Busulfex

(busulfan)

ApprovedSmall molecule

Busulfan is used to treat certain blood cancers, particularly chronic myeloid leukemia, and is also used to prepare patients for bone marrow or stem cell transplants by destroying cancer cells and suppressing the immune system.[2] Busulfex is currently approved and in use across 85 indications[4].[1]

Busulfex is one of 1126 small molecule programs targeting Glioma. It is currently in Approved.

Indications

446

Total Trials

Active Trials

1,125

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[4][1]

Indication

GliomaActive, not recruiting

HIV InfectionApproved

Systemic Lupus ErythematosusActive, not recruiting

Non-Hodgkins LymphomaApproved

X-Linked Combined Immunodeficiency DiseasesApproved

Primary Systemic AmyloidosisActive, not recruiting

FucosidosisApproved

Primitive Neuroectodermal TumorActive, not recruiting

Aplastic AnemiaActive, not recruiting

Thrombotic DiseaseApproved

GliomaPreclinical

Active, not recruiting

HIV InfectionApproved

Approved

Systemic Lupus ErythematosusPhase 1

Active, not recruiting

Non-Hodgkins LymphomaApproved

Approved

X-Linked Combined Immunodeficiency DiseasesApproved

Approved

Primary Systemic AmyloidosisPhase 2

Active, not recruiting

FucosidosisApproved

Approved

Primitive Neuroectodermal TumorPhase 1

Active, not recruiting

Aplastic AnemiaPhase 2/Phase 3

Active, not recruiting

Thrombotic DiseaseApproved

Approved

Showing 10 of 85 indications

Competition

Competitive Landscape

2828 competitors in Breast Cancer

#DrugCompanyPhaseTrials

Busulfex

Akero Therapeutics

Approved

3 2

Tecentriq

Roche

Preclinical

36 3

DATROWAY

Daiichi Sankyo

Preclinical

4 4

Blenoxane

Bristol-Myers Squibb

Preclinical

1 5

Lexiscan

Astellas

Preclinical

1 6

Adenocard

Astellas

Preclinical

1 7

Sporanox

SSebela Pharmaceuticals Development LLC

Preclinical

1 8

Aquasol E

NNational Eye Institute (NEI)

Preclinical

—9

Apatinib Mesylate

BBukwang Pharmaceutical

Preclinical

3 10

Metfomin

UUniversity of Texas Southwestern Medical Center

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2026

Phase 1Active, not recruiting

Lymphoma

NCT01961063

Phase 3Recruiting

Neuroblastoma

NCT01704716

Q4 2026

Phase 1/2Active, not recruiting

Study completion

NCT03983850

Phase 2Active, not recruiting

Malignancy

NCT03674411

N/AActive, not recruiting

Hematopoietic Stem Cell Transplant

NCT01652092

Q2 2027

Phase 2Active, not recruiting

Graft Versus Host Disease

NCT04859946

Clinical

Trials by Indication[1]

Stem Cell Transplant17 trials

High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors

Completed·RRoswell Park Cancer Institute·N/A·NCT00536601·174 enrolled

Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders

Active, not recruiting·—·Phase 2·NCT00544115·260 enrolled

Early Administration of ATG Followed by Cyclophosphamide, Busulfan and Fludarabine Before a Donor Stem Cell Transplant in Patients With Hematological Cancer

Completed·NNorthside Hospital, Inc.·Phase 2·NCT00787761·24 enrolled

Show 14 more trials

Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer

Completed·—·Phase 2·NCT00054327·34 enrolled

Carfilzomib/SAHA Combined With High-Dose Gemcitabine/Busulfan/Melphalan With Autologous Stem Cell Transplant in Myeloma

Withdrawn·MM.D. Anderson Cancer Center·Phase 2·NCT02114502

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

Completed·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT00357396·10 enrolled

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

Completed·UUniversity of California, San Francisco·Phase 2·NCT00301834·35 enrolled

Clofarabine Pre-conditioning Followed by Stem Cell Transplant for Non-remission AML

Terminated·MMilton S. Hershey Medical Center·Phase 2·NCT04002115·2 enrolled

Alemtuzumab, Busulfan, and Cyclophosphamide Followed By a Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer

Terminated·FFred Hutchinson Cancer Center·Phase 1/2·NCT00555048·1 enrolled

Donor Peripheral Stem Cell Transplant, Fludarabine, and Busulfan in Treating Patients With Hematologic Cancers

Completed·UUniversity of California, Davis·Phase 2·NCT00619645·8 enrolled

Busulfan, Cyclophosphamide, & Antithymocyte Globulin Followed by Stem Cell Transplant in Treating Hematologic Cancer

Completed·UUniversity of Nebraska·Phase 2·NCT00611351·5 enrolled

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Withdrawn·MM.D. Anderson Cancer Center·Phase 1·NCT04776850

Thiotepa-Clofarabine-Busulfan With Allogeneic Stem Cell Transplant for High Risk Malignancies

