臨床ケアを改善するための PCD 診断の有用性 (PCD)
原発性毛様体ジスキネジア（PCD）の診断に使用される検査の有用性に関するデータを収集する臨床研究
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).
調査の概要
詳細な説明
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.
研究の種類
入学 (推定)
連絡先と場所
研究連絡先
- 名前：Melanie S Collins, MD
- 電話番号：860-545-9440
- メール：mcollin@connecticutchildrens.org
研究連絡先のバックアップ
- 名前：Sigrid M Almeida, BS
- 電話番号：8608375343
- メール：salmeida@connecticutchildrens.org
研究場所
-
-
Connecticut
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Hartford、Connecticut、アメリカ、06111
- 募集
- Pulmonary Division
-
コンタクト：
- Sigrid M Almeida, BS
- 電話番号：8608375343
- メール：salmeida@connecticutchildrens.org
-
コンタクト：
- Melanie S Collins, MD
- 電話番号：8605459440
- メール：mcollin@connecticutchildrens.org
-
-
参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
サンプリング方法
調査対象母集団
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.
説明
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
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 観測モデル：COHORT
- 時間の展望：CROSS_SECTIONAL
コホートと介入
グループ/コホート |
介入・治療 |
|---|---|
|
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
他の名前：
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Creation of Nasal NO REDCap registry
時間枠：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
|
|
Evaluation of utility of PCD diagnostic testing
時間枠：2-3 years
|
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
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Refinement and Improvement of PCD Diagnostic Testing
時間枠：1-2 years
|
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
|
協力者と研究者
協力者
捜査官
- 主任研究者：Melanie S Collins, MD、Connecticut Children's Medical Center
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- 23-052-CCMC
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
米国で製造され、米国から輸出された製品。
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