NCT00491634

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2007

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2007

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

June 25, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 26, 2007

Completed
6.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2014

Completed
Last Updated

December 2, 2015

Status Verified

November 1, 2015

Enrollment Period

7 years

First QC Date

June 25, 2007

Last Update Submit

November 30, 2015

Conditions

Acute Myeloid LeukemiaMyelodysplastic Syndrome

Keywords

acute myeloid leukemiamyelodysplastic syndromeallogeneic stem cell transplantationreduced-intensity conditioningtreosulfan

Outcome Measures

Primary Outcomes (1)

  • disease-free survival

    2 years after transplantation

Secondary Outcomes (1)

  • treatment-related mortality, GVHD, relapse, overall survival

    2 year after transplantation

Study Arms (1)

1

EXPERIMENTAL

treosulfan

Drug: treosulfan

Interventions

TreosulfanDRUG

12 g/m2 x 3 days

1

Eligibility Criteria

Age18 Years - 68 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age less than physiologic 68 years.
  • 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).
  • 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
  • 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 -

You may not qualify if:

  • Bilirubin \> 3.0 mg/dl, transaminases \> 3 times upper normal limit
  • Creatinine \> 2.0 mg/dl
  • ECOG-Performance status \> 2
  • Uncontrolled infection
  • Pregnancy or lactation
  • Abnormal lung diffusion capacity (DLCO \< 40% predicted)
  • Severe cardiovascular disease
  • CNS disease involvement
  • Pleural effusion or ascites \> 1 liter
  • Known hypersensitivity to fludarabine or treosulfan
  • Psychiatric conditions/disease that impair the ability to give informed consent or to adequately co-operate -

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Chaim Sheba Medical Center

Tel Litwinsky, Israel

Location

Related Publications (1)

  • Kroger 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. doi: 10.1038/sj.bmt.1705259.

    PMID: 16415898BACKGROUND

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteMyelodysplastic Syndromes

Interventions

treosulfan

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Diseases

Study Officials

  • Arnon Nagler, MD

    Chaim Sheba Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Dr. Avichai Shimoni

Study Record Dates

First Submitted

June 25, 2007

First Posted

June 26, 2007

Study Start

June 1, 2007

Primary Completion

June 1, 2014

Study Completion

June 1, 2014

Last Updated

December 2, 2015

Record last verified: 2015-11

Locations

IL(1)