Treosulfan
drugOn this page
Also known as GrafapexGraftrevoKepcondiNSC-39069OvastatThreosulphanTreosulfanoTrecondiTrecondyvTreograft
Summary
Treosulfan (CHEMBL455186) is an approved small molecule (ATC L01AB02); indicated across 19 conditions including neoplasm and acute myeloid leukemia.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: L01AB02
- Indications: 19 conditions
- Clinical trials: 40
- Chemistry: 278.3 Da · C6H14O8S2
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL455186 |
| Name | Treosulfan |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 9882105 |
| ATC | L01AB02 |
| Molecular formula | C6H14O8S2 |
| Molecular weight | 278.3 |
| InChIKey | YCPOZVAOBBQLRI-WDSKDSINSA-N |
SMILES: CS(=O)(=O)OC[C@@H]([C@H](COS(=O)(=O)C)O)O
IUPAC name: [(2S,3S)-2,3-dihydroxy-4-methylsulfonyloxybutyl] methanesulfonate
Also known as: Grafapex, Graftrevo, Kepcondi, NSC-39069, Ovastat, Threosulphan, Treosulfan, Treosulfano, Trecondi, Trecondyv, Treograft, TREOSULFAN
Patent coverage: 137 distinct patent families (240 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
No target linkage available.
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
19 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| neoplasm | 4 | MONDO:0005070 | EFO:0000616 |
| acute myeloid leukemia | 3 | MONDO:0018874 | EFO:0000222 |
| Ewing sarcoma | 3 | MONDO:0012817 | EFO:0000174 |
| myelodysplastic syndrome | 3 | MONDO:0018881 | EFO:0000198 |
| acute lymphoblastic leukemia | 3 | MONDO:0004967 | EFO:0000220 |
| melanoma | 3 | MONDO:0005105 | EFO:0000756 |
| ovarian cancer | 3 | MONDO:0008170 | MONDO:0008170 |
| Hodgkins lymphoma | 2 | MONDO:0004952 | EFO:0000183 |
| squamous cell lung carcinoma | 2 | MONDO:0005097 | EFO:0000708 |
| plasma cell myeloma | 2 | MONDO:0009693 | EFO:0001378 |
| lymphoid neoplasm | 2 | MONDO:0005157 | EFO:0001642 |
| non-small cell lung carcinoma | 2 | MONDO:0005233 | EFO:0003060 |
| non-Hodgkin lymphoma | 2 | MONDO:0018908 | EFO:0005952 |
| graft versus host disease | 2 | MONDO:0013730 | MONDO:0013730 |
| hemoglobinuria | 2 | MONDO:0003656 | MONDO:0100244 |
| Nijmegen breakage syndrome | 2 | MONDO:0009623 | MONDO:0009623 |
3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 40.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 28 |
| PHASE3 | 6 |
| PHASE1/PHASE2 | 3 |
| PHASE2/PHASE3 | 1 |
| PHASE1 | 1 |
| Not specified | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT01949129 | PHASE2/PHASE3 | RECRUITING | Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia |
| NCT00170690 | PHASE3 | COMPLETED | Preference Study With Elderly Patients Recurrent Ovarian Cancer |
| NCT00779714 | PHASE3 | UNKNOWN | Comparative Study of Individualized Sensitivity-Directed Chemotherapy Versus DTIC |
| NCT00822393 | PHASE3 | COMPLETED | Clinical Phase III Trial Treosulfan-based Conditioning Versus Reduced-intensity Conditioning (RIC) |
| NCT00987636 | PHASE3 | COMPLETED | Study in Localized and Disseminated Ewing Sarcoma |
| NCT01423500 | PHASE3 | UNKNOWN | ALL-SCT BFM International- HSCT in Children and Adolescents With ALL |
| NCT01423747 | PHASE3 | UNKNOWN | Allogeneic Stem Cell Transplantation in Children and Adolescents With Acute Lymphoblastic Leukaemia |
| NCT03980769 | PHASE2 | RECRUITING | Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders |
| NCT04195633 | PHASE2 | RECRUITING | Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies |
| NCT04994808 | PHASE2 | ACTIVE_NOT_RECRUITING | Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia |
| NCT05636787 | PHASE2 | RECRUITING | Clinical Trial Investigating the Chemotherapeutic Compound Treosulfan (Trecondi® Ideogen) in Myeloma Patients |
