鎌状赤血球症および肺高血圧症の小児および若者に対するヒドロキシ尿素
鎌状赤血球症の小児および若年成人における肺高血圧症の治療のためのヒドロキシ尿素のパイロット研究
The goal of this study is to test the hypothesis that hydroxyurea is effective for the specific treatment of secondary pulmonary hypertension found on screening in children and young adults with sickle cell disease.
調査の概要
詳細な説明
Increasing evidence suggests that pulmonary hypertension, defined by an elevated tricuspid regurgitant jet velocity (TRJV) on echocardiogram, is a major cause of morbidity and mortality in adults with sickle cell disease (SCD). However, both the prevalence and optimal treatment of pulmonary hypertension in children and young adults with SCD are unknown.
We hypothesize that short term therapy with hydroxyurea will decrease TRJV in children and young adults with pulmonary hypertension found on screening. Patients eligible for treatment will have had evidence of pulmonary hypertension on at least 2 screening echocardiograms. Baseline laboratory tests will be obtained and other causes of secondary pulmonary hypertension will be excluded prior to initiation of treatment. Patients will be treated with hydroxyurea according to a standard dose escalation schedule for a total of 12 months. A clinic visit will be required every 2 months and standard screening for toxicity will be performed monthly. There will be an interim analysis of the primary outcome at 6 months following therapy.
研究の種類
入学 (実際)
段階
- フェーズ2
- フェーズ 1
連絡先と場所
研究場所
-
-
Illinois
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Chicago、Illinois、アメリカ、60611
- Ann & Robert H Lurie Children's Hospital of Chicago
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-
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:
- Age between 10 and 25 years old
- Sickle cell disease with hemoglobin SS, SC or S-B^0 thalassemia confirmed on hemoglobin electrophoresis
- Tricuspid regurgitant jet velocity (TRJV) equal to or greater than 2.5 m/sec on 2 baseline Doppler echocardiograms at least 3 months apart
Exclusion Criteria:
- Patients already being treated with hydroxyurea
- Patients on a chronic transfusion protocol
- Patients with evidence of hepatic (alanine aminotransferase [ALT] equal to or greater than 2 SD above normal) or renal dysfunction (creatinine [Cr] equal to or greater than 2 SD above normal)
- Patients who are pregnant
- Patients with documented causes of severe pulmonary hypertension other than from SCD
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：処理
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：Hydroxyurea
Single arm pilot of hydroxyurea in the experimental arm with starting dose of 20 mg/kg/day po and dose escalation every 2 months until maximum tolerated dose. |
Drug to be initiated at standard starting dose with dose escalation until maximum tolerated dose reached.
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 |
時間枠 |
|---|---|
|
Mean Reduction in TRJV at M6
時間枠：6 months after initiation of hydroxyurea
|
6 months after initiation of hydroxyurea
|
|
Mean Reduction in TRJV at M12
時間枠：12 months after initiation of hydroxyurea
|
12 months after initiation of hydroxyurea
|
二次結果の測定
結果測定 |
時間枠 |
|---|---|
|
Mean Increase Hb F% at M6
時間枠：6 months after initiation of hydroxyurea
|
6 months after initiation of hydroxyurea
|
|
Mean Increase in HbF% at M12
時間枠：12 months after initiation of hydroxyurea
|
12 months after initiation of hydroxyurea
|
|
Mean Reduction in LDH at M6
時間枠：6 months after initiation of hydroxyurea
|
6 months after initiation of hydroxyurea
|
|
Mean Reduction in LDH at M12
時間枠：12 months after initiation of hydroxyurea
|
12 months after initiation of hydroxyurea
|
|
Mean Reduction in Retic% at M6
時間枠：6 months after initiation of hydroxyurea
|
6 months after initiation of hydroxyurea
|
|
Mean Reduction in Retic% at M12
時間枠：12 months after initiation of hydroxyurea
|
12 months after initiation of hydroxyurea
|
協力者と研究者
捜査官
- 主任研究者：Robert I Liem, MD、Ann & Robert H Lurie Children's Hospital of Chicago
研究記録日
主要日程の研究
研究開始
一次修了 (実際)
研究の完了 (実際)
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 (推定)
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
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。