Pentostatin has been evaluated across numerous clinical trials spanning Phase 1 through Phase 3 for diverse hematologic malignancies and immunological disorders, including leukemias, non-Hodgkin lymphoma, graft-versus-host disease, and primary T-cell dysregulation disorders. Sponsored by academic centers, federal institutions, and industry partners, trial statuses range from withdrawn or terminated studies to active and completed protocols assessing post-transplantation chimerism and disease prophylaxis.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT05757310 | RECRUITING | Recurrent Severe Aplastic Anemia; Refractory Severe Aplastic Anemia |
City of Hope Medical Center |
2024-11-15 | PHASE1 |
| NCT03249831 | ACTIVE_NOT_RECRUITING | Sickle Cell Disease; Sickle Cell Disorder; Hemoglobinopathies; Thalassemia; Anemia, Sickle Cell |
City of Hope Medical Center |
2019-01-04 | PHASE1 |
| NCT03663933 | ACTIVE_NOT_RECRUITING | Lymphoproliferative Disorders; Autoimmune Lymphoproliferative; Primary T-cell Immunodeficiency Disorders; Immune System Diseases; Common Variable Immunodeficiency |
National Cancer Institute (NCI) |
2018-09-04 | PHASE2 |
| NCT02105766 | ACTIVE_NOT_RECRUITING | Sickle Cell Disease; Thalassemia; Stem Cell Transplantation; Graft vs Host Disease |
National Heart, Lung, and Blood Institute (NHLBI) |
2014-04-21 | PHASE2 |
| NCT03077542 | ACTIVE_NOT_RECRUITING | Sickle Cell Disease |
National Heart, Lung, and Blood Institute (NHLBI) |
2017-04-06 | PHASE1; PHASE2 |
| NCT01059786 | ACTIVE_NOT_RECRUITING | Hairy Cell Leukemia |
National Cancer Institute (NCI) |
2010-07-01 | PHASE2 |
| NCT03112603 | COMPLETED | Graft-versus-host Disease (GVHD) |
Incyte Corporation |
2017-06-29 | PHASE3 |
| NCT01416974 | COMPLETED | Leukemia |
Memorial Sloan Kettering Cancer Center |
2011-08-22 | PHASE1 |
| NCT00074282 | COMPLETED | Leukemia |
Eastern Cooperative Oncology Group |
2005-04-14 | PHASE2 |
| NCT01362790 | COMPLETED | Mesothelioma; Adenocarcinoma of Lung; Pancreatic Neoplasms |
National Cancer Institute (NCI) |
2011-05-11 | PHASE1; PHASE2 |
| NCT01352312 | TERMINATED | Chronic Lymphocytic Leukemia; B-Cell Non-Hodgkin's Lymphoma |
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University |
2011-05-25 | PHASE1 |
| NCT00541034 | COMPLETED | Leukemia; Lymphoma |
Memorial Sloan Kettering Cancer Center |
2005-05 | PHASE2 |
| NCT01681563 | COMPLETED | Chronic Lymphocytic Leukemia |
Niguarda Hospital |
2011-09 | PHASE2 |
| NCT00923845 | COMPLETED | Renal Cell Carcinoma; Graft-Versus-Host Disease; Engraftment Syndrome |
National Cancer Institute (NCI) |
2008-03-01 | PHASE2 |
| NCT00096161 | COMPLETED | Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia, BCR-ABL1 Positive; Graft Versus Host Disease; Hodgkin Lymphoma; Myelodysplastic/Myeloproliferative Neoplasm; Non-Hodgkin Lymphoma; Plasma Cell Myeloma; Waldenstrom Macroglobulinemia |
Fred Hutchinson Cancer Center |
2003-05 | PHASE2 |
| NCT01460602 | WITHDRAWN | Follicular Lymphoma; Marginal Zone Lymphoma; Mantle Cell Lymphoma |
Louisiana State University Health Sciences Center Shreveport |
2010-05 | PHASE1; PHASE2 |
| NCT00546377 | COMPLETED | Leukemia; Lymphoma |
Memorial Sloan Kettering Cancer Center |
2005-07 | PHASE1; PHASE2 |
| NCT00045305 | COMPLETED | Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms |
Eastern Cooperative Oncology Group |
2006-10-24 | PHASE2 |
| NCT00816595 | COMPLETED | Leukemia; Lymphoma |
Mayo Clinic |
2009-02 | PHASE2 |
| NCT01024010 | COMPLETED | Chronic Lymphocytic Leukemia; Stage 0 Chronic Lymphocytic Leukemia; Stage I Chronic Lymphocytic Leukemia; Stage I Small Lymphocytic Lymphoma; Stage II Chronic Lymphocytic Leukemia; Stage II Small Lymphocytic Lymphoma; Stage III Chronic Lymphocytic Leukemia; Stage III Small Lymphocytic Lymphoma; Stage IV Chronic Lymphocytic Leukemia; Stage IV Small Lymphocytic Lymphoma |