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00857389·60 enrolled

Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease

No longer available·BBarbara Ann Karmanos Cancer Institute··NCT00423826

Fludarabine Phosphate, Busulfan, and Anti-Thymocyte Globulin Followed By Donor Peripheral Blood Stem Cell Transplant, Tacrolimus, and Methotrexate in Treating Patients With Myeloid Malignancies

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT01056614·23 enrolled

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Active, not recruiting·DDiane George·Phase 1/2·NCT02061800·14 enrolled

Reduced-Intensity Busulfan and Fludarabine With or Without Antithymocyte Globulin Followed by Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease

Completed·UUNC Lineberger Comprehensive Cancer Center·Phase 2·NCT00448201·71 enrolled

Lymphoma17 trials

Tandem Auto-Allo Transplant for Lymphoma

Completed·MMassachusetts General Hospital·Phase 2·NCT01181271·42 enrolled

Belinostat Combined With Azacitidine/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Refractory or Relapsed Lymphoma

Withdrawn·MM.D. Anderson Cancer Center·Phase 1/2·NCT02701673

Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma

Active, not recruiting·—·Phase 1·NCT01961063·3 enrolled

Show 14 more trials

VP-16, Ifosfamide, Dexamethasone, L-asparaginase Chemotherapy in Patients With Extranodal Natural Killer T Cell Lymphoma (VIDL+ASCT)

Unknown status·SSamsung Medical Center·Phase 2·NCT02544425·27 enrolled

Graft-Versus-Host Disease in Treating Patients With Recurrent or Refractory Lymphoma or Hodgkin's Disease

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 3·NCT00003414·50 enrolled

Chemotherapy Followed By Peripheral Stem Cell Transplantation in Treating Patients With Recurrent or Refractory AIDS-Related Lymphoma

Completed·AAIDS Malignancy Consortium·Phase 2·NCT00005824

Aza-SAHA-GBM With AutoSCT for Refractory Lymphoma

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT01983969·61 enrolled

Peripheral Stem Cell Transplantation Following Radiation Therapy in Treating Patients With Hodgkin's Disease or Non-Hodgkin's Lymphoma

Completed·NNorthwestern University·Phase 2·NCT00004171

Chemotherapy and Radiation Therapy Plus Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Relapsed or Refractory T-cell Lymphoma, Hodgkin's Lymphoma, or Non-Hodgkin's Lymphoma

Completed·NNorthwestern University·Phase 1/2·NCT00004907

Combination Chemotherapy, Peripheral Stem Cell Transplantation, and Biological Therapy in Treating Patients With Solid Tumors or Lymphoma

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00027937

Dose Monitoring of Busulfan and Combination Chemotherapy in Hodgkin or Non-Hodgkin Lymphoma Undergoing Stem Cell Transplant

Completed·—·Phase 1·NCT01959477·33 enrolled

CHOEP + High Dose Therapy + Auto SCT for T-Cell Lymphoma

Terminated·—·Phase 2·NCT01746173·5 enrolled

BuCE Versus BuME as Conditioning Therapy in Non-Hodgkin's Lymphoma

Completed·SSoonchunhyang University Hospital·Phase 2·NCT03794167·75 enrolled

Fludeoxyglucose F 18 Positron Emission Tomography in Predicting Risk of Relapse in Patients With Non-Hodgkin's Lymphoma Who Are Undergoing Combination Chemotherapy With or Without Autologous Stem Cell or Bone Marrow Transplant

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 2·NCT00238368·59 enrolled

Chidamide Combined With Clad/Gem/Bu With AutoSCT in R/R Diffuse Large B Cell Lymphoma

Unknown status·SSichuan University·Phase 2·NCT03151876·93 enrolled

Autologous Stem Cell Transplant in Treating Patients With Progressive or Recurrent Hodgkin's Lymphoma

Completed·—·Phase 2·NCT00265889·42 enrolled

Thiotepa, Busulfan and Fludarabin for pt With Refractory/Early Relapsed Aggressive B-cell Non Hodgkin Lymphomas

Unknown status·AAzienda Ospedaliera San Giovanni Battista·Phase 2·NCT01786018·42 enrolled

Conditioning12 trials

IDA+BUCY vs BUCY Conditioning Regimen for Intermediate-risk AML Undergoing Auto-HSCT

Completed·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT02671708·153 enrolled

Mel vs BUCY+VP-16 Conditioning Regimen for MM Undergoing Auto-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT03385096·122 enrolled

BUCYE Conditioning Regimen for PCNSL Undergoing Auto-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT03733327·20 enrolled

Show 9 more trials

Decitabine + BUCY vs BUCY Conditioning Regimen for Myeloid Tumors Undergoing Allo-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT04098653·196 enrolled

Decitabine + BUCY vs BUCY Conditioning Regimen for TP53+ Myeloid Tumors Undergoing Allo-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT04123392·196 enrolled

Dec+Flu+Bu Conditioning Regimen for Elderly AML in CR Undergoing Allo-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT03530085·60 enrolled

