: 268 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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268 participants in 3 patient groups, including a placebo group

Efzofitimod 3 mg/kg

Experimental group

Treatment:

Drug: Efzofitimod 3 mg/kg

Efzofitimod 5 mg/kg

Experimental group

Treatment:

Drug: Efzofitimod 5 mg/kg

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo