Clinical Trials

Numerous clinical trials spanning Early Phase 1 through Phase 3 have extensively evaluated Thiotepa in combined conditioning and therapeutic regimens across primary CNS lymphoma, acute leukemias, solid tumors, breast cancer, and non-malignant disorders such as Fanconi anemia. Sponsored by prominent academic and medical research institutions—including St. Jude Children's Research Hospital, the University of California San Francisco, and M.D. Anderson Cancer Center—these investigations encompass diverse recruitment statuses, ranging from completed and active not recruiting to currently recruiting.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07616154 RECRUITING
Sickle Cell Disease
St. Jude Children's Research Hospital
2026-09 PHASE2
NCT05565105 NOT_YET_RECRUITING
Leukemia, Myeloid, Acute; Leukemia, Lymphocytic, Acute
Guenther Koehne
2026-06 PHASE2
NCT07759102 NOT_YET_RECRUITING
Recurrent Multiple Myeloma; Refractory Multiple Myeloma
University of California, Davis
2026-09-01 PHASE1
NCT07257419 RECRUITING
Relapsed Pediatric ALL; Hematopoietic Cell Transplantation; Hematologic Malignancy
St. Jude Children's Research Hospital
2026-10-03 PHASE1
NCT06013423 RECRUITING
Acute Leukemia of Ambiguous Lineage; Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Blastic Plasmacytoid Dendritic Cell Neoplasm; Hematopoietic and Lymphatic System Neoplasm; Mixed Phenotype Acute Leukemia; Myelodysplastic Syndrome; Myeloproliferative Neoplasm; Non-Hodgkin Lymphoma; Chronic Myeloid Leukemia, BCR-ABL1 Positive
Fred Hutchinson Cancer Center
2024-07-23 PHASE2
NCT02870907 ACTIVE_NOT_RECRUITING
Retinoblastoma
Institut Curie
2010-03-18 PHASE2
NCT06942039 RECRUITING
CNS Embryonal Tumor; CNS, Medulloblastoma; Atypical Teratoid Rhabdoid Tumor; Medulloblastoma, Childhood; Medulloblastoma, Group 3; Medulloblastoma, Group 4; Pineoblastoma; Neuroblastoma; Embryonal Tumor With Multilayered Rosettes; Embryonal Tumor With Abundant Neuropil and True Rosettes; Ependymoblastoma; Medulloepithelioma; CNS Embryonal Tumor With Rhabdoid Features; CNS Embryonal Tumor, Nos
C17 Council
2025-09-23 EARLY_PHASE1
NCT03126916 RECRUITING
Ganglioneuroblastoma; Ganglioneuroblastoma, Nodular; Neuroblastoma
Children's Oncology Group
2018-05-14 PHASE3
NCT01949129 RECRUITING
Acute Lymphoblastic Leukaemia
St. Anna Kinderkrebsforschung
2013-04 PHASE2; PHASE3
NCT07566377 RECRUITING
Acute Myelogenous Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndromes; Non-hodgkin Lymphoma; Hodgkin Lymphoma; Leukemia; Lymphoma; Graft-versus-host Disease
Memorial Sloan Kettering Cancer Center
2026-04-28 PHASE2
NCT06195891 RECRUITING
Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Myelodysplastic Syndrome
City of Hope Medical Center
2024-05-24 PHASE1
NCT04282174 WITHDRAWN
Hematologic Diseases; Hematologic Malignancy; Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Chronic Myelogenous Leukemia; Chronic Lymphocytic Leukemia
Baptist Health South Florida
2022-09 PHASE2
NCT07074470 RECRUITING
Primary Central Nervous System Lymphoma (PCNSL)
The First Affiliated Hospital of Soochow University
2025-01-02
NCT06172296 RECRUITING
Ganglioneuroblastoma, Nodular; Neuroblastoma
National Cancer Institute (NCI)
2024-04-19 PHASE3
NCT07276737 NOT_YET_RECRUITING
NK T-Cell Lymphoma
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
2025-12-30 PHASE2
NCT04684368 RECRUITING
Central Nervous System Nongerminomatous Germ Cell Tumor; Choriocarcinoma; Embryonal Carcinoma; Immature Teratoma; Malignant Teratoma; Mixed Germ Cell Tumor; Pineal Region Germ Cell Tumor; Pineal Region Immature Teratoma; Pineal Region Yolk Sac Tumor; Suprasellar Germ Cell Tumor
Children's Oncology Group
2021-07-13 PHASE2
NCT07259161 NOT_YET_RECRUITING
CML,Blast Phase; HSCT
Institute of Hematology & Blood Diseases Hospital, China
2025-12-15 PHASE2
NCT07565220 RECRUITING
Acute Lymphocytic Leukemia (ALL); Acute Myeloid Leukemia (AML); Acute Leukemia; Myelodysplastic Syndrome(MDS); Myeloproliferative Neoplasm (MPN)
Sawa Ito, MD
2026-07-27 PHASE1
NCT07563920 NOT_YET_RECRUITING
Acute Myeloid Leukemia
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
2026-06
NCT03779854 ACTIVE_NOT_RECRUITING
Acute Biphenotypic Leukemia; Acute Leukemia; Acute Leukemia of Ambiguous Lineage; Acute Lymphoblastic Leukemia; Acute Undifferentiated Leukemia; Allogeneic Hematopoietic Stem Cell Transplantation Recipient; Blastic Plasmacytoid Dendritic Cell Neoplasm; Blasts Under 25 Percent of Bone Marrow Nucleated Cells; Blasts Under 5 Percent of Bone Marrow Nucleated Cells; Mixed Phenotype Acute Leukemia; Myelodysplastic Syndrome With Excess Blasts-1; Myelodysplastic Syndrome/Acute Myeloid Leukemia; Burkitt Leukemia; Chronic Monocytic Leukemia; Lymphoblastic Lymphoma; Mast Cell Leukemia; Myeloproliferative Neoplasm
Fred Hutchinson Cancer Center
2019-08-29 PHASE2
NCT07052370 RECRUITING
Hematologic Malignancy
St. Jude Children's Research Hospital
2025-09-25 PHASE1
NCT06158828 RECRUITING
AML, Childhood; Aml; Acute Myeloid Leukemia, Pediatric; Acute Myeloid Leukemia
Washington University School of Medicine
2024-11-15 PHASE1; PHASE2
NCT02861417 ACTIVE_NOT_RECRUITING
Hematopoietic and Lymphoid Cell Neoplasm; High Risk Acute Myeloid Leukemia; High Risk Myelodysplastic Syndrome; Lymphoproliferative Disorder; Myelodysplastic Syndrome; Myelodysplastic/Myeloproliferative Neoplasm; Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Recurrent High Risk Myelodysplastic Syndrome; Recurrent Hodgkin Lymphoma; Recurrent Myelodysplastic Syndrome; Recurrent Non-Hodgkin Lymphoma; Recurrent Plasma Cell Myeloma