G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for High-risk MDS Undergoing Allo-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT05453552·242 enrolled

G-CSF+Decitabine+BUCY vs BUCY Conditioning Regimen for RAEB-1, REAB-2 and AML Secondary to MDS Undergoing Allo-HSCT

Completed·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT02744742·202 enrolled

BuCY vs TBICY Conditioning Regimen for Standard-risk ALL Undergoing Allo-HSCT

Completed·NNanfang Hospital, Southern Medical University·Phase 3·NCT02670252·550 enrolled

G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for RAEB-1,REAB-2 and AML Secondary to MDS Undergoing Allo-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT04713956·242 enrolled

Sequential and Personalized PK-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allo-HSCT in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning

Not yet recruiting·IInstitut Paoli-Calmettes·Phase 2·NCT04451200·82 enrolled

BUCY+VP-16 vs BUCY Conditioning Regimen for DLBCL Undergoing Auto-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT03229616·122 enrolled

Bone Marrow Transplantation9 trials

Chemotherapy and Bone Marrow Transplantation in Treating Patients Acute Myeloid With Leukemia or Myelodysplastic Syndrome

Completed·BBarbara Ann Karmanos Cancer Institute·Phase 2·NCT00002547·280 enrolled

Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP

Terminated·TTami D. John·N/A·NCT00578344·8 enrolled

Combination Chemotherapy and Bone Marrow Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome

Completed·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT00002502

Show 6 more trials

Monoclonal Antibody Followed by Bone Marrow Transplantation in Treating Patients With Acute Myelogenous Leukemia in Remission or First Relapse

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00002554·30 enrolled

Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Advanced Hematologic Cancer

Completed·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT00014469

Removal of T Cells to Prevent Graft-Versus-Host Disease in Patients Undergoing Bone Marrow Transplantation

Terminated·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT00005641

Chemotherapy Plus Bone Marrow Transplantation and Filgrastim in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

Completed·NNorthwestern University·Phase 2·NCT00004899

Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

Completed·NNational Cancer Institute (NCI)·Phase 3·NCT00002798·880 enrolled

Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers

Terminated·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 2·NCT01135329·15 enrolled

Acute Myeloid Leukemia8 trials

G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for Secondary Acute Myeloid Leukemia Undergoing Allo-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT05449899·232 enrolled

Allogeneic or Haploidentical Stem Cell Transplant Followed By High-Dose Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Terminated·WWashington University School of Medicine·Phase 1·NCT02057770·25 enrolled

Gemtuzumab Ozogamicin in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia Undergoing Remission Induction and Intensification Therapy

Completed·CChildren's Oncology Group·Phase 2·NCT00070174·350 enrolled

Show 5 more trials

Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia

Terminated·MMasonic Cancer Center, University of Minnesota·Phase 2/3·NCT00630565·12 enrolled

Busulfan, Fludarabine, Clofarabine With Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00469014·72 enrolled

Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Acute Myeloid Leukemia in Second Remission

Completed·AAlliance for Clinical Trials in Oncology·Phase 2·NCT00002768·51 enrolled

Combination Chemotherapy With or Without PSC 833, Peripheral Stem Cell Transplantation, and/or Interleukin-2 in Treating Patients With Acute Myeloid Leukemia

Completed·NNational Cancer Institute (NCI)·Phase 3·NCT00006363·720 enrolled

Decitabine and Total-Body Irradiation Followed By Donor Bone Marrow Transplant and Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Completed·UUniversity of Wisconsin, Madison·Phase 2·NCT01707004·20 enrolled

73 more indications

Myelodysplastic Syndrome7 trials

Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00475020·63 enrolled

CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome

Suspended·GGuenther Koehne·Phase 2·NCT05617625·50 enrolled

Fludarabine-IV Busulfan ± Clofarabine and Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Completed·MM.D. Anderson Cancer Center·Phase 3·NCT01471444·256 enrolled

Show 4 more trials

Busulfan, Fludarabine Phosphate, and Anti-Thymocyte Globulin Followed By Donor Stem Cell Transplant and Azacitidine in Treating Patients With High-Risk Myelodysplastic Syndrome and Older Patients With Acute Myeloid Leukemia

Completed·NNational Cancer Institute (NCI)·Phase 2·NCT01168219·68 enrolled

Alloreactive NK Cells for Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT00402558·15 enrolled

Venetoclax and Sequential Busulfan, Cladribine, and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

Active, not recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT02250937·116 enrolled

Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome

Completed·FFred Hutchinson Cancer Center·N/A·NCT00445744·52 enrolled

Neuroblastoma7 trials

Therapy for Children With Advanced Stage Neuroblastoma

Active, not recruiting·SSt. Jude Children's Research Hospital·Phase 2·NCT01857934·153 enrolled

Induction Therapy Including 131 I-MIBG and Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma Undergoing Stem Cell Transplant, Radiation Therapy, and Maintenance Therapy With Isotretinoin

