Emapalumab for the Treatment of Anti-MDA5 Antibody Positive Rapidly Progressive Interstitial Lung Disease
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 2
- 主要终点
- Forced Vital Capacity (FVC)
研究概览
简要总结
This is a proof of concept study to determine if Emapalumab appears effective for the treatment of anti-MDA5 antibody positive rapidly progressive interstitial lung disease (MDA5 RP-ILD). Emapalumab is a medication that is currently used for a severe problem with the immune system, called macrophage activation syndrome, and this disease shares some similar features with MDA5 RP-ILD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Progressive interstitial lung disease as determined by at least 2/4 of the following:
- •worsening respiratory symptoms;
- •worsening or new oxygen requirement;
- •worsening disease on CT chest;
- •worsening Forced Vital Capacity (FVC1) or Diffusing Capacity for Carbon Monoxide (DLCO) on pulmonary function tests
- •MDA5 antibodies present
- •Elevated ferritin above the upper limit of normal (ULN)
- •Participant at least 18 years old
排除标准
- •Active, untreated bacterial, mycobacterial or fungal infection
- •Active herpes zoster infection
- •Currently requiring extracorporeal membrane oxygenation (ECMO)
- •Participant refusal to participate in the study
- •Pregnant women
- •Prisoners
研究组 & 干预措施
Emapalumab
Participants will receive emapalumab administered intravenously at a dose of 6 mg/kg on Day 1, followed by 3 mg/kg every 3 days for 2 weeks, and then 3 mg/kg twice weekly for an additional 2 weeks. Total treatment duration is 12 weeks.
干预措施: Emapalumab (Drug)
结局指标
主要结局
Forced Vital Capacity (FVC)
时间窗: Baseline, 12 weeks
Pulmonary function assessed using forced vital capacity (FVC) measured by standard pulmonary function testing (spirometry). The outcome is expressed as percent change from baseline (%) in FVC.
Change in oxygen requirement
时间窗: Baseline, 4 weeks, 12 weeks
Supplemental oxygen requirement assessed as the oxygen flow rate (reported in liters per minute \[L/min\]) while the participant is at rest and, when feasible, while the participant is ambulating during the 6-Minute Walk Test (6MWT). Values are compared with baseline to assess change over time.
Forced Expiratory Volume in 1 Second (FEV₁)
时间窗: Baseline, 12 weeks
Pulmonary function assessed using forced expiratory volume in 1 second (FEV₁) measured by standard pulmonary function testing (spirometry). The outcome is expressed as percent change from baseline (%) in FEV₁.
Diffusing capacity of the lungs for carbon monoxide (DLCO)
时间窗: Baseline, 12 weeks
Pulmonary gas exchange capacity assessed using diffusing capacity of the lungs for carbon monoxide (DLCO) measured by standard pulmonary function testing. The outcome is expressed as percent change from baseline (%) in DLCO
Change in chest computed tomography (CT) consolidations
时间窗: Baseline, 4 weeks, 12 weeks
Pulmonary consolidations assessed on chest computed tomography (CT) scans. CT images are reviewed by a radiologist and pulmonologist, and percentage of lung affected by consolidations will be documented. Percentage change of affected lung will be evaluated.
次要结局
- Change in Ferritin level(Baseline, 4 weeks, 12 weeks)
- Change in MDA5 antibody level(Baseline, 12 weeks)
- New infections during treatment(Baseline, 12 weeks)
研究者
Kelly Corbitt
Assistant Professor of Clinical Medicine
University of Miami