M.D. Anderson Cancer Center
2016-08-05 PHASE2
NCT04678401 RECRUITING
Stem Cell Transplant Complications; Graft Vs Host Disease; Myeloid Leukemia, Acute; Myeloid Leukemia in Relapse (Disorder); Myelodysplastic Syndromes
Dana-Farber Cancer Institute
2021-01-12 PHASE1
NCT07416890 RECRUITING
Pirtobrutinib; Sintilimab; Central Nervous System Lymphoma
Zhejiang Cancer Hospital
2026-02-10 PHASE2
NCT03630211 RECRUITING
Systemic Sclerosis; Diffuse Sclerosis Systemic; Interstitial Lung Disease; Pulmonary Hypertension
Paul Szabolcs
2018-07-31 PHASE2
NCT03653338 RECRUITING
Sickle Cell Anemia; Beta-thalassemia Major; Diamond-blackfan Anemia
Paul Szabolcs
2018-08-02 PHASE1; PHASE2
NCT06687772 RECRUITING
Diffuse Large B Cell Lymphoma
Washington University School of Medicine
2025-01-16 PHASE2
NCT03980769 RECRUITING
Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder
Fred Hutchinson Cancer Center
2021-05-05 PHASE2
NCT07604987 RECRUITING
Primary Central Nervous System (CNS) Lymphoma
Zou Dehui
2026-05-20 PHASE2
NCT07588594 RECRUITING
Chronic Myelomonocytic Leukemia (CMML)
Peking University People's Hospital
2026-02-13
NCT05088356 ACTIVE_NOT_RECRUITING
Allogeneic Hematopoietic Cell Transplantation (HCT); Advanced Hematologic Malignancies; Acute Leukemia; Chronic Myelogenous Leukemia; Myelodysplastic Syndromes; Myeloproliferative Disorders
Stanford University
2021-09-07 PHASE1
NCT07542418 NOT_YET_RECRUITING
Multiple Myeloma
First Affiliated Hospital Xi'an Jiaotong University
2026-04-20
NCT06869265 RECRUITING
Acute Myeloid Leukemia (AML); High Risk Acute Myeloid Leukemia(AML); Relapsed/Refractory Acute Myeloid Leukemia (AML)
Peking University People's Hospital
2025-05-01 PHASE2
NCT07481669 RECRUITING
Lymphoma Patients With High-risk of Central Nervous System Relapse
Second Affiliated Hospital, Zhejiang University, School of Medicine
2026-03-01 PHASE2
NCT07511725 RECRUITING
Glioblastoma (GBM)
Second Affiliated Hospital, Zhejiang University, School of Medicine
2026-01-04 PHASE2
NCT06646211 NOT_YET_RECRUITING
Primary Central Nervous System Lymphoma (PCNSL); Non Hodgkin Lymphoma (NHL)
Sun Yat-sen University
2025-01-01 PHASE2
NCT03970096 RECRUITING
Acute Leukemia; Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Myelodysplastic Syndrome; Myeloproliferative Neoplasm
Fred Hutchinson Cancer Center
2019-11-19 PHASE2
NCT04708054 RECRUITING
Acute Myeloid Leukemia; Chronic Myelomonocytic Leukemia; Myelodysplastic Syndrome
M.D. Anderson Cancer Center
2021-10-21 PHASE2; PHASE3
NCT07441967 NOT_YET_RECRUITING
Hematologic Malignancies
Institute of Hematology & Blood Diseases Hospital, China
2026-02-20 PHASE2
NCT06946407 RECRUITING
Primary Central Nervous System Lymphoma (PCNSL); CNS Lymphoma Treatment
FengYan Jin
2022-12-02
NCT03500731 RECRUITING
Idiopathic Pulmonary Fibrosis; Emphysema or COPD
Paul Szabolcs
2018-04-19 PHASE1; PHASE2
NCT06807606 RECRUITING
Hematologic Malignancies
M.D. Anderson Cancer Center
2025-03-03 PHASE2
NCT06680661 RECRUITING
Acute Myelogenous Leukemia; Acute Lymphatic Leukemia; Chronic Myelogenous Leukemia; Myelodysplastic Syndromes; Myelodysplastic Syndrome Other; Chronic Myelomonocytic Leukemia; Lymphoma; Hodgkin Lymphoma
Leland Metheny
2025-02-25 PHASE2
NCT07263425 RECRUITING
Leptomeningeal Metastasis of HER2-positive Breast Cancer
The First Affiliated Hospital with Nanjing Medical University
2025-10-27 PHASE2
NCT06252870 RECRUITING
Graft Versus Host Disease; Hematologic Malignancy
Nantes University Hospital
2024-07-18 PHASE2
NCT07191431 RECRUITING
Lymphoma Cns
Ruijin Hospital
2025-09-22
NCT07778940 NOT_YET_RECRUITING
Leptomeningeal Metastasis
Tianjin Medical University Cancer Institute and Hospital
2026-09 PHASE2
NCT05303727 NOT_YET_RECRUITING
Neuroblastoma
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
2022-08 PHASE2
NCT03597594 ACTIVE_NOT_RECRUITING
Severe Combined Immunodeficiency
St. Jude Children's Research Hospital
2021-09-02 PHASE1; PHASE2
NCT04859946 ACTIVE_NOT_RECRUITING
Hematologic and Lymphocytic Disorder
M.D. Anderson Cancer Center
2022-01-11 PHASE2
NCT07193654 RECRUITING
Glioblastoma (GBM)
Second Affiliated Hospital, Zhejiang University, School of Medicine
2025-04-01 PHASE2
NCT04872595 ACTIVE_NOT_RECRUITING
Acute Myeloid Leukemia (AML); Acute Lymphoid Leukemia (ALL); Myelodysplastic Syndromes (MDS)
Memorial Sloan Kettering Cancer Center
2021-04-30 PHASE2
NCT06832267 RECRUITING
Primary Central Nervous System Lymphoma
Zhejiang Cancer Hospital
2025-02-14
NCT06385808 NOT_YET_RECRUITING
Relapse Leukemia; Refractory Acute Myeloid Leukemia; Conditioning; Hematopoietic Stem Cell Transplantation
First Affiliated Hospital Xi'an Jiaotong University
2024-05-01
NCT07014943 RECRUITING
Primary Central Nervous System Lymphoma (PCNSL)
Sichuan University
2022-01-01 PHASE2
NCT06543992 RECRUITING
Leptomeningeal Metastasis of Breast Cancer
The First Affiliated Hospital with Nanjing Medical University
2024-08-08 PHASE2
NCT01746849 ACTIVE_NOT_RECRUITING
Leukemia; Multiple Myeloma; Myelodysplastic Syndrome; Non-Hodgkin's Lymphoma
Memorial Sloan Kettering Cancer Center
2012-12 PHASE2
NCT00692939 RECRUITING
Crohn's Disease
Paul Szabolcs
2012-06-26 PHASE1; PHASE2
NCT04221035 RECRUITING
High-Risk Neuroblastoma; Patient With Insufficient Response Chemoimmunotherapy
Gustave Roussy, Cancer Campus, Grand Paris
2019-11-05 PHASE3
NCT01962415 RECRUITING
Primary Immunodeficiency (PID); Congenital Bone Marrow Failure Syndromes; Inherited Metabolic Disorders (IMD); Hereditary Anemias; Inflammatory Conditions; Systemic Juvenile Idiopathic Arthritis (sJIA); Juvenile Rheumatoid Arthritis (JRA)
