{"id":"treosulfan-treo","safety":{"commonSideEffects":[{"rate":null,"effect":"Myelosuppression"},{"rate":null,"effect":"Mucositis"},{"rate":null,"effect":"Nausea and vomiting"},{"rate":null,"effect":"Hepatotoxicity"},{"rate":null,"effect":"Infection"}]},"_chembl":null,"_dailymed":{"setId":"2d06b03c-17aa-0492-e063-6294a90abd8f","title":"GRAFAPEX (TREOSULFAN) INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION [MEDEXUS PHARMA, INC.]"},"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Treosulfan is a prodrug that is metabolized to active diepoxide metabolites, which alkylate DNA by forming inter- and intra-strand cross-links. This leads to cell cycle arrest and apoptosis. It is used as a conditioning regimen in hematopoietic stem cell transplantation, particularly in pediatric patients, due to its reduced toxicity profile compared to traditional alkylating agents like busulfan.","oneSentence":"Treosulfan is an alkylating agent that cross-links DNA, causing cell death and used primarily as a myeloablative conditioning agent before hematopoietic stem cell transplantation.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T04:06:41.749Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Conditioning agent for hematopoietic stem cell transplantation in pediatric patients with hematologic malignancies and non-malignant disorders"}]},"trialDetails":[{"nctId":"NCT07316595","phase":"PHASE2","title":"Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome","status":"NOT_YET_RECRUITING","sponsor":"Federal Research Institute of Pediatric Hematology, Oncology and Immunology","startDate":"2025-12-30","conditions":"HSCT, Nijmegen Breakage Syndrome, Treosulfan Based Conditioning","enrollment":24},{"nctId":"NCT07025824","phase":"PHASE2","title":"Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation","status":"NOT_YET_RECRUITING","sponsor":"Technische Universität Dresden","startDate":"2025-09","conditions":"AML - Acute Myeloid Leukemia, MDS (Myelodysplastic Syndrome)","enrollment":220},{"nctId":"NCT06861257","phase":"","title":"Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients","status":"RECRUITING","sponsor":"Fondazione IRCCS Policlinico San Matteo di Pavia","startDate":"2021-02-24","conditions":"Pediatric Hematopoietic Stem Cell Transplantation, Malignant Disorders, Non-malignant Disorders","enrollment":70},{"nctId":"NCT01949129","phase":"PHASE2, PHASE3","title":"Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia","status":"RECRUITING","sponsor":"St. Anna Kinderkrebsforschung","startDate":"2013-04","conditions":"Acute Lymphoblastic Leukaemia","enrollment":1800},{"nctId":"NCT01894477","phase":"PHASE2","title":"Treo/Flu/TBI With Donor Stem Cell Transplant for Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia","status":"UNKNOWN","sponsor":"Fred Hutchinson Cancer Center","startDate":"2013-11","conditions":"Acute Myeloid Leukemia in Remission, Chronic Myelomonocytic Leukemia, Minimal Residual Disease","enrollment":102}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":55,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"marketed","status":"active","brandName":"Treosulfan (Treo)","genericName":"Treosulfan (Treo)","companyName":"Federal Research Institute of Pediatric Hematology, Oncology and Immunology","companyId":"federal-research-institute-of-pediatric-hematology-oncology-and-immunology","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Treosulfan (Treo) is a small molecule used as a cross-linking agent that targets DNA. It is being studied in clinical trials for various conditions, including pediatric hematopoietic stem cell transplantation, malignant and non-malignant disorders, acute myeloid leukemia, and myelodysplastic syndrome.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}