Drugs / Treosulfan
last change Jul 2026 re-read 2 minutes ago

Treosulfan

Small molecule

Developed for
myelodysplastic syndrome · acute myeloid leukemia · disease related to hematopoietic stem cell transplant · chronic myeloid leukemia · chronic myelomonocytic leukemia · Hodgkins lymphoma · acute lymphoblastic leukemia · bone marrow failure syndrome
+47 more · chronic lymphocytic leukemia/small lymphocytic lymphoma · diffuse large B-cell lymphoma · hematologic disorder · hematopoietic and lymphoid cell neoplasm · Nijmegen breakage syndrome · non-Hodgkin lymphoma · plasma cell myeloma · acute biphenotypic leukemia · acute myeloid leukemia with 11q23 abnormalities · acute myeloid leukemia with t(8;21)(q22;q22) translocation · acute myeloid leukemia, del(5q31-q32) · acute myeloid leukemia, inv(16)(p13.1;q22) · acute myeloid leukemia, t(15;17)(q24;q21) · acute myeloid leukemia, t(16;16)(p13.1;q22) · adult acute lymphoblastic leukemia · anaplastic large cell lymphoma · blast phase chronic myelogenous leukemia, BCR-ABL1 positive · Burkitt lymphoma · childhood acute lymphoblastic leukemia · childhood acute myeloid leukemia · childhood myelodysplastic syndrome · congenital amegakaryocytic thrombocytopenia · Diamond-Blackfan anemia · Ewing sarcoma · GATA2 deficiency with susceptibility to MDS/AML · hemoglobinopathy · immunodeficiency disease · inborn errors of metabolism · inherited sideroblastic anemia · juvenile myelomonocytic leukemia · leukemia · leukemia, myeloid, accelerated-phase · lymphoblastic lymphoma · lymphoplasmacytic lymphoma · mantle cell lymphoma · marginal zone lymphoma · melanoma · mixed phenotype acute leukemia · non-small cell lung carcinoma · ocular melanoma · ovarian cancer · paroxysmal nocturnal hemoglobinuria · prolymphocytic leukemia · SAMD9-related spectrum and myeloid neoplasm risk · SAMD9L-related spectrum and myeloid neoplasm risk · Shwachman-Diamond syndrome · squamous cell lung carcinoma
Investigated by
medac GmbH · Fred Hutchinson Cancer Center · National Cancer Institute (NCI) · National Heart, Lung, and Blood Institute (NHLBI) · Celerion · Federal Research Institute of Pediatric Hematology, Oncology and Immunology
+42 more · National Marrow Donor Program · Syneos Health · ALL SCTped Forum · ALL-BFM Study Group · Assistance Publique - Hôpitaux de Paris · Australian & New Zealand Children's Haematology/Oncology Group · Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia · Blood and Marrow Transplant Clinical Trials Network · Center for International Blood and Marrow Transplant Research · Charite University, Berlin, Germany · Ciceri Fabio · DCS Innovative Diagnostik Systeme · Dr. Avichai Shimoni MD · Dutch Childhood Oncology Group · European Society for Blood and Marrow Transplantation · Hiege-Stiftung gegen Hautkrebs · Hospices Civils de Lyon · IRCCS San Raffaele · Insel Gruppe AG, University Hospital Bern · Institut Paoli-Calmettes · Masonic Cancer Center, University of Minnesota · Medexus Pharma, Inc. · Neovii Biotech · North Eastern German Society of Gynaecological Oncology · OHSU Knight Cancer Institute · Pediatric Blood and Marrow Transplant Consortium · Pfizer · Prof. Dr. med. Wolfgang Bethge · Resource for Clinical Investigation in Blood and Marrow Transplantation (RCI BMT) · Sheba Medical Center · St. Anna Children's Cancer Research · St. Baldrick's Foundation · Swiss Pediatric Oncology Group · Synteract, Inc. · Technical University Dresden · The Anticancer Fund · University Hospital Muenster · University Hospital Regensburg · University Hospital Tuebingen · University of Magdeburg · University of Wuerzburg · Venn Life Sciences

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved US (FDA) GRAFAPEX 2025-01-21 fda.gov
Approved EU (EMA) Trecondi 2019-06-20 europa.eu

