Clofarabine has been evaluated in numerous clinical trials spanning phase 1 through phase 3, primarily targeting hematologic malignancies such as acute myeloid leukemia, acute lymphocytic leukemia, myelodysplastic syndromes, and non-Hodgkin's lymphoma. Assessing clofarabine as both monotherapy and in combination regimens, this research is supported by academic institutions, consortia, and industry sponsors including Nantes University Hospital, Indiana University, and Genzyme. Across these studies, trial recruitment statuses range from active and recruiting to completed and terminated protocols.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT06986382 | NOT_YET_RECRUITING | Crohn Disease |
Stanford University |
2025-07 | PHASE1; PHASE2 |
| 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 |
| NCT05477589 | RECRUITING | Acute Myeloid Leukemia (AML) in Remission; Stem Cell Transplantation |
Vastra Gotaland Region |
2022-06-07 | PHASE3 |
| NCT07644481 | NOT_YET_RECRUITING | AML (Acute Myelogenous Leukemia); Allogeneic Hematopoietic Stem Cell Transplantation Recipient |
Institute of Hematology & Blood Diseases Hospital, China |
2026-07-20 | |
| NCT07034053 | RECRUITING | Advanced Urothelial Carcinoma; Metastatic Urothelial Cancer |
Ekaterina Laukhtina |
2023-10-24 | PHASE1; PHASE2 |
| NCT05917405 | RECRUITING | Acute Myeloid Leukemia in Remission |
Nantes University Hospital |
2023-09-14 | PHASE2 |
| NCT07022834 | RECRUITING | Langerhans Cell Histiocytosis (LCH) |
West China Second University Hospital |
2025-06-01 | PHASE2 |
| NCT06252870 | RECRUITING | Graft Versus Host Disease; Hematologic Malignancy |
Nantes University Hospital |
2024-07-18 | PHASE2 |
| NCT03136146 | RECRUITING | Recurrent Acute Lymphoblastic Leukemia; Recurrent Adult Lymphoblastic Lymphoma; Recurrent Burkitt Leukemia; Recurrent Burkitt Lymphoma; Recurrent Childhood Lymphoblastic Lymphoma; Recurrent High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements; Refractory Acute Lymphoblastic Leukemia; Refractory Burkitt Leukemia; Refractory Burkitt Lymphoma; Refractory High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements; Refractory Lymphoblastic Lymphoma |
M.D. Anderson Cancer Center |
2017-08-09 | PHASE2 |
| NCT02727803 | RECRUITING | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Biphenotypic Leukemia; Acute Lymphoblastic Leukemia; Acute Lymphoblastic Leukemia in Remission; Acute Myeloid Leukemia With Myelodysplasia-Related Changes; Acute Myeloid Leukemia With Variant MLL Translocations; B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1; Chemotherapy-Related Leukemia; Chronic Myelomonocytic Leukemia; Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements; ISS Stage II Plasma Cell Myeloma; ISS Stage III Plasma Cell Myeloma; Myelodysplastic Syndrome; Myelodysplastic Syndrome With Excess Blasts; Myelodysplastic Syndrome With Gene Mutation; Myelodysplastic/Myeloproliferative Neoplasm; Previously Treated Myelodysplastic Syndrome; Recurrent Acute Myeloid Leukemia; Recurrent Adult Acute Myeloid Leukemia; Recurrent Hodgkin Lymphoma; Recurrent Non-Hodgkin Lymphoma; Refractory Acute Lymphoblastic Leukemia; Refractory Adult Acute Lymphoblastic Leukemia; Secondary Acute Myeloid Leukemia; Therapy-Related Myelodysplastic Syndrome |
M.D. Anderson Cancer Center |
2016-05-19 | PHASE2 |
| NCT07156435 | RECRUITING | Relapsed Pediatric AML; Refractory Pediatric AML |
Princess Maxima Center for Pediatric Oncology |
2020-11-06 | PHASE1 |
| NCT04644016 | RECRUITING | AML; ALL; MDS; MPD Withou Myelofibrosis; NHL or HL; Inherited Metabolic Disorders; Hemoglobinopathies; Bone Marrow Failure; HLH |
Memorial Sloan Kettering Cancer Center |
2020-11-20 | PHASE2 |
| NCT03117751 | ACTIVE_NOT_RECRUITING | Acute Lymphoblastic Leukemia; Acute Lymphoblastic Lymphoma |
