Treosulfan-based Conditioning for Transplantation in AML/MDS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00491634
Recruitment Status : Completed
First Posted : June 26, 2007
Last Update Posted : December 2, 2015
Information provided by (Responsible Party):
Dr. Avichai Shimoni MD, Sheba Medical Center

June 25, 2007
June 26, 2007
December 2, 2015
June 2007
June 2014   (Final data collection date for primary outcome measure)
disease-free survival [ Time Frame: 2 years after transplantation ]
Same as current
Complete list of historical versions of study NCT00491634 on Archive Site
treatment-related mortality, GVHD, relapse, overall survival [ Time Frame: 2 year after transplantation ]
Same as current
Not Provided
Not Provided
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.
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.
Not Provided
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Acute Myeloid Leukemia
  • Myelodysplastic Syndrome
  • Drug: treosulfan
    12 g/m2 x 3 days
  • Drug: Treosulfan
    12 g/m2 x 3
Experimental: 1
  • Drug: treosulfan
  • Drug: Treosulfan
Kröger N, Shimoni A, Zabelina T, Schieder H, Panse J, Ayuk F, Wolschke C, Renges H, Dahlke J, Atanackovic D, Nagler A, Zander A. Reduced-toxicity conditioning with treosulfan, fludarabine and ATG as preparative regimen for allogeneic stem cell transplantation (alloSCT) in elderly patients with secondary acute myeloid leukemia (sAML) or myelodysplastic syndrome (MDS). Bone Marrow Transplant. 2006 Feb;37(4):339-44.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
June 2014
June 2014   (Final data collection date for primary outcome measure)

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 -
Sexes Eligible for Study: All
18 Years to 68 Years   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
Not Provided
Not Provided
Dr. Avichai Shimoni MD, Sheba Medical Center
Dr. Avichai Shimoni MD
Not Provided
Principal Investigator: Arnon Nagler, MD Chaim Sheba Medical Center
Sheba Medical Center
November 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP