Clinical trial · Interventional
Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)
Clinical Phase II Trial to Evaluate the Safety and Efficacy of Treosulfan Based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome (MDS)
NCT01062490CI-TRIAL-00003104completedPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This is a multicenter, multinational, non-randomized, non-controlled open-label phase II trial to evaluate the safety and efficacy of treosulfan in a combination regimen with fludarabine as conditioning therapy prior to allogeneic stem cell transplantation (SCT) in patients with MDS. The aim is to demonstrate a clinical benefit compared to historical data with intravenous busulfan.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myelodysplastic Syndrome | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Treosulfan | Drug | Treosulfan | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Treosulfan
- description
- Patients with myelodysplastic syndrome, (MDS) according to WHO classification (\< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) indicated for allogeneic transplantation
- interventionNames
- Drug: Treosulfan
Primary outcomes (2)
- measure
- Efficacy: Evaluation of engraftment
- timeFrame
- 4 years
- measure
- Safety: Evaluation of CTC grade 3 and 4 adverse events between Day -6 and Day +28: hyperbilirubinemia and mucositis/stomatitis, veno-occlusive disease, seizures
- timeFrame
- 4 years
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 60 Years
Show eligibility criteria text
Inclusion Criteria: 1. Patients with myelodysplastic syndrome, (MDS) according to WHO classification (\< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) indicated for allogeneic transplantation 2. Availability of an HLA-identical sibling donor (MRD) or HLA-identical unrelated donor (MUD) HLA-identity defined by the following markers: HLA-A, -B, -DRB1, DQB1. 3. Target graft size (unmanipulated) bone marrow: 2 to 10 x 106 CD34+ cells/kg BW recipient or at least 2 x 108 nucleated cells /kg BW or peripheral blood: 4 to 10 x 106 CD34+ cells/kg BW recipient 4. Age \> 18 and \< 60 years 5. Karnofsky Index \> 80 % 6. Adequate contraception in female patients of child-bearing potential 7. Written informed consent Exclusion Criteria: 1. 'Secondary' or therapy-related MDS with known history of exposure to cytotoxic alkylating drugs and/or radiation therapy 2. Previous AML-induction therapy with more than two courses (e.g. in case of blast excess) 3. Previous allogeneic transplantation 4. Severe concomitant illnesses / medical conditions (e.g. impaired respiratory and/or cardiac function) 5. Known and manifested malignant involvement of the CNS 6. Active infectious disease 7. HIV- positivity or active hepatitis infection 8. Impaired liver function (Bilirubin \> upper normal limit; Transaminases \> 3.0 x upper normal limit) 9. Impaired renal function (Creatinine-clearance \< 60 ml/min; Serum Creatinine \> 1.5 x upper normal limit). 10. Pleural effusion or ascites \> 1.0 L 11. Pregnancy or lactation 12. Known hypersensitivity to treosulfan and/or fludarabine 13. Participation in another experimental drug trial within 4 weeks before study 14. Non-co-operative behaviour or non-compliance 15. Psychiatric diseases or conditions that might impair the ability to give informed consent
References
Publications (1)
- DERIVEDRuutu T, Volin L, Beelen DW, Trenschel R, Finke J, Schnitzler M, Holowiecki J, Giebel S, Markiewicz M, Uharek L, Blau IW, Kienast J, Stelljes M, Larsson K, Zander AR, Gramatzki M, Repp R, Einsele H, Stuhler G, Baumgart J, Mylius HA, Pichlmeier U, Freund M, Casper J. Reduced-toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes: final results of an international prospective phase II trial. Haematologica. 2011 Sep;96(9):1344-50. doi: 10.3324/haematol.2011.043810. Epub 2011 Jun 9. PMID 21659356