Paul Szabolcs
2014-02-04 PHASE2
NCT06901999 NOT_YET_RECRUITING
CNS Lymphoma
Ruijin Hospital
2025-04 PHASE2
NCT02605421 RECRUITING
Neuroblastoma
Masonic Cancer Center, University of Minnesota
2016-06 PHASE2
NCT06060782 RECRUITING
Burkitt Lymphoma; Lymphoblastic Lymphoma, Adult; Acute Lymphoblastic Leukemia, Adult
Fifth Affiliated Hospital, Sun Yat-Sen University
2024-03-06 PHASE1
NCT06468267 UNKNOWN
Peripheral T Cell Lymphoma
Xianmin Song, MD
2024-07-15 PHASE2
NCT02061800 UNKNOWN
Chronic Myeloid Leukemia (CML); Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Juvenile Myelomonocytic Leukemia (JMML); Acute Lymphoblastic Leukemia (ALL); Lymphoma (Hodgkin's and Non-Hodgkin's)
Diane George
2013-06-03 PHASE1; PHASE2
NCT05457556 ACTIVE_NOT_RECRUITING
Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Mixed Phenotype Acute Leukemia; Myelodysplastic Syndrome
Children's Oncology Group
2023-03-15 PHASE3
NCT04362293 ACTIVE_NOT_RECRUITING
Sickle Cell Disease
St. Jude Children's Research Hospital
2020-04-30 PHASE2
NCT04217278 ACTIVE_NOT_RECRUITING
Acute Myeloid Leukaemia; High-risk Myelodysplastic Syndrome
University of Birmingham
2020-01-27 PHASE2; PHASE3
NCT06376292 COMPLETED
Solid Tumor Leptomeningeal Metastasis
Second Affiliated Hospital, Zhejiang University, School of Medicine
2024-01-06 PHASE2
NCT02790515 ACTIVE_NOT_RECRUITING
Acute Lymphoblastic Leukemia (ALL); Acute Myeloid Leukemia (AML); Myeloid Sarcoma; Chronic Myeloid Leukemia (CML); Juvenile Myelomonocytic Leukemia (JMML); Myelodysplastic Syndrome (MDS); Non-Hodgkin Lymphoma (NHL)
St. Jude Children's Research Hospital
2016-07-14 PHASE2
NCT03399773 ACTIVE_NOT_RECRUITING
Acute Biphenotypic Leukemia; Acute Lymphoblastic Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Hematopoietic and Lymphoid Cell Neoplasm; Myelodysplastic Syndrome; Myelodysplastic Syndrome With Excess Blasts
Fred Hutchinson Cancer Center
2022-05-10 PHASE2
NCT01821781 ACTIVE_NOT_RECRUITING
Immune Deficiency Disorders; Severe Combined Immunodeficiency; Chronic Granulomatous Disease; X-linked Agammaglobulinemia; Wiskott-Aldrich Syndrome; Hyper-IgM; DiGeorge Syndrome; Chediak-Higashi Syndrome; Common Variable Immune Deficiency; Immune Dysregulatory Disorders; Hemophagocytic Lymphohistiocytosis; IPEX; Autoimmune Lymphoproliferative Syndrome; X-linked Lymphoproliferative Syndrome
Washington University School of Medicine
2013-04-29 PHASE2
NCT02220985 COMPLETED
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Biphenotypic Leukemia; Acute Leukemia of Ambiguous Lineage; Acute Undifferentiated Leukemia; Allogeneic Hematopoietic Stem Cell Transplant Recipient; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Blastic Plasmacytoid Dendritic Cell Neoplasm; Childhood Acute Lymphoblastic Leukemia in Remission; Childhood Acute Myeloid Leukemia in Remission; Donor; Lymphoblastic Lymphoma; Myelodysplastic Syndrome With Excess Blasts; Myelodysplastic Syndrome With Excess Blasts-1; Myelodysplastic Syndrome With Excess Blasts-2; Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia; Recurrent Childhood Acute Lymphoblastic Leukemia; Recurrent Childhood Acute Myeloid Leukemia; Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Refractory Acute Lymphoblastic Leukemia; Refractory Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia in Remission; Acute Myeloid Leukemia in Remission
Fred Hutchinson Cancer Center
2015-03-23 PHASE2
NCT03263559 COMPLETED
Sickle Cell Disease
Medical College of Wisconsin
2017-10-03 PHASE2
NCT05931328 RECRUITING
Relapsed or Refractory (R/R) Primary Central Nervous System Lymphoma
The First Affiliated Hospital with Nanjing Medical University
2022-10-15 PHASE2
NCT06625359 TERMINATED
Central Nervous System Lymphoma
Seoul National University Hospital
2015-06-01 PHASE2
NCT05598593 UNKNOWN
Cytarabine+Thiotepa + Fludarabine + Busulfan; T Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma
First Affiliated Hospital of Zhejiang University
2022-10-23 PHASE2
NCT04530487 TERMINATED
Desmoplastic Small Round Cell Tumor; Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor; Recurrent Desmoplastic Small Round Cell Tumor; Recurrent Malignant Peripheral Nerve Sheath Tumor; Recurrent Malignant Solid Neoplasm; Recurrent Neuroblastoma; Recurrent Rhabdomyosarcoma; Refractory Desmoplastic Small Round Cell Tumor; Refractory Malignant Peripheral Nerve Sheath Tumor; Refractory Malignant Solid Neoplasm; Refractory Neuroblastoma; Refractory Rhabdomyosarcoma
M.D. Anderson Cancer Center
2020-08-19 PHASE2
NCT03849651 ACTIVE_NOT_RECRUITING
Acute Lymphoblastic Leukemia (ALL); Acute Myeloid Leukemia (AML); Myelodysplastic Syndromes (MDS); NK-Cell Leukemia; Hodgkin Lymphoma; Non Hodgkin Lymphoma (NHL); Juvenile Myelomonocytic Leukemia (JMML); Chronic Myeloid Leukemia (CML)
St. Jude Children's Research Hospital
2019-01-31 PHASE2
NCT03346096 COMPLETED
Malignant Lymphoma
Sheba Medical Center
2018-10-09 PHASE2
NCT03342196 COMPLETED
Leukemia
Case Comprehensive Cancer Center
2018-03-21 PHASE2
NCT05854225 UNKNOWN
HLH
Beijing Friendship Hospital
2022-01-01
NCT05579769 TERMINATED
Hematologic Malignancy; Myeloid Malignancy
St. Jude Children's Research Hospital
2023-03-14 PHASE2
NCT03615105 TERMINATED
Acute Lymphoid Leukemia (ALL); Acute Myeloid Leukemia (AML); Chronic Myeloid Leukemia (CML); Hodgkin Lymphoma; Non-Hodgkin Lymphoma; Chronic Lymphocytic Leukemia
Memorial Sloan Kettering Cancer Center
2018-07-25 PHASE2
NCT01505569 COMPLETED