Completed·CChildren's Oncology Group·Phase 1·NCT01175356·99 enrolled

Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma

Completed·CChildren's Oncology Group·Phase 1·NCT01798004·150 enrolled

Show 4 more trials

High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)

Recruiting·GGustave Roussy, Cancer Campus, Grand Paris·Phase 3·NCT04221035·800 enrolled

Combination Chemotherapy in Treating Infants With Newly Diagnosed Neuroblastoma Who Are Undergoing Surgery With or Without Autologous Bone Marrow or Peripheral Stem Cell Transplant

Unknown status·CChildren's Cancer and Leukaemia Group·Phase 3·NCT00417053

Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma

Not yet recruiting·SSun Yat-Sen Memorial Hospital of Sun Yat-Sen University·Phase 2·NCT05303727·64 enrolled

High Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)

Recruiting·SSt. Anna Kinderkrebsforschung·Phase 3·NCT01704716·3,300 enrolled

Leukemia6 trials

CD34+ Transplants for Leukemia and Lymphoma

Not yet recruiting·GGuenther Koehne·Phase 2·NCT05565105·100 enrolled

GVHD Prophylaxis With Post-transplantation Bendamustine in Refractory Leukemia

Completed·SSt. Petersburg State Pavlov Medical University·Phase 1/2·NCT02799147·27 enrolled

Radiolabeled BC8 Antibody, Busulfan, Cyclophosphamide Followed by Donor Stem Cell Transplant in Treating Patients With Acute Myelogenous Leukemia in First Remission

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00005940·18 enrolled

Show 3 more trials

Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

Completed·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT00357565·34 enrolled

Umbilical Cord Blood Transplant With Added Sugar and Chemotherapy and Radiation Therapy in Treating Patients With Leukemia or Lymphoma

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT03096782·6 enrolled

Combination Chemotherapy With or Without Monoclonal Antibody Therapy in Treating Patients With AML Leukemia

Completed·EEastern Cooperative Oncology Group·Phase 3·NCT00049517·657 enrolled

Hematologic Cancer6 trials

Umbilical Cord Blood Transplantation in Treating Patients With High-Risk Hematologic Cancer

Completed·—·Phase 2·NCT00003335·44 enrolled

Low-Dose Fludarabine, Busulfan, and Anti-Thymocyte Globulin Followed By Donor Umbilical Cord Blood Transplant in Treating Patients With Advanced Hematologic Cancer

Completed·UUniversity of California, San Francisco·Phase 1·NCT00301951·7 enrolled

Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer

Completed·RRoswell Park Cancer Institute·Phase 2·NCT00003661·3 enrolled

Show 3 more trials

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

Terminated·MMasonic Cancer Center, University of Minnesota·N/A·NCT00290628·43 enrolled

Donor Umbilical Cord Blood Transplant in Treating Patients With Advanced Hematologic Cancer

Completed·UUniversity of California, San Francisco·Phase 1·NCT00304018·5 enrolled

Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer

Completed·RRoswell Park Cancer Institute·Phase 2·NCT00003270·20 enrolled

Hematologic Malignancies5 trials

CD34+ Enriched Transplants From HLA-Compatible Patients With Hematologic Malignancies

Withdrawn·BBaptist Health South Florida·Phase 2·NCT04282174

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

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 2·NCT02120157·35 enrolled

Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Completed·—·Phase 2·NCT01093586·14 enrolled

Show 2 more trials

Conditioning Treatment With Umbilical Cord Blood Transplant for Hematologic Malignancies

Terminated·UUniversity of Virginia·Phase 2·NCT01622556·6 enrolled

Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Malignancies or Secondary Myelodysplasia Previously Treated With High-Dose Chemotherapy and Autologous Stem Cell Transplant

Terminated·AAlliance for Clinical Trials in Oncology·Phase 2·NCT01118013·6 enrolled

Multiple Myeloma5 trials

Busulfan, Cyclophosphamide, and Autologous Stem Cell Transplant in Treating Patients With Multiple Myeloma

Completed·—·Phase 2·NCT00941720·71 enrolled

Pre-transplant Purging and Post-transplant MRD-guided Maintenance Therapy With Elranatamab in Patients With High-risk Multiple Myeloma

Recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT06207799·40 enrolled

Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan Before Stem Cell Transplant in Treating Patients With Refractory or Relapsed Multiple Myeloma

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT02506959·83 enrolled

Show 2 more trials

High Dose Busulfan and Bortezomib in Treating Patients With High Risk Multiple Myeloma Undergoing Stem Cell Transplant

Terminated·BBarbara Ann Karmanos Cancer Institute·Phase 2·NCT01534143·1 enrolled

Busulfan, Melphalan, and Bortezomib Before First-Line Stem Cell Transplant in Treating Patients With Multiple Myeloma

Completed·—·Phase 2·NCT01605032·19 enrolled

Chronic Myelogenous Leukemia5 trials

Chemotherapy and Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia

Completed·—·Phase 2·NCT00002592·40 enrolled

Combination Chemotherapy Followed by Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous Leukemia or Myelodysplastic Syndrome

