G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome (G-CSF PMRD)
Feasibility Study of Using G-CSF Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome With Partially Mismatched Related Donors
The purposes of this study are:
- To examine the engraftment rate in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
- To evaluate the incidence and severity of acute and chronic graft-versus-host disease in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
調査の概要
詳細な説明
This study is a single-arm, non-randomized feasibility study. Patients meeting the criteria for this study will be entered sequentially until completion or closure of the study. Early stopping rules will be employed to ascertain whether an unacceptable rate of toxicity (non-engraftment, and/or acute GVHD) occurs.
Patients will be prepared for transplant through the administration of the following conditioning regimen based on their primary disease:
- Total body irradiation (1400 rads in 8 fractionated doses) and high dose chemotherapy, including cytosine arabinoside, etoposide, and cyclophosphamide. Patients with bone marrow failure syndrome will not receive etoposide in the conditioning regimen.
- Post transplant immunosuppression prophylaxis against acute GVHD will include sequential administration of cyclosporine, methotrexate, basiliximab and mycophenolate.
- The donor will receive 3 daily G-CSF injections prior to marrow harvest starting on day -3. The injections may be initiated by the donor's primary physician prior to donor's arrival, or by the BMT service at Children's Healthcare of Atlanta.
- Patients will receive daily GM-CSF injections (250 mcg/m2) starting from day +7 post transplant until absolute neutrophil count (ANC) is greater than 2,000/µL for three days.
研究の種類
入学 (実際)
段階
- 適用できない
連絡先と場所
研究場所
-
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Georgia
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Atlanta、Georgia、アメリカ、30322
- Children's Healthcare of Atlanta/Emory University
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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:
Patients with hematologic malignancies or bone marrow failure syndrome who are candidates for allogeneic bone marrow transplantation are eligible for this study. Hematologic malignancies indicated for transplantation:
- Acute lymphoblastic leukemia (ALL) in first remission (with high risk feature), 2nd or greater remission.
- Acute myeloid leukemia (AML) in first remission (with high risk feature), 2nd or greater remission.
- Chronic myeloid leukemia (CML) in 2nd chronic phase or accelerated phase.
- Juvenile myelomonocytic leukemia (JMML).
- Myelodysplastic syndrome.
- Biphenotypic leukemia in first (with high risk feature), 2nd or greater remission.
- Induction failure leukemia.
- Refractory relapsed leukemia.
- Bone marrow failure syndrome.
- Severe aplastic anemia failed immunotherapy.
- Patients who do not have a 6 out of 6 matched related or unrelated donor or 4/6 and 5/6 matched cord blood will be eligible for this study.
- Partially mismatched related donor availability as defined by molecular typing with 3 to 5 HLA matches.
- Patients who are under 22 years of age.
Exclusion Criteria:
- Patients will not be excluded based on sex, racial, or ethnic background.
Patients will be excluded if they demonstrate significant functional deficits in major organs, which would obviously interfere with successful outcome following bone marrow transplant utilizing the following guidelines.
- Evidence of active, deep-seated, life-threatening infections for which there is no known effective therapy (certain fungal species, HIV, etc.).
- Patients who have been treated for infections must have appropriate responses as documented by 2 (two) consecutive negative cultures and/or stable radiographic examinations.
- Patients who have active central nervous system (CNS) leukemic disease.
- Patients will be excluded if they are women of childbearing potential who are currently pregnant (beta-HCG+) or who are not practicing adequate contraception.
- Patients who have had a previous hematopoietic stem cell transplant will be excluded.
- Donors will be excluded if they are sensitive to E. coli-derived protein.
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 |
|---|---|
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他の：Granulocyte Colony Stimulating Factor (G-CSF) stimulation
Participants will receive bone marrow from donors who undergo Granulocyte Colony Stimulating Factor (G-CSF) stimulation prior to bone marrow collection.
|
FILGRASTIM: G-CSF (NEUPOGEN®) is administered as a short IV infusion over 30 minutes or subcutaneously. It is given beginning on day -3 for 3 days to the donor prior to the bone marrow harvest. Drug Information: FILGRASTIM: G-CSF (Neupogen®) Formulation: G-CSF is available as a preservative-free solution for injection in 1.0 ml and 1.6 ml vials containing 300 mcg/ml. Administration: G-CSF 5 mcg/kg/d will be given subcutaneously or as a short I.V. infusion over 30 minutes. Recombinant GM-CSF at the dose of 250 mcg/m2 will be given intravenously from day +7 to help white counts recovery. The drug will be diluted in NS at a concentration of at least 10 mcg/ml. Drug Information: Sargramostim (Leukine) Formulation: 250 mcg, 500 mcg lyophlized powder for injection |
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 |
|---|---|---|
|
Engraftment Rate
時間枠：Day 45
|
The number of participants who received in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donor who reached engraftment by Day 45.
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Day 45
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Incidence of Acute Graft-versus-host Disease
時間枠：Day 100
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The number of participants diagnosed with new acute graft-versus-host disease (GVHD).
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Day 100
|
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Incidence of Chronic Graft-versus-host Disease
時間枠：Duration of Study (Up to two years)
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The number of participants diagnosed with chronic graft-versus-host disease (GVHD).
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Duration of Study (Up to two years)
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協力者と研究者
スポンサー
捜査官
- 主任研究者：Kuang-Yueh Chiang, M.D.、Children's Healthcare of Atlanta/Emory University
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 (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
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