Ewing's Family Tumors; Renal Tumors; Hepatoblastoma; Rhabdomyosarcoma; Soft Tissue Sarcoma; Primary Malignant Brain Neoplasms; Retinoblastoma; Medulloblastoma; Supra-tentorial Primative Neuro-Ectodermal Tumor (PNET); Atypical Teratoid/Rhabdoid Tumor (AT/RT); CNS Tumors; Germ Cell Tumors
Masonic Cancer Center, University of Minnesota
2011-10-20
NCT00719888 COMPLETED
Acute Biphenotypic Leukemia; Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Burkitt Lymphoma; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Follicular Lymphoma; Lymphoblastic Lymphoma; Lymphoplasmacytic Lymphoma; Mantle Cell Lymphoma; Marginal Zone Lymphoma; Myelodysplastic Syndrome; Myelofibrosis; Non-Hodgkin Lymphoma; Plasma Cell Myeloma; Prolymphocytic Leukemia; Refractory Anemia; Small Lymphocytic Lymphoma
Fred Hutchinson Cancer Center
2005-11-18 PHASE2
NCT02880293 COMPLETED
Hematologic Malignancy
Memorial Sloan Kettering Cancer Center
2016-08-23
NCT04151706 COMPLETED
Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndromes; Acute Leukemia; Chronic Myeloid Leukemia
Robert Lowsky
2020-02-27 PHASE2
NCT07493486 COMPLETED
Diffuse Large B-Cell Lymphoma (DLBCL); Intrathecal Chemotherapy
Zhejiang Cancer Hospital
2022-02-01 PHASE2
NCT03165292 TERMINATED
Very High Risk Neuroblastoma
Gustave Roussy, Cancer Campus, Grand Paris
2018-10-01 PHASE2
NCT01057069 ACTIVE_NOT_RECRUITING
Breast Cancer
The Netherlands Cancer Institute
2010-01 PHASE2; PHASE3
NCT00588523 COMPLETED
CNS Cancer; CNS BRAIN
Memorial Sloan Kettering Cancer Center
2002-09 PHASE2
NCT00596154 COMPLETED
CNS Lymphoma; CNS Brain Cancer; Non-Hodgkin's Lymphoma
Memorial Sloan Kettering Cancer Center
2004-12 PHASE2
NCT01858740 COMPLETED
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Biphenotypic Leukemia; Acute Leukemia of Ambiguous Lineage; Acute Undifferentiated Leukemia; Adult Acute Lymphoblastic Leukemia in Remission; Adult Acute Myeloid Leukemia in Remission; Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Childhood Acute Lymphoblastic Leukemia in Remission; Childhood Acute Myeloid Leukemia in Remission; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Myelodysplastic Syndrome With Excess Blasts-1; Myelodysplastic Syndrome With Excess Blasts-2; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Acute Myeloid Leukemia; Recurrent Childhood Acute Lymphoblastic Leukemia; Recurrent Childhood Acute Myeloid Leukemia; Refractory Adult Acute Lymphoblastic Leukemia; Refractory Childhood Acute Lymphoblastic Leukemia
Fred Hutchinson Cancer Center
2014-04-10 PHASE2
NCT01646034 ACTIVE_NOT_RECRUITING
Breast Cancer
The Netherlands Cancer Institute
2014-09 PHASE3
NCT00790413 UNKNOWN
Neuroblastoma
Lund University Hospital
2005-08 EARLY_PHASE1
NCT04521946 WITHDRAWN
Anaplastic Ependymoma; Atypical Teratoid/Rhabdoid Tumor; Central Nervous System Germ Cell Tumor; Choroid Plexus Carcinoma; Intracranial Myeloid Sarcoma; Malignant Brain Neoplasm; Malignant Glioma; Medulloblastoma; Primitive Neuroectodermal Tumor; Recurrent Anaplastic Ependymoma; Recurrent Atypical Teratoid/Rhabdoid Tumor; Recurrent Malignant Brain Neoplasm; Recurrent Malignant Glioma; Recurrent Medulloblastoma; Recurrent Primitive Neuroectodermal Tumor
M.D. Anderson Cancer Center
2021-01-14 PHASE1
NCT05021770 UNKNOWN
Refractory and Relapsed Primary CNS Lymphoma; PCNSL; Non Hodgkin Lymphoma
Huiqiang Huang
2021-12 PHASE1; PHASE2
NCT04083170 TERMINATED
Acute Erythroid Leukemia; Acute Lymphoblastic Leukemia; Acute Megakaryoblastic Leukemia; Acute Myeloid Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Hematopoietic and Lymphoid Cell Neoplasm; HIV Infection; Myelodysplastic Syndrome; Myelodysplastic Syndrome With Excess Blasts; Non-Hodgkin Lymphoma; Refractory Anemia
Fred Hutchinson Cancer Center
2022-10-06 PHASE2
NCT03513328 COMPLETED
Bone Marrow Failure Syndrome; Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Acquired Neutropenia in Newborn; Acquired Anemia Hemolytic; Acquired Thrombocytopenia; Hemophagocytic Lymphohistiocytoses; Wiskott-Aldrich Syndrome; Chronic Granulomatous Disease; Common Variable Immunodeficiency; X-linked Lymphoproliferative Disease; Severe Combined Immunodeficiency; Hurler Syndrome; Mannosidosis; Adrenoleukodystrophy
University of Florida
2018-06-15 PHASE1; PHASE2
NCT03786783 COMPLETED
Ganglioneuroblastoma; High Risk Neuroblastoma
National Cancer Institute (NCI)
2019-03-04 PHASE2
NCT02867800 COMPLETED
Sickle Cell Disease; Graft Versus Host Disease
Monica Bhatia
2016-07 PHASE1
NCT02757885 COMPLETED
Sickle Cell Disease
Emory University
2019-07-10 PHASE2
NCT05021770 Not yet recruiting
Refractory and Relapsed Primary CNS Lymphoma|PCNSL|Non Hodgkin Lymphoma
Huiqiang Huang|Guangdong 999 Brain Hospital|Sun Yat-sen University
2021-12 Phase 1|Phase 2
NCT00739141 COMPLETED
Leukemia; Myelodysplastic Syndrome; Non-Hodgkins Lymphoma; Chronic Myelogenous Leukemia; Hodgkins Lymphoma
Memorial Sloan Kettering Cancer Center
2008-08-12 PHASE2
NCT01807611 COMPLETED
Leukemia; Lymphoma
St. Jude Children's Research Hospital
2013-05-16 PHASE2
NCT00423514 COMPLETED
Graft Versus Host Disease; Leukemia; Myelodysplastic Syndromes
Memorial Sloan Kettering Cancer Center
2006-11-20 PHASE1; PHASE2
NCT01119066 COMPLETED
Acute Lymphoblastic Leukemia; Acute Myelogenous Leukemia; Myelodysplastic Syndrome; Multiple Myeloma
Memorial Sloan Kettering Cancer Center
2010-05-03 PHASE2
NCT03885947 COMPLETED
Hematological Malignancy; Acute Leukemia in Remission; Acute Lymphoblastic Leukemia in Remission; Myelodysplastic Syndromes; Non-Hodgkin Lymphoma; Hodgkin Lymphoma