Trials 38 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT05534620 Feb 2024 → May 2025 acute myeloid leukemia, disease related to hematopoietic stem cell transplant, myelodysplastic syndrome medac GmbH Completed No outcome recorded
Phase 1/23 trials
Phase 1/2 NCT00253513 Jun 2005 → Oct 2009 leukemia, myelodysplastic syndrome OHSU Knight Cancer Institute Completed No outcome recorded
Phase 1/2 NCT00384553 Jun 2004 → Jun 2010 non-Hodgkin lymphoma University of Magdeburg Unknown No outcome recorded
Phase 1/2 NCT01063647 Nov 2001 → Jun 2005 hematopoietic and lymphoid cell neoplasm medac GmbH Completed No outcome recorded
Phase 229 trials
Phase 2 NCT07598110 Oct 2026 → Oct 2030 expected acute myeloid leukemia Institut Paoli-Calmettes Not yet recruiting No outcome recorded
Phase 2 NCT07493538 Apr 2026 → Mar 2030 expected acute myeloid leukemia, myelodysplastic syndrome Masonic Cancer Center, University of Minnesota Recruiting No outcome recorded
Phase 2 NCT07214064 Jan 2026 → Aug 2027 expected acute myeloid leukemia, myelodysplastic syndrome University Hospital Tuebingen Not yet recruiting No outcome recorded
Phase 2 NCT07025824 Sep 2025 → Dec 2028 expected acute myeloid leukemia, myelodysplastic syndrome Technical University Dresden Not yet recruiting No outcome recorded
Phase 2 NCT06198842 Nov 2023 → Jan 2027 expected Nijmegen breakage syndrome Federal Research Institute of Pediatric Hematology, Oncology and Immunology Recruiting No outcome recorded
Phase 2 NCT04994808 Aug 2023 → Jan 2026 overdue acute myeloid leukemia, myelodysplastic syndrome Fred Hutchinson Cancer Center Active not recruiting No outcome recorded
Phase 2 NCT05636787 Jun 2023 → Jan 2028 expected plasma cell myeloma Insel Gruppe AG, University Hospital Bern Recruiting No outcome recorded
Phase 2 NCT07232953 Nov 2022 → Jun 2026 overdue acute myeloid leukemia, disease related to hematopoietic stem cell transplant Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia Active not recruiting No outcome recorded
Phase 2 NCT04965597 Apr 2022 → Feb 2026 Diamond-Blackfan anemia, GATA2 deficiency with susceptibility to MDS/AML, SAMD9-related spectrum and myeloid neoplasm risk, SAMD9L-related spectrum and myeloid neoplasm risk +5 Fred Hutchinson Cancer Center Completed No outcome recorded
Phase 2 NCT03980769 May 2021 → Jul 2028 expected hematologic disorder Fred Hutchinson Cancer Center Recruiting No outcome recorded
Phase 2/31 trial
Phase 2/3 NCT01949129 Apr 2013 → Jun 2025 overdue acute lymphoblastic leukemia St. Anna Children's Cancer Research Recruiting No outcome recorded
Phase 34 trials
Phase 3 NCT00987636 Oct 2009 → Mar 2019 Ewing sarcoma University Hospital Muenster Completed No outcome recorded
Phase 3 NCT00822393 Nov 2008 → Jan 2018 acute myeloid leukemia, myelodysplastic syndrome medac GmbH Completed No outcome recorded
Phase 3 NCT00779714 Oct 2008 → Oct 2012 melanoma University of Wuerzburg Unknown No outcome recorded
Phase 3 NCT00170690 Aug 2004 → Dec 2011 ovarian cancer North Eastern German Society of Gynaecological Oncology Completed No outcome recorded
Also used as a comparator or background therapy in 9 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT03963024 Background Feb 2014 → Jan 2019 acute leukemia, bone marrow disorder, graft versus host disease, plasma cell myeloma IRCCS San Raffaele Terminated No outcome recorded
Phase 1/21 trial
Phase 1/2 NCT01010243 Background Oct 2009 → Oct 2013 plasma cell myeloma University of Regensburg Unknown No outcome recorded
Phase 23 trials
Phase 2 NCT07316595 Comparator Dec 2025 → Dec 2028 expected Nijmegen breakage syndrome, disease related to hematopoietic stem cell transplant Federal Research Institute of Pediatric Hematology, Oncology and Immunology Not yet recruiting No outcome recorded
Phase 2 NCT03434704 Background Jun 2018 → Oct 2019 acute leukemia, fungal infectious disease, graft versus host disease, myelodysplastic syndrome Ciceri Fabio Completed No outcome recorded
Phase 2 NCT01810926 Background Sep 2011 → Aug 2015 graft versus host disease Franco Locatelli Unknown No outcome recorded
Phase 2/31 trial
Phase 2/3 NCT06756152 Background Jul 2024 → Jul 2026 overdue acute biphenotypic leukemia, bilineal acute myeloid leukemia, myelodysplastic syndrome Federal Research Institute of Pediatric Hematology, Oncology and Immunology Recruiting No outcome recorded
Phase 33 trials
Phase 3 NCT04000698 Background Oct 2019 → Jul 2022 acute lymphoblastic leukemia, acute myeloid leukemia Federal Research Institute of Pediatric Hematology, Oncology and Immunology Unknown No outcome recorded
Phase 3 NCT01423500 Background Jan 2007 → Sep 2011 childhood acute lymphoblastic leukemia St. Anna Children's Cancer Research Unknown No outcome recorded
Phase 3 NCT01423747 Background Jul 2003 → Sep 2011 childhood acute lymphoblastic leukemia St. Anna Children's Cancer Research Unknown No outcome recorded