Mayo Clinic |
2010-03 | PHASE2 |
| NCT00669318 | COMPLETED | Leukemia; Lymphoma |
Mayo Clinic |
2008-07 | PHASE2 |
| NCT00496340 | COMPLETED | Hematologic Malignancies |
H. Lee Moffitt Cancer Center and Research Institute |
2007-07 | PHASE2 |
| NCT01188798 | COMPLETED | Acute Lymphoblastic Leukemia; Acute Myelocytic Leukemia; Chronic Myelocytic Leukemia; Hodgkin's Disease; Myelodysplastic Syndrome |
St. Jude Children's Research Hospital |
2010-09 | PHASE3 |
| NCT00254163 | COMPLETED | B-Cell Chronic Lymphocytic Leukemia |
US Oncology Research |
2003-12 | PHASE3 |
| NCT01001780 | WITHDRAWN | Active Chronic Graft Versus Host Disease |
University of Rochester |
2009-08 | PHASE2 |
| NCT00602836 | COMPLETED | Leukemia; Lymphoma |
Mayo Clinic |
2008-02 | PHASE2 |
| NCT00709215 | UNKNOWN | Refractory TdT-Positive Leukemia |
OncoVista, Inc. |
2008-06 | PHASE1; PHASE2 |
| NCT01188798 | Completed | Acute Lymphoblastic Leukemia|Acute Myelocytic Leukemia|Chronic Myelocytic Leukemia|Hodgkin''s Disease|Myelodysplastic Syndrome |
St. Jude Children''s Research Hospital |
2010-09 | Phase 3 |
| NCT00453193 | TERMINATED | Lymphoma; Leukemia |
M.D. Anderson Cancer Center |
2004-09 | PHASE2 |
| NCT00816413 | WITHDRAWN | Chronic Myeloproliferative Disorders; Leukemia; Lymphoma; Myelodysplastic Syndromes; Nonmalignant Neoplasm |
University of Nebraska |
2008-09 | PHASE1; PHASE2 |
| NCT00506922 | COMPLETED | Leukemia; Lymphoma |
M.D. Anderson Cancer Center |
2000-09 | PHASE1; PHASE2 |
| NCT00402714 | COMPLETED | Hematologic Malignancies |
Yale University |
2006-07 | PHASE2 |
| NCT00698685 | TERMINATED | Leukemia; Lymphoma; Hodgkin's Disease; Hematologic Neoplasms; Multiple Myeloma; Carcinoma, Renal Cell |
University of Arizona |
2006-01-23 | PHASE2 |
| NCT00224874 | COMPLETED | Graft vs Host Disease; Immune System Disorders |
National Heart, Lung, and Blood Institute (NHLBI) |
2005-09 | PHASE2 |
| NCT00927797 | UNKNOWN | Immunocytoma/Morbus Waldenström; B-Cell Non-Hodgkin's Lymphoma; B-Cell Chronic Lymphocytic Leukemia |
Heidelberg University |
2005-02 | PHASE2 |
| NCT00571662 | COMPLETED | Acute Myelogenous Leukemia; Acute Lymphocytic Leukemia; Chronic Myelogenous Leukemia; Chronic Lymphocytic Leukemia; Myelodysplastic Syndromes; Multiple Myeloma; Non-Hodgkins Lymphoma; Hodgkins Disease; Peripheral T-cell Lymphoma |
University of Nebraska |
2000-12-08 | PHASE2 |
| NCT00144430 | COMPLETED | Graft vs Host Disease |
Ann & Robert H Lurie Children's Hospital of Chicago |
2004-01 | PHASE2 |
| NCT00074035 | COMPLETED | Graft Versus Host Disease |
Alliance for Clinical Trials in Oncology |
2003-12 | PHASE2 |
| NCT00709215 | Unknown status | Refractory TdT-Positive Leukemia |
OncoVista Inc.|AAIPharma |
2008-06 | Phase 1|Phase 2 |
| NCT00057954 | TERMINATED | Lymphoma |
Eastern Cooperative Oncology Group |
2005-11-09 | PHASE2 |
| NCT00201786 | COMPLETED | Graft-Versus-Host Disease |
Ohio State University Comprehensive Cancer Center |
2003-07 | PHASE2 |
| NCT00201721 | COMPLETED | B-Chronic Lymphocytic Leukemia |
Ohio State University Comprehensive Cancer Center |
2002-07 | PHASE2 |
| NCT00032773 | TERMINATED | Acute Graft Versus Host Disease |
Astex Pharmaceuticals, Inc. |
2002-01-30 | PHASE1; PHASE2 |
| NCT00003658 | COMPLETED | Leukemia; Lymphoma |
Memorial Sloan Kettering Cancer Center |
1998-09 | PHASE2 |
| NCT00049413 | COMPLETED | Leukemia; Lymphoma |
Hoag Memorial Hospital Presbyterian |
2002-06 | PHASE2 |
| NCT00006968 | COMPLETED | Kidney Cancer |
Jonsson Comprehensive Cancer Center |
2000-09 | PHASE1; PHASE2 |
| NCT00026351 | COMPLETED | Leukemia; Lymphoma |
Pharmatech Oncology |
2000-12 | PHASE2 |
(data from https://clinicaltrials.gov, updated on 2026-01-05)
Check the
Pentostatin
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
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>).
Note: Technical data last updated: Sep 1, 2026.