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

27. dubna 2026 aktualizováno: 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).

Přehled studie

Detailní popis

Allogeneic stem cell transplantation (alloSCT) offers potential cures for patients with high-risk hematologic malignancies. Establishing appropriate immune tolerance between the donor and recipient is crucial to prevent graft-versus-host disease (GVHD) and graft rejection. Over the last decade, the introduction of post-graft cyclophosphamide (PTCy)1,2 as a strategy for inducing immune tolerance has significantly transformed the landscape of alloSCT.

This trial will optimize the PTCy regimen through two main strategies: 1) de-escalating the PTCy dose to reduce toxicities, and 2) incorporating thiotepa to enhance the anti-leukemia effect. We hypothesize that this optimization will improve transplant outcomes, specifically with respect to GVHD and relapse-free survival (GRFS), for recipients of HLA-matched donor alloSCT who have high-risk hematologic malignancies. Additionally, in our exploratory aim, we will investigate potential pre-transplant biomarkers that can help stratify toxicity and relapse risks, allowing us to personalize the optimal regimen intensity for individual recipients.

Typ studie

Intervenční

Zápis (Odhadovaný)

48

Fáze

  • Fáze 1

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

  • Jméno: Amy Rogers, RN, BSN
  • Telefonní číslo: 412-623-4036
  • E-mail: rodgera@upmc.edu

Studijní záloha kontaktů

  • Jméno: Linda Elias, RN, BSN
  • Telefonní číslo: 412-623-6037
  • E-mail: eliaslj@upmc.edu

Studijní místa

    • Pennsylvania
      • Pittsburgh, Pennsylvania, Spojené státy, 15213
        • UMPC Hillman Cancer Center
        • Vrchní vyšetřovatel:
          • Sawa Ito, MD
        • Kontakt:
        • Kontakt:
          • Linda Elias, RN, BSN
          • Telefonní číslo: 412-623-6037
          • E-mail: eliaslj@upmc.edu

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

  • Dospělý
  • Starší dospělý

Přijímá zdravé dobrovolníky

Ne

Popis

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.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Nerandomizované
  • Intervenční model: Paralelní přiřazení
  • Maskování: Žádné (otevřený štítek)

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: 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.
Ostatní jména:
  • Tepadina
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.
Experimentální: 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.
Ostatní jména:
  • Tepadina
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.

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
GVHD-free, relapse-free survival (GRFS)
Časové okno: 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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Median time to neutrophil engraftment
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Vyšetřovatelé

  • Vrchní vyšetřovatel: Sawa M Ito, MD, UPMC Hillman Cancer Center

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Odhadovaný)

1. června 2026

Primární dokončení (Odhadovaný)

1. června 2029

Dokončení studie (Odhadovaný)

1. listopadu 2030

Termíny zápisu do studia

První předloženo

27. dubna 2026

První předloženo, které splnilo kritéria kontroly kvality

27. dubna 2026

První zveřejněno (Aktuální)

4. května 2026

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

4. května 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

27. dubna 2026

Naposledy ověřeno

1. dubna 2026

Více informací

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