Treosulfan-based Conditioning for Transplantation in AML/MDS

November 30, 2015 updated by: Dr. Avichai Shimoni MD

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

24

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Israel
      • Tel-Hashomer, Israel
        • Chaim Sheba Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 68 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 -

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1
treosulfan
Drug: treosulfan
12 g/m2 x 3 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
disease-free survival
Time Frame: 2 years after transplantation
2 years after transplantation

Secondary Outcome Measures

Outcome Measure
Time Frame
treatment-related mortality, GVHD, relapse, overall survival
Time Frame: 2 year after transplantation
2 year after transplantation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Dr. Avichai Shimoni MD

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2007

Primary Completion (Actual)

June 1, 2014

Study Completion (Actual)

June 1, 2014

Study Registration Dates

First Submitted

June 25, 2007

First Submitted That Met QC Criteria

June 25, 2007

First Posted (Estimate)

June 26, 2007

Study Record Updates

Last Update Posted (Estimate)

December 2, 2015

Last Update Submitted That Met QC Criteria

November 30, 2015

Last Verified

November 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SHEBA-07-3116-AN-CTIL

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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