Drugs / Pentostatin
last change Aug 2026 re-read 9 days ago

Pentostatin

Small molecule targets ADA via inhibition

Developed for
leukemia · lymphoma · B-cell chronic lymphocytic leukemia · chronic lymphocytic leukemia/small lymphocytic lymphoma · graft versus host disease · Hodgkins lymphoma · myelodysplastic syndrome · plasma cell myeloma
+23 more · acute myeloid leukemia · chronic graft versus host disease · peripheral T-cell lymphoma, not otherwise specified · sickle cell disease · acute graft versus host disease · acute lymphoblastic leukemia · aplastic anemia · chronic myeloid leukemia · renal cell carcinoma · thalassemia · Waldenstrom macroglobulinemia · acute leukemia of ambiguous lineage · benign neoplasm · follicular lymphoma · hairy cell leukemia · hematopoietic and lymphoid cell neoplasm · kidney cancer · mantle cell lymphoma · marginal zone lymphoma · myeloproliferative neoplasm · primary cutaneous T-cell lymphoma · urinary bladder cancer · urothelial carcinoma
Investigated by
National Cancer Institute (NCI) · Astex Pharmaceuticals, Inc. · M.D. Anderson Cancer Center · Eastern Cooperative Oncology Group · Mayo Clinic · National Heart, Lung, and Blood Institute (NHLBI)
+41 more · City of Hope Medical Center · GlaxoSmithKline · Memorial Sloan Kettering Cancer Center · Ohio State University Comprehensive Cancer Center · Pharmatech Oncology · University of Nebraska · Alliance for Clinical Trials in Oncology · Ann & Robert H Lurie Children's Hospital of Chicago · Bayer · Blood and Marrow Transplant Clinical Trials Network · Boston Medical Center · California Institute for Regenerative Medicine (CIRM) · Cancer Biotherapy Research Group · Diakonie Krankenhaus Schwäbisch Hall, Germany · Diakonie-Klinikum Stuttgart · East Valley Hematology and Oncology Medical Group · Fred Hutchinson Cancer Center · Gemeinschaftspraxis Porowski & Koniczek, Heilbronn, Germany · H. Lee Moffitt Cancer Center and Research Institute · Heidelberg University · Hoag Memorial Hospital Presbyterian · Jonsson Comprehensive Cancer Center · Klinikum am Plattenwald, Bad Friedrichshall, Germany · Louisiana State University Health Sciences Center Shreveport · Ludwig-Maximilians - University of Munich · Mena, Raul, M.D. · Millennium Pharmaceuticals, Inc. · National Institutes of Health Clinical Center (CC) · Niguarda Hospital · Novartis · Pfizer · Pharmatech · Regione Lombardia · Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University · St. Jude Children's Research Hospital · Tufts Medical Center · US Oncology Research · University Medical Center Mannheim · University of Arizona · University of Rochester · University of Washington

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansion US (FDA) NIPENT — 1993-09-29 fda.gov ↗
Approved US (FDA) NIPENT — 1991-10-11 fda.gov ↗

