Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the growth of abnormal cells by stopping them from dividing or killing them. Giving colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib followed by stem cell transplant works in treating patients with primary systemic amyloidosis.
調査の概要
詳細な説明
OBJECTIVES:
- To determine if hematologic responses to high-dose melphalan and autologous stem cell transplantation increase with addition of bortezomib in the conditioning regimen in patients with primary systemic amyloidosis.
OUTLINE:
- Autologous stem cell mobilization and collection: Patients receive filgrastim to mobilize stem cells, which are then collected.
- Conditioning regimen: Patients receive bortezomib intravenously on days -6, -3, 1, and 4 and oral high-dose melphalan on days -2 and -1.
- Stem cell transplantation: Patients undergo autologous stem cell transplantation on day 0.
After completion of study therapy, patients are followed every 6 months for 1 year and annually thereafter.
研究の種類
入学 (実際)
段階
- フェーズ2
連絡先と場所
研究場所
-
-
Massachusetts
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Boston、Massachusetts、アメリカ、02118
- Boston University Cancer Research Center
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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:
DISEASE CHARACTERISTICS:
Histologically confirmed primary systemic amyloidosis based on the following criteria:
- Amyloid light-chain disease
- Deposition of amyloid material by congo red stain showing characteristic green birefringence
- Monoclonal light chain protein (Bence Jones protein) in the serum or urine, immunohistochemical studies, or serum free light chain assay
- Evidence of tissue involvement other than carpal tunnel syndrome (i.e., positive immunohistochemical staining of bone marrow demonstrating clonal plasma cells); tissue amyloid deposits with anti-kappa or anti-lambda anti-serum; evidence for a plasma cell dyscrasia by serum/urine or bone marrow; or overwhelmingly convincing clinical features (e.g., macroglossia) associated with other systemic manifestations
PATIENT CHARACTERISTICS:
- Southwest Oncology Group performance status 0-1
- Fertile patients must use effective contraception
- Left ventricular ejection fraction ≥ 45% by Echocardiogram within the past 60 days
- diffusion capacity of lung for carbon monoxide ≥ 50%
PRIOR CONCURRENT THERAPY:
- Prior chemotherapy with alkylating agent allowed provided there is no morphological or cytogenetic evidence of myelodysplastic syndromes
- Prior total cumulative dose of oral melphalan < 300 mg
- At least 4 weeks since prior cytotoxic therapy and fully recovered
Exclusion criteria:
- No senile, secondary, localized, dialysis-related, or familial amyloidosis
- No overt multiple myeloma (> 30% of bone marrow plasmacytosis, extensive [> 2] lytic lesions, or hypercalcemia)
- Not pregnant or nursing
- No myocardial infarction within the past 6 months, congestive heart failure, or arrhythmia refractory to therapy
No prior malignancy except for any of the following:
- Adequately treated basal cell or squamous cell skin cancer
- In situ cervical cancer
- Adequately treated stage I or II cancer currently in complete remission
- Any cancer from which the patient has been disease-free ≥ 5 years
- No advanced (grade 3-4) pre-existing neuropathy
- No HIV positivity
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：Stem Cell Transplant with Bortezomib and Melphalan
Mobilization with Filgrastim Stem Cell Collection Bortezomib Melphalan Stem Cell infusion
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16 mcg/kg daily beginning 3 days before stem cell collection through day before final stem cell collection
他の名前：
1.0 mg/m2/dose D -6, D-3, D +1, D + 4
他の名前：
100 mg/m2/dose D -2, D -1
他の名前：
infusion of previously collected autologous stem cells
|
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 With Hematologic Response
時間枠：one year
|
complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy <5% plasma cells with no clonal predominance of kappa or lambda isotype. Partial response: any one of the following
|
one year
|
二次結果の測定
結果測定 |
時間枠 |
|---|---|
|
Number of Participants Surviving at 100 Days From Transplant
時間枠：100 Days from transplant date
|
100 Days from transplant date
|
|
Number of Participants Surviving at 1 Year
時間枠：one year from transplant
|
one year from transplant
|
|
Number of Participants Surviving at 2 Years
時間枠：2 years from transplant
|
2 years from transplant
|
協力者と研究者
スポンサー
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 (見積もり)
学習記録の更新
投稿された最後の更新 (見積もり)
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
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- CDR0000618857
- BUMC-H-27277 (その他の識別子：Boston University Medical Center IRB)
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?
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