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Showing 1–24 of 99 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Myeloablative Busulfan Clear all
Terminated Phase 2Phase 3 Interventional Results available

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

NCT00176826
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
Interventions
Stem Cell Transplant, Myeloablative conditioning regimen
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 22, 2018 · Synced Sep 7, 2026, 2:14 AM EDT
Recruiting Phase 2 Interventional

Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

NCT03314974
View directory record Official record
Conditions
Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Lymphoma, Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myeloproliferative Neoplasms, Myelofibrosis, Myelodysplasia, Refractory Anemia, High Risk Anemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Diffuse Large Cell Non Hodgkins Lymphoma, Lymphoblastic Lymphoma, Burkitt Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult, Multiple Myeloma, Juvenile Myelomonocytic Leukemia, Biphenotypic/Undifferentiated/Prolymphocytic Leukemias, MRD Positive Leukemia, Natural Killer Cell Malignancies, Acquired Bone Marrow Failure Syndromes
Interventions
HSCT with TBI Regimen, HSCT with Non-TBI Regimen
Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 60 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Sep 7, 2026, 2:14 AM EDT
Recruiting Not applicable Interventional

CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT

NCT03810196
View directory record Official record
Conditions
Acute Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndromes, Acute Lymphoblastic Leukemia, Mixed Lineage Leukemia, Lymphoblastic Lymphoma, Burkitt Lymphoma, Juvenile Myelomonocytic Leukemia
Interventions
CliniMACS Cell Processing System for TCRαβ + T Cell and CD45RA Depleted Peripheral Stem Cell Addback
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 25 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional Results available

A Phase II Study Evaluating the Safety and Efficacy of Subcutaneous Plerixafor

NCT01696461
View directory record Official record
Conditions
Related Donors Donating Peripheral Blood Stem Cells (PBSC) to a Family Member, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia, Non-Hodgkin's Lymphoma, Hodgkin's Disease, Chronic Lymphocytic Leukemia
Interventions
Plerixafor
Drug
Lead sponsor
Center for International Blood and Marrow Transplant Research
Network
Eligibility
18 Years to 65 Years
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
12
States / cities
Tampa, Florida • Atlanta, Georgia • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional Results available

Myeloablative Haploidentical BMT With Post-transplant Cyclophosphamide for Pediatric Patients With Hematologic Malignancies

NCT02120157
View directory record Official record
Conditions
Myeloablative Conditioning, HLA-mismatched Bone Marrow Transplantation, Graft Survival, Transplantation, Bone Marrow
Interventions
Cyclophosphamide, TBI, Busulfan, Unmanipulated Bone Marrow, Tacrolimus, Mycophenolate mofetil
Drug · Radiation · Other
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
6 Months to 25 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
7
States / cities
Aurora, Colorado • Wilmington, Delaware • St. Petersburg, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 25, 2021 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Myeloid-Derived Suppressor Cells and Checkpoint Immune Regulators' Expression in Allogeneic SCT Using FluBuATG

NCT02916979
View directory record Official record
Conditions
Leukemia, Lymphoid, Leukemia, Myeloid, Myelodysplastic Syndromes, Myelofibrosis, Lymphoma, Malignant, Multiple Myeloma, Waldenstrom Macroglobulinemia
Interventions
Fludarabine, Busulfan, Rabbit ATG, Methotrexate
Drug · Biological
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2022
U.S. locations
1
States / cities
Lebanon, New Hampshire
Source: ClinicalTrials.gov public record
Updated Oct 17, 2023 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCT03207009
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Sep 7, 2026, 2:14 AM EDT
Terminated Not applicable Interventional Results available

Stem Cell Transplantation for Children Affected With Osteopetrosis

NCT00145587
View directory record Official record
Conditions
Osteopetrosis
Interventions
Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies
Procedure · Device · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional Results available

Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer

NCT00134017
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
Busulfan, Cyclophosphamide
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
6 Months to 65 Years
Enrollment
142 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 30, 2018 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 1 Interventional

Cord Blood Expansion on Mesenchymal Stem Cells

NCT00498316
View directory record Official record
Conditions
Myelodysplastic Syndrome, Leukemia
Interventions
Cord Blood Infusion, Busulfan, Fludarabine, Rituximab, ATG, Cyclophosphamide, Clofarabine, Total Body Irradiation (TBI), Melphalan, Tacrolimus, Mycophenolate Mofetil, G-CSF
Procedure · Drug · Other + 1 more
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
1 Year to 80 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 27, 2020 · Synced Sep 7, 2026, 2:14 AM EDT
Recruiting Phase 2 Interventional

The EndRAD Trial: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults With B-ALL

NCT03509961
View directory record Official record
Conditions
B-cell Acute Lymphoblastic Leukemia
Interventions
NGS-MRD, Myeloablative allogeneic HCT with a non-TBI conditioning regimen
Diagnostic Test · Drug
Lead sponsor
Pediatric Transplantation & Cellular Therapy Consortium
Other
Eligibility
1 Year to 25 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
24
States / cities
Birmingham, Alabama • Phoenix, Arizona • Duarte, California + 19 more
Source: ClinicalTrials.gov public record
Updated May 3, 2025 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 3 Interventional Results available

