Treosulfan-based Conditioning for Transplantation in AML/MDS

Phase II Trial of Fludarabine Combined With Intravenous Treosulfan and Allogeneic Hematopoietic Stem-cell Transplantation in Patients With Chemo-refractory or Previously Untreated Acute Myeloid Leukemia and Myelodysplastic Syndrome.

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00491634

Enrollment

Registered

2007-06-26

Start date

2007-06-30

Completion date

2014-06-30

Last updated

2015-12-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acute Myeloid Leukemia, Myelodysplastic Syndrome

Keywords

acute myeloid leukemia, myelodysplastic syndrome, allogeneic stem cell transplantation, reduced-intensity conditioning, treosulfan

Brief summary

The study hypotheses is that the introduction of dose escalated treosulfan, in substitution to busulfan, will reduce toxicity after allogeneic transplantation while improving myeloablation and and disease control in patients with AML and MDS not eligible for standard transplantation.

Interventions

DRUGtreosulfan

12 g/m2 x 3 days

DRUGTreosulfan

12 g/m2 x 3

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 68 Years

Healthy volunteers

Inclusion criteria

1. Age less than physiologic 68 years. 2. Patients with AML and MDS not eligible for standard TBI- or Busulfan-based myeloablative conditioning due to age, concurrent medical condition, or extensive prior therapy (e.g. age \> 55 years for HLA-matched sibling transplants or \> 50 for matched unrelated donor transplants, prior / concomitant pulmonary, liver, or other organ complications). 3. This study will only include patients with chemo-refractory disease or previously untreated active disease. A. acute myeloid leukemias (AML) according to WHO classification (\> 20% myeloblasts in peripheral blood or bone marrow at diagnosis) in induction failure, PR, untreated or chemo-refractory relapse. Patients must have \> 10% marrow blasts at the time of transplantation. B. myelodysplastic syndromes (MDS) according to WHO classification (\< 20% myeloblasts in peripheral blood and bone marrow at diagnosis), indicated for allogeneic transplantation: \- refractory anaemia with excess blasts (RAEB-1 and RAEB-2) with no prior therapy 4. Patients must have an HLA matched related or unrelated donor willing to donate either peripheral blood stem cells or bone marrow. Matching is based on high-resolution class I (HLA-A, -B, -C) and class II (HLA-DRB1, -DQB1) typing. The goal is to transplant \> 3 x 106 CD34+ cells per kg body weight of the recipient -

Exclusion criteria

1. Bilirubin \> 3.0 mg/dl, transaminases \> 3 times upper normal limit 2. Creatinine \> 2.0 mg/dl 3. ECOG-Performance status \> 2 4. Uncontrolled infection 5. Pregnancy or lactation 6. Abnormal lung diffusion capacity (DLCO \< 40% predicted) 7. Severe cardiovascular disease 8. CNS disease involvement 9. Pleural effusion or ascites \> 1 liter 10. Known hypersensitivity to fludarabine or treosulfan 11. Psychiatric conditions/disease that impair the ability to give informed consent or to adequately co-operate -

Design outcomes

Primary

Measure	Time frame
disease-free survival	2 years after transplantation

Secondary

Measure	Time frame
treatment-related mortality, GVHD, relapse, overall survival	2 year after transplantation

Countries

Israel

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026