St. Jude Children's Research Hospital |
2017-03-29 | PHASE2; PHASE3 |
| NCT04994808 | TERMINATED | Acute Myeloid Leukemia; Myelodysplastic Syndrome |
Fred Hutchinson Cancer Center |
2023-08-11 | PHASE2 |
| NCT02211755 | COMPLETED | Neoplasms; Myelodysplastic Syndromes; Lymphomas |
National Cancer Institute (NCI) |
2014-10-06 | PHASE1 |
| NCT04989335 | UNKNOWN | Myelogenous Leukemia, Acute |
Sheba Medical Center |
2021-08-02 | PHASE2 |
| NCT01701986 | COMPLETED | Hematopoietic Cell Transplantation Recipient; Refractory B-Cell Non-Hodgkin Lymphoma; Refractory Hodgkin Lymphoma; Refractory T-Cell Non-Hodgkin Lymphoma |
M.D. Anderson Cancer Center |
2012-10-25 | PHASE1; 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 |
| NCT05569512 | TERMINATED | Acute Myeloid Leukemia; Pediatric Cancer |
Malika Kapadia |
2022-10-06 | PHASE1 |
| NCT05917405 | Recruiting | Acute Myeloid Leukemia in Remission |
Nantes University Hospital |
2023-09-14 | Phase 2 |
| NCT05065736 | TERMINATED | Metastatic Cancer; Carcinoma; Adenocarcinoma |
Roberto Vargas |
2023-03-06 | EARLY_PHASE1 |
| NCT02135874 | COMPLETED | Acute Bilineal Leukemia; Acute Biphenotypic Leukemia; Acute Leukemia of Ambiguous Lineage; Acute Undifferentiated Leukemia; Mixed Phenotype Acute Leukemia; Mixed Phenotype Acute Leukemia, B/Myeloid, Not Otherwise Specified; Mixed Phenotype Acute Leukemia, T/Myeloid, Not Otherwise Specified; Recurrent Mixed Phenotype Acute Leukemia |
M.D. Anderson Cancer Center |
2014-10-27 | PHASE2 |
| NCT02883049 | COMPLETED | B Acute Lymphoblastic Leukemia; B Acute Lymphoblastic Leukemia, BCR-ABL1-Like; Central Nervous System Leukemia; Testicular Leukemia |
National Cancer Institute (NCI) |
2012-02-29 | PHASE3 |
| NCT03096782 | COMPLETED | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Acute Biphenotypic Leukemia; Acute Leukemia; Acute Lymphoblastic Leukemia; Acute Lymphoblastic Leukemia in Remission; Acute Myeloid Leukemia; Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome; Chemotherapy-Related Leukemia; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Chronic Myelomonocytic Leukemia; Hodgkin Lymphoma; Langerhans Cell Histiocytosis; Minimal Residual Disease; Myelodysplastic Syndrome; Myelodysplastic Syndrome With Excess Blasts; Non-Hodgkin Lymphoma; Recurrent Hodgkin Lymphoma; Refractory Acute Lymphoblastic Leukemia; Refractory Myelodysplastic Syndrome; Small Lymphocytic Lymphoma; Therapy-Related Myelodysplastic Syndrome |
M.D. Anderson Cancer Center |
2017-10-13 | PHASE2 |
| NCT04002115 | TERMINATED | Acute Myeloid Leukemia |
Milton S. Hershey Medical Center |
2020-06-03 | PHASE2 |
| NCT02083250 | COMPLETED | Acute Lymphoblastic Leukemia in Remission; Acute Myeloid Leukemia in Remission; Allogeneic Hematopoietic Stem Cell Transplantation Recipient; Myelodysplastic Syndrome; Previously Treated Myelodysplastic Syndrome; Recurrent Acute Lymphoblastic Leukemia; Recurrent Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2014-03-06 | PHASE1 |
| NCT04220008 | WITHDRAWN | Recurrent Aggressive Non-Hodgkin Lymphoma; Recurrent B-Cell Non-Hodgkin Lymphoma; Recurrent Burkitt Lymphoma; Recurrent Diffuse Large B-Cell Lymphoma; Recurrent High Grade B-Cell Lymphoma; Recurrent T-Cell Non-Hodgkin Lymphoma; Recurrent Transformed B-Cell Non-Hodgkin Lymphoma |
M.D. Anderson Cancer Center |
2021-10-29 | PHASE2 |
| NCT03263767 | TERMINATED | Graft Versus Host Disease |
Nantes University Hospital |
2018-01-15 | PHASE2 |
| NCT01842672 | COMPLETED | Acute Lymphoblastic Leukemia; Acute Myelogenous Leukemia; Lymphoblastic Lymphoma; Diffuse Large B-cell Lymphoma; Burkitt Lymphoma/Leukemia |
New York Medical College |
2013-03 | PHASE1; PHASE2 |