Trials 47

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 14 trials
Phase 1 NCT05757310 Nov 2024 → Jun 2027 expected aplastic anemia City of Hope Medical Center Recruiting No outcome recorded
Phase 1 NCT03249831 Jan 2019 → Jan 2027 expected sickle cell disease, thalassemia City of Hope Medical Center Active not recruiting No outcome recorded
Phase 1 NCT01352312 May 2011 → Apr 2016 B-cell chronic lymphocytic leukemia Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University Terminated No outcome recorded Stop: Enrollment
Phase 1 NCT00003005 Dec 1997 → Mar 2001 leukemia Boston Medical Center Completed No outcome recorded
Phase 1/27 trials
Phase 1/2 NCT03077542 Apr 2017 → Jul 2023 overdue sickle cell disease National Heart, Lung, and Blood Institute (NHLBI) Active not recruiting No outcome recorded
Phase 1/2 NCT01362790 May 2011 → Aug 2016 lung adenocarcinoma, mesothelioma, pancreatic neoplasm National Cancer Institute (NCI) Completed No outcome recorded
Phase 1/2 NCT01460602 May 2010 → Oct 2014 follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma Louisiana State University Health Sciences Center Shreveport Withdrawn No outcome recorded
Phase 1/2 NCT00816413 Sep 2008 → Feb 2010 benign neoplasm, leukemia, lymphoma, myelodysplastic syndrome +1 University of Nebraska Withdrawn No outcome recorded Stop: Enrollment
Phase 1/2 NCT00032773 Jan 2002 → Nov 2005 acute graft versus host disease Astex Pharmaceuticals, Inc. Terminated No outcome recorded
Phase 1/2 NCT00006968 Sep 2000 → Sep 2004 kidney cancer Jonsson Comprehensive Cancer Center Completed No outcome recorded
Phase 1/2 NCT00506922 Sep 2000 → Nov 2009 leukemia, lymphoma M.D. Anderson Cancer Center Completed No outcome recorded
Phase 232 trials · 1 met primary
Phase 2 NCT03922724 Apr 2019 → Jun 2027 expected lymphoproliferative syndrome, peripheral T-cell lymphoma, not otherwise specified National Cancer Institute (NCI) Active not recruiting No outcome recorded
Phase 2 NCT02788201 Mar 2017 → Oct 2019 urinary bladder cancer, urothelial carcinoma National Cancer Institute (NCI) Completed No outcome recorded
Phase 2 NCT02579967 Nov 2015 → Dec 2031 expected T-cell immunodeficiency, autoimmune lymphoproliferative syndrome, common variable immunodeficiency National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 2 NCT02105766 Apr 2014 → Dec 2023 overdue graft versus host disease, sickle cell disease, thalassemia National Heart, Lung, and Blood Institute (NHLBI) Active not recruiting No outcome recorded
Phase 2 NCT01681563 Sep 2011 → Nov 2015 B-cell chronic lymphocytic leukemia Niguarda Hospital Completed No outcome recorded
Phase 2 NCT01059786 Jul 2010 → Dec 2022 overdue hairy cell leukemia National Cancer Institute (NCI) Active not recruiting Met primary
Phase 2 NCT01024010 Mar 2010 → May 2013 chronic lymphocytic leukemia/small lymphocytic lymphoma Mayo Clinic Completed No outcome recorded
Phase 2 NCT01001780 Aug 2009 → Jan 2011 chronic graft versus host disease University of Rochester Withdrawn No outcome recorded Stop: Enrollment
Phase 2 NCT00669318 Jul 2008 → May 2013 leukemia, lymphoma Mayo Clinic Completed No outcome recorded
Phase 2 NCT00923845 Mar 2008 → Jun 2015 engraftment syndrome, graft versus host disease, renal cell carcinoma National Cancer Institute (NCI) Completed No outcome recorded
Phase 32 trials
Phase 3 NCT01188798 Sep 2010 → Feb 2012 Hodgkins lymphoma, acute lymphoblastic leukemia, acute myeloid leukemia, chronic myeloid leukemia +1 St. Jude Children's Research Hospital Completed No outcome recorded
Phase 3 NCT00254163 Dec 2003 → Sep 2011 B-cell chronic lymphocytic leukemia US Oncology Research Completed No outcome recorded
Phase 41 trial
Phase 4 NCT00131313 Jan 2003 → Apr 2009 B-cell chronic lymphocytic leukemia East Valley Hematology and Oncology Medical Group Unknown No outcome recorded
Phase not applicable1 trial
— NCT02551718 Sep 2015 → Jun 2020 acute leukemia of ambiguous lineage, acute lymphoblastic leukemia University of Washington Completed No outcome recorded
Comparator or background therapy10 trials
Phase 1/2 NCT05470491 Background Jan 2023 → Jul 2027 expected HIV infectious disease, hematopoietic and lymphoid cell neoplasm National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 1/2 NCT02032446 Background Sep 2013 → Sep 2016 hematopoietic and lymphoid cell neoplasm A.O. Ospedale Papa Giovanni XXIII Unknown No outcome recorded
Phase 1/2 NCT00709215 Background Jun 2008 → Dec 2010 acute lymphoblastic leukemia OncoVista, Inc. Unknown No outcome recorded
Phase 1/2 NCT00546377 Background Jul 2005 → May 2014 leukemia, lymphoma Memorial Sloan Kettering Cancer Center Completed No outcome recorded
Phase 2 NCT03663933 Background Sep 2018 → Apr 2025 overdue T-cell immunodeficiency, autoimmune lymphoproliferative syndrome, common variable immunodeficiency National Cancer Institute (NCI) Active not recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 6