Evidence & citations 20 cited values

Every value below carries the sentence it was read from. 51 sources stand behind the page.

FieldValueCited text
Known as Treosulfan “Venetoclax (Venclyxto®): 400 mg abs./d day -8 to -2, Fludarabine 30 mg/m2 BSA day -6 to day -2, Treosulfan 10 g/m2 BSA day -4 to day -2” NCT07214064
46

“followed by high dose chemotherapy with treosulfan, etoposide and cisplatin (TEC)” NCT00384553

“Conditioning with treosulfan 14 g/m2 day -6 to -4” NCT01582048

“* Vecanoid Condition * treosulfan * fludarabine” NCT04000698

NCT01423500

NCT00598624

NCT06198842

NCT04195633

NCT03980769

NCT01949129

NCT06756152

NCT03434704

NCT02852083

NCT00779714

NCT04994808

NCT07493538

NCT00168870

NCT00919503

NCT00796068

NCT01010243

NCT07232953

NCT01894477

NCT01810926

NCT00491634

NCT02349906

NCT04400045

NCT05534620

NCT05636787

NCT00822393

NCT03961919

NCT01063647

NCT01062490

NCT00987636

NCT03963024

NCT02333058

NCT07598110

NCT07316595

NCT00170690

NCT07025824

NCT00129155

NCT01423747

NCT01772953

NCT04965597

NCT01063660

NCT01079013

NCT00860574

NCT00253513

Known as dihydroxybusulfan “Treosulfan (L-threitol-1,4-bis-methanesulfonate; dihydroxybusulfan) was initially used in the treatment of certain solid tumors.” PMID 21478921 Apr 2011
Known as Grafapex ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as Graftrevo ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as Kepcondi ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as L-threitol 1,4-dimethanesulfonate ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as L-threitol-1,4-bis-methanesulfonate “Treosulfan (L-threitol-1,4-bis-methanesulfonate; dihydroxybusulfan) was initially used in the treatment of certain solid tumors.” PMID 21478921 Apr 2011
Known as L-threitol-1,4-bis-methanesulphonate “Treosulfan (L-threitol-1,4-bis-methanesulphonate; Ovastat(R)) is a bifunctional alkylating drug indicated for the treatment of advanced ovarian carcinoma.” PMID 12651206 Apr 2003
Known as NSC-39069 ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as Ovastat ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as Ovastat(R) “Treosulfan (L-threitol-1,4-bis-methanesulphonate; Ovastat(R)) is a bifunctional alkylating drug indicated for the treatment of advanced ovarian carcinoma.” PMID 12651206 Apr 2003
Known as Threosulphan ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as Trecondi ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as Trecondi Ideogen “The present study aims to combine high-dose melphalan (Melphalan Ideogen) with treosulfan (Trecondi® Ideogen), a well established bifunctional alkylating agent.” NCT05636787
Known as Trecondyv ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as TREO “Although the use of treosulfan (TREO) in conventional donor hematopoietic cell transplantation (HCT) has been extensively evaluated, its use in cord blood transplantation (CBT)...” PMID 32706891 Jul 2020
2

Treosulfan (Treo)” NCT07316595

Treosulfan (Treo)” NCT07025824

Known as Treograft ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Known as Treosulfano ChEMBL registry synonym — accepted as the source's own label CHEMBL455186
Modality Small molecule “Treosulfan (powder for solution for infusion) is a marketed alkylating medication” NCT07598110
Route Oral “Treosulfan 600 mg/m² p.o. on daý 1-28, 57-84, etc” NCT00170690