| NCT01700946 | COMPLETED | Recurrent B-Cell Childhood Acute Lymphoblastic Leukemia; Recurrent Childhood B-Lymphoblastic Lymphoma |
St. Jude Children's Research Hospital |
2013-04-15 | PHASE2 |
| NCT00423514 | COMPLETED | Graft Versus Host Disease; Leukemia; Myelodysplastic Syndromes |
Memorial Sloan Kettering Cancer Center |
2006-11-20 | PHASE1; PHASE2 |
| NCT01451515 | COMPLETED | Lymphoblastic Lymphoma |
St. Jude Children's Research Hospital |
2012-05-25 | PHASE2 |
| NCT01119066 | COMPLETED | Acute Lymphoblastic Leukemia; Acute Myelogenous Leukemia; Myelodysplastic Syndrome; Multiple Myeloma |
Memorial Sloan Kettering Cancer Center |
2010-05-03 | PHASE2 |
| NCT00416351 | COMPLETED | Leukemia; Lymphoma; Small Intestine Cancer |
Memorial Sloan Kettering Cancer Center |
2006-06-27 | PHASE1; PHASE2 |
| NCT02085408 | COMPLETED | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome; Adult Acute Megakaryoblastic Leukemia (M7); Adult Acute Minimally Differentiated Myeloid Leukemia (M0); Adult Acute Monoblastic Leukemia (M5a); Adult Acute Monocytic Leukemia (M5b); Adult Acute Myeloblastic Leukemia With Maturation (M2); Adult Acute Myeloblastic Leukemia Without Maturation (M1); 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(16;16)(p13;q22); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Adult Acute Myelomonocytic Leukemia (M4); Adult Erythroleukemia (M6a); Adult Pure Erythroid Leukemia (M6b); Secondary Acute Myeloid Leukemia; Untreated Adult Acute Myeloid Leukemia |
ECOG-ACRIN Cancer Research Group |
2011-02-04 | PHASE3 |
| NCT01471444 | COMPLETED | Disorder Related to Bone Marrow Transplantation; Leukemia; Transplantation Infection |
M.D. Anderson Cancer Center |
2011-11-02 | PHASE3 |
| NCT00697684 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndrome; Acute Lymphocytic Leukemia; Relapsed/Refractory Chronic Lymphocytic Leukemia; Relapsed/Refractory Non Hodgkin's Lymphoma; Hodgkins Disease; Relapsed Refractory Multiple Myeloma |
Beth Israel Deaconess Medical Center |
2008-06 | PHASE1 |
| NCT03615144 | TERMINATED | Non-Malignant Hematologic Disorders |
Memorial Sloan Kettering Cancer Center |
2018-07-23 | PHASE2 |
| NCT00549848 | COMPLETED | Acute Lymphoblastic Leukemia |
St. Jude Children's Research Hospital |
2007-10-29 | PHASE3 |
| NCT02441803 | COMPLETED | Leukemia |
M.D. Anderson Cancer Center |
2015-09-14 | PHASE2 |
| NCT02425904 | ACTIVE_NOT_RECRUITING | Langerhans Cell Histiocytosis |
Dana-Farber Cancer Institute |
2015-05 | PHASE2 |
| NCT01885689 | ACTIVE_NOT_RECRUITING | Adult Acute Lymphoblastic Leukemia in Remission; Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome; Adult Acute Myeloid Leukemia in Remission; Myelodysplastic Syndrome; Secondary Myelodysplastic Syndrome; Chronic Myelomonocytic Leukemia; Therapy-Related Myelodysplastic Syndrome |
City of Hope Medical Center |
2014-02-10 | PHASE2 |
| NCT01228331 | COMPLETED | Leukemia |
Universitätsklinikum Hamburg-Eppendorf |
2010-10 | PHASE2; PHASE3 |
| NCT01596699 | TERMINATED | Myeloid Malignancy; Bone Marrow Failure Syndrome; Transfusion-dependent Red Blood Cell (RBC) Defect; Congenital Immunodeficiency; Metabolic Disease; Severe Immune Dysregulation |
University of California, San Francisco |
2012-05-24 | PHASE2 |
| NCT00857389 | COMPLETED | Stem Cell Transplantation; Leukemia; Lymphoma |
M.D. Anderson Cancer Center |
2009-03-02 | PHASE2 |
| NCT01252667 | COMPLETED | Acute Myeloid Leukemia |
Fred Hutchinson Cancer Center |
2011-01-25 | PHASE2 |
| NCT02925351 | COMPLETED | Autoimmune Disease; Crohn Disease; Inflammatory Disorder; Rheumatoid Arthritis; Systemic Lupus Erythematosus; Takayasu Arteritis |
Jonsson Comprehensive Cancer Center |
2016-01-25 | EARLY_PHASE1 |
