特発性後腹膜線維症におけるシロリムスの研究
特発性後腹膜線維症の治療におけるプレドニゾンとシロリムスの前向き研究
Retroperitoneal fibrosis refers to a group of diseases characterized by hyperplasia of the fibrosclerotic tissues in the retroperitoneal space, which can compress the surrounding ureters and inferior vena cava and cause serious complications such as aortic aneurysm, renal failure, and even death. The lesion is diffuse and difficult to resect. corticosteroid is the first-line medication, but the recurrence rate of the disease is high, especially after dose reduction of corticosteroid. Therefore, the combined use of immunosuppressants is very important in preventing disease recurrence and reducing the toxic and side effects of long-term corticosteroid. Sirolimus plays dual roles in inhibiting lymphocyte activation and fibroblast proliferation. It is inferred from its mechanism that sirolimus is a good potential treatment option for idiopathic retroperitoneal fibrosis. Therefore, we conducted this RCT on patients with idiopathic retroperitoneal fibrosis to determine the efficacy and safety of sirolimus.
調査の概要
詳細な説明
Retroperitoneal fibrosis refers to a group of diseases characterized by hyperplasia of the fibrosclerotic tissues in the retroperitoneal space, which mostly involve the abdominal aorta and iliac artery distal to the kidneys. The hyperplastic tissues can compress the surrounding ureters and inferior vena cava and cause serious complications such as aortic aneurysm, renal failure, and even death. There is no clear boundary between the lesion and its surrounding tissues. The lesion is diffuse and difficult to resect. corticosteroid is the first-line medication, but the recurrence rate of the disease is high, especially after dose reduction of corticosteroid. Therefore, the combined use of immunosuppressants is very important in preventing disease recurrence and reducing the toxic and side effects of long-term high- and medium-dose corticosteroid. In recent years, as the immunological characteristics of retroperitoneal fibrosis have gradually been recognized, some rheumatologists and immunologists have attempted to use immunosuppressants commonly used for autoimmune diseases in this population, including biologics. However, high-level evidences of evidence-based medicine such as randomized controlled trials (RCTs) were still lacking. Sirolimus plays dual roles in inhibiting T lymphocyte activation and fibroblast proliferation. It is inferred from its mechanism that sirolimus is a good potential treatment option for idiopathic retroperitoneal fibrosis. Therefore, we conducted this RCT on patients with idiopathic retroperitoneal fibrosis to determine the efficacy and safety of sirolimus.
研究の種類
入学 (実際)
段階
- フェーズ2
- フェーズ 3
連絡先と場所
研究場所
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Beijing Municipality
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Beijing、Beijing Municipality、中国、100000
- Peking University International Hospital
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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:
- Idiopathic retroperitoneal fibrosis diagnosed on CT or MRI. For patients with suspected secondary retroperitoneal fibrosis or atypical idiopathic retroperitoneal fibrosis suggested by imaging, idiopathic retroperitoneal fibrosis should be confirmed by puncture biopsy
- Increased ESR and CRP levels caused by this disease and/or active lesions suggested on imaging
Exclusion Criteria:
- Secondary retroperitoneal fibrosis
- Having used corticosteroid (equivalent to >10 mg per day of prednisone), immunosuppressant, or biologic within 3 months prior to enrollment
- Having any contraindication of prednisone or sirolimus, or allergy to sirolimus, or having experienced serious adverse reactions from the previous use of any of the above drugs
- Massive proteinuria (24-hour urine protein quantitation ≥3 g), moderate-to-severe anemia (hemoglobin <90 g/L), agranulocytosis (white blood cell count <1.5×10^9/L or neutrophil count <0.5×10^9/L), platelet count <50×10^9/L, interstitial pneumonia
- Uncontrollable diabetes, hypertension, hyperlipidemia, infection, or heart failure, or other serious complications
- Malignancy
- Pregnancy or need for pregnancy in the near future
- Unable to adhere to follow-up or refuses to provide consent
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：ランダム化
- 介入モデル：並列代入
- 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 |
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実験的：sirolimus group
Sirolimus: 2 mg/day for the first 3 days and 1 mg/day thereafter. The plasma drug concentration was monitored at 14 days, 12 weeks, and 48 weeks of medication to maintain a plasma drug concentration of 4-15 ug/L. Prednisone: 0.8 mg/kg/d (maximum dose: 60 mg/d), reduced by 5 mg every 14 days, by 2.5 mg every 2 weeks after 30 mg/d until discontinuation. |
The efficacy is evaluated at 12 weeks, and treatment will be adjusted according to the control of disease and adverse effects.For experimental group, if a patient is assessed as treatment failure (TS), the patient should be withdrawn from the study and receive rescue treatment. Whereas, a patient would be transferred to the control group if he/ she cann't stand the side effects of sirolimus but not serious adverse event (SAE). The efficacy is evaluated at 12 weeks, and treatment will be adjusted according to the control of disease and adverse effects. For active comparator group, if a patient is assessed as treatment failure (TF), the patient should be withdrawn from the study and receive rescue treatment.
他の名前：
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アクティブコンパレータ：corticosteroid group
Prednisone: 0.8 mg/kg/d (maximum dose: 60 mg/d), reduced by 5 mg every 14 days, by 2.5 mg every 2 weeks after 30 mg/d until 5-7.5 mg/d. |
The efficacy is evaluated at 12 weeks, and treatment will be adjusted according to the control of disease and adverse effects. For active comparator group, if a patient is assessed as treatment failure (TF), the patient should be withdrawn from the study and receive rescue treatment.
他の名前：
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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 |
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再発率
時間枠：研究完了まで、平均1年
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研究完了まで、平均1年
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二次結果の測定
結果測定 |
時間枠 |
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治療失敗率
時間枠：12週間の治療
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12週間の治療
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プレドニゾンの累積投与量
時間枠：48週間
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48週間
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後腹膜線維性腫瘤のサイズ
時間枠：48週間
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48週間
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協力者と研究者
捜査官
- 主任研究者：Hui Gao, Doctor、Peking University International Hospital
研究記録日
主要日程の研究
研究開始 (実際)
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 (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
- Sirolimus for RPF
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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