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Treosulfan Based Conditioning Acute Myeloid Leukaemia (AML)

This study has been completed.
Information provided by:
medac GmbH Identifier:
First received: February 3, 2010
Last updated: February 4, 2010
Last verified: February 2010

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.

Condition Intervention Phase
Acute Myeloid Leukaemia Drug: Treosulfan Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: Clinical Phase II Trial to Evaluate the Safety and Efficacy of Treosulfan Based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With Acute Myeloid Leukaemia

Resource links provided by NLM:

Further study details as provided by medac GmbH:

Primary Outcome Measures:
  • 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 [ Time Frame: 3.5 years ]

Secondary Outcome Measures:
  • 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 [ Time Frame: 3.5 years ]

Enrollment: 75
Study Start Date: March 2004
Study Completion Date: July 2007
Arms Assigned Interventions
Experimental: Treosulfan
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
14 g/m²/d day -6 to -4
Other Name: Ovastat


Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • Non-co-operative behaviour or non-compliance
  • Psychiatric diseases or conditions that might impair the ability to give informed consent
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Please refer to this study by its identifier: NCT01063660

University of Rostock
Rostock, Germany, 18057
Sponsors and Collaborators
medac GmbH
Principal Investigator: Mathias Freund, MD University of Rostock
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Prof. Dr. Mathias Freund, University of Rostock Identifier: NCT01063660     History of Changes
Other Study ID Numbers: MC-FludT.7/AML
Study First Received: February 3, 2010
Last Updated: February 4, 2010

Keywords provided by medac GmbH:

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Myeloablative Agonists processed this record on September 21, 2017