DateIssuerRelease
2006-03-14 Astex Pharmaceuticals, Inc. Results SuperGen Announces Results From Combination Study in CLL Published in the Journal of Clinical Oncology astx.com ↗
2005-12-09 Astex Pharmaceuticals, Inc. Results Studies of Nipent® in Combination Cancer Therapies for Treatment of Chronic Lymphocytic Leukemia and Graft-versus Host Disease (GVHD) Presented at American Society of Hematology Annual Meeting astx.com ↗
The response frequencies were virtually identical in both treatment groups with responses seen in 75% of PCR treated patients and 73% of FCR treated patients and a complete response (CR) achieved in 25% in both studies.
2004-12-06 Astex Pharmaceuticals, Inc. Results Studies Exploring Nipent® in Combination Cancer Therapies in the Treatment of Graft-Versus Host Disease (GVHD) Presented at American Society of Hematology Annual Meeting astx.com ↗
The drug showed particular activity in the treatment of children and adolescents with chronic graft versus host disease (cGVHD), and in high -risk patients undergoing reduced intensity transplantation regimens.
2004-06-10 Astex Pharmaceuticals, Inc. Results SuperGen Reports Results of Ongoing Studies of Nipent® in Combination Therapies for Chronic Lymphocytic Leukemia and Low Grade B-cell Lymphomas astx.com ↗
The three studies and their conclusions, which were presented in the General Poster Session, demonstrated that pentostatin used in combination with other chemotherapeutic agents was active, well -tolerated and did not increase the toxicity of the combination regimen.
2004-05-21 Astex Pharmaceuticals, Inc. Results SuperGen Announces Study of Novel Regimen Using Nipent® for Hematopoietic Stem Cell Transplantation Published in Journal of Bone Marrow Transplantation astx.com ↗
The study showed that the treatment was well tolerated and associated with a low incidence of transplant -related mortality and acute GVHD.
2004-01-23 Astex Pharmaceuticals, Inc. Results Published Data Suggests SuperGen’s Nipent®, as Part of a Combination Preparative Regimen, is Active in Allogeneic Bone Marrow Transplant Patients with Myelodysplastic Syndrome astx.com ↗
Published Data Suggests SuperGen's Nipent ®, as Part of a Combination Preparative Regimen, is Active in Allogeneic Bone Marrow Transplant Patients with Myelodysplastic Syndrome

All press releases naming this drug 11 releases

DateIssuerRelease

Evidence & citations 16 cited values

Every value below carries the sentence it was read from. 59 sources stand behind the page.

FieldValueCited text
Known as pentostatin “Assess the CR and ORR following treatment with VELCADE (bortezomib), Nipent (pentostatin) and Rituxan (rituximab) (VNR) in subjects with follicular lymphoma (FL), marginal zone...” NCT01460602 ↗
52

“Pentostatin 4 mg/m2/day IV on days -9 and -5, cyclophosphamide 5 mg/kg orally daily on days -9 through -2 (Closed with amendment L)” NCT02579967 ↗

“such as Etanercept and infliximab, pentostatin, sirolimus, mycophenolate mofetil and extracorporeal photopheresis” NCT02694770 ↗

“Pentostatin will be given by intravenous infusion at a dose of 1 mg/m2 for 3 consecutive days.” NCT02032446 ↗

“e-ATG 40 mg/kg/day IV on days -14 and -13, pentostatin 4 mg/m2/day IV on days -11 and -7” NCT03922724 ↗

“pentostatin 4 mg/kg/d (continuous infusion days -5 \& -4)” NCT00676806 ↗

“Pentostatin 4 mg/m2/day IV on days -11 and -7.” NCT05470491 ↗

“Pazopanib, Pentostatin, Romidepsin” NCT02788201 ↗

“Pazopanib Pentostatin Ponatinib” NCT02551718 ↗

“• Pentostatin” NCT06821542 ↗

NCT01024010 ↗

Show 42 more sources

Known as 2'-Deoxycoformycin “A Phase I Study of Cordycepin (NSC 63984) Plus 2'-Deoxycoformycin (NSC 218321) in Patients With Refractory TdT-Positive Leukemia” NCT00003005 ↗
Known as CI-825 ChEMBL registry synonym — accepted as the source's own label CHEMBL1580 ↗
Known as DCF “Deoxycoformycin (DCF)” NCT00038025 ↗
Known as Deoxycoformycin ClinicalTrials.gov intervention name — accepted as the source's own label NCT00038025 ↗
1

NCT00003005 ↗

Known as Nipent “Pentostatin (Nipent®) and Alemtuzumab (Campath®)” NCT00698685 ↗
3

NCT00496873 ↗

NCT00131313 ↗

NCT01460602 ↗

Known as NSC 218321 “A Phase I Study of Cordycepin (NSC 63984) Plus 2'-Deoxycoformycin (NSC 218321) in Patients With Refractory TdT-Positive Leukemia” NCT00003005 ↗
Known as PD 81565 ChEMBL registry synonym — accepted as the source's own label CHEMBL1580 ↗
Known as Pentostatin - Nipent “Pentostatin - Nipent” NCT01001780 ↗
Known as Pentostatina ChEMBL registry synonym — accepted as the source's own label CHEMBL1580 ↗
Known as Pentostatine ChEMBL registry synonym — accepted as the source's own label CHEMBL1580 ↗
Known as YK-176 ChEMBL registry synonym — accepted as the source's own label CHEMBL1580 ↗
Action Inhibit “the adenosine deaminase inhibitor pentostatin” NCT00003005 ↗
Modality Small molecule “purine nucleoside analogue” PMID 20187101 ↗ May 2010
Route Intravenous “pentostatin is administered over 5 minutes by IV bolus” NCT00003005 ↗
Target ADA “It is a potent inhibitor of the enzyme adenosine deaminase (ADA)” NCT00496340 ↗