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00027924

Autologous Transplantation for Chronic Myelogenous Leukemia

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT01003054·24 enrolled

Show 2 more trials

Chemotherapy Followed by Peripheral Stem Cell Transplantation And Biological Therapy in Treating Patients With Chronic Myelogenous Leukemia

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00005948

Interferon-alfa With or Without Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Newly Diagnosed Chronic Myelogenous Leukemia

Completed·EEastern Cooperative Oncology Group·Phase 3·NCT00002868·744 enrolled

Graft Versus Host Disease5 trials

Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)

Recruiting·FFred Hutchinson Cancer Center·Phase 2·NCT03970096·120 enrolled

OTI-010 for Graft-Versus-Host Disease Prophylaxis in Treating Patients Who Are Undergoing Donor Peripheral Stem Cell Transplantation for Hematologic Malignancies

Withdrawn·MMesoblast International Sàrl·Phase 2·NCT00081055

Itacitinib for the Prevention of Graft Versus Host Disease in Patients Undergoing Donor Stem Cell Transplantation

Withdrawn·MM.D. Anderson Cancer Center·Phase 2·NCT04127721

Show 2 more trials

Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplant

Completed·BBarbara Ann Karmanos Cancer Institute·Phase 2·NCT00691015·48 enrolled

Itacitinib for the Prevention of Graft Versus Host Disease

Active, not recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT04859946·31 enrolled

Allogeneic Hematopoietic Stem Cell Transplantation4 trials

Busulfan Plus Clofarabine Followed by Allogeneic Hematopoietic Stem Cell Transplantation

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00990249·120 enrolled

Human Leukocyte Antigen (HLA) Mismatched Related Allogeneic Hematopoietic Stem Cell Transplantation

Recruiting·HHe Huang·Phase 1·NCT06809699·29 enrolled

Allogeneic Hematopoietic Stem Cell Transplantation for Relapsed or Refractory High-Risk NBL.

Withdrawn·NNationwide Children's Hospital·Phase 2·NCT00874315

Show 1 more trial

Human Leukocyte Antigen (HLA) Mismatched Unrelated Allogeneic Hematopoietic Stem Cell Transplantation

Recruiting·HHe Huang·Phase 1·NCT06809712·29 enrolled

Sickle Cell Disease4 trials

A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

Completed·SSangamo Therapeutics·Phase 1/2·NCT03653247·7 enrolled

Gene Editing For Sickle Cell Disease

Recruiting·SSt. Jude Children's Research Hospital·Phase 1·NCT06506461·25 enrolled

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Active, not recruiting·—·Phase 2·NCT00408447·53 enrolled

Show 1 more trial

Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

Terminated·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00034528·2 enrolled

Transplantation3 trials

Study Testing Two Conditioning Regimen With a Single Prophylaxis of GVHD by Cyclophosphamide and Methotrexate Post-transplant in Patients Eligible for Matched-donor Allograft Transplantation

Recruiting·NNantes University Hospital·Phase 2·NCT06252870·82 enrolled

Allogeneic Transplantation Using Timed Sequential Busulfan and Fludarabine Conditioning

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT01572662·201 enrolled

Umbilical Cord Blood Transplantation From Unrelated Donors

Recruiting·UUniversity of Rochester··NCT03016806·30 enrolled

Acute Leukemia3 trials

Ex Vivo Expansion of Mafosfamide Purged CD34+ Cells in Patients With Acute Leukemia

Terminated·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1·NCT00245115·7 enrolled

Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous or Acute Leukemia

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT00002831·24 enrolled

Decitabine+BUCY vs BUCY Conditioning Regimen for MLL+ Acute Leukemia Undergoing Allo-HSCT

Unknown status·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT03596892·122 enrolled

Allogeneic Stem Cell Transplantation3 trials

Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation

Completed·MMassachusetts General Hospital·Phase 2·NCT01643668·34 enrolled

Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia

Recruiting·SSt. Anna Kinderkrebsforschung·Phase 2/3·NCT01949129·1,800 enrolled

Busulfan and Fludarabine as Conditioning for Allogeneic Stem Cell Transplantation: a Pharmacokinetic Study

Unknown status·AAlberta Health services·N/A·NCT00156858·200 enrolled

Severe Combined Immunodeficiency2 trials

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

Suspended·SSt. Jude Children's Research Hospital·Phase 1/2·NCT01512888·28 enrolled

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency

Unknown status·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT03315078·13 enrolled

Withdrawn·—·N/A·NCT01795430

A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Relapsed or Therapy Refractory Ewing Sarcoma

Withdrawn·UUniversity of Louisville·Phase 1·NCT01969942

Myeloproliferative Disorder2 trials

Combination Chemotherapy Plus Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Myeloproliferative Disorders

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00002792·20 enrolled

Combination Chemotherapy and Antithymocyte Globulin in Reducing Graft-Versus-Host Disease in Patients Undergoing Donor Stem Cell Transplantation For Myelodysplastic Syndrome or Myeloproliferative Disorder

