Clinical trials: Busulfan
DOCK8 Deficiency
Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency
Background: -DOCK8 deficiency is a genetic disorder that affects the immune system and can lead to severe recurrent infections and possible death from infections or certain types of cancers, including blood cancers.
- Trial name
- NCT01176006
- Testing
- Reduced-intensity hematopoietic stem cell, Fludarabine(Fludara, Berlex Laboratories), Cyclophosphamide(CTX, Cytoxan)
- Where
- United States
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
SCID
Conditioning SCID Infants Diagnosed Early
The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation.
- Trial name
- CSIDE (NCT03619551)
- Testing
- Busulfan, Cell processing for TCRαβ+/CD19+ depletion
- Where
- 38 sites in United States, Canada
- Volunteers
- 56 people
- Sponsor
- Center for International Blood and Marrow Transplant Research
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
This MyeloMATCH Master Screening and Reassessment Protocol (MSRP) evaluates the use of a screening tool and specific laboratory tests to help improve participants' ability to register to clinical trials throughout the course of…
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia, Myelodysplasia-Related
- Trial name
- NCT05564390
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Azacitidine, Best Practice
- Where
- 360 sites in United States, Canada and 1 more
- Volunteers
- 2,000 people
- Sponsor
- National Cancer Institute (NCI)
Core Binding Factor Alpha Subunits, Hematologic Neoplasms
Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers
Background: Some blood cancers can be caused by germline variants (changes) in a person s RUNX1 gene. Germline variants are genetic inherited changes a person is born with.
- Conditions
- Core Binding Factor Alpha Subunits, Hematologic Neoplasms, Leukemia, Lymphoma
- Trial name
- NCT07524530
- Testing
- Cyclophosphamide, Busulfan, Fludarabine
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- National Cancer Institute (NCI)
WHIM, Warts
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM
Background: Warts, hypogammaglobulinemia, infections and myelokathexis syndrome (WHIMs) is a rare disorder that affects the immune system. People with WHIMs can have severe infections all over their body.
- Conditions
- WHIM, Warts, Hypogammaglobulinemia, Immunodeficiency
- Trial name
- NCT07775313
- Testing
- Busulfan, Palifermin, Plerixafor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hematologic Malignancies
Giving Chemotherapy for a Shortened Amount of Time Before a Stem Cell Transplantation
The purpose of this study is to see if a condensed version of the chemotherapy regimen busulfan, melphalan, fludarabine (bu/mel/flu) and the drug antithymocyte globulin (ATG-also referred to as rATG or thymoglobulin) can have the…
- Trial name
- NCT04098393
- Testing
- Busulfan 3.2 mg/kg/day, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
X-linked Severe Combined Immunodeficiency (XSCID)
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficiency (XSCID, also known as SCID-X1) using a self-inactivating lentiviral…
- Trial name
- NCT01306019
- Testing
- Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Acute Leukemia, Acute Lymphoblastic Leukemia
Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)
This phase II trial investigates two strategies and how well they work for the reduction of graft versus host disease in patients with acute leukemia or MDS in remission.
- Conditions
- Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT03970096
- Testing
- Total-Body Irradiation, Thiotepa, Fludarabine
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- Fred Hutchinson Cancer Center
Acute Myeloid Leukemia
Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)
This phase II MyeloMATCH treatment trial compares the effect of adding venetoclax or placebo to reduced intensity conditioning chemotherapy with fludarabine and busulfan or melphalan, with or without total body irradiation…
- Trial name
- NCT06954987
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Biospecimen Collection, Bone Marrow Biopsy
- Volunteers
- 244 people
- Sponsor
- National Cancer Institute (NCI)
Lymphoproliferative Disorders, Autoimmune Lymphoproliferative
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies.
- Conditions
- Lymphoproliferative Disorders, Autoimmune Lymphoproliferative, Immune System Diseases, Common Variable Immunodeficiency
- Trial name
- NCT04339777
- Testing
- Busulfan test dose, Fludarabine, Busulfan
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- National Cancer Institute (NCI)
Myelodysplastic/Myeloproliferative Neoplasm, Primary Myelofibrosis
Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes
This phase II trial tests the effect of adding ruxolitinib to standard graft versus host disease (GVHD) prevention in treating older patients with myelofibrosis (MF) or myelodysplastic syndrome/myeloproliferative neoplasm…
- Conditions
- Myelodysplastic/Myeloproliferative Neoplasm, Primary Myelofibrosis, Secondary Myelofibrosis
- Trial name
- NCT07228624
- Testing
- Ruxolitinib, Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Fred Hutchinson Cancer Center
GATA2, Immunodeficiency
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Background: GATA2 deficiency is a disease caused by mutations in the GATA2 gene. It can cause different types of leukemia and other diseases. Researchers want to see if a stem cell transplant can be used to treat this condition.
- Conditions
- GATA2, Immunodeficiency, MDS
- Trial name
- NCT01861106
- Testing
- Allogeneic HSCT, Busulfan Test dose, Fludarabine (Fludara, Berlex Laboratories)
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Granulomatous Disease Transplant
High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
Chronic granulomatous disease (CGD) affects white blood cell function. Currently, the only curative treatment is bone marrow transplant to replace the abnormal stem cells with new ones (donor cells) capable of making a normal…
- Trial name
- NCT02629120
- Testing
- Alemtuzumab, Busulfan, Sirolimus
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease
Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide
Background: CGD causes infections and inflammation. The only cure currently is a bone marrow transplant. Most often a perfectly matched bone marrow donor is used.
- Trial name
- NCT03910452
- Testing
- Busulfan, Alemtuzumab, Cyclophosphamide
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Immunodeficiency, Hematopoietic Stem Cell Transplantation
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
Background: Allogeneic hematopoietic stem cell transplant involves taking blood stem cells from a donor and giving them to a recipient. The transplants are used to treat certain diseases and cancers.
- Trial name
- NCT05027945
- Testing
- Allogeneic HSCT, Busulfan test dose, Mycophenolate mofetil (MMF)
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- National Cancer Institute (NCI)
Hematologic Neoplasms
Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies
Background: People with blood cancers often receive blood or bone marrow transplants. But even with these treatments, the risk of relapse is high.
- Trial name
- NCT05327023
- Testing
- donor lymphocyte infusion, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 430 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
Gene Editing For Sickle Cell Disease
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a…
- Trial name
- NCT06506461
- Testing
- Plerixafor, Busulfan, Gene-modified CD34+ cells
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- St. Jude Children's Research Hospital
Acute Myeloid Leukemia, Myelodysplastic Syndromes
Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromes
The study is a Phase II clinical trial. Patients will receive intensity modulated total marrow irradiation (TMI) at a dose of 9 Gy with standard myeloablative fludarabine/ i.v.
- Trial name
- NCT03121014
- Testing
- Fludarabine, Busulfan, ATG
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- University of Illinois at Chicago
Chronic Granulomatous Disease
Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists
Background: Chronic granulomatous disease (CGD) affects the immune system. People with CGD are more likely to get infections. Drugs can help control infections, but these treatments can cause side effects including kidney failure…
- Trial name
- NCT05463133
- Testing
- Sirolimus, Cyclophosphamide, Alemtuzumab
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute
CD34+ Transplants for Myeloid Malignancies
This study will evaluate whether processing blood stem cell transplants using an investigational device (the CliniMACS system) results in less complications for patients undergoing transplant for treatment of a blood malignancy…
- Trial name
- NCT05565105
- Testing
- Busulfan, Melphalan, Fludarabine
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Guenther Koehne