Treosulfan Based Conditioning Acute Myeloid Leukaemia (AML)

Acute Myeloid Leukaemia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01063660
• Other study ID #: MC-FludT.7/AML
• Status: Completed
• Phase: Phase 2
• First received: February 3, 2010
• Last updated: February 4, 2010
• Start date: March 2004
• Est. completion date: July 2007

Study information

• Verified date: February 2010
• Source: medac GmbH
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Federal Institute for Drugs and Medical Devices
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 75
• Est. completion date: July 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 60 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Related Conditions & MeSH terms

• Acute Myeloid Leukaemia
• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute

Intervention

Drug:
• Treosulfan
14 g/m²/d day -6 to -4

Locations

Country	Name	City	State
Germany	University of Rostock	Rostock

Sponsors (1)

Lead Sponsor	Collaborator
medac GmbH

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	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	Yes