Randomized, Double-blind, Placebo-controlled Proof-of-Concept (PoC) Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Absolute change from baseline in forced vital capacity (FVC) in mL
研究概览
简要总结
This is a 2-Part study with Part A, a double-blind, randomized, placebo-controlled, PoC study to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with SSc-ILD. The primary objective of the study is to evaluate the PoC for efficacy in a population with SSc-ILD. While improvement of ILD is the outcome of interest, the study will also evaluate changes in the skin. After initial screening (up to 4 weeks), approximately 25 eligible participants will be randomized 2:2:1 to 1 of 2 active (experimental) dose arms or placebo, administered every 4 weeks up to and including Week 20. Part B is an optional open-label extension to Part A in which participants can receive 450 mg efzofitimod every 4 weeks for 6 doses.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of SSc based on ACR/ EULAR criteria (2013)
- •Overall duration of SSc < 84 months from the first non-Raynaud symptom manifestation prior to Day 1
- •HRCT obtained at the Screening Visit or within the 3 months prior to Screening consistent with SSc-ILD (adjudicated by a central reader) AND with pulmonary involvement > 10%
- •Clinical presentation at Screening consistent with lcSSc (up to 40% of patients) or dcSSc
- •MMF of ≥ 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 3 months prior to Day 1 OR When documented intolerance to mycophenolates (in discussion with the Medical Monitor): treatment with maximum tolerated dose of MMF is acceptable, if < 2 gm/day, provided the cumulative duration of dosing has exceeded 3 months, OR An adequate dose and duration of an alternate immunosuppressant with a stable dose for the 4 weeks prior to baseline is also allowed.
排除标准
- •Pulmonary disease with FVC %pred ≤ 45% OR DLco %pred ≤ 30%; FEV1/FVC ratio < 0.7
- •Participants with pulmonary artery hypertension on parenteral therapy or with clinical evidence of right heart failure
- •HRCT obtained in the 3 months prior to Screening consistent with other confounding pathology.
- •Treatment with corticosteroids (> 10 mg/day of prednisone or equivalent) within 2 weeks prior to Day 1
- •Treatment with more than 1 immunosuppressant (e.g., MMF, methotrexate [MTX], azathioprine [AZA], or leflunomide)
- •Any treatment in the 12 months prior to Day 1 with any of the following: rituximab, intravenous immune globulin (IVIG), tocilizumab, cyclophosphamide, pirfenidone, tyrosine-kinase inhibitors (e.g., imatinib, nilotinib, dasatinib)
- •Rheumatic autoimmune disease other than SSc, Is an active, heavy smoker of tobacco/nicotine-containing products
- •History of (anti-Jo-1) anti-synthetase syndrome or Jo-1 positive at Screening
研究组 & 干预措施
efzofitimod 450 mg
Administered IV infusion
干预措施: efzofitimod 450 mg (Drug)
efzofitimod 270 mg
Administered IV infusion
干预措施: efzofitimod 270 mg (Drug)
Placebo
Administered IV infusion
干预措施: Placebo (Drug)
结局指标
主要结局
Absolute change from baseline in forced vital capacity (FVC) in mL
时间窗: 24 weeks
Annual rate of decline in FVC in mL
时间窗: 24 weeks
Annual rate of decline in FVC in percent predicted
时间窗: 24 weeks
Change in HRCT fibrosis score
时间窗: Baseline to Week 24
次要结局
未报告次要终点