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

27 aprile 2026 aggiornato da: 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).

Panoramica dello studio

Descrizione dettagliata

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.

Tipo di studio

Interventistico

Iscrizione (Stimato)

48

Fase

  • Fase 1

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

  • Nome: Amy Rogers, RN, BSN
  • Numero di telefono: 412-623-4036
  • Email: rodgera@upmc.edu

Backup dei contatti dello studio

  • Nome: Linda Elias, RN, BSN
  • Numero di telefono: 412-623-6037
  • Email: eliaslj@upmc.edu

Luoghi di studio

    • Pennsylvania
      • Pittsburgh, Pennsylvania, Stati Uniti, 15213
        • UMPC Hillman Cancer Center
        • Investigatore principale:
          • Sawa Ito, MD
        • Contatto:
        • Contatto:

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Adulto
  • Adulto più anziano

Accetta volontari sani

No

Descrizione

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.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: 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.
Altri nomi:
  • 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.
Sperimentale: 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.
Altri nomi:
  • 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.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
GVHD-free, relapse-free survival (GRFS)
Lasso di tempo: 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

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Median time to neutrophil engraftment
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Investigatori

  • Investigatore principale: Sawa M Ito, MD, UPMC Hillman Cancer Center

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Stimato)

1 giugno 2026

Completamento primario (Stimato)

1 giugno 2029

Completamento dello studio (Stimato)

1 novembre 2030

Date di iscrizione allo studio

Primo inviato

27 aprile 2026

Primo inviato che soddisfa i criteri di controllo qualità

27 aprile 2026

Primo Inserito (Effettivo)

4 maggio 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

4 maggio 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

27 aprile 2026

Ultimo verificato

1 aprile 2026

Maggiori informazioni

Termini relativi a questo studio

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

prodotto fabbricato ed esportato dagli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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