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Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies

2026年8月17日 更新者:Sawa Ito, MD

Phase 1 Trial of Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies

This phase 1 trial will investigate the safety and effectiveness of Thiotepa, Busulfan, and Fludarabine (TBF) conditioning regimen with post-transplant cyclophosphamide (PTCy) in HLA-matched related or unrelated donor allogeneic stem cell transplantation (alloSCT).

調査の概要

詳細な説明

Allogeneic stem cell transplantation (alloSCT) offers potential curative therapy for individuals with high-risk hematologic malignancies. Establishing appropriate immune tolerance between donor and recipient is essential to prevent graft-versus-host disease (GVHD) and graft rejection. Over the past decade, the introduction of post-graft cyclophosphamide (PTCy) as an immune-tolerance-inducing strategy has substantially reshaped the alloSCT landscape.

This trial aims to optimize the PTCy regimen through two primary approaches: 1) de-escalation of PTCy dosing to reduce toxicities, and 2) incorporation of thiotepa to strengthen anti-leukemia activity. The central hypothesis is that regimen optimization will improve transplant outcomes-particularly graft-versus-host disease and relapse-free survival (GRFS)-for recipients of HLA-matched donor alloSCT with high-risk hematologic malignancies. An exploratory objective will evaluate pre-transplant biomarkers capable of stratifying toxicity and relapse risk, enabling personalization of regimen intensity for each transplant recipient.

研究の種類

介入

入学 (推定)

48

段階

  • フェーズ 1

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。

研究連絡先

  • 名前:Amy Rogers, RN, BSN
  • 電話番号:412-623-4036
  • メール:rodgera@upmc.edu

研究連絡先のバックアップ

  • 名前:Linda Elias, RN, BSN
  • 電話番号:412-623-6037
  • メール:eliaslj@upmc.edu

研究場所

    • Pennsylvania
      • Pittsburgh、Pennsylvania、アメリカ、15213
        • 募集
        • UMPC Hillman Cancer Center
        • 主任研究者:
          • Sawa Ito, MD
        • コンタクト:
        • コンタクト:

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

  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

説明

Inclusion Criteria:

  1. Patients must be considered appropriate candidates for either the low- or high-intensity conditioning regimen for allogeneic hematopoietic stem cell transplantation based on the following age-related criteria:

    1. Age 50-70 years old or
    2. Age 18-49 and unfit for a conventional myeloablative conditioning regimen per the treating physician
  2. Patients have one of the following diagnoses:

    1. Acute lymphocytic leukemia (ALL) in first or subsequent morphological remission (<5% marrow blasts by morphology).
    2. Acute myeloid leukemia (AML) in first or subsequent morphological remission (<5% marrow blasts by morphology) with or without hematologic recovery.
    3. Other acute leukemia or related neoplasm (including but not limited to 'mixed phenotype' 'biphenotypic', 'acute undifferentiated' or 'ambiguous lineage' acute leukemia, blastic plasmacytoid dendritic cell neoplasm, lymphoblastic lymphoma, Burkitt leukemia/lymphoma, mast cell leukemia or chronic myeloid leukemia with blast crisis) in first or subsequent morphological remission (<5% marrow blasts by morphology) with or without hematologic recovery.
    4. Myelodysplastic syndrome (MDS) with a history of excess blasts, with >5% marrow blasts by morphology after receiving at least one cycle of treatment, including but not limited to hypomethylating agent, BCL-2 inhibitor, cytoreductive chemotherapy.
    5. High-risk myeloproliferative neoplasm (MPN) with no evidence of high-grade bone marrow fibrosis or massive splenomegaly at the time of enrollment.
  3. Patients with an 8/8 HLA-matched (HLA-A, B, C, DRB1) related or unrelated donor capable of donating peripheral blood stem cells (PBSC)
  4. Provision of signed and dated informed consent form
  5. Sexually active fertile subjects and their partners must agree to use highly effective methods of contraception prior to study entry, during the course of the study, and until tacrolimus or other immunosuppressive therapy for GVHD is discontinued (whichever is later). An additional contraceptive method, such as a barrier method (e.g., condom), is required. In addition, men must agree not to donate sperm and women must agree not to donate eggs (ova, oocyte) for the purpose of reproduction during these same periods.
  6. Female subjects of childbearing potential must not be pregnant or breastfeeding at screening. Female subjects are considered to be of childbearing potential unless one of the following criteria is met:

    1. Permanent sterilization (hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) or documented postmenopausal status (defined as 12 months of amenorrhea in a woman > 45 years-of-age in the absence of other biological or physiological causes). Note: Documentation may include review of medical records, medical examination, or medical history interview by study site staff.

Exclusion Criteria:

Subjects will be excluded from the study if they meet any of the following criteria.