Alla Keyzner
2019-02-21 PHASE1
NCT02784054 UNKNOWN
Intracranial Non-germinomatous Germ Cell Tumor
Samsung Medical Center
2014-04 PHASE2
NCT00432094 COMPLETED
Childhood Germ Cell Tumor; Ovarian Cancer; Teratoma
Masonic Cancer Center, University of Minnesota
2006-12-19 PHASE2
NCT01511562 ACTIVE_NOT_RECRUITING
Lymphoma
Alliance for Clinical Trials in Oncology
2012-09 PHASE2
NCT02707393 COMPLETED
Chronic Myeloid Leukemia
St. Anna Kinderkrebsforschung
2009-04-30 PHASE2; PHASE3
NCT03615144 TERMINATED
Non-Malignant Hematologic Disorders
Memorial Sloan Kettering Cancer Center
2018-07-23 PHASE2
NCT02025881 TERMINATED
High-risk Medulloblastoma
Gustave Roussy, Cancer Campus, Grand Paris
2013-09-14 PHASE1; PHASE2
NCT02784015 UNKNOWN
Unresectable Localized Soft Tissue Sarcoma
Samsung Medical Center
2016-05 PHASE2
NCT00867178 COMPLETED
Medulloblastoma; Pineoblastoma; Supratentorial Embryonal Tumor, Not Otherwise Specified
National Cancer Institute (NCI)
2009-02-25 PHASE1
NCT00914940 TERMINATED
Graft Versus Host Disease; Leukemia; Myelodysplastic Syndromes
Fred Hutchinson Cancer Center
2009-12-17 PHASE2
NCT02724904 WITHDRAWN
Lymphoma
Massachusetts General Hospital
2017-05 PHASE1; PHASE2
NCT02660281 COMPLETED
Hematological Disease; Immune Deficiencies; Solid Tumors; Myelofibrosis; Multiple Myeloma; Lymphoma
University of Rochester
2015-10 PHASE1
NCT03434704 COMPLETED
Transplant-Related Hematologic Malignancy; Leukemia, Acute; Myelodysplastic Syndromes; Graft Vs Host Disease; Fungal Infection
Ciceri Fabio
2018-06-18 PHASE2
NCT02329080 COMPLETED
Diffuse Large B-cell Lymphoma
International Extranodal Lymphoma Study Group (IELSG)
2014-12 PHASE2
NCT00003816 COMPLETED
Chronic Myeloproliferative Disorders; Leukemia; Lymphoma; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Diseases; Nonmalignant Neoplasm; Unspecified Adult Solid Tumor, Protocol Specific; Unspecified Childhood Solid Tumor, Protocol Specific
Roswell Park Cancer Institute
1998-10-19 PHASE2; PHASE3
NCT00857389 COMPLETED
Stem Cell Transplantation; Leukemia; Lymphoma
M.D. Anderson Cancer Center
2009-03-02 PHASE2
NCT00978471 COMPLETED
Osteosarcoma
Centre Leon Berard
2009-07 PHASE2
NCT01690520 COMPLETED
Acute Biphenotypic Leukemia; Acute Lymphoblastic Leukemia in Remission; Acute Myeloid Leukemia in Remission; Chronic Myelogenous Leukemia; Myelodysplastic Syndrome
Nohla Therapeutics, Inc.
2012-12-11 PHASE2
NCT00943800 COMPLETED
Leukemia; Myelodysplastic Syndrome; Multiple Myeloma; Lymphoma
University of Chicago
2006-10-09
NCT00536601 COMPLETED
Adult Acute Lymphoblastic Leukemia in Remission; 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); Adult Nasal Type Extranodal NK/T-cell Lymphoma; Childhood Acute Lymphoblastic Leukemia in Remission; Childhood Acute Myeloid Leukemia in Remission; Childhood Burkitt Lymphoma; Childhood Diffuse Large Cell Lymphoma; Childhood Immunoblastic Large Cell Lymphoma; Childhood Nasal Type Extranodal NK/T-cell Lymphoma; Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor (PNET); Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue; Hepatosplenic T-cell Lymphoma; Intraocular Lymphoma; Nodal Marginal Zone B-cell Lymphoma; Peripheral T-cell Lymphoma; Plasma Cell Neoplasm; Primary Systemic Amyloidosis; Recurrent Adult Acute Lymphoblastic Leukemia; Recurrent Adult Acute Myeloid Leukemia; Recurrent Adult Burkitt Lymphoma; Recurrent Adult Diffuse Large Cell Lymphoma; Recurrent Adult Diffuse Mixed Cell Lymphoma; Recurrent Adult Diffuse Small Cleaved Cell Lymphoma; Recurrent Adult Grade III Lymphomatoid Granulomatosis; Recurrent Adult Hodgkin Lymphoma; Recurrent Adult Immunoblastic Large Cell Lymphoma; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Childhood Acute Lymphoblastic Leukemia; Recurrent Childhood Acute Myeloid Leukemia; Recurrent Childhood Anaplastic Large Cell Lymphoma; Recurrent Childhood Grade III Lymphomatoid Granulomatosis; Recurrent Childhood Large Cell Lymphoma; Recurrent Childhood Lymphoblastic Lymphoma; Recurrent Childhood Small Noncleaved Cell Lymphoma; Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma; Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor; Recurrent Grade 1 Follicular Lymphoma; Recurrent Grade 2 Follicular Lymphoma; Recurrent Grade 3 Follicular Lymphoma; Recurrent Malignant Testicular Germ Cell Tumor; Recurrent Mantle Cell Lymphoma; Recurrent Marginal Zone Lymphoma; Recurrent Neuroblastoma; Recurrent Small Lymphocytic Lymphoma; Recurrent/Refractory Childhood Hodgkin Lymphoma; Refractory Chronic Lymphocytic Leukemia; Refractory Multiple Myeloma; Regional Neuroblastoma; Splenic Marginal Zone Lymphoma; Testicular Lymphoma; Unspecified Adult Solid Tumor, Protocol Specific; Unspecified Childhood Solid Tumor, Protocol Specific; Waldenström Macroglobulinemia
Roswell Park Cancer Institute
2006-06-29
NCT00554788 COMPLETED
Extraocular Retinoblastoma
Children's Oncology Group
2008-05-06 PHASE3
NCT02333838 UNKNOWN
Leukemia, Myeloid
Assistance Publique - Hôpitaux de Paris
2015-05 PHASE2
NCT01598025 TERMINATED
Acute Leukemia; Chronic Leukemia; Myelodysplastic Syndrome; Non-Hodgkins Lymphoma
Memorial Sloan Kettering Cancer Center
2012-05-02
NCT01010217 COMPLETED
Blood Stem Cell Transplant Failure; Leukemia; Hematologic Malignancies
M.D. Anderson Cancer Center
2009-11-05 PHASE2
NCT02653196 TERMINATED
Neuroepithelial Tumor; Solid Tumor; Allogeneic Hematopoietic Stem Cell Transplantation
Montefiore Medical Center
2015-09 EARLY_PHASE1
NCT02507479 UNKNOWN