| NCT01629511 | TERMINATED | Allogeneic Hematopoietic Stem Cell Transplantation Recipient; Chronic Lymphocytic Leukemia; Prolymphocytic Leukemia; Richter Syndrome |
M.D. Anderson Cancer Center |
2012-11-21 | PHASE1; PHASE2 |
| NCT02686593 | COMPLETED | Acute Myeloid Leukemia |
The University of Hong Kong |
2016-02-01 | PHASE2 |
| NCT02518750 | TERMINATED | Acute Lymphoblastic Leukemia; Lymphoma, Non-Hodgkin's; Leukemia, T-Cell; Leukemia, B-Cell |
St. Jude Children's Research Hospital |
2016-11-23 | PHASE2 |
| NCT01794702 | COMPLETED | Leukemia |
M.D. Anderson Cancer Center |
2013-02-20 | PHASE1; PHASE2 |
| NCT01796405 | WITHDRAWN | Langerhans Cell Histiocytosis |
Dana-Farber Cancer Institute |
PHASE2 | |
| NCT01289457 | COMPLETED | Leukemia |
M.D. Anderson Cancer Center |
2011-02-02 | PHASE1; PHASE2 |
| NCT01471067 | COMPLETED | Blood And Marrow Transplantation; Leukemia; Lymphoma; Transplantation Infection; Transplantation, Bone Marrow |
M.D. Anderson Cancer Center |
2012-07-13 | PHASE1 |
| NCT00703820 | COMPLETED | Acute Myeloid Leukemia |
St. Jude Children's Research Hospital |
2008-08-04 | PHASE3 |
| NCT02349178 | TERMINATED | Leukemia, Acute Lymphoblastic; Acute Myeloid Leukemia |
Medical College of Wisconsin |
2014-12-08 | PHASE2 |
| NCT01462253 | COMPLETED | Acute Lymphoblastic Leukemia |
Gruppo Italiano Malattie EMatologiche dell'Adulto |
2012-10 | PHASE2 |
| NCT01444742 | COMPLETED | Leukemia; Myeloproliferative Diseases |
M.D. Anderson Cancer Center |
2011-11-16 | PHASE2 |
| NCT00498316 | COMPLETED | Myelodysplastic Syndrome; Leukemia |
M.D. Anderson Cancer Center |
2007-07-03 | PHASE1 |
| NCT01643668 | COMPLETED | Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome |
Massachusetts General Hospital |
2012-07 | PHASE2 |
| NCT00990249 | COMPLETED | Leukemia; Lymphoma; Allogeneic Haematopoietic Stem Cell Transplantation; Acute Lymphoblastic Leukemia |
M.D. Anderson Cancer Center |
2009-10 | PHASE2 |
| NCT01900509 | COMPLETED | Hodgkin Lymphoma; Non-Hodgkin Lymphoma; Acute Leukemia |
St. Jude Children's Research Hospital |
2013-08 | PHASE1 |
| NCT00932412 | COMPLETED | Acute Myeloid Leukemia |
Hospices Civils de Lyon |
2009-03 | PHASE2 |
| NCT01160354 | TERMINATED | Acute Myelogenous Leukemia |
M.D. Anderson Cancer Center |
2010-08 | PHASE1; PHASE2 |
| NCT01677949 | WITHDRAWN | Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia |
Masonic Cancer Center, University of Minnesota |
2013-12 | PHASE2 |
| NCT00529360 | COMPLETED | Acute Lymphoblastic Leukemia; Acute Myelogenous Leukemia |
New York Medical College |
2007-06 | PHASE1; PHASE2 |
| NCT03609814 | Completed | Hematologic Malignancies|Nonmalignant Diseases|Immunodeficiencies|Hemoglobinopathies|Genetic Inborn Errors of Metabolism|Fanconi''s Anemia|Thalassemia|Sickle Cell Disease |
University of California San Francisco |
2016-01-26 | -- |
| NCT01621477 | TERMINATED | Acute Lymphoblastic Leukemia; Acute Myelocytic Leukemia; Chronic Myelocytic Leukemia; Juvenile Myelomonocytic Leukemia; Myelodysplastic Syndrome; Hodgkin or Non-Hodgkin Lymphoma; Sarcoma, Myeloid |
St. Jude Children's Research Hospital |
2012-08 | PHASE2 |
| NCT01629082 | COMPLETED | Myeldysplastic Syndrome (MDS); Chronic Myelomonocytic Leukemia; Bone Marrow Diseases; Neutropenia; Acute Myeloid Leukemia (AML) |
National Heart, Lung, and Blood Institute (NHLBI) |
2012-06-06 | PHASE1 |
| NCT00908167 | COMPLETED | Acute Myeloid Leukemia; Infantile Leukemia (Both AML and ALL); Myelodysplastic Syndrome; Myelodysplastic/Myeloproliferative Neoplasms; Biphenotypic Leukemia |
St. Jude Children's Research Hospital |
2009-09 | PHASE1 |
| NCT00903760 | COMPLETED | Myelodysplastic Syndrome |
M.D. Anderson Cancer Center |
2010-01 | PHASE2 |
| NCT01960387 | TERMINATED | Acute Myeloid Leukemia |
University of Pittsburgh |
2013-10 | PHASE2 |
