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- Klinische proef NCT07565220
Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies
Phase 1 Trial of Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies
Studie Overzicht
Toestand
Conditie
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Inschrijving (Geschat)
Fase
- Fase 1
Contacten en locaties
Studiecontact
- Naam: Amy Rogers, RN, BSN
- Telefoonnummer: 412-623-4036
- E-mail: rodgera@upmc.edu
Studie Contact Back-up
- Naam: Linda Elias, RN, BSN
- Telefoonnummer: 412-623-6037
- E-mail: eliaslj@upmc.edu
Studie Locaties
-
-
Pennsylvania
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Pittsburgh, Pennsylvania, Verenigde Staten, 15213
- Werving
- UMPC Hillman Cancer Center
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Hoofdonderzoeker:
- Sawa Ito, MD
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Contact:
- Amy Rogers, RN, BSN
- Telefoonnummer: 412-623-4036
- E-mail: rodgera@upmc.edu
-
Contact:
- Linda Elias, RN, BSN
- Telefoonnummer: 412-623-6037
- E-mail: eliaslj@upmc.edu
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-
Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Beschrijving
Inclusion Criteria:
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:
- Age 50-70 years old or
- Age 18-49 and unfit for a conventional myeloablative conditioning regimen per the treating physician
Patients have one of the following diagnoses:
- Acute lymphocytic leukemia (ALL) in first or subsequent morphological remission (<5% marrow blasts by morphology).
- Acute myeloid leukemia (AML) in first or subsequent morphological remission (<5% marrow blasts by morphology) with or without hematologic recovery.
- 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.
- 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.
- High-risk myeloproliferative neoplasm (MPN) with no evidence of high-grade bone marrow fibrosis or massive splenomegaly at the time of enrollment.
- Patients with an 8/8 HLA-matched (HLA-A, B, C, DRB1) related or unrelated donor capable of donating peripheral blood stem cells (PBSC)
- Provision of signed and dated informed consent form
- 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.
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:
- 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:
- Poor performance status with Karnofsky Score <70%
- Center for International Blood and Marrow Transplant Research (CIBMTR) hematopoietic cell transplant co-morbidity index (HCT-CI) score >5
- Patients with active central nervous system (CNS) involvement refractory to intrathecal chemotherapy and/or standard craniospinal radiation.
- Patients who are positive for HIV-1, HIV-2, HTLV1 or HTLV2.
- Patients with uncontrolled infections for whom alloSCT is considered contraindicated by the consulting infectious disease physician.
Patients with organ dysfunction, including:
- Renal insufficiency creatinine clearance <45 ml/min/1.72m2 measured by 24-hr urine specimen
- Left ventricular ejection fraction <45%
- Diffusing capacity of the lung for carbon monoxide (DLCO) corrected <50% or FEV1 <50%
- 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.
- Patients who have received previous allogeneic transplantation.
- Patients with a life expectancy <12 months due to co-existing diseases other than hematologic malignancies.
- Patients with any other significant medical conditions that would make them unsuitable for transplantation, as determined by the PI.
- Patients with a known hypersensitivity to cyclophosphamide, thiotepa, fludarabine, busulfan, tacrolimus, or mycophenolate mofetil (MMF).
- Patients who have received checkpoint inhibitors within three months of transplantation, unless an exception is made by the PI.
For low-intensity regimen
- Poor performance status with Karnofsky Score <60%
- Patients with active CNS involvement refractory to intrathecal chemotherapy and/or standard craniospinal radiation.
- Patients who are positive for HIV-1, HIV-2, HTLV1 or HTLV2.
- Patients with uncontrolled infections for whom alloSCT is considered contraindicated by the consulting infectious disease physician.
Patients with organ dysfunction, including:
- Renal insufficiency creatinine clearance <40 ml/min/1.72m2 measured by 24-hr urine specimen
- Left ventricular ejection fraction <40%
- DLCO corrected< 50% or FEV1<50%
- 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.
