A Prospective Study of the Utility of Spirometry to Identify and Manage Immunoglobulin Replacement Dosage in Primary Antibody Deficiency in Patients With Potentially Reversible Airway Disease
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 22
- 试验地点
- 2
- 主要终点
- FEF25-75% at 3 months
研究概览
简要总结
Although there is evidence in the literature that gammaglobulin replacement therapy can lead to a reduction in the prevalence of pulmonary infection and improved lung function, there is no published study to guide immunologists regarding the use of spirometry in titrating IG therapy to assist in the management of immunodeficiency patients with regards to gammaglobulin replacement therapy.
The investigators propose to study the use of spirometry to identify patients that could potentially benefit from an increase in IGRT. The investigators will identify 22 common variable immune deficiency (CVID) study subjects on stable IGRT replacement therapy equivalent to 0.40 to 0.60 gm/kg per 4 weeks who have evidence of mild to moderate obstruction as assessed by an FEF25-75% between 50% and 80% of predicted. Patients who are on Hizentra will be preferentially recruited. Of these 22, 11 will be identified at random and treated for 6 months at their current dose (control population). The remaining 11 study subjects (treatment group) will have their level of IGRT increased by the equivalent of 0.05 gm/kg in dose per 4 weeks, adjusted for bioavailability as per manufacturer's instructions. On average, rounded up to the nearest gram, this will typically increase their dose of Hizentra by 2 gm per week.
详细描述
The key finding of the published retrospective study was that common variable immune deficiency (CVID) patients with moderate, presumed reversible, obstruction on stable, therapeutic doses of IgG who exhibited a decline in lung function from one clinic visit to the next responded to an increased dose of IgG with an improvement in lung function as assessed by spirometry.
The investigators now wish perform a clinical trial to assess whether primary antibody deficiency patients receiving IGRT who fit in this range of obstruction, i.e. an FEF25-75% that is 50-80% of predicted, will demonstrate an increase in lung function, as assessed by spirometry, after increasing the dose of IGRT. The presumption is that obstruction at this level is most likely due to the effects of subclinical infections that can be reduced or avoided by increasing the amount of gammaglobulin received by the patients.
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
- None
入排标准
- 年龄范围
- 21 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients who meet criteria for common variable immune deficiency (CVID) who are on stable IGRT for at least 3 months and who have an FEF25-75% between 50% and 80% of predicted.
- •Patients who are already on Hizentra will be preferred.
排除标准
- •Age <21 or cannot perform spirometry.
- •Smokers with 20 pack years or more, and active smokers will not be included among the study subjects, but will be considered separately as an ancillary study.
- •Patients with specific antigen-specific antibody deficiencies or X-linked agammaglobulinemia on IGRT will not be included among the 20 study subjects, but will be considered separately in ancillary studies.
- •Patients with heart failure, TB, bronchiolitis, or lymphangioleiomyomatosis.
研究组 & 干预措施
Treatment Group
11 subjects will have their level of immunoglobulin replacement therapy increased by the equivalent of 0.05 gm/kg in dose per 4 weeks, adjusted for bioavailability as per manufacturer's instructions. On average, rounded up to the nearest gram, this will typically increase their dose of Hizentra by 2 gm per week.
干预措施: Hizentra (Drug)
Control Group
11 subjects will be treated for 6 months at their current dose of Hizentra
结局指标
主要结局
FEF25-75% at 3 months
时间窗: 3 months
Pulmonary function will be measured by forced expiratory flow at 25 and 75% of the pulmonary volume (FEF25-75%) at 3 months.
FEV1 at 3 months
时间窗: 3 months
Pulmonary function will be measured by forced expiratory volume in one second (FEV1) at three months into the study.
FEF25-75% at baseline
时间窗: baseline
Pulmonary function will be measured by forced expiratory flow at 25 and 75% of the pulmonary volume (FEF25-75%) at baseline.
FEV1/FVC ratio at baseline
时间窗: baseline
FEV1/FVC ratio will be calculated at baseline. The FEV1/FVC ratio is the ratio of the forced expiratory volume in the first one second (FEV1) to the forced vital capacity (FVC) of the lungs.
FOT at 6 months.
时间窗: 6 months
Forced Oscillation Technique (FOT) will be measured at 6 months. Forced Oscillation Technique (FOT) measures lung impedance during tidal breathing.
FVC at baseline
时间窗: baseline
Pulmonary function will be measured by forced vital capacity (FVC) at baseline.
FVC at 6 months.
时间窗: 6 months
Pulmonary function will be measured by forced vital capacity (FVC) at six months.
FEF25-75% at 6 months
时间窗: 6 months
Pulmonary function will be measured by forced expiratory flow at 25 and 75% of the pulmonary volume (FEF25-75%) at six months.
FEV1/FVC ratio at 6 months
时间窗: 6 months
FEV1/FVC ratio will be calculated at 6 months. The FEV1/FVC ratio is the ratio of the forced expiratory volume in the first one second (FEV1) to the forced vital capacity (FVC) of the lungs.
FEV1 at 6 months.
时间窗: 6 months
Pulmonary function will be measured by forced expiratory volume in one second (FEV1) at six months into the study.
FVC at 3 months
时间窗: 3 months
Pulmonary function will be measured by forced vital capacity (FVC) at three months.
FOT at 3 months.
时间窗: 3 months
Forced Oscillation Technique (FOT) will be measured at 3 months. Forced Oscillation Technique (FOT) measures lung impedance during tidal breathing.
FEV1 at baseline
时间窗: baseline
Pulmonary function will be measured by forced expiratory volume in one second (FEV1) at baseline.
FEV1/FVC ratio at 3 months
时间窗: 3 months
FEV1/FVC ratio will be calculated at 3 months. The FEV1/FVC ratio is the ratio of the forced expiratory volume in the first one second (FEV1) to the forced vital capacity (FVC) of the lungs.
FOT at baseline.
时间窗: baseline
Forced Oscillation Technique (FOT) will be measured at baseline. Forced Oscillation Technique (FOT) measures lung impedance during tidal breathing.
次要结局
- FACIT score at baseline and monthly on therapy(6 months)
- St. George's Respiratory Questionnaire at baseline and monthly on therapy(6 months)
- PADQOL-16 at baseline and monthly on therapy(6 months)
研究者
Harry W. Schroeder, Jr., MD PhD
Principal Investigator
University of Alabama at Birmingham