BEGIN 新規イメージングバイオマーカー (BEGINNING)
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).
調査の概要
詳細な説明
The overall hypothesis is that multi-organ MRI will provide more sensitive, robust outcome measures in young CF patients than traditional measures employed in the BEGIN study and that these novel measures will be more sensitive to treatment effects, tested here by comparison before and after triple-combination modulator therapy. By understanding the nature of early lung obstruction and characteristic changes in the liver and pancreas over time, we continue to lay the groundwork for more personalized medicine in the future.
Assessing treatment response and clinical benefit in children with CF who are clinically normal per standard outcomes (e.g., spirometry, pancreatic function) will become paramount as triplecombination therapy is extended to younger patients with milder CF clinical presentation than their historic peers. Here the sensitivity and profile free of ionizing-radiation exposure of MRI can be leveraged to follow an individual with CF over time to quantify changes with therapy-with additional spatial resolution unavailable from standard clinical testing.
研究の種類
入学 (実際)
段階
- フェーズ 4
連絡先と場所
研究場所
-
-
Kansas
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Kansas City、Kansas、アメリカ、66160
- University of Kansas Medical Center
-
-
Ohio
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Cincinnati、Ohio、アメリカ、45229
- Carrie Stevens
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Virginia
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Charlottesville、Virginia、アメリカ、22903
- University of Virginia
-
-
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
説明
Inclusion Criteria:
-
Written informed consent (and assent where appropriate) obtained from the subject or subject's legal representative.
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Willingness to adhere to the study-visit schedule and other protocol requirements.
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Ages 6-8 years old at baseline MRI visit (may be enrolled up to 60 days before 6th birthday).
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Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- Sweat chloride equal to or greater than 60 mEq/liter by quantitative pilocarpine iontophoresis test
- Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
-
Physician intent to prescribe triple-combination therapy
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Clinically-stable with no respiratory tract infection at the time of enrollment.
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No change in chronic maintenance therapies in the 28 days prior to enrollment.
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Ability to cooperate with MRI procedures
Exclusion Criteria:
- Individuals currently on ivacaftor therapy (including Kalydeco, Orkambi, and Symdeko) and with at least one gating mutation. Gating mutations include G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D.
- Acute respiratory symptoms (e.g. wheezing) at the time of the MRI.
- Acute respiratory infection, defined as increased cough, wheezing or respiratory rate in the 28 days prior to enrollment.
- Chronic lung disease not related to CF
- Chronic liver disease not related to CF
- Acute pancreatitis, defined by clinical criteria (45).
- Chronic pancreatic disease not related to CF.
- Physical findings that would compromise the safety of the subject or the quality of the study data as determined at the discretion of the site investigator.
- Any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
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
- 主な目的：診断
- 割り当て：非ランダム化
- 介入モデル：クロスオーバー割り当て
- マスキング：独身
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：Pre Trikafta
129Xe MRI |
Rapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation. |
|
実験的：Post Trikafta
129Xe MRI |
Rapid spatial mapping of lung, liver, and pancreatic structure and function is now possible with a combination of hyperpolarized 129Xe and traditional proton MRI, all absent sedation and ionizing radiation. |
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 |
|---|---|---|
|
Ventilation Defect Percentage change from baseline
時間枠：1 year
|
For pulmonary MRI, the primary outcome measure is the change in 129Xe ventilation defect percentage (VDP) from pre-therapy baseline to the one-year follow-up visit. |
1 year
|
|
Pancreas volume
時間枠：1 year
|
For pancreatic MRI, the primary outcome measure is change in pancreas volume normalized to BSA between pre-therapy baseline and one-year follow-up visit. |
1 year
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Abdominal T1 values
時間枠：1 year
|
Changes in MRI T1 average in the liver and pancreas, from baseline to follow up at 1 year |
1 year
|
|
Lung reader score
時間枠：1 year
|
Changes in reader score for visible structural defects from proton MRI, from baseline to follow up at 1 year |
1 year
|
協力者と研究者
捜査官
- 主任研究者：Jason Woods, PhD、Children's Hospital Medical Center, Cincinnati
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
- 2021-0325
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
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