Non Hodgkin Lymphoma; Hodgkin Lymphoma; Chronic Lymphocytic Leukemia; Multiple Myeloma
Sheba Medical Center
2015-09 PHASE2
NCT00528437 COMPLETED
Brain Tumors
NYU Langone Health
2005-10 PHASE2
NCT02199041 TERMINATED
Hematological Malignancies
St. Jude Children's Research Hospital
2014-07-11 PHASE2
NCT03609840 Completed
Hematologic Malignancies|Nonmalignant Diseases|Immune Deficiency|Hemoglobinopathies|Genetic Inborn Errors of Metabolism|Fanconi Anemia|Thalassemia|Sickle Cell Disease
University of California San Francisco
2017-05-24 --
NCT00336024 COMPLETED
Anaplastic Medulloblastoma; Medulloblastoma; Supratentorial Embryonal Tumor, Not Otherwise Specified; Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor
Children's Oncology Group
2007-10-22 PHASE3
NCT01814475 COMPLETED
Myelofibrosis
Gruppo Italiano Trapianto di Midollo Osseo
2011-07 PHASE2
NCT00653068 COMPLETED
Childhood Atypical Teratoid/Rhabdoid Tumor
Children's Oncology Group
2009-02-10 PHASE3
NCT00593554 TERMINATED
Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplasia; Chronic Myeloid Leukemia
Sherif S. Farag
2007-08-07 PHASE2
NCT02049580 UNKNOWN
Lymphoma
Istituto Clinico Humanitas
2013-07 PHASE2
NCT00067002 COMPLETED
Leukemia, Lymphocytic, Acute; Leukemia, Myelocytic, Acute; Leukemia, Myeloid, Chronic; Lymphoma, Non-Hodgkin
M.D. Anderson Cancer Center
2003-04 PHASE2
NCT00961142 TERMINATED
Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia
University Hospital Tuebingen
2009-06 PHASE2
NCT01182415 COMPLETED
Non-Hodgkin's Lymphoma; CNS Lymphoma
Massachusetts General Hospital
2010-06 PHASE2
NCT01810926 UNKNOWN
Graft Versus Host Disease
Franco Locatelli
2011-09 PHASE2
NCT00238433 COMPLETED
Lymphoma
OHSU Knight Cancer Institute
2005-03 PHASE2
NCT02259348 TERMINATED
Acute Lymphoblastic Leukemia (ALL); Acute Myeloid Leukemia (AML); Myeloid Sarcoma; Chronic Myelogenous Leukemia (CML); Juvenile Myelomonocytic Leukemia (JMML); Myelodysplastic Syndrome (MDS); Non-Hodgkin Lymphoma (NHL)
St. Jude Children's Research Hospital
2014-10 PHASE2
NCT01011920 COMPLETED
Central Nervous System Lymphoma
International Extranodal Lymphoma Study Group (IELSG)
2009-11 PHASE2
NCT00567567 COMPLETED
Localized Resectable Neuroblastoma; Localized Unresectable Neuroblastoma; Recurrent Neuroblastoma; Regional Neuroblastoma; Stage 4 Neuroblastoma; Stage 4S Neuroblastoma
Children's Oncology Group
2007-11-05 PHASE3
NCT01786018 UNKNOWN
B-cell Lymphoma Refractory
Azienda Ospedaliera San Giovanni Battista
2013-02 PHASE2
NCT00749723 COMPLETED
Recurrent Brain Tumors; Supratentorial PNETs; Medulloblastomas; Ependymomas
University Hospital, Bonn
2006-02-01 PHASE2; PHASE3
NCT00793845 COMPLETED
Neuroblastoma
Samsung Medical Center
2008-08 PHASE2
NCT00182793 COMPLETED
Breast Cancer
City of Hope Medical Center
2005-07 PHASE2
NCT00317408 UNKNOWN
Lymphoma
European Inter-group Cooperation on Childhood and Adolescent Non Hodgkin Lymphoma
2004-04
NCT03061656 UNKNOWN
High Risk Neuroblastoma
Samsung Medical Center
2009-01-01 PHASE2
NCT00004092 COMPLETED
Breast Cancer
City of Hope Medical Center
1999-05 PHASE2
NCT00303810 COMPLETED
Brain and Central Nervous System Tumors
Universitätsklinikum Hamburg-Eppendorf
2001-01
NCT00462787 COMPLETED
Leukemia
Memorial Sloan Kettering Cancer Center
2007-04 PHASE1
NCT00647049 UNKNOWN
Primary Non Hodgkin Lymphoma of the Central Nervous System
University Hospital Freiburg
2007-01 PHASE2
NCT00020722 TERMINATED
Breast Cancer
Barbara Ann Karmanos Cancer Institute
2007-08 PHASE2
NCT01025778 COMPLETED
Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia
Lund University Hospital
2009-12 PHASE2
NCT00824135 COMPLETED
Hematologic Malignancies
St. Jude Children's Research Hospital
2009-01 PHASE1
NCT01050764 TERMINATED
Leukemia, Acute; Chronic Myelogenous Leukemia (CML); Myelodysplastic Syndrome (MDS); Non-Hodgkin Lymphoma (NHL); Chronic Lymphocytic Leukemia (CLL); Acute Myelogenous Leukemia (AML); Acute Lymphoblastic Leukemia (ALL)
Everett Meyer
2009-06 PHASE1; PHASE2
NCT00623077 TERMINATED
Brain and Central Nervous System Tumors; Kidney Cancer; Liver Cancer; Retinoblastoma; Sarcoma
Masonic Cancer Center, University of Minnesota
2005-08 PHASE1
NCT01350258 TERMINATED
Hematologic Malignancies; Acute Leukemia; Myelodysplastic Syndromes (MDS) Other Than RA or RARS Subtypes; Hodgkin's Lymphoma; Non-Hodgkin's Lymphoma; Myeloma; Chronic Myelogenous (or Myeloid) Leukemia (CML) Resistant to STI Therapy
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
2011-04 PHASE1; PHASE2
NCT00916045 TERMINATED
Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Leukemia, Lymphoblastic, Acute; Lymphoma, Non-Hodgkin; Hodgkin Disease; Chronic Lymphocytic Leukemia
King's College Hospital NHS Trust
2009-09 PHASE2
NCT00429182 COMPLETED
Breast Cancer; Metastatic Breast Carcinoma; Invasive Breast Carcinoma
M.D. Anderson Cancer Center
2007-06 PHASE2
NCT00936156 COMPLETED
Metastatic, Cerebral Primitive Neuroectodermal Tumors
Gustave Roussy, Cancer Campus, Grand Paris
2009-01 PHASE2
NCT00295971 COMPLETED
Congenital Amegakaryocytic Thrombocytopenia; Leukemia; Myelodysplastic Syndromes; Severe Congenital Neutropenia
University of California, San Francisco
2005-04 PHASE1
NCT00972101 WITHDRAWN
Stem Cell Transplantation; Leukemia; Lymphoma; Pediatric Disorders
M.D. Anderson Cancer Center
2009-09 PHASE1
NCT01423747 UNKNOWN
Lymphoblastic Leukemia, Acute, Childhood;
St. Anna Kinderkrebsforschung
2003-07 PHASE3
NCT01423500 UNKNOWN
Lymphoblastic Leukemia, Acute, Childhood;
St. Anna Kinderkrebsforschung
2007-01 PHASE3
NCT00382109 COMPLETED
