Clinical Trials

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)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Intracellularly phosphorylated clofarabine inhibits ribonucleotide reductase (IC50 = 65 nM) and DNA polymerase, thereby blocking DNA replication and repair while disrupting mitochondrial membrane integrity to trigger pro-apoptotic factor release and cellular apoptosis. By driving apoptotic cell death in rapidly dividing blast cells, this mechanism inhibits neoplastic cell expansion and provides the therapeutic rationale for treating hematologic malignancies such as acute myeloid leukemia and acute lymphocytic leukemia.

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.