| NCT00295841 | COMPLETED | Leukemia |
Wake Forest University Health Sciences |
2005-02 | PHASE1 |
| NCT00778375 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndrome |
M.D. Anderson Cancer Center |
2008-10 | PHASE2 |
| NCT01423175 | UNKNOWN | Acute Myeloid Leukemia; MDS |
Hannover Medical School |
2011-07 | PHASE2 |
| NCT01188174 | COMPLETED | AML |
Nantes University Hospital |
2010-07 | PHASE2 |
| NCT00973752 | COMPLETED | Acute Lymphoblastic Leukemia |
Massachusetts General Hospital |
2009-08 | PHASE2 |
| NCT00708721 | TERMINATED | Cancer; Chronic Myelomonocytic Leukemia |
University of Utah |
2008-03 | PHASE1; PHASE2 |
| NCT01457885 | COMPLETED | Acute Myeloblastic Leukemia |
University of Michigan Rogel Cancer Center |
2011-11 | PHASE2 |
| NCT00617929 | TERMINATED | Cancer |
Masonic Cancer Center, University of Minnesota |
2008-01 | PHASE2 |
| NCT00383448 | COMPLETED | Adrenoleukodystrophy; Metachromatic Leukodystrophy; Globoid Cell Leukodystrophy; Tay Sachs Disease; Sandhoffs Disease; Wolman Disease; I-Cell Disease; Sanfilippo Syndrome; GM1 Gangliosidosis |
Masonic Cancer Center, University of Minnesota |
2006-09 | PHASE2 |
| NCT01041508 | COMPLETED | Leukemia Lymphoblastic, Acute; Acute Myeloid Leukemia; Neoplasm Recurrent |
Therapeutic Advances in Childhood Leukemia Consortium |
2010-01-29 | PHASE1 |
| NCT01132573 | COMPLETED | Acute Leukemias of Ambiguous Lineage; Philadelphia Chromosome Negative Adult Precursor Acute Lymphoblastic Leukemia; Recurrent Adult Acute Lymphoblastic Leukemia; Untreated Adult Acute Lymphoblastic Leukemia |
National Cancer Institute (NCI) |
2010-04 | PHASE1 |
| NCT01422603 | COMPLETED | Acute Myeloid Leukaemia; Myelodysplasia |
University Hospital Southampton NHS Foundation Trust |
2011-02 | PHASE1; PHASE2 |
| NCT01063257 | COMPLETED | Myelodysplastic Syndrome |
Groupe Francophone des Myelodysplasies |
2010-04 | PHASE1; PHASE2 |
| NCT01983761 | UNKNOWN | Blood And Marrow Transplantation; Leukemia; Lymphoma; Transplantation Infection; Myelodysplastic Syndrome (MDS); Non-Hodgkin's Lymphoma (NHL); Hodgkin's Lymphoma |
Targazyme, Inc. |
2013-11 | PHASE1; PHASE2 |
| NCT00644189 | COMPLETED | Follicular Lymphoma; Marginal Zone Lymphoma; Mantle Cell Lymphoma; Small Lymphocytic Lymphoma; Lymphoplasmacytic Lymphoma; Low Grade B-cell Lymphoma, Not Otherwise Specified; Diffuse Large B-cell Lymphoma; Peripheral T-cell Lymphoma; Angioimmunoblastic T-cell Lymphoma; Anaplastic Large-cell Lymphoma |
Massachusetts General Hospital |
2008-06 | PHASE1; PHASE2 |
| NCT01626092 | COMPLETED | Lysosomal Storage Disease; Peroxisomal Disorder |
Masonic Cancer Center, University of Minnesota |
2012-07-11 | |
| NCT01500161 | TERMINATED | Acute Myelogenous Leukemia; Acute Lymphocytic Leukemia; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia; Hodgkins Disease; Non-Hodgkins Lymphoma; Aplastic Anemia; Multiple Myeloma; Myelodysplastic Syndrome |
Texas Oncology Cancer Center |
2011-11 | PHASE2 |
| NCT00462787 | COMPLETED | Leukemia |
Memorial Sloan Kettering Cancer Center |
2007-04 | PHASE1 |
| NCT01295307 | COMPLETED | Acute Myeloid Leukemia |
Technische Universität Dresden |
2011-03 | PHASE2 |
| NCT00814164 | TERMINATED | Leukemia |
Roswell Park Cancer Institute |
2008-12 | PHASE2 |
| NCT01003678 | TERMINATED | Myelodysplastic Syndrome |
Roswell Park Cancer Institute |
2009-10 | PHASE1 |
| NCT00067028 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndrome; Chronic Myeloid Leukemia |
M.D. Anderson Cancer Center |
2003-12 | PHASE2 |
| NCT00863148 | COMPLETED | MDS; AML; ALL; BAL |
Nantes University Hospital |
2009-10 | PHASE2 |
| NCT01101880 | COMPLETED | 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); Chronic Myelomonocytic Leukemia; de Novo Myelodysplastic Syndromes; Refractory Anemia With Excess Blasts; Untreated Adult Acute Myeloid Leukemia; Myeloproliferative Neoplasm With 10% Blasts or Higher |
