Sirolimus as Secondary Therapy in Chronic Graft-Versus-Host Disease Not Responding To Prior Treatment
2017年5月23日 更新者：Paul Carpenter、Fred Hutchinson Cancer Center
A Phase II Clinical Trial to Evaluate the Safety and Efficacy of Sirolimus for Secondary Treatment of Chronic Graft-versus-Host Disease
This phase II trial studies the side effects and how well sirolimus works as secondary therapy in treating patients with chronic graft-versus-host disease (GVHD) that did not respond to prior treatment.
Sirolimus may be an effective treatment for chronic GVHD
調査の概要
詳細な説明
PRIMARY OBJECTIVES:
I. To assess the safety of sirolimus administered at a dose which provides steady-state, whole blood trough levels of 5-10 ng/mL in patients with chronic GVHD.
II. To determine whether administration of sirolimus provides benefit for patients with chronic GVHD that has not responded adequately to previous systemic treatment.
OUTLINE:
Patients receive sirolimus orally (PO) once daily (QD). Patients continue to receive prednisone and cyclosporine or tacrolimus at the discretion of the managing physician.
After completion of study treatment, patients are followed up periodically.
研究の種類
介入
入学 (実際)
44
段階
- フェーズ2
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究場所
-
-
Washington
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Seattle、Washington、アメリカ、98109
- Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
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-
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
いいえ
受講資格のある性別
全て
説明
Inclusion Criteria:
Biopsy-confirmed diagnosis of clinical extensive chronic GVHD with inadequate response to previous treatment and where secondary systemic therapy is indicated because of
- Clinical progression of signs and symptoms of chronic GVHD in a previously involved organ, or
- Development of signs and symptoms of chronic GVHD in a previously uninvolved organ, or
- Absence of improvement after 3 months of primary treatment, or
- Continued need for treatment with prednisone at doses >= 1.0 mg/kg/day for more than 2 months, without qualification for type of donor, graft or conditioning regimen
- Patient or guardian able and willing to provide informed consent
- Stated willingness to use contraception in women of child-bearing potential (Food and Drug Administration [FDA] requirement)
- Stated willingness of the patient to comply with study procedures and reporting requirements
- Stated willingness of the physician most involved in management of chronic GVHD (the "managing physician,") to comply with study procedures and reporting requirements
Exclusion Criteria:
- Fungal or viral infection with no radiographic evidence of improvement during continued appropriate antimicrobial therapy
- Cytomegalovirus (CMV) antigenemia unresponsive to antiviral therapy
- Active disseminated varicella zoster virus (VZV) infection with persistent non-crusted lesions
- Inability to tolerate oral medications
- Absolute neutrophil count (ANC) < 1500/uL
- Platelet count < 50,000/uL
- Persistent or recurrent malignancy, including histopathologic evidence of myeloma or lymphoma; patients with breakpoint cluster region-abelson (bcr/abl) detected by polymerase chain reaction (PCR) assay as the only evidence of persistent chronic myeloid leukemia may be enrolled
- Pregnancy
- Known history of hypersensitivity to sirolimus
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
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：支持療法
- 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 |
|---|---|
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実験的：Sirolimus
Study participants receive sirolimus added once daily to their baseline combination therapy of prednisone plus either cyclosporine or tacrolimus at the discretion of the managing physician.
Treatment other than cyclosporine (or tacrolimus) and prednisone must be discontinued when administration of sirolimus is started.
Topical therapy, including psoralen and UVA irradiation (PUVA), glucocorticoid creams, topical tacrolimus, oral beclomethasone, topical azathioprine and ophthalmic glucocorticoids may be given at the discretion of the managing physician in consultation with the transplant center.
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与えられたPO
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 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Number of Participants Experiencing Treatment Success
時間枠：Approximately 7 years
|
Defined as the absence of any immunosuppressive treatment, including sirolimus, with resolution of all reversible manifestations of chronic GVHD and no additional systemic therapy.
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Approximately 7 years
|
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Number of Participants Experiencing Treatment Failure
時間枠：Approximately 7 years
|
Defined as the initiation of additional systemic therapy, development of bronchiolitis obliterans, or death from causes other than recurrent malignancy during primary treatment for chronic GVHD, whichever occurs first.
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Approximately 7 years
|
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Number of Participants Needing Additional Systemic Therapy
時間枠：Approximately 7 years
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Includes any intervention intended to control chronic GVHD through an immunosuppressive effect from oral or parenteral administration of any systemic medication not originally given under auspices of this protocol.
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Approximately 7 years
|
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Number of Participants With Recurrent Malignancy
時間枠：Approximately 7 years
|
Defined as clinical or histopathologic evidence demonstrating the presence of any malignancy considered as the indication for transplant.
Recurrent malignancy will also be defined as any post-transplant intervention not routinely used to prevent the development of overt recurrence, prompted by laboratory evidence of persisting malignant cells but without clinical or histopathologic evidence of recurrence.
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Approximately 7 years
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Proportion of Patients Who Discontinue Administration of Sirolimus Because of Toxicity
時間枠：Approximately 7 years
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Approximately 7 years
|
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Proportion With Infections Categorized by Organism
時間枠：Approximately 7 years
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Approximately 7 years
|
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Secondary Malignancies
時間枠：Up to 7 years
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Proportion of participants who developed at least one secondary malignancy by 7 years
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Up to 7 years
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Duration of Treatment With Prednisone
時間枠：Approximately 7 years
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Approximately 7 years
|
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Probability of Survival Without Recurrent Malignancy
時間枠：Approximately 7 years
|
Kaplan-Meier estimate assessed at 7 years for probability of survival without recurrent malignancy.
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Approximately 7 years
|
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Probability of Overall Survival
時間枠：Approximately 7 years
|
Kaplan-Meier estimate assessed at 7 years
|
Approximately 7 years
|
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Probability of Cumulative Incidence of Death Without Recurrent Malignancy
時間枠：Approximately 7 years
|
Analyzed with recurrent malignancy as a competing risk factor.
Assessed at 7 years.
|
Approximately 7 years
|
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Probability of Cumulative Incidence of Recurrent Malignancy
時間枠：Approximately 7 years
|
Analyzed with death as a competing risk factor.
Assessed at 7 years.
|
Approximately 7 years
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協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始
2002年4月1日
一次修了 (実際)
2009年7月1日
研究の完了 (実際)
2010年6月10日
試験登録日
最初に提出
2004年3月8日
QC基準を満たした最初の提出物
2004年3月9日
最初の投稿 (見積もり)
2004年3月10日
学習記録の更新
投稿された最後の更新 (実際)
2017年6月20日
QC基準を満たした最後の更新が送信されました
2017年5月23日
最終確認日
2017年5月1日
詳しくは
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