PCD 诊断改善临床护理的效用 (PCD)
收集有关用于诊断原发性纤毛运动障碍 (PCD) 的测试效用的数据的临床研究
This cross-sectional and longitudinal observational study is to gather data on the utility of tests that are used to make a diagnosis of primary ciliary dyskinesia (PCD). There is new testing available, called nasal nitric oxide testing, that non-invasively measures nitric oxide levels in the sinus cavity. Individuals with PCD characteristically have low levels, but this testing does not have extensive data from everyday clinical practice. The objective of this proposal is to improve the diagnostic approach to children and adults with clinical concerns for primary ciliary dyskinesia (PCD).
研究概览
详细说明
This is an observational study to gather data on the utility of tests that are used to make a diagnosis of primary ciliary dyskinesia (PCD). Participant selection will be on a referral basis for the consideration of a clinical suspicion for PCD. Participants will be identified by pulmonary providers that consider testing for PCD based on the clinical symptoms of the participant. These providers will then refer for enrollment in the study based on the clinical considerations for a diagnosis of PCD. Participants will be consented during the time of testing at the Pulmonary department. Any patient referred by a clinician for concerns for PCD, including those who have had PCD genetics sent, or a ciliary biopsy performed will be enrolled in the study. In addition, any participant who is referred for nasal nitric oxide testing and meets the inclusion and exclusion criteria for this testing, will also need to provide informed consent and be enrolled in the Nasal NO REDCap registry. This equipment is not currently FDA approved or CLIA certified for PCD diagnostic testing and therefore referring clinicians will be informed that the results of this test should not influence clinical decision making regarding the diagnosis of PCD. However, there is growing evidence that this testing has utility as a non-invasive screening tool for PCD and continued data from the general pediatric pulmonary population is needed.
研究类型
注册 (估计的)
联系人和位置
学习联系方式
- 姓名：Melanie S Collins, MD
- 电话号码：860-545-9440
- 邮箱：mcollin@connecticutchildrens.org
研究联系人备份
- 姓名：Sigrid M Almeida, BS
- 电话号码：8608375343
- 邮箱：salmeida@connecticutchildrens.org
学习地点
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Connecticut
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Hartford、Connecticut、美国、06111
- 招聘中
- Pulmonary Division
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接触：
- Sigrid M Almeida, BS
- 电话号码：8608375343
- 邮箱：salmeida@connecticutchildrens.org
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接触：
- Melanie S Collins, MD
- 电话号码：8605459440
- 邮箱：mcollin@connecticutchildrens.org
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-
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
取样方法
研究人群
The target population are ages 2 and up. Participant selection will be on a referral basis for the consideration of a clinical suspicion for PCD. Participants will be identified by pulmonary providers that consider testing for PCD based on the clinical symptoms of the participant. These providers will thenrefer for enrollment in the study based on the clinical considerations for a diagnosis of PCD.
描述
Inclusion Criteria:
- Age ≥ 2 years of age
- Must have two of the following clinical history points:
- Neonatal respiratory distress
- Chronic nasal congestion/runny nose
- Chronic cough
- Situs/laterality defects
- Bronchiectasis
- Ability to provide informed consent or consent of parent/guardian and ass
Exclusion Criteria:
- Recent history of sinus surgery or bloody nose in the past week
- Age < 2 years of age
- Inability to tolerate probe in nose
- Sinusitis or other respiratory exacerbation currently being treated with antibiotics
- Admitted to hospital for respiratory exacerbation (inpatient status)
- Inability to understand the requirements of the study or be unwilling to provide written informed consent (as evidenced by signature on an informed consent document approved by the IRB)
- Any other reason for which the study investigators feel the patient is not a good candidate to complete the testing
学习计划
研究是如何设计的？
设计细节
- 观测模型：COHORT
- 时间观点：CROSS_SECTIONAL
队列和干预
团体/队列 |
干预/治疗 |
|---|---|
|
Nasal Nitric Oxide
Participants who are referred by his/her clinician for nasal NO testing and meet the inclusion and exclusion criteria will undergo testing. Clinical information regarding prior diagnostic testing will be collected at time of enrollment. Participants that have a confirmed diagnosis of PCD by genetics or ciliary biopsy at time of study entry |
Collection of already performed clinical data and nNO testing
其他名称：
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Creation of Nasal NO REDCap registry
大体时间：2-3 years
|
To establish a research registry to characterize the features of individuals that undergo PCD diagnostic testing at Connecticut Children's Medical Center and UCONN. |
2-3 years
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Evaluation of utility of PCD diagnostic testing
大体时间：2-3 years
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To evaluate the utility of PCD diagnostic testing, including potential use of nasal nitric oxide in comparison to genetic testing and ciliary biopsy, in pulmonary practice at Connecticut Children's Medical Center and UCONN. |
2-3 years
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Refinement and Improvement of PCD Diagnostic Testing
大体时间：1-2 years
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To refine and improve current diagnostic processes for PCD diagnosis at CCMC using information gathered from the research registry established through Primary aims 1 and 2. |
1-2 years
|
合作者和调查者
合作者
调查人员
- 首席研究员：Melanie S Collins, MD、Connecticut Children's Medical Center
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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