Completed·FFred Hutchinson Cancer Center·Phase 1/2·NCT00054340

Hematological Malignancies2 trials

A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies

Completed·SSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University·Phase 2·NCT01384513·40 enrolled

Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies

Completed·—·N/A·NCT01247701·16 enrolled

Myelofibrosis2 trials

High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly

Recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT06345495·30 enrolled

JAK Inhibitor Before Donor Stem Cell Transplant in Treating Patients With Primary or Secondary Myelofibrosis

Active, not recruiting·FFred Hutchinson Cancer Center·Phase 2·NCT02251821·61 enrolled

Efficacy and Safety2 trials

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

Completed·UUniversity of California, Los Angeles·Phase 1/2·NCT02999984·10 enrolled

Compare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR

Not yet recruiting·RRuijin Hospital·Phase 3·NCT06297772·220 enrolled

Fanconi Anemia2 trials

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Recruiting·—·Phase 2·NCT02143830·70 enrolled

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

Terminated·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT03600909·3 enrolled

Hematopoietic Stem Cell Transplant2 trials

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Active, not recruiting·MMasonic Cancer Center, University of Minnesota·N/A·NCT01652092·57 enrolled

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT04339777·66 enrolled

Bone Marrow Transplant2 trials

A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant

Active, not recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT04872595·59 enrolled

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

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 2·NCT00134017·142 enrolled

T-cell non-Hodgkin lymphoma2 trials

Gemcitabine Hydrochloride, Clofarabine, and Busulfan Before Donor Stem Cell Transplant in Treating Patients With Refractory B-Cell or T-Cell Non-Hodgkin Lymphoma or Hodgkin Lymphoma

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT01701986·64 enrolled

Vorinostat and Combination Chemotherapy Before Donor Stem Cell Transplantation for the Treatment of Relapsed Aggressive B-cell or T-cell Non-Hodgkin Lymphoma

Withdrawn·MM.D. Anderson Cancer Center·Phase 2·NCT04220008

Stem Cell Transplantation1 trial

Donor Stem Cell Transplantation for Congenital Immunodeficiencies

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT00426517·48 enrolled

Systemic Lupus Erythematosus1 trial

Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc)

Terminated·NNew York Medical College·Phase 1·NCT00684255·1 enrolled

Malignancy1 trial

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy

Active, not recruiting·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT03674411·22 enrolled

Peripheral Blood Stem Cell Transplantation1 trial

Study of Busulfan and FLAG Conditioning Regimen for Allogeneic Peripheral Blood Stem Cell Transplantation

Unknown status·—·Phase 4·NCT02784561·80 enrolled

G-CSF1 trial

Plerixafor and Granulocyte Colony-stimulating Factor (G-CSF) With Busulfan, Fludarabine and Thymoglobulin

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT00822770·47 enrolled

TED1 trial

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Recruiting·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT03579875·48 enrolled

Hematopoietic Stem Cell Transplantation1 trial

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Terminated·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT00668564·18 enrolled

Intensity-modulated Radiation Therapy1 trial

Busulfan, Etoposide, and Intensity-Modulated Radiation Therapy Followed By Donor Stem Cell Transplant in Treating Patients With Advanced Myeloid Cancer

Terminated·—·Phase 1/2·NCT00540995·25 enrolled

Metabolic Disease1 trial

Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases

Completed·RRoswell Park Cancer Institute·Phase 2·NCT00003662·90 enrolled

Trial1 trial

Phase I/II Trial of Fludarabine Plus Busulfan and Allogeneic Progenitor Cell Support

Completed·MM.D. Anderson Cancer Center·Phase 1/2·NCT00506857·82 enrolled

Glioma1 trial

Combination Chemotherapy and Radiation Therapy in Treating Younger Patients Who Are Undergoing an Autologous Stem Cell Transplant for Newly Diagnosed Gliomas

Completed·—·N/A·NCT00253487

Hematological Malignancy1 trial

Stem Cell Transplant for Hematological Malignancy

Terminated·MMasonic Cancer Center, University of Minnesota·N/A·NCT00176930·330 enrolled

Outcome1 trial

Outcomes of Patients After Allo-HSCT With Decitabine and NAC

Unknown status·TThe First Affiliated Hospital of Soochow University·Phase 3·NCT04945096·100 enrolled

Chronic Lymphocytic Leukemia1 trial

Fludarabine and Cyclophosphamide in Treating Patients Who Are Undergoing Donor Stem Cell Transplant for Chronic Lymphocytic Leukemia or Waldenstrom's Macroglobulinemia

Completed·GGerman CLL Study Group·Phase 1/2·NCT00281983·100 enrolled

Hemoglobinopathies1 trial

Allo HSCT for High Risk Hemoglobinopathies

Recruiting·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT06872333·62 enrolled

Epidermolysis Bullosa1 trial

Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

Terminated·MMasonic Cancer Center, University of Minnesota·N/A·NCT00478244·7 enrolled

