New transplant cocktail aims to tame blood cancer without severe side effects
NCT ID NCT07565220
First seen Jun 26, 2026 · Last updated Aug 19, 2026 · Updated 5 times
Summary
This early-stage trial is testing a new combination of chemotherapy drugs (thiotepa, busulfan, and fludarabine) followed by a stem cell transplant for people with certain blood cancers like leukemia and myelodysplastic syndrome. The goal is to see if this approach can reduce the dose of a post-transplant drug (cyclophosphamide) that helps prevent graft-versus-host disease, a serious complication. The study will enroll 48 adults aged 18-70 who are candidates for a stem cell transplant.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Thiotepa, Fludarabine, Busulfan (chemotherapy drugs) and a stem cell transplant
- What this could lead to
- If successful, this could lead to a safer and more effective transplant procedure for people with blood cancers, reducing the risk of graft-versus-host disease and relapse.
- What could go wrong
- This is an early Phase 1 trial with only 48 participants, so the results may not apply to everyone. The treatment involves strong chemotherapy and carries risks like infection, organ damage, and graft-versus-host disease.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 48 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jul 2026
- Expected to finish
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Nov 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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UMPC Hillman Cancer Center
RECRUITINGPittsburgh, Pennsylvania, 15213, United States
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