For high-intensity regimen:

  1. Poor performance status with Karnofsky Score <70%
  2. Center for International Blood and Marrow Transplant Research (CIBMTR) hematopoietic cell transplant co-morbidity index (HCT-CI) score >5
  3. Patients with active central nervous system (CNS) involvement refractory to intrathecal chemotherapy and/or standard craniospinal radiation.
  4. Patients who are positive for HIV-1, HIV-2, HTLV1 or HTLV2.
  5. Patients with uncontrolled infections for whom alloSCT is considered contraindicated by the consulting infectious disease physician.
  6. Patients with organ dysfunction, including:

    1. Renal insufficiency creatinine clearance <45 ml/min/1.72m2 measured by 24-hr urine specimen
    2. Left ventricular ejection fraction <45%
    3. Diffusing capacity of the lung for carbon monoxide (DLCO) corrected <50% or FEV1 <50%
    4. Liver function abnormality: total bilirubin, aspartate aminotransferase (AST), and alanine aminotransferase (ALT) >5 times the upper limit of normal should be evaluated by a gastroenterologist. If a gastroenterologist considers that alloSCT is contraindicated, the patient will be excluded from the protocol.
  7. Patients who have received previous allogeneic transplantation.
  8. Patients with a life expectancy <12 months due to co-existing diseases other than hematologic malignancies.
  9. Patients with any other significant medical conditions that would make them unsuitable for transplantation, as determined by the PI.
  10. Patients with a known hypersensitivity to cyclophosphamide, thiotepa, fludarabine, busulfan, tacrolimus, or mycophenolate mofetil (MMF).
  11. Patients who have received checkpoint inhibitors within three months of transplantation, unless an exception is made by the PI.

For low-intensity regimen

  1. Poor performance status with Karnofsky Score <60%
  2. Patients with active CNS involvement refractory to intrathecal chemotherapy and/or standard craniospinal radiation.
  3. Patients who are positive for HIV-1, HIV-2, HTLV1 or HTLV2.
  4. Patients with uncontrolled infections for whom alloSCT is considered contraindicated by the consulting infectious disease physician.
  5. Patients with organ dysfunction, including:

    1. Renal insufficiency creatinine clearance <40 ml/min/1.72m2 measured by 24-hr urine specimen
    2. Left ventricular ejection fraction <40%
    3. DLCO corrected< 50% or FEV1<50%
    4. Liver function abnormality: total bilirubin, AST, ALT>5 times the upper limit of normal should be evaluated by a gastroenterologist. If a gastroenterologist considers that alloSCT is contraindicated, the patient will be excluded from the protocol.
  6. Patients who have received previous allogeneic transplantation.
  7. Patients with a life expectancy <12 months from co-existing disease other than hematologic malignancies
  8. Patients with any other significant medical conditions that would make them unsuitable for transplantation, as determined by the PI.
  9. Patients with a known hypersensitivity to cyclophosphamide, thiotepa, fludarabine, busulfan, tacrolimus, or mycophenolate mofetil (MMF).
  10. Patients who have received checkpoint inhibitors within three months of transplantation, unless an exception is made by the PI.

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:処理
  • 割り当て:非ランダム化
  • 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
実験的:Cohort 1: T1FluBu2 (low-intensity)
Thiotepa: 5 mg/kg - administered on Day -5 prior to PBSC infusion Fludarabine: 30 mg/m2/day - administered on Day -4, Day -3, Day -2 and Day -1 prior to PBSC infusion Busulfan: 3.2 mg/kg/day IV - administered on Day -4 and Day -3 prior to PBSC infusion PBSC infusion: given on Day 0
Thiotepa is an alkylating agent used in combination with other chemotherapy agents to treat cancer.
他の名前:
  • テパディナ
Fludarabine is a chemotherapy drug used in the treatment of chronic lymphocytic leukemia. It acts at DNA polymerase alpha, ribonucleotide reductase and DNA primase, results in the inhibition of DNA synthesis, and destroys the cancer cells.
Busulfan is a chemotherapy drug used in preparation for a stem cell transplant.
Peripheral Blood Stem Cell (PBSC) infusion is a medical procedure used to replace diseased or damaged stem cells in patients, particularly after cancer treatments.
実験的:Cohort 2: T2FluBu2 (high-intensity)
Thiotepa: 5 mg/kg - administered on Day -5 prior to PBSC infusion Fludarabine: 40 mg/m2/day - administered on Day -4, Day -3, Day -2 and Day -1 prior to PBSC infusion Busulfan: 3.2 mg/kg/day IV - administered on Day -4 and Day -3 prior to PBSC infusion PBSC infusion: given on Day 0
Thiotepa is an alkylating agent used in combination with other chemotherapy agents to treat cancer.
他の名前:
  • テパディナ
Fludarabine is a chemotherapy drug used in the treatment of chronic lymphocytic leukemia. It acts at DNA polymerase alpha, ribonucleotide reductase and DNA primase, results in the inhibition of DNA synthesis, and destroys the cancer cells.
Busulfan is a chemotherapy drug used in preparation for a stem cell transplant.
Peripheral Blood Stem Cell (PBSC) infusion is a medical procedure used to replace diseased or damaged stem cells in patients, particularly after cancer treatments.