University of Washington |
2010-08 | PHASE2 |
| NCT01534702 | UNKNOWN | Acute Myeloid Leukemia |
Hannover Medical School |
2012-01 | PHASE1; PHASE2 |
| NCT00469014 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndrome; Chronic Myeloid Leukemia |
M.D. Anderson Cancer Center |
2006-09 | PHASE2 |
| NCT00945815 | COMPLETED | Leukemia |
SWOG Cancer Research Network |
2010-09 | PHASE2 |
| NCT01025154 | COMPLETED | Acute Myeloid Leukemia |
M.D. Anderson Cancer Center |
2010-01 | PHASE2 |
| NCT00697671 | COMPLETED | Acute Lymphoblastic Leukemia; Chronic Myelogenous Leukemia; Juvenile Myelomonocytic Leukemia; Myelodysplastic Syndrome; Non-Hodgkin's Lymphoma |
St. Jude Children's Research Hospital |
2007-03 | PHASE1 |
| NCT01025778 | COMPLETED | Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia |
Lund University Hospital |
2009-12 | PHASE2 |
| NCT00838240 | UNKNOWN | Leukemia; Myelodysplastic Syndromes |
European Organisation for Research and Treatment of Cancer - EORTC |
2008-11 | PHASE1; PHASE2 |
| NCT00839982 | COMPLETED | 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(16;16)(p13;q22); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Myelodysplastic Syndrome With Isolated Del(5q); Previously Treated Myelodysplastic Syndromes; Recurrent Adult Acute Myeloid Leukemia |
Fred Hutchinson Cancer Center |
2008-11 | PHASE1; PHASE2 |
| NCT00299156 | COMPLETED | Myelodysplastic Syndrome; Chronic Myelomonocytic Leukemia |
M.D. Anderson Cancer Center |
2006-03 | PHASE2 |
| NCT01158885 | TERMINATED | Minimal Residual Disease; Leukemia, Lymphoblastic, Acute; Leukemia, Myelogenous, Acute |
Therapeutic Advances in Childhood Leukemia Consortium |
2010-08 | PHASE2 |
| NCT00824135 | COMPLETED | Hematologic Malignancies |
St. Jude Children's Research Hospital |
2009-01 | PHASE1 |
| NCT01031368 | COMPLETED | Adult Acute Megakaryoblastic Leukemia (M7); Adult Acute Minimally Differentiated Myeloid Leukemia (M0); Adult Acute Monoblastic Leukemia (M5a); Adult Acute Monocytic Leukemia (M5b); Adult Acute Myeloblastic Leukemia With Maturation (M2); Adult Acute Myeloblastic Leukemia Without Maturation (M1); 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 Acute Myelomonocytic Leukemia (M4); Adult Acute Promyelocytic Leukemia (M3); Adult Erythroleukemia (M6a); Adult Pure Erythroid Leukemia (M6b); Recurrent Adult Acute Myeloid Leukemia; Untreated Adult Acute Myeloid Leukemia |
Nohla Therapeutics, Inc. |
2009-12 | PHASE1 |
| NCT00727766 | COMPLETED | Leukemia, Myeloid, Acute |
Washington University School of Medicine |
2009-01 | PHASE1 |
| NCT00454480 | COMPLETED | Leukemia; Myelodysplastic Syndromes |
The University of New South Wales |
2006-08 | PHASE2; PHASE3 |
| NCT00372619 | COMPLETED | Leukemia |
Children's Oncology Group |
2007-03 | PHASE1; PHASE2 |
| NCT00775593 | COMPLETED | Leukemia |
Gruppo Italiano Malattie EMatologiche dell'Adulto |
2008-12 | PHASE2 |
| NCT01279096 | COMPLETED | Acute Lymphoid Leukemia Relapse; Acute Lymphoid Leukemia Relapse After Bone Marrow Transplant |
University Hospital, Lille |
2010-01 | PHASE1 |
| NCT00412243 | COMPLETED | Burkitt's Lymphoma; Lymphoblastic Lymphoma; Acute Lymphoblastic Leukemia |
M.D. Anderson Cancer Center |
2006-03 | PHASE1; PHASE2 |
| NCT01169012 | COMPLETED | Myelodysplastic Syndrome; Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Chronic Myelomonocytic Leukemia |
UNC Lineberger Comprehensive Cancer Center |
2010-09 | PHASE1 |
| NCT01193400 | TERMINATED | Acute Myelogenous Leukemia |
PETHEMA Foundation |
2010-09 | PHASE2 |
| NCT00187096 | COMPLETED | Acute Myeloid Leukemia |
St. Jude Children's Research Hospital |