B-cell Childhood Acute Lymphoblastic Leukemia; Childhood Acute Lymphoblastic Leukemia in Remission; Graft Versus Host Disease; L1 Childhood Acute Lymphoblastic Leukemia; L2 Childhood Acute Lymphoblastic Leukemia; T-cell Childhood Acute Lymphoblastic Leukemia
Children's Oncology Group
2007-03 PHASE3
NCT00392782 TERMINATED
Leukemia; Myelodysplastic Syndromes
Masonic Cancer Center, University of Minnesota
2005-07 PHASE2
NCT00587054 COMPLETED
Allogeneic Stem Cell Transplant; Leukemia; Non-Hodgkins; Lymphoblastic Lymphoma; Myelodysplastic Syndrome; Paroxysmal Nocturnal Hemoglobinuria (PNH)
Memorial Sloan Kettering Cancer Center
2001-06 PHASE2
NCT00644371 COMPLETED
Non-Hodgkin Lymphoma
Grupo Español de Linfomas y Transplante Autólogo de Médula Ósea
2007-11 PHASE2
NCT05167370 Terminated
Solid Tumors|Brain Tumors
Children''s Hospital Medical Center Cincinnati
2010-12-13 Phase 2
NCT00392886 UNKNOWN
Brain and Central Nervous System Tumors
Children's Hospital Los Angeles
2004-03 PHASE3
NCT00607984 UNKNOWN
Central Nervous System Tumors; Tumors
Rabin Medical Center
2006-06
NCT00450983 TERMINATED
Graft Versus Host Disease; Leukemia; Myelodysplastic Syndromes
Fred Hutchinson Cancer Center
2006-12 PHASE2
NCT00376480 COMPLETED
Leukemia; Myelodysplastic Syndromes
Dana-Farber Cancer Institute
2005-06 PHASE1
NCT01068301 Completed
Acute Lymphoblastic Leukemia|Acute Myeloid Leukemia|Chronic Myeloid Leukemia|Myelodysplastic Syndrome|Non-Hodgkin''s Lymphoma
St. Jude Children''s Research Hospital|Genzyme a Sanofi Company
2010-05 Phase 1
NCT00448266 TERMINATED
Breast Cancer
The Netherlands Cancer Institute
2007-05 PHASE2; PHASE3
NCT00003199 COMPLETED
Estrogen Receptor-negative Breast Cancer; Estrogen Receptor-positive Breast Cancer; Inflammatory Breast Cancer; Male Breast Cancer; Progesterone Receptor-negative Breast Cancer; Progesterone Receptor-positive Breast Cancer; Stage IIIB Breast Cancer; Stage IV Breast Cancer
Fred Hutchinson Cancer Center
1997-11 PHASE2
NCT00357396 COMPLETED
Sarcoma
Memorial Sloan Kettering Cancer Center
2005-06 PHASE2
NCT00202917 COMPLETED
Hematologic Malignancies
University Hospital Tuebingen
2004-02 PHASE1; PHASE2
NCT00038857 COMPLETED
Leukemia; Lymphoma
M.D. Anderson Cancer Center
2001-09 PHASE2
NCT00801931 TERMINATED
Leukemia; Lymphoma; Neuroblastoma; Immunodeficiencies; Anemia
Columbia University
2007-09-06 PHASE1; PHASE2
NCT00047320 COMPLETED
Brain Tumor; Central Nervous System Tumors; Childhood Germ Cell Tumor
Children's Oncology Group
2004-01 PHASE2
NCT00621036 WITHDRAWN
Brain and Central Nervous System Tumors; Lymphoma; Lymphoproliferative Disorder; Small Intestine Cancer
University of Texas Southwestern Medical Center
2007-10-19 PHASE2
NCT00160355 COMPLETED
Wiskott-Aldrich Syndrome
St. Jude Children's Research Hospital
2005-05 PHASE1
NCT00004174 COMPLETED
Breast Cancer
Northwestern University
1999-10 PHASE1
NCT00104975 COMPLETED
Chronic Myeloproliferative Disorders; Leukemia; Lymphoma; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms
Yale University
2005-02 PHASE1
NCT00003141 COMPLETED
Brain Tumors; Central Nervous System Tumors; Neuroblastoma; Sarcoma
Children's Oncology Group
1998-03 PHASE1
NCT00025558 COMPLETED
Brain and Central Nervous System Tumors
NYU Langone Health
2000-10 PHASE1
NCT00040872 COMPLETED
Neuroblastoma
Memorial Sloan Kettering Cancer Center
2000-06 PHASE2
NCT00078988 COMPLETED
Brain Tumor; Central Nervous System Tumor
Children's Oncology Group
2004-10 PHASE3
NCT00060255 COMPLETED
Breast Cancer; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Testicular Germ Cell Tumor; Unspecified Adult Solid Tumor, Protocol Specific; Unspecified Childhood Solid Tumor, Protocol Specific
Roswell Park Cancer Institute
1991-12 PHASE2
NCT00028730 COMPLETED
Leukemia; Lymphoma; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms
Memorial Sloan Kettering Cancer Center
2001-08 PHASE2
NCT00002836 COMPLETED
Breast Cancer
M.D. Anderson Cancer Center
1995-09-26 PHASE3
NCT00008203 COMPLETED
Breast Cancer
Herbert Irving Comprehensive Cancer Center
1996-05 PHASE3
NCT00017368 COMPLETED
Neuroblastoma
Children's Oncology Group
2001-04 PHASE2
NCT00008008 COMPLETED
Brain and Central Nervous System Tumors
Herbert Irving Comprehensive Cancer Center
1997-09 PHASE2
NCT00002515 COMPLETED
Childhood Germ Cell Tumor; Extragonadal Germ Cell Tumor; Head and Neck Cancer; Kidney Cancer; Liver Cancer; Lymphoma; Neuroblastoma; Ovarian Cancer; Retinoblastoma; Sarcoma; Testicular Germ Cell Tumor
Memorial Sloan Kettering Cancer Center
1992-10 PHASE2
NCT00007982 COMPLETED
Brain and Central Nervous System Tumors; Head and Neck Cancer; Lymphoma
Herbert Irving Comprehensive Cancer Center
1999-04 PHASE2
NCT00003846 COMPLETED
Brain and Central Nervous System Tumors; Neuroblastoma
Children's Oncology Group
1999-07 PHASE2
NCT00002704 COMPLETED
Leukemia
National Cancer Institute (NCI)
1996-01 PHASE2
NCT00002772 TERMINATED
Breast Cancer
SWOG Cancer Research Network
1996-07 PHASE3
NCT00002718 COMPLETED
Leukemia; Lymphoma; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms
Memorial Sloan Kettering Cancer Center
1995-11 PHASE2
NCT00004172 COMPLETED
Breast Cancer
Northwestern University
1999-10 PHASE2
NCT00003173 COMPLETED
Brain and Central Nervous System Tumors; Childhood Germ Cell Tumor; Extragonadal Germ Cell Tumor; Ovarian Cancer; Retinoblastoma; Testicular Germ Cell Tumor; Unspecified Adult Solid Tumor, Protocol Specific; Unspecified Childhood Solid Tumor, Protocol Specific
Memorial Sloan Kettering Cancer Center
1997-09 PHASE2
NCT00002534 COMPLETED