| NCT06198842 | PHASE2 | RECRUITING | Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome |
| NCT07025824 | PHASE2 | NOT_YET_RECRUITING | Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation |
| NCT07232953 | PHASE2 | ACTIVE_NOT_RECRUITING | Safety and Efficacy of FT14 Conditioning for Allogeneic HSCT in Acute Myeloid Leukemia |
| NCT07316595 | PHASE2 | NOT_YET_RECRUITING | Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome |
| NCT07493538 | PHASE2 | RECRUITING | MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases |
| NCT07598110 | PHASE2 | NOT_YET_RECRUITING | FLUDARABINE-TREOSULFAN REDUCED INTENSITY CONDITIONING REGIMEN PRIOR HAPLOIDENTICAL STEM CELL TRANSPLANTATION WITH POST TRANSPLANTATION CYCLOPHOSPHAMIDE FOR OLDER AND/OR FRAIL PATIENTS WITH AML |
| NCT00129155 | PHASE2 | UNKNOWN | MiniMUD Study - Unrelated Reduced Intensity Conditioning With Treosulfan® for Allogeneic Stem Cell Transplantation in Patients With Hematological Malignancies |
| NCT00168870 | PHASE2 | UNKNOWN | Study of Metastatic Ocular Melanoma |
| NCT00253513 | PHASE1/PHASE2 | COMPLETED | Treosulfan and Fludarabine in Treating Younger Patients Who Are Undergoing a Donor Stem Cell Transplant for Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Myelodysplastic Syndrome |
| NCT00491634 | PHASE2 | COMPLETED | Treosulfan-based Conditioning for Transplantation in AML/MDS |
| NCT00796068 | PHASE2 | COMPLETED | Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients With Hematological Cancer Who Are Undergoing Umbilical Cord Blood Transplant |
| NCT00860574 | PHASE2 | COMPLETED | Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia |
| NCT00919503 | PHASE2 | COMPLETED | Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders |
| NCT01010243 | PHASE1/PHASE2 | UNKNOWN | Third-line Therapy of Multiple Myeloma a Prospective Phase I /II Trial |
| NCT01062490 | PHASE2 | COMPLETED | Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS) |
| NCT01063647 | PHASE1/PHASE2 | COMPLETED | Dose-range Finding Treosulfan-based Conditioning |
| NCT01063660 | PHASE2 | COMPLETED | Treosulfan Based Conditioning Acute Myeloid Leukaemia (AML) |
| NCT01079013 | PHASE2 | UNKNOWN | Treosulfan-based Conditioning for Allogeneic Stem-cell Transplantation (SCT) in Lymphoid Malignancies |
| NCT01772953 | PHASE2 | COMPLETED | Treosulfan/Fludarabine/Low Dose TBI as a Preparative Regimen for Children With AML/MDS Undergoing Allo HCT |
| NCT01810926 | PHASE2 | UNKNOWN | T&B Depletion Non Malignant |
| NCT01894477 | PHASE2 | UNKNOWN | Treo/Flu/TBI With Donor Stem Cell Transplant for Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia |
| NCT02333058 | PHASE2 | COMPLETED | Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies |
| NCT02349906 | PHASE2 | COMPLETED | Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases |
| NCT02852083 | PHASE2 | UNKNOWN | A Trial With Metronomic Low-dose Treosulfan, Pioglitazone and Clarithromycin Versus Standard Treatment in NSCLC |
| NCT03961919 | PHASE2 | COMPLETED | Phase II Trial in Elderly Patients With AML or MDS in Complete Remission Not Eligible for Allogenic Transplant |
| NCT04400045 | PHASE2 | UNKNOWN | Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage Syndrome |
| NCT04965597 | PHASE2 | COMPLETED | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) |
| NCT05534620 | PHASE1 | COMPLETED | Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics |
| NCT02048800 | Not specified | COMPLETED | Treosulfan Pharmacokinetics in Children Undergoing Allogeneic HSCT |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).