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DRUG:

busulfan

Company:
Generic mfg.
Drug class:
Alkylating agent
1d
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (clinicaltrials.gov)
P1/2, N=10, Recruiting, National Institute of Allergy and Infectious Diseases (NIAID) | Enrolling by invitation --> Recruiting
Enrollment status
|
CD34 (CD34 molecule)
|
busulfan • Kepivance (palifermin) • Neupogen (filgrastim) • plerixafor
6d
Efficacy and Safety of MTBF Conditioning Regimen for Salvageable Allo-HSCT in the Treatment of R/R AML (clinicaltrials.gov)
P=N/A, N=37, Not yet recruiting, First Affiliated Hospital Xi'an Jiaotong University
New trial
|
fludarabine IV • thiotepa • busulfan • Duoenda (mitoxantrone liposomal)
6d
Exagamglogene Autotemcel for Severe Sickle Cell Disease. (PubMed, N Engl J Med)
Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with sickle cell disease for a period of 12 months or more. (CLIMB SCD-121; ClinicalTrials.gov number, NCT03745287.).
Journal
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CD34 (CD34 molecule)
|
busulfan
6d
Enrollment closed
|
FLT3 positive
|
sorafenib • cyclophosphamide • fludarabine IV • busulfan • Neupogen (filgrastim)
7d
Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia. (PubMed, N Engl J Med)
Treatment with exa-cel, preceded by myeloablation, resulted in transfusion independence in 91% of patients with transfusion-dependent β-thalassemia. (Supported by Vertex Pharmaceuticals and CRISPR Therapeutics; CLIMB THAL-111 ClinicalTrials.gov number, NCT03655678.).
Journal
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CD34 (CD34 molecule)
|
busulfan
14d
Enrollment closed
|
TP53 (Tumor protein P53) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule) • CD4 (CD4 Molecule)
|
TP53 mutation
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cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
23d
Enrollment change • Post-transplantation
|
cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan
23d
CD34+ Transplants for Leukemia and Lymphoma (clinicaltrials.gov)
P2, N=100, Not yet recruiting, Guenther Koehne | Trial completion date: Mar 2031 --> Jun 2031 | Trial primary completion date: Mar 2031 --> Jun 2031
Trial completion date • Trial primary completion date
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CD34 (CD34 molecule)
|
cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
23d
Successful bridging therapy with alectinib prior to allogeneic stem cell transplantation for refractory ALK-positive anaplastic large cell lymphoma (PubMed, Rinsho Ketsueki)
Brentuximab vedotin led to partial response and high-dose chemotherapy combined with autologous HSCT was performed...After cessation of alectinib, allogeneic bone marrow transplantation from an HLA 1-locus mismatched unrelated donor was performed after conditioning with fludarabine, busulfan, and total body irradiation. GVHD prophylaxis consisted of tacrolimus and short-term methotrexate...The lymphoma has not recurred for 2 years after allo-HSCT without resuming alectinib. The clinical course of our case suggests that alectinib bridging therapy and allo-HSCT are effective in relapsed/refractory ALK-positive ALCL.