Leukemia
Memorial Sloan Kettering Cancer Center
1993-05 PHASE3
NCT00003972 COMPLETED
Breast Cancer
Fred Hutchinson Cancer Center
1998-07 PHASE3
NCT00002471 COMPLETED
Leukemia; Lymphoma
Memorial Sloan Kettering Cancer Center
1990-02 PHASE2
NCT00003194 TERMINATED
Unspecified Childhood Solid Tumor, Protocol Specific
Seattle Children's Hospital
1997-07 PHASE1
NCT00005092 COMPLETED
Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes
M.D. Anderson Cancer Center
1999-05-28 PHASE1
NCT00003926 TERMINATED
Brain and Central Nervous System Tumors; Childhood Germ Cell Tumor; Chordoma; Kidney Cancer; Liver Cancer; Neuroblastoma; Ovarian Cancer; Retinoblastoma; Sarcoma
Masonic Cancer Center, University of Minnesota
1998-11 PHASE1
NCT00003101 COMPLETED
Brain and Central Nervous System Tumors
Memorial Sloan Kettering Cancer Center
1997-08 PHASE2
NCT00002870 COMPLETED
Breast Cancer
UNICANCER
1994-12 PHASE3
NCT00003081 COMPLETED
Sarcoma
Fred Hutchinson Cancer Center
1998-03 PHASE1
NCT00002977 COMPLETED
Ovarian Cancer
Fred Hutchinson Cancer Center
1997-01 PHASE1
NCT00002508 COMPLETED
Extragonadal Germ Cell Tumor; Ovarian Cancer; Testicular Germ Cell Tumor
Temple University
1990-11 PHASE1; PHASE2
NCT00005798 COMPLETED
Breast Cancer
H. Lee Moffitt Cancer Center and Research Institute
1995-07 PHASE2
NCT00003146 COMPLETED
Multiple Myeloma and Plasma Cell Neoplasm
Fred Hutchinson Cancer Center
1997-11 PHASE2
NCT00003080 COMPLETED
Ovarian Cancer
Fred Hutchinson Cancer Center
1996-09 PHASE1
NCT00003086 TERMINATED
Breast Cancer
Louisiana State University Health Sciences Center Shreveport
1997-03 PHASE1; PHASE2
NCT00003297 COMPLETED
Ovarian Cancer
Georgetown University
1997-12 PHASE1; PHASE2
NCT00002680 COMPLETED
Breast Cancer
Yale University
1994-02 PHASE2
NCT00006123 WITHDRAWN
Breast Cancer
Beth Israel Deaconess Medical Center
2000-07 PHASE1; PHASE2
NCT00003398 COMPLETED
Anemia; Graft Versus Host Disease; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes
University of Maryland, Baltimore
1998-09 PHASE4
NCT00002619 COMPLETED
Brain and Central Nervous System Tumors
NYU Langone Health
1994-09 PHASE2
NCT00003852 TERMINATED
Childhood Germ Cell Tumor; Extragonadal Germ Cell Tumor; Ovarian Cancer; Testicular Germ Cell Tumor
UNICANCER
1998-03 PHASE2
NCT00003877 COMPLETED
Breast Cancer
Roswell Park Cancer Institute
1998-09 PHASE1; PHASE2
NCT00002942 COMPLETED
Breast Cancer
Wake Forest University Health Sciences
1996-06 PHASE3
NCT00313521 UNKNOWN
Brain and Central Nervous System Tumors
Children's Cancer and Leukaemia Group
1995-06 PHASE2
NCT00066417 TERMINATED
Chronic Myeloproliferative Disorders; Leukemia; Myelodysplastic/Myeloproliferative Diseases
National Heart, Lung, and Blood Institute (NHLBI)
PHASE2
NCT00112567 COMPLETED
Leukemia; Myelodysplastic Syndromes
Fred Hutchinson Cancer Center
2003-04 PHASE1; PHASE2
NCT00002755 COMPLETED
Breast Cancer
Scottish Cancer Therapy Network
1995-11 PHASE3
NCT00003068 COMPLETED
Breast Cancer; Drug/Agent Toxicity by Tissue/Organ
University of Arizona
1997-06 PHASE2
NCT00101140 WITHDRAWN
Adult Acute Erythroid Leukemia; Adult Acute Monoblastic and Acute Monocytic Leukemia; Adult Acute Myeloid Leukemia
Eastern Cooperative Oncology Group
PHASE2
NCT00003680 UNKNOWN
Breast Cancer
Scottish Cancer Therapy Network
1998-11 PHASE3
NCT00002616 UNKNOWN
Breast Cancer
University of Illinois at Chicago
1995-02 PHASE1
NCT00003899 COMPLETED
Unspecified Adult Solid Tumor, Protocol Specific
Fred Hutchinson Cancer Center
1999-01 PHASE1
NCT00004906 COMPLETED
Breast Cancer
Hackensack Meridian Health
1999-10 PHASE2
NCT00003412 UNKNOWN
Breast Cancer
Beth Israel Deaconess Medical Center
1998-06 PHASE1
NCT00045539 COMPLETED
Lymphoma
New Approaches to Brain Tumor Therapy Consortium
2002-10 PHASE2
NCT00025077 COMPLETED
Brain and Central Nervous System Tumors
Children's Cancer and Leukaemia Group
2000-01 PHASE2
NCT00025324 UNKNOWN
Brain and Central Nervous System Tumors
Children's Hospital Los Angeles
2000-12 PHASE2
NCT00004900 COMPLETED
Breast Cancer
Hackensack Meridian Health
1999-10 PHASE2
NCT00027937 COMPLETED
Lymphoma; Unspecified Adult Solid Tumor, Protocol Specific; Unspecified Childhood Solid Tumor, Protocol Specific
Fred Hutchinson Cancer Center
2001-08 PHASE2
NCT00003064 UNKNOWN
Fallopian Tube Cancer; Ovarian Cancer; Primary Peritoneal Cavity Cancer
Herbert Irving Comprehensive Cancer Center
1997-01 PHASE1; PHASE2
NCT00002780 UNKNOWN
Breast Cancer
St. Luke's Medical Center
1996-05 PHASE1; PHASE2
NCT00180791 UNKNOWN
Medulloblastoma; Brain Tumors; Neuroectodermal Tumors, Primitive
Gustave Roussy, Cancer Campus, Grand Paris
2002-07 PHASE2
NCT00012311 UNKNOWN
Breast Cancer
Herbert Irving Comprehensive Cancer Center
2000-01 PHASE2
NCT00003273 WITHDRAWN
Brain and Central Nervous System Tumors; Neuroblastoma; Retinoblastoma; Sarcoma
NYU Langone Health
1997-11 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-07-23)

Check the Thiotepa product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

As an aziridine-derived alkylating agent, Thiotepa forms reactive ethyleneimmonium intermediates that covalently cross-link DNA strands, thereby disrupting nucleic acid synthesis and inducing apoptotic cell death. This potent inhibition of cellular proliferation provides the biological basis for suppressing tumor growth and conditioning patients in clinical studies for hematologic malignancies, solid tumors, breast cancer, and ovarian cancer.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

Note: Technical data last updated: Sep 1, 2026.