Treosulfan Based Conditioning Acute Myeloid Leukaemia (AML)

This study has been completed.
Sponsor:
Information provided by:
medac GmbH
ClinicalTrials.gov Identifier:
NCT01063660
First received: February 3, 2010
Last updated: February 4, 2010
Last verified: February 2010
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 ] [ Designated as safety issue: Yes ]

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 ] [ Designated as safety issue: Yes ]

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

  Eligibility

Ages Eligible for Study:   18 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01063660

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

No publications provided by medac GmbH

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Prof. Dr. Mathias Freund, University of Rostock
ClinicalTrials.gov Identifier: NCT01063660     History of Changes
Other Study ID Numbers: MC-FludT.7/AML
Study First Received: February 3, 2010
Last Updated: February 4, 2010
Health Authority: Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by medac GmbH:
Treosulfan
Fludarabine
ATG
AML

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Leukemia
Neoplasms by Histologic Type
Neoplasms
Treosulfan
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on October 16, 2014