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

NCT03852498
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 23, 2024 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional Results available

Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases

NCT02248597
View directory record Official record
Conditions
Graft Versus Host Disease, Hematopoietic/Lymphoid Cancer
Interventions
fludarabine phosphate, busulfan, cyclophosphamide, allogeneic hematopoietic stem cell transplantation, tacrolimus, mycophenolate mofetil
Drug · Procedure
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 69 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 10, 2023 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 1 Interventional Results available

Rasburicase in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer or Other Disease Undergoing Donor Stem Cell Transplant

NCT00513474
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
busulfan, cyclophosphamide, cyclosporin-A, etoposide, methotrexate, rasburicase, sirolimus, tacrolimus, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, total-body irradiation, fludarabine, allopurinol
Drug · Procedure · Radiation
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 24, 2017 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional

Sirolimus & Mycophenolate Mofetil as GVHD Prophylaxis in Myeloablative, Matched Related Donor HCT

NCT00350181
View directory record Official record
Conditions
Leukemia, Lymphoma, Non-Hodgkin, Hematologic Diseases, Acute GVHD
Interventions
Sirolimus, MMF, BCNU, VP-16, CY, FTBI, BU
Drug
Lead sponsor
Stanford University
Other
Eligibility
2 Years to 60 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Sep 22, 2021 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional Results available

Tacrolimus and Sirolimus as Prophylaxis After Allogenic Non-myeloablative Peripheral Blood Stem Cell Transplantation

NCT00282282
View directory record Official record
Conditions
Graft Versus Host Disease, GVHD
Interventions
tacrolimus, sirolimus, fludarabine, busulfex
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 11, 2014 · Synced Sep 7, 2026, 2:14 AM EDT
Recruiting Phase 3 Interventional

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Charlotte, North Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 22, 2026 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

NCT02999984
View directory record Official record
Conditions
Severe Combined Immunodeficiency Due to ADA Deficiency
Interventions
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
Genetic · Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
30 Days to 17 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2022 · Synced Sep 7, 2026, 2:14 AM EDT
Terminated Phase 2 Interventional Results available

A Phase II Study of Umbilical Cord Blood Transplantation

NCT00676806
View directory record Official record
Conditions
Leukemia, Lymphoma, Multiple Myeloma, Aplastic Anemia
Interventions
Umbilical Cord Blood After Myeloablative Conditioning, Umbilical Cord Blood After Reduced-Intensity Conditioning
Biological
Lead sponsor
Tufts Medical Center
Other
Eligibility
Up to 70 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 31, 2017 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs

NCT01033552
View directory record Official record
Conditions
Epidermolysis Bullosa
Interventions
Cyclophosphamide, Fludarabine, Anti-thymocyte globulin, Myeloablative Busulfan, Mesenchymal stem cell transplantation, Total body irradiation, Bone marrow or umbilical cord blood (UCG) stem cell transplantation
Drug · Procedure · Radiation
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 25 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2021
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional

Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease

NCT00186810
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Busulfan, Cyclophosphamide, Horse ATG, Allogeneic stem cell transplant
Drug · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 28, 2009 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 3 Interventional Results available

Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia

NCT00322101
View directory record Official record
Conditions
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Acute Myeloid Leukemia/Transient Myeloproliferative Disorder, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Childhood Acute Myeloid Leukemia in Remission, Childhood Myelodysplastic Syndromes, de Novo Myelodysplastic Syndromes, Myelodysplastic Syndrome With Isolated Del(5q), Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes
Interventions
total-body irradiation, allogeneic hematopoietic stem cell transplantation, cyclophosphamide, mycophenolate mofetil, busulfan, cyclosporine, fludarabine phosphate, peripheral blood stem cell transplantation, nonmyeloablative allogeneic hematopoietic stem cell transplantation, laboratory biomarker analysis, cytogenetic analysis, flow cytometry, fluorescence in situ hybridization, pharmacological study, polymorphism analysis, tacrolimus, methotrexate
Radiation · Procedure · Drug + 2 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 65 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
7
States / cities
Denver, Colorado • Altanta, Georgia • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2014 · Synced Sep 7, 2026, 2:14 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies

NCT05589896
View directory record Official record
Conditions
Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Biphenotypic Leukemia, Acute Undifferentiated Leukemia, CLL (Chronic Lymphocytic Leukemia), Chronic Myeloid Leukemia (CML), MDS (Myelodysplastic Syndrome), Non-Hodgkin Lymphomas, Hodgkins Lymphoma, Cutaneous T Cell Lymphomas (CTCL)
Interventions
Ossium HPC Marrow, Bone Marrow Transplant, Pre-transplant conditioning - Myeloablative (MAC), Pre-transplant conditioning - Reduced Intensity (RIC), Post-transplant treatment
Other
Lead sponsor
Ossium Health, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
9
States / cities
Duarte, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 7, 2026, 2:14 AM EDT
Completed Phase 2 Interventional Results available

Busulfan, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk Ewing's Tumors

NCT00357396
View directory record Official record
Conditions
Sarcoma
Interventions
graft versus host disease prophylaxis/therapy, busulfan, melphalan, thiotepa, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 24, 2015 · Synced Sep 7, 2026, 2:14 AM EDT