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Treosulfan-based Conditioning for Transplantation in AML/MDS

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00491634
First Posted: June 26, 2007
Last Update Posted: December 2, 2015
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.
Information provided by (Responsible Party):
Dr. Avichai Shimoni MD, Sheba Medical Center
  Purpose
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
treosulfan
Drug: treosulfan
12 g/m2 x 3 days
Drug: Treosulfan
12 g/m2 x 3

  Eligibility

Information from the National Library of Medicine

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

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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00491634


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

Publications:
Responsible Party: Dr. Avichai Shimoni MD, Dr. Avichai Shimoni, Sheba Medical Center
ClinicalTrials.gov Identifier: NCT00491634     History of Changes
Other Study ID Numbers: SHEBA-07-3116-AN-CTIL
First Submitted: June 25, 2007
First Posted: June 26, 2007
Last Update Posted: December 2, 2015
Last Verified: November 2015

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

Additional relevant MeSH terms:
Syndrome
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Preleukemia
Disease
Pathologic Processes
Neoplasms by Histologic Type
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Treosulfan
Busulfan
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Myeloablative Agonists