Myeloid1 trial

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

Completed·—·Phase 1·NCT02916979·20 enrolled

Metastatic1 trial

Intrathecal Busulfan in Treating Patients With Recurrent, Refractory, or Metastatic Leptomeningeal Tumors

Completed·—·Phase 1·NCT00003462·20 enrolled

Bone Marrow Failure Syndrome1 trial

Stem Cell Transplant for Bone Marrow Failure Syndromes

Completed·MMasonic Cancer Center, University of Minnesota·Phase 2/3·NCT00176878·10 enrolled

Malignant Solid Tumors1 trial

Busulfan, Melphalan, and Antithymocyte Globulin Followed By Umbilical Cord Blood Transplant in Treating Young Patients With Refractory or Relapsed Malignant Solid Tumors

Unknown status·MMilton S. Hershey Medical Center·Phase 1·NCT00436761·20 enrolled

Acute Lymphoblastic Leukemia1 trial

Chemotherapy With or Without Total-Body Irradiation Prior to Bone Marrow Transplantation in Treating Children With Acute Lymphoblastic Leukemia

Terminated·—·Phase 3·NCT00002961·43 enrolled

Solid Tumors1 trial

MT2004-30: Tomotherapy for Solid Tumors

Terminated·MMasonic Cancer Center, University of Minnesota·Phase 1·NCT00623077·23 enrolled

Donor1 trial

URMC Related Haplo-identical Donor BMT

Completed·UUniversity of Rochester·Phase 1·NCT02660281·74 enrolled

Pilot Study1 trial

Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions

Completed·—·Phase 2·NCT00003336·6 enrolled

Recurrent or Metastatic1 trial

Donor Stem Cell Transplant After Conditioning Therapy in Treating Patients With Hematologic Cancer, Recurrent or Metastatic Solid Tumor, or Other Disease

Completed·—·N/A·NCT00521430·30 enrolled

Phase II1 trial

Phase II Trial of Pentostatin and Targeted Busulfan

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT00496340·42 enrolled

Aplastic Anemia1 trial

Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer, Metastatic Kidney Cancer, or Aplastic Anemia

Unknown status·UUniversity of California, San Francisco·N/A·NCT00295997·35 enrolled

Allogeneic Hematopoietic Cell Transplant1 trial

Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation

Terminated·UUniversity of California, San Francisco·Phase 2·NCT01596699·16 enrolled

Non-malignant Disorders1 trial

Double Cord Blood Transplant for Patients With Malignant and Non-malignant Disorders

Terminated·—·Phase 1/2·NCT00801931·1 enrolled

Haploidentical Stem Cell Transplantation1 trial

Benadamustine, Fludarabine and Busulfan Conditioning in Recipients of Haploidentical Stem Cell Transplantation (FluBuBe)

Completed·SSt. Petersburg State Pavlov Medical University·Phase 2·NCT04942730·50 enrolled

Hodgkin's Disease1 trial

High-Dose Gemcitabine, Busulfan and Melphalan for Patients With Refactory Hodgkin's Disease

Completed·MM.D. Anderson Cancer Center·Phase 2·NCT01200329·81 enrolled

Pediatric Disorder1 trial

Umbilical Cord Blood Transplant for Congenital Pediatric Disorders

Completed·—·N/A·NCT00950846·40 enrolled

Memory1 trial

Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML

Recruiting·WWashington University School of Medicine·Phase 1/2·NCT06158828·68 enrolled

Hematological Diseases1 trial

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

Completed·WWake Forest University Health Sciences·Phase 2·NCT02248597·27 enrolled

Cell Therapy1 trial

Personalized NK Cell Therapy in CBT

Recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT02727803·100 enrolled

MDS/AML1 trial

GVAX vs. Placebo for MDS/AML After Allo HSCT

Terminated·—·Phase 2·NCT01773395·123 enrolled

Efficacy1 trial

Study Comparing the Efficacy of 2 RIC Regimens (Clofarabine vs Fludarabine) in Adults With AML Eligible to Allo-SCT

Recruiting·NNantes University Hospital·Phase 2·NCT05917405·302 enrolled

Tic Disorder1 trial

Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders

Unknown status·—·N/A·NCT00006056·40 enrolled

Chemotherapy1 trial

Respiratory Side Effects of Busulfan High Dose Chemotherapy in a Pediatric Population

Unknown status·UUniversity Hospital, Strasbourg, France·Phase 2·NCT00328029·35 enrolled

CNS Tumors1 trial

Auto Transplant for High Risk or Relapsed Solid or CNS Tumors

Completed·MMasonic Cancer Center, University of Minnesota·N/A·NCT01505569·44 enrolled

PrEP1 trial

Targeted, Dose-Escalation Busulfan-Etoposide as Prep Regimen

Completed·UUniversity of California, San Francisco·Phase 1·NCT01048827·12 enrolled