- Patients who have received previous allogeneic transplantation.
- Patients with a life expectancy <12 months from co-existing disease other than hematologic malignancies
- Patients with any other significant medical conditions that would make them unsuitable for transplantation, as determined by the PI.
- Patients with a known hypersensitivity to cyclophosphamide, thiotepa, fludarabine, busulfan, tacrolimus, or mycophenolate mofetil (MMF).
- Patients who have received checkpoint inhibitors within three months of transplantation, unless an exception is made by the PI.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Niet-gerandomiseerd
- Interventioneel model: Parallelle opdracht
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
|
Experimenteel: 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.
Andere namen:
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.
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|
Experimenteel: 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.
Andere namen:
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.
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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GVHD-free, relapse-free survival (GRFS)
Tijdsspanne: At 1 year
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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.
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At 1 year
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Median time to neutrophil engraftment
Tijdsspanne: 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%.
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Up to 30 days
|
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Median time to platelet engraftment
Tijdsspanne: Up to 30 days
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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.
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Up to 30 days
|
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Frequency of severe mucositis
Tijdsspanne: Up to 30 days post-transplant
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Percentage of patients experiencing severe mucositis, defined as grade 3-4 by WHO criteria.
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Up to 30 days post-transplant
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Frequency of total parental nutrition
Tijdsspanne: Up to 30 days post-transplant
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Percentage of patients who require TPN at any time from the start of conditioning through day +30 post-transplant.
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Up to 30 days post-transplant
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Frequency of severe pulmonary complications requiring ICU-level support
Tijdsspanne: Up to 30 days
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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%.
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Up to 30 days
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Cumulative incidence of infectious disease complications
Tijdsspanne: Up to 1-year post-transplant
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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.
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Up to 1-year post-transplant
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Cumulative incidence of thrombotic microangiopathy
Tijdsspanne: Up to 180 days post-transplant
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Up to 180 days post-transplant
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Grade III-IV acute GVHD-free survival
Tijdsspanne: Up to 100 days post-transplant
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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.
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Up to 100 days post-transplant
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Moderate to severe chronic GVHD-free survival
Tijdsspanne: At 1 year1 year post-transplant
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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.
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At 1 year1 year post-transplant
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Primary graft failure
Tijdsspanne: Up to Day 42
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Failure to achieve an absolute neutrophil count (ANC) > 0.5 x 109/L by day +42 after stem cell infusion.
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Up to Day 42
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Medewerkers en onderzoekers
Sponsor
Onderzoekers
- Hoofdonderzoeker: Sawa M Ito, MD, UPMC Hillman Cancer Center
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
- Neoplasmata
- Ziekten van het immuunsysteem
- Neoplasmata per histologisch type
- Hematologische ziekten
- Lymfatische ziekten
- Lymfoproliferatieve aandoeningen
- Immunoproliferatieve aandoeningen
- Leukemie, myeloïde
- Beenmergziekten
- Leukemie, Lymfoïde
- Leukemie
- Hemische en lymfatische ziekten
- Leukemie, myeloïde, acuut
- Voorlopercel lymfoblastische leukemie-lymfoom
- Myelodysplastische syndromen
- Myeloproliferatieve aandoeningen
- Graft vs Host-ziekte
- Zwavelverbindingen
- Organische chemicaliën
- Heterocyclische verbindingen, 1-ring
- Heterocyclische verbindingen
- Koolwaterstoffen, acyclisch
- Koolwaterstoffen
- Alkanes
- Alcohol
- Butyleenglycolen
- Glycolen
- Mesyleert
- Alkanesulfonaten
- Alkanesulfonzuren
- Sulfonzuren
- Zwavelzuren
- Fosforamides
- Organofosforverbindingen
- Triethylepenefosforamide
- Aziridines
- Azirines
- Busulfan
- Thiotepa
- fludarabine
Andere studie-ID-nummers
- HCC 26-008
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