Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis
Purpose
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
- Pulmonary Sarcoidosis
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Efzofitimod 3 mg/kg |
|
|
|
Experimental Efzofitimod 5 mg/kg |
|
|
|
Placebo Comparator Placebo |
|
Recruiting Locations
More Details
- NCT ID
- NCT05415137
- Status
- Active, not recruiting
- Sponsor
- aTyr Pharma, Inc.