Journal
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ALK (Anaplastic lymphoma kinase)
|
ALK positive
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Alecensa (alectinib) • methotrexate • Adcetris (brentuximab vedotin) • fludarabine IV • busulfan
27d
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (clinicaltrials.gov)
P1/2, N=10, Enrolling by invitation, National Institute of Allergy and Infectious Diseases (NIAID) | Not yet recruiting --> Enrolling by invitation
Enrollment open
|
CD34 (CD34 molecule)
|
busulfan • Kepivance (palifermin) • Neupogen (filgrastim) • plerixafor
27d
Auto BMT for Non-M3 AML in 1st Remission in Pts </=60y of Age Using Busulfan/FTBI & VP16 as a Prep R (clinicaltrials.gov)
P2, N=60, Active, not recruiting, City of Hope Medical Center | Trial completion date: Dec 2023 --> Dec 2024
Trial completion date
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cytarabine • etoposide IV • idarubicin hydrochloride • Proleukin (aldesleukin) • busulfan • Neupogen (filgrastim)
27d
Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders (clinicaltrials.gov)
P2, N=260, Active, not recruiting, City of Hope Medical Center | Trial completion date: Dec 2023 --> Dec 2024
Trial completion date • Metastases
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B)
|
cyclophosphamide • etoposide IV • sirolimus • melphalan • fludarabine IV • busulfan
29d
New P2 trial
|
Jakafi (ruxolitinib) • cyclophosphamide • fludarabine IV • Promacta (eltrombopag) • busulfan • Nplate (romiplostim)
1m
New P2 trial
|
Venclexta (venetoclax) • decitabine • fludarabine IV • busulfan
1m
RIC Regimen for Elderly or High Comorbidity Burden Patients Receiving Haplo-HSCT (clinicaltrials.gov)
P2, N=50, Recruiting, Peking University People's Hospital | Unknown status --> Recruiting | Trial completion date: Sep 2021 --> Sep 2025 | Trial primary completion date: Sep 2021 --> Sep 2025
Enrollment open • Trial completion date • Trial primary completion date
|
cytarabine • cyclophosphamide • fludarabine IV • busulfan
1m
NCI-2018-01752: Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant (clinicaltrials.gov)
P2, N=68, Recruiting, Fred Hutchinson Cancer Center | Trial completion date: Dec 2024 --> Dec 2025 | Trial primary completion date: Dec 2024 --> Dec 2025
Trial completion date • Trial primary completion date
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
|
cyclophosphamide • methotrexate • fludarabine IV • thiotepa • busulfan
1m
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (clinicaltrials.gov)
P1/2, N=10, Not yet recruiting, National Institute of Allergy and Infectious Diseases (NIAID)
New P1/2 trial
|
CD34 (CD34 molecule)
|
busulfan • Kepivance (palifermin) • Neupogen (filgrastim) • plerixafor
1m
Itacitinib for the Prevention of Graft Versus Host Disease (clinicaltrials.gov)
P1, N=31, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting
Enrollment closed
|
cyclophosphamide • fludarabine IV • thiotepa • busulfan • itacitinib (INCB039110)
1m
Directing the migration of serum-free, ex vivo-expanded Vγ9Vδ2 T cells. (PubMed, Front Immunol)
To better understand the migration of these cells and possibly influence their migration, NSG mice were conditioned with agents to clear BM cellular compartments, i.e., busulfan or total body irradiation (TBI), or promote T-cell migration to inflamed BM, i.e., incomplete Freund's adjuvant (IFA), prior to administering γδ T cells...Using an established neuroblastoma NSG mouse model, we show that intratumorally-injected γMSCs increase the homing of γδ T cells to this tumor. These studies provide insight into the migration of serum-free, ex vivo-expanded Vγ9Vδ2 T cells in NSG mice, which is critical to understanding the fundamental properties of these cells.
Preclinical • Journal
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IFNG (Interferon, gamma) • CCL2 (Chemokine (C-C motif) ligand 2) • CCR2 (C-C Motif Chemokine Receptor 2)
|
IFNG expression
|
busulfan • ET206
1m
Busulfan, Melphalan, Topotecan Hydrochloride, and a Stem Cell Transplant in Treating Patients With Newly Diagnosed or Relapsed Solid Tumor (clinicaltrials.gov)
P1, N=25, Active, not recruiting, City of Hope Medical Center | Trial completion date: Dec 2023 --> Dec 2024
Trial completion date • Metastases
|
topotecan • melphalan • busulfan • Neupogen (filgrastim)
1m
Journal • Combination therapy
|
CD34 (CD34 molecule)
|
cyclophosphamide • cladribine • busulfan
1m
ABT199/venetoclax synergism with thiotepa enhances the cytotoxicity of fludarabine, cladribine and busulfan in AML cells. (PubMed, Oncotarget)
Enhanced activation of apoptosis was observed in leukemia patient-derived cell samples exposed to the five-drug combination, suggesting a clinical relevance. The results provide a rationale for clinical trials using these two- and five-drug combinations as part of a conditioning regimen for AML patients undergoing HSCT.