Primary Systemic Amyloidosis1 trial

Chemotherapy Plus Peripheral Stem Cell Transplant in Treating Patients Who Have Multiple Myeloma or Primary Systemic Amyloidosis

Completed·HHerbert Irving Comprehensive Cancer Center·Phase 2·NCT00007995·75 enrolled

Hematopoietic Cell Transplantation1 trial

Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies

Completed·UUniversity of Utah·Phase 2·NCT00795132·47 enrolled

Recessive Dystrophic Epidermolysis Bullosa1 trial

Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Terminated·—·Phase 1·NCT00881556·3 enrolled

Relapsed or Refractory1 trial

Vorinostat With Gemcitabine, Busulfan, and Melphalan With Stem Cell Transplant (SCT) in Relapsed or Refractory Lymphoid Malignancies

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT01421173·78 enrolled

Lenalidomide1 trial

Bortezomib (Velcade®), Lenalidomide (Revlimid®) and IV Busulfan (Busilvex®) in Patients Under 65 Years Old

Completed·PPETHEMA Foundation·Phase 3·NCT01916252·460 enrolled

Myeloid Malignancies1 trial

VEN+DAC+Bu2Flu4 vs Bu2Flu5 Conditioning Regimen for Elderly Myeloid Malignancies Undergoing Allo-HSCT

Not yet recruiting·NNanfang Hospital, Southern Medical University·Phase 2/3·NCT07052422·160 enrolled

Unspecified3 trials

Twice-per-weekSelinexor, 2 Days Melphalan

Not yet recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 3·NCT06613035·126 enrolled

Optimizing PTCy Dose and Timing

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 1/2·NCT03983850·105 enrolled

Ruxolitinib in Combination With Autotransplant

Withdrawn·MMarina Kremyanskaya·N/A·NCT02469974

Regulatory

FDA Approval Timeline[5]

Jan 1, 1982

MYLERAN

NDA NDA009386

Feb 4, 1999

BUSULFEX

NDA NDA020954

Sources

References

[1]
ClinicalTrials.gov — 446 trial records for busulfan. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Busulfex drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — busulfan compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for busulfan. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA020954). U.S. Food & Drug Administration.
[6]
SEC EDGAR — Akero Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: Akero Therapeutics

Busulfex

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q3 2026

Q4 2026

Q2 2027

Trials by Indication[1]

Stem Cell Transplant17 trials

Lymphoma17 trials

Conditioning12 trials

Bone Marrow Transplantation9 trials

Acute Myeloid Leukemia8 trials

Myelodysplastic Syndrome7 trials

Neuroblastoma7 trials

Leukemia6 trials

Hematologic Cancer6 trials

Hematologic Malignancies5 trials

Multiple Myeloma5 trials

Chronic Myelogenous Leukemia5 trials

Graft Versus Host Disease5 trials

Allogeneic Hematopoietic Stem Cell Transplantation4 trials

Sickle Cell Disease4 trials

Transplantation3 trials

Acute Leukemia3 trials

Allogeneic Stem Cell Transplantation3 trials

Severe Combined Immunodeficiency2 trials

Thalassemia2 trials

Chronic Granulomatous Disease2 trials

Ewing sarcoma2 trials

Myeloproliferative Disorder2 trials

Hematological Malignancies2 trials

Myelofibrosis2 trials

Efficacy and Safety2 trials

Fanconi Anemia2 trials

Hematopoietic Stem Cell Transplant2 trials

Bone Marrow Transplant2 trials

T-cell non-Hodgkin lymphoma2 trials

Stem Cell Transplantation1 trial

Systemic Lupus Erythematosus1 trial

Malignancy1 trial

Peripheral Blood Stem Cell Transplantation1 trial

G-CSF1 trial

TED1 trial

Hematopoietic Stem Cell Transplantation1 trial

Intensity-modulated Radiation Therapy1 trial

Metabolic Disease1 trial

Trial1 trial

Glioma1 trial

Hematological Malignancy1 trial

Outcome1 trial

Chronic Lymphocytic Leukemia1 trial

Hemoglobinopathies1 trial

Epidermolysis Bullosa1 trial

Myeloid1 trial

Metastatic1 trial

Bone Marrow Failure Syndrome1 trial

Malignant Solid Tumors1 trial

Acute Lymphoblastic Leukemia1 trial

Solid Tumors1 trial

Donor1 trial

Pilot Study1 trial

Recurrent or Metastatic1 trial

Phase II1 trial

Aplastic Anemia1 trial

Allogeneic Hematopoietic Cell Transplant1 trial

Non-malignant Disorders1 trial

Haploidentical Stem Cell Transplantation1 trial

Hodgkin's Disease1 trial

Pediatric Disorder1 trial

Memory1 trial

Hematological Diseases1 trial

Cell Therapy1 trial

MDS/AML1 trial

Efficacy1 trial

Tic Disorder1 trial

Chemotherapy1 trial

CNS Tumors1 trial

PrEP1 trial

Primary Systemic Amyloidosis1 trial