Back to resultsTreosulfan Based Conditioning Myelodysplastic Syndrome (MDS)
Primary Purpose
Myelodysplastic Syndrome
Status
Completed
Phase
Phase 2
Locations
Finland
Study Type
Interventional
Intervention
Treosulfan
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring Treosulfan, MDS, allogeneic stem cell transplantation, leukemia, Conditioning
Eligibility Criteria
Inclusion Criteria:
- Patients with myelodysplastic syndrome, (MDS) according to WHO classification (< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) 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: HLA-A, -B, -DRB1, DQB1.
- Target graft size (unmanipulated) bone marrow: 2 to 10 x 106 CD34+ cells/kg BW recipient or at least 2 x 108 nucleated cells /kg BW or peripheral blood: 4 to 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:
- 'Secondary' or therapy-related MDS with known history of exposure to cytotoxic alkylating drugs and/or radiation therapy