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
GVHD-free, relapse-free survival (GRFS)
時間枠:At 1 year
Graft Versus Host Disease (GVHD)-free, relapse-free survival (GRFS) is defined by survival without a qualifying event including Death, Relapse of primary disease, Grade III-IV acute graft-versus-host disease (GVHD), graded via MAGIC criteria, Chronic moderate or severe GVHD requiring systemic immunosuppression, graded via NIH consensus criteria.
At 1 year

二次結果の測定

結果測定
メジャーの説明
時間枠
Median time to neutrophil engraftment
時間枠:Up to 30 days
Median time to neutrophil engraftment will be defined as the median number of days from transplant at which the cumulative engraftment rate reaches 50%.
Up to 30 days
Median time to platelet engraftment
時間枠:Up to 30 days
Median number of days from transplant at which the cumulative engraftment rate of platelet recovery to 20,000/mm3 and 50,000/mm3 reaches 50% respectively.
Up to 30 days
Frequency of severe mucositis
時間枠:Up to 30 days post-transplant
Percentage of patients experiencing severe mucositis, defined as grade 3-4 by WHO criteria.
Up to 30 days post-transplant
Frequency of total parental nutrition
時間枠:Up to 30 days post-transplant
Percentage of patients who require TPN at any time from the start of conditioning through day +30 post-transplant.
Up to 30 days post-transplant
Frequency of severe pulmonary complications requiring ICU-level support
時間枠:Up to 30 days
Percentage of patients with first occurrence of any respiratory failure, severe infectious lower respiratory tract infection, or noninfectious acute lung injury, requiring ICU-level support between Day 0 and Day +30 after stem cell infusion. ICU-level support is defined by either 1) ICU admission due to respiratory failure, 2) need for new non-invasive ventilation (BiPAP or CPAP), or 3) requirement of high-flow nasal cannula >30L/min at FiO2>40%.
Up to 30 days
Cumulative incidence of infectious disease complications
時間枠:Up to 1-year post-transplant
Cumulative incidence of all Grade II and higher infections will be reported according to Blood and Marrow Transplant Clinical Trials Network (BMT-CTN) Infection Grading Criteria. The BMT CTN grading system provides a standardized approach for capturing and monitoring infectious complications in clinical trials.
Up to 1-year post-transplant
Cumulative incidence of thrombotic microangiopathy
時間枠:Up to 180 days post-transplant
  1. . Microangiopathic hemolytic anemia, defined by: a. Evidence of schistocytes on peripheral blood smear, and b. Elevated lactate dehydrogenase (LDH) above the upper limit of normal, and c. Decreased haptoglobin or other laboratory evidence of hemolysis (e.g., indirect hyperbilirubinemia), and d. Negative Coombs (direct antiglobulin) test.
  2. Thrombocytopenia: a. New or progressive thrombocytopenia not explained by disease relapse, infection, drug effect, or disseminated intravascular coagulation (DIC).
  3. Renal and/or neurologic dysfunction temporally associated with hemolysis, not fully explained by other causes: a. Rising serum creatinine or new/worsening hypertension, and/or b. New-onset or worsening neurologic signs/symptoms (e.g., confusion, seizures, focal deficits).
  4. No alternative explanation: a. ADAMTS13 activity not suggestive of TTP (if available) and clinical evaluation not consistent with DIC, severe sepsis, or malignant hypertension as the primary cause.
Up to 180 days post-transplant
Grade III-IV acute GVHD-free survival
時間枠:Up to 100 days post-transplant
Time from date of stem cell infusion to the first occurrence of grade III or IV acute Graft Versus Host Disease (GVHD), with follow-up through 100 days post-transplant or death.
Up to 100 days post-transplant
Moderate to severe chronic GVHD-free survival
時間枠:At 1 year1 year post-transplant
Time from date of stem cell infusion to the first occurrence of moderate-to-severe chronic Graft Versus Host Disease (GVHD) with follow up through 1-year post-transplant or death.
At 1 year1 year post-transplant
Primary graft failure
時間枠:Up to Day 42
Failure to achieve an absolute neutrophil count (ANC) > 0.5 x 109/L by day +42 after stem cell infusion.
Up to Day 42

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。

スポンサー

捜査官

  • 主任研究者:Sawa M Ito, MD、UPMC Hillman Cancer Center

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 (実際)

2026年7月27日

一次修了 (推定)

2029年6月1日

研究の完了 (推定)

2030年11月1日

試験登録日

最初に提出

2026年4月27日

QC基準を満たした最初の提出物

2026年4月27日

最初の投稿 (実際)

2026年5月4日

学習記録の更新

投稿された最後の更新 (実際)

2026年8月18日

QC基準を満たした最後の更新が送信されました

2026年8月17日

最終確認日

2026年8月1日

詳しくは

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