This site became the new on June 19th. Learn more.
Show more Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu
Give us feedback

Treosulfan-based Conditioning for Transplantation in AML/MDS

This study has been completed.
Information provided by (Responsible Party):
Dr. Avichai Shimoni MD, Sheba Medical Center Identifier:
First received: June 25, 2007
Last updated: November 30, 2015
Last verified: November 2015
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.

Condition Intervention Phase
Acute Myeloid Leukemia Myelodysplastic Syndrome Drug: treosulfan Drug: Treosulfan Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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.

Resource links provided by NLM:

Further study details as provided by Dr. Avichai Shimoni MD, Sheba Medical Center:

Primary Outcome Measures:
  • disease-free survival [ Time Frame: 2 years after transplantation ]

Secondary Outcome Measures:
  • treatment-related mortality, GVHD, relapse, overall survival [ Time Frame: 2 year after transplantation ]

Estimated Enrollment: 24
Study Start Date: June 2007
Study Completion Date: June 2014
Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Drug: treosulfan
12 g/m2 x 3 days
Drug: Treosulfan
12 g/m2 x 3


Ages Eligible for Study:   18 Years to 68 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 -
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00491634

Chaim Sheba Medical Center
Tel-Hashomer, Israel
Sponsors and Collaborators
Dr. Avichai Shimoni MD
Principal Investigator: Arnon Nagler, MD Chaim Sheba Medical Center
  More Information

Responsible Party: Dr. Avichai Shimoni MD, Dr. Avichai Shimoni, Sheba Medical Center Identifier: NCT00491634     History of Changes
Other Study ID Numbers: SHEBA-07-3116-AN-CTIL
Study First Received: June 25, 2007
Last Updated: November 30, 2015

Keywords provided by Dr. Avichai Shimoni MD, Sheba Medical Center:
acute myeloid leukemia
myelodysplastic syndrome
allogeneic stem cell transplantation
reduced-intensity conditioning

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Myeloablative Agonists processed this record on September 21, 2017