2005-09 | |
| NCT00317642 | COMPLETED | Acute Myelogenous Leukemia |
Genzyme, a Sanofi Company |
2006-08 | PHASE3 |
| NCT00422032 | COMPLETED | Myelodysplastic Syndrome; Leukemia |
M.D. Anderson Cancer Center |
2006-01 | PHASE2 |
| NCT02544789 | COMPLETED | Acute Lymphoblastic Leukemia |
Betta Pharmaceuticals Co., Ltd. |
2009-06 | PHASE2 |
| NCT00983528 | TERMINATED | Acute Lymphoblastic Leukemia |
University of California, San Diego |
2009-09 | PHASE1; PHASE2 |
| NCT00852163 | COMPLETED | Leukemia; Myelodysplastic Syndrome |
Baylor Research Institute |
2007-03 | PHASE2 |
| NCT00939653 | TERMINATED | Relapsed Acute Myelogenous Leukemia |
Therapeutic Advances in Childhood Leukemia Consortium |
2009-07-10 | PHASE2 |
| NCT00556452 | COMPLETED | Leukemia; Hodgkin Lymphoma; Non-Hodgkin Lymphoma; Multiple Myeloma; Myelodysplastic Syndrome |
University of Michigan Rogel Cancer Center |
2007-10 | PHASE1; PHASE2 |
| NCT00531232 | COMPLETED | Myelodysplastic Syndromes; Secondary Acute Myeloid Leukemia (AML) |
Genzyme, a Sanofi Company |
2007-05-07 | PHASE2 |
| NCT01196013 | COMPLETED | Acute Lymphoblastic Leukemia |
Genzyme, a Sanofi Company |
2010-08 | PHASE1 |
| NCT01090167 | COMPLETED | Acute Myelogenous Leukemia |
Genzyme, a Sanofi Company |
2010-02 | PHASE1 |
| NCT00991133 | COMPLETED | Lymphoblastic Leukemia, Acute, Childhood |
Genzyme, a Sanofi Company |
2009-10 | PHASE1 |
| NCT00943592 | COMPLETED | Advanced Hematologic Malignancies; Leukemia; Preleukemia |
University of Chicago |
2006-03 | PHASE1; PHASE2 |
| NCT00882076 | TERMINATED | Leukemia |
Milton S. Hershey Medical Center |
2009-03 | PHASE1 |
| NCT00863434 | TERMINATED | Adult Acute Myeloid Leukemia in Remission; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; 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); Recurrent Adult Acute Myeloid Leukemia |
University of Washington |
2009-02 | PHASE2 |
| NCT00641030 | COMPLETED | Leukemia; Myelodysplastic Syndromes |
City of Hope Medical Center |
2007-07 | PHASE1 |
| NCT00477945 | COMPLETED | Non Hodgkin's Lymphoma |
Indiana University |
2007-05 | PHASE1 |
| NCT00477542 | COMPLETED | Hematologic Malignancies |
Indiana University |
2007-05 | PHASE1 |
| NCT00724009 | COMPLETED | Leukemia |
University of Chicago |
2007-12 | PHASE2 |
| NCT00577694 | TERMINATED | Leukemia |
UNC Lineberger Comprehensive Cancer Center |
2007-11 | PHASE1 |
| NCT00315705 | COMPLETED | Acute Lymphoblastic Leukemia; Acute Myelogenous Leukemia; Relapsed Leukemia |
Genzyme, a Sanofi Company |
2006-03 | PHASE1; PHASE2 |
| NCT00156013 | COMPLETED | Lymphoma, B-Cell; Lymphoma, Non-Hodgkin |
Oncology Specialists, S.C. |
2005-09 | PHASE1; PHASE2 |
| NCT00293410 | COMPLETED | Chronic Myeloproliferative Disorders; Leukemia; Myelodysplastic/Myeloproliferative Diseases |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
2005-11 | PHASE1 |
| NCT00338494 | COMPLETED | B-Cell Lymphoma |
Yale University |
2005-10 | PHASE1 |
| NCT00700011 | TERMINATED | Myelodysplastic Syndromes |
Texas Oncology Cancer Center |
2008-03 | PHASE2 |
| NCT00602225 | COMPLETED | Acute Myeloid Leukemia; Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities; 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 Acute Promyelocytic Leukemia (M3); Recurrent Adult Acute Myeloid Leukemia |
University of Washington |
2007-12 | PHASE1; PHASE2 |
| NCT00626626 | TERMINATED | Leukemia; Myelodysplastic Syndrome; Chronic Myelogenous Leukemia; Lymphoma; Hodgkin's Lymphoma; Multiple Myeloma |
Milton S. Hershey Medical Center |
2007-05 | PHASE1; PHASE2 |
| NCT01385891 | COMPLETED | Acute Leukemia |
Istituto Giannina Gaslini |
2008-08 | PHASE2; PHASE3 |
| NCT00852709 | TERMINATED | Myelodysplastic Syndrome; Acute Myeloid Leukemia; Myeloproliferative Disorders; Acute Lymphocytic Leukemia; Acute Promyelocytic Leukemia; Acute Leukemia; Chronic Myelogenous Leukemia; Myelofibrosis; Chronic Myelomonocytic Leukemia; Juvenile Myelomonocytic Leukemia |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
2007-09-01 | PHASE1 |
| NCT00503880 | TERMINATED | Myelodysplastic Syndromes |
University of Nebraska |
2007-05-07 | PHASE1; PHASE2 |
| NCT00081822 | COMPLETED | Acute Myeloid Leukemia |
University of Alabama at Birmingham |
2004-01 | PHASE1 |
| NCT00800566 | WITHDRAWN | Leukemia; Chronic Lymphocytic Leukemia |
M.D. Anderson Cancer Center |
2008-11 | PHASE1 |
| NCT00691652 | TERMINATED | Lymphoma |
OHSU Knight Cancer Institute |
2008-05 | PHASE1; PHASE2 |
| NCT00337168 | COMPLETED | Leukemia |
SWOG Cancer Research Network |
2006-10 | PHASE2 |
| NCT00593645 | TERMINATED | Myelodysplastic Syndromes; Acute Myeloid Leukemia |
Washington University School of Medicine |
2007-11 | PHASE2 |
| NCT01656031 | COMPLETED | Leukemia |
Wake Forest University Health Sciences |
2005-02 | PHASE2 |
| NCT00750334 | TERMINATED | Myelodysplastic Syndromes |
Genzyme, a Sanofi Company |
2008-09 | PHASE1 |
| NCT00638820 | TERMINATED | Osteopetrosis |
Masonic Cancer Center, University of Minnesota |
2007-09 | PHASE2 |
| NCT00373529 | COMPLETED | Acute Myelogenous Leukemia; Acute Myeloid Leukemia |
Genzyme, a Sanofi Company |
2006-10 | PHASE2 |
| NCT00088218 | COMPLETED | Acute Myeloid Leukemia; Myelodysplastic Syndrome |
M.D. Anderson Cancer Center |
2004-07 | PHASE2 |
| NCT00081887 | TERMINATED | Chronic Lymphocytic Leukemia; Leukemia |
M.D. Anderson Cancer Center |
2004-05 | PHASE1 |
| NCT00334074 | COMPLETED | Acute Myelogenous Leukemia; Myelodysplastic Syndromes; Chronic Myelogenous Leukemia |
Baylor Research Institute |
2005-08 | PHASE2 |
| NCT00924443 | COMPLETED | Acute Myeloid Leukemia |
Genzyme, a Sanofi Company |
2004-06 | PHASE2 |
| NCT00201669 | COMPLETED | Non-Hodgkin's Lymphoma |
Ohio State University Comprehensive Cancer Center |
2004-10 | PHASE2 |
| NCT00065143 | COMPLETED | Leukemia, Myeloid; Myelodysplastic Syndromes |
M.D. Anderson Cancer Center |
2003-06-23 | PHASE2 |
| NCT00241371 | TERMINATED | Multiple Myeloma |
Washington University School of Medicine |
2005-03 | PHASE2 |
| NCT00125827 | COMPLETED | Solid Tumors |
Genzyme, a Sanofi Company |
2003-10 | PHASE1 |
| NCT00028418 | COMPLETED | Hematologic Neoplasms; Lymphoproliferative Disorders; Leukemia; Leukemia, Lymphocytic, Chronic |
M.D. Anderson Cancer Center |
1999-02 | PHASE1 |
| NCT00098033 | COMPLETED | Acute Myelogenous Leukemia; Acute Lymphocytic Leukemia; Chronic Myelogenous Leukemia |
University of Texas |
2002-09 | PHASE2 |
| NCT01065545 | WITHDRAWN | Acute Myeloid Leukemia |
Northwestern University |
2011-06 | PHASE1 |
| NCT01302106 | WITHDRAWN | Myelodysplastic Syndromes |
Fondazione Italiana Sindromi Mielodisplastiche-ETS |
PHASE2 | |
| NCT00125840 | COMPLETED | Solid Tumors; Leukemia, Lymphocytic, Acute, Pediatric; Leukemia, Lymphocytic, Acute, Adult; Leukemia, Myelocytic, Acute, Pediatric; Leukemia, Myelocytic, Acute, Adult; Myelodysplastic Syndromes, Adult |
Genzyme, a Sanofi Company |
2002-08 | PHASE1 |
| NCT00044889 | COMPLETED | Acute Myelogenous Leukemia |
Genzyme, a Sanofi Company |
2002-05 | PHASE2 |
| NCT00042341 | COMPLETED | Leukemia, Lymphoblastic, Acute, Pediatric |
Genzyme, a Sanofi Company |
2002-05 | PHASE2 |
| NCT00930098 | COMPLETED | Acute Lymphoblastic Leukemia |
Genzyme, a Sanofi Company |
2003-12 | PHASE2 |
| NCT00042354 | COMPLETED | Leukemia, Myelocytic, Acute, Pediatric |
Genzyme, a Sanofi Company |
2002-05 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2025-05-23)
Mechanism and Biochemical Profile
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>).