Overview

This trial is active, not recruiting.

Conditions acute myeloid leukemia, myelodysplastic syndrome
Treatments treosulfan
Phase phase 2
Sponsor Dr. Avichai Shimoni MD
Start date June 2007
End date June 2014
Trial size 24 participants
Trial identifier NCT00491634, SHEBA-07-3116-AN-CTIL

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
treosulfan
treosulfan
12 g/m2 x 3 days
treosulfan
12 g/m2 x 3

Primary Outcomes

Measure
disease-free survival
time frame: 2 years after transplantation

Secondary Outcomes

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

Eligibility Criteria

Male or female participants from 18 years up to 68 years old.

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 -

Additional Information

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.
Principal investigator Arnon Nagler, MD
Trial information was received from ClinicalTrials.gov and was last updated in October 2013.
Information provided to ClinicalTrials.gov by Sheba Medical Center.