Journal
|
FLT3 (Fms-related tyrosine kinase 3) • BCL2 (B-cell CLL/lymphoma 2) • MCL1 (Myeloid cell leukemia 1) • BCL2L1 (BCL2-like 1) • CHEK2 (Checkpoint kinase 2) • CHEK1 (Checkpoint kinase 1) • CASP3 (Caspase 3) • ANXA5 (Annexin A5) • HSP90AA1 (Heat Shock Protein 90 Alpha Family Class A Member 1Heat Shock Protein 90 Alpha Family Class A Member 1)
|
Venclexta (venetoclax) • cladribine • fludarabine IV • thiotepa • busulfan
2ms
Itacitinib for the Prevention of Graft Versus Host Disease (clinicaltrials.gov)
P1, N=31, Recruiting, M.D. Anderson Cancer Center | Active, not recruiting --> Recruiting
Enrollment open
|
cyclophosphamide • fludarabine IV • thiotepa • busulfan • itacitinib (INCB039110)
2ms
Donor Stem Cell Transplantation Using α/β+ T-lymphocyte Depleted Grafts From HLA Mismatched Donors (clinicaltrials.gov)
P2, N=9, Terminated, Memorial Sloan Kettering Cancer Center | Trial completion date: Jul 2024 --> Mar 2024 | Active, not recruiting --> Terminated | Trial primary completion date: Jul 2024 --> Mar 2024; Low accrual
Trial completion date • Trial termination • Trial primary completion date
|
TP53 (Tumor protein P53) • ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • RUNX1 (RUNX Family Transcription Factor 1) • KMT2A (Lysine Methyltransferase 2A) • CRLF2 (Cytokine Receptor Like Factor 2) • IKZF1 (IKAROS Family Zinc Finger 1) • NUP214 (Nucleoporin 214) • CDKN1A (Cyclin-dependent kinase inhibitor 1A) • DEK (DEK Proto-Oncogene)
|
TP53 mutation • RUNX1 mutation • KMT2A rearrangement • IKZF1 mutation • MLL mutation • Chr t(9;11)
|
Rituxan (rituximab) • cyclophosphamide • clofarabine • melphalan • fludarabine IV • thiotepa • busulfan
2ms
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) (clinicaltrials.gov)
P1/2, N=40, Recruiting, National Institute of Allergy and Infectious Diseases (NIAID) | Trial completion date: Dec 2024 --> Dec 2032 | Trial primary completion date: Dec 2024 --> Dec 2032
Trial completion date • Trial primary completion date
|
busulfan • Kepivance (palifermin)
2ms
Beneficial effects of adipose-derived stromal vascular fraction on testicular injury caused by busulfan. (PubMed, Drug Chem Toxicol)
In the future, clinical applications should bring higher benefits. Since SVF is the patient's own tissue, being harmless, it will offer an advantageous supportive treatment option for patients already weakened by cancer and anticancer therapy.
Journal • Stroma
|
BAX (BCL2-associated X protein) • TGFB1 (Transforming Growth Factor Beta 1) • ATG5 (Autophagy Related 5)
|
busulfan
2ms
Effects of Perilla frutescens Var. Acuta in Busulfan-Induced Spermatogenesis Dysfunction Mouse Model. (PubMed, World J Mens Health)
This study shows that PFA exerts a protective effect against testicular damage by attenuating OS induced by busulfan. Our results suggest that PFA is a potentially relevant drug used to decrease the side effects induced by busulfan on testicular function and sperm during cancer chemotherapy.
Preclinical • Journal
|
SOD1 (Superoxide Dismutase 1)
|
busulfan
2ms
Enrollment change
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
|
Jakafi (ruxolitinib) • cyclophosphamide • methotrexate • melphalan • fludarabine IV • busulfan
2ms
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders (clinicaltrials.gov)
P2, N=75, Active, not recruiting, Children's Hospital of Philadelphia | Recruiting --> Active, not recruiting
Enrollment closed
|
Campath (alemtuzumab) • melphalan • busulfan
2ms
Auto Transplant for High Risk or Relapsed Solid or CNS Tumors (clinicaltrials.gov)
P=N/A, N=44, Completed, Masonic Cancer Center, University of Minnesota | Recruiting --> Completed | N=20 --> 44
Trial completion • Enrollment change
|
MYCN (MYCN Proto-Oncogene BHLH Transcription Factor) • CD34 (CD34 molecule)
|
LDH elevation • MYCN amplification • MYCN expression
|
carboplatin • paclitaxel • ifosfamide • etoposide IV • melphalan • thiotepa • busulfan
2ms
Itacitinib for the Prevention of Graft Versus Host Disease (clinicaltrials.gov)
P1, N=30, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Feb 2027 --> Apr 2025 | Trial primary completion date: Feb 2027 --> Apr 2025
Trial completion date • Trial primary completion date
|
cyclophosphamide • fludarabine IV • thiotepa • busulfan • itacitinib (INCB039110)
2ms
Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma (clinicaltrials.gov)
P1, N=3, Active, not recruiting, City of Hope Medical Center | Trial completion date: Dec 2023 --> Dec 2024 | Trial primary completion date: Dec 2023 --> Dec 2024
Trial completion date • Trial primary completion date
|
CD34 (CD34 molecule)
|
busulfan
2ms
US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov)
P1/2, N=12, Recruiting, ExCellThera inc. | Trial completion date: Dec 2025 --> Jun 2026 | Trial primary completion date: Dec 2023 --> Jun 2026
Trial completion date • Trial primary completion date
|
HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule)
|
cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan • ECT-001-CB
2ms
Trial completion date • Metastases
|
Chr del(5q)
|
etoposide IV • busulfan
2ms
Pap Smear Abnormalities and Human Papillomavirus (HPV) Infection in Allogenic Hematopoietic Stem Cell Transplantation Recipients (USCAP 2024)
Women undergoing allo-HSCT are at higher risk of HPV infection and premalignant lesions, and consequently, screening programs should be strictly followed- However, pathologists should be aware of the cytological abnormalities associated with busulfan, which may closely mimic HPV-associated lesions.
cobas® HPV test
|
busulfan
2ms
The Effects of Busulfan on Xenogeneic Transplantation of Human Peripheral Blood Mononuclear Cells in Recipient Mice. (PubMed, Transplant Proc)
Our findings demonstrate that busulfan treatment is a beneficial alternative for simple and efficient PBMC engraftment in a rodent model, possibly helping to evaluate human immunity in preclinical studies.
Preclinical • Journal
|
IFNG (Interferon, gamma) • IL6 (Interleukin 6) • TNFA (Tumor Necrosis Factor-Alpha) • CXCL8 (Chemokine (C-X-C motif) ligand 8) • IL2 (Interleukin 2) • IL10 (Interleukin 10) • IL22 (Interleukin 22) • IL4 (Interleukin 4) • IL5 (Interleukin 5) • PRKDC (Protein Kinase, DNA-Activated, Catalytic Subunit)
|
busulfan
3ms
Blood Samples to Identify Biomarkers of Busulfan (clinicaltrials.gov)
P=N/A, N=139, Active, not recruiting, Fred Hutchinson Cancer Center | Trial completion date: May 2024 --> May 2025
Trial completion date
|
busulfan
3ms
New P2 trial • Post-transplantation
|
cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan
3ms
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency. (PubMed, Nat Med)
Most adverse events/reactions were related to disease background, busulfan conditioning or immune reconstitution; the safety profile of the real world experience was in line with premarketing cohort...These long-term data suggest that the risk-benefit of GT in ADA remains favorable and warrant for continuing long-term safety monitoring. Clinical trial registration: NCT00598481 , NCT03478670 .
Journal • Real-world evidence • Gene therapy • Real-world
|
CD34 (CD34 molecule)
|
busulfan
3ms
Itacitinib for the Prevention of Graft Versus Host Disease (clinicaltrials.gov)
P1, N=30, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting
Enrollment closed
|
cyclophosphamide • fludarabine IV • thiotepa • busulfan • itacitinib (INCB039110)
3ms
Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS (clinicaltrials.gov)
P2, N=103, Active, not recruiting, M.D. Anderson Cancer Center | Recruiting --> Active, not recruiting
Enrollment closed
|
KRAS (KRAS proto-oncogene GTPase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • DNMT3A (DNA methyltransferase 1) • NF1 (Neurofibromin 1) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • PTPN1 (Protein Tyrosine Phosphatase Non-Receptor Type 1)
|
TP53 mutation • KRAS mutation • NRAS mutation • RAS mutation
|
Venclexta (venetoclax) • cladribine • fludarabine IV • thiotepa • busulfan
3ms
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations (clinicaltrials.gov)
P2, N=144, Recruiting, National Cancer Institute (NCI) | Trial completion date: Dec 2024 --> Dec 2025
Trial completion date
|
GATA2 (GATA Binding Protein 2)
|
GATA2 mutation
|
cyclophosphamide • fludarabine IV • busulfan