NCT01063660

Brief Summary

This is a multicenter, multinational, non-randomized, non-controlled open-label phase II trial to evaluate the safety and efficacy of treosulfan in a combination regimen with fludarabine as conditioning therapy prior to allogeneic stem cell transplantation (SCT) in patients with AML. The aim is to demonstrate a clinical benefit compared with historical data on intravenous busulfan (BusulfexTM, BusilvexTM), the only drug so far registered in the indication conditioning before allogeneic stem cell transplantation.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
75

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Mar 2004

Typical duration 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

March 1, 2004

Completed
3.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2007

Completed
2.6 years until next milestone

First Submitted

Initial submission to the registry

February 3, 2010

Completed
2 days until next milestone

First Posted

Study publicly available on registry

February 5, 2010

Completed
Last Updated

February 5, 2010

Status Verified

February 1, 2010

First QC Date

February 3, 2010

Last Update Submit

February 4, 2010

Conditions

Acute Myeloid Leukaemia

Keywords

TreosulfanFludarabineATGAML

Outcome Measures

Primary Outcomes (1)

  • Efficacy - Evaluation of engraftment. Safety - Evaluation of the incidence of the following CTC grade 3 and 4 adverse events between day -6 and day +28 - hyperbilirubinemia and mucositis / stomatitis - veno-occlusive disease - seizures

    3.5 years

Secondary Outcomes (1)

  • Efficacy - Evaluation of disease free survival (DFS) - Evaluation of overall survival (OS) - Evaluation of relapse incidence (RI) - Donor chimerism on day +28, +56 and +100. Safety - Evaluation of NRM on days +28 and +100

    3.5 years

Study Arms (1)

Treosulfan

EXPERIMENTAL

Patients with acute myeloid leukaemia (AML) according to WHO classification (\> 20% myeloblasts in peripheral blood or bone marrow at initial diagnosis) with \< 5% myeloblasts in the bone marrow, indicated for allogeneic transplantation

Drug: Treosulfan

Interventions

TreosulfanDRUG

14 g/m²/d day -6 to -4

Also known as: Ovastat
Treosulfan

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Patients with acute myeloid leukaemia (AML) according to WHO classification (\> 20% myeloblasts in peripheral blood or bone marrow at initial diagnosis) with \< 5% myeloblast in the bone marrow, indicated for allogeneic transplantation
  • Availability of an HLA-identical sibling donor (MRD) or HLA-identical unrelated donor (MUD) HLA-identity defined by the following markers: A, B, DRB1, DQB1.
  • Target graft size (unmanipulated)
  • bone marrow: 2 - 10 x 106 CD34+ cells/kg BW recipient or \> 2 x 108 nucleated cells/kg BW recipient or
  • peripheral blood: 4 - 10 x 106 CD34+ cells/kg BW recipient
  • Age \> 18 and \< 60 years
  • Karnofsky Index \> 80 %
  • Adequate contraception in female patients of child-bearing potential
  • Written informed consent

You may not qualify if:

  • Therapy related secondary AML
  • AML with t(8;21)(q22;q22) in CR1
  • Acute promyelocytic leukaemia with t(15;17)(q22;q12) in CR1
  • Secondary malignancies
  • Previous allogeneic transplantation
  • Severe concomitant illnesses / medical conditions (e.g. impaired respiratory and/or cardiac function)
  • Known and manifested malignant involvement of the CNS
  • Active infectious disease
  • HIV- positivity or active hepatitis infection
  • Impaired liver function (Bilirubin \> upper normal limit; Transaminases \> 3.0 x upper normal limit)
  • Impaired renal function (Creatinine-clearance \< 60 ml/min; Serum Creatinine \> 1.5 x upper normal limit).
  • Pleural effusion or ascites \> 1.0 L
  • Pregnancy or lactation
  • Known hypersensitivity to treosulfan and/or fludarabine
  • Participation in another experimental drug trial within 4 weeks before day -6
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Rostock

Rostock, 18057, Germany

Location

Related Publications (1)

  • Casper J, Holowiecki J, Trenschel R, Wandt H, Schaefer-Eckart K, Ruutu T, Volin L, Einsele H, Stuhler G, Uharek L, Blau I, Bornhaeuser M, Zander AR, Larsson K, Markiewicz M, Giebel S, Kruzel T, Mylius HA, Baumgart J, Pichlmeier U, Freund M, Beelen DW. Allogeneic hematopoietic SCT in patients with AML following treosulfan/fludarabine conditioning. Bone Marrow Transplant. 2012 Sep;47(9):1171-7. doi: 10.1038/bmt.2011.242. Epub 2011 Dec 12.

    PMID: 22158386DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

treosulfan

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Mathias Freund, MD

    University of Rostock

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
SUPPORTIVE CARE
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

February 3, 2010

First Posted

February 5, 2010

Study Start

March 1, 2004

Study Completion

July 1, 2007

Last Updated

February 5, 2010

Record last verified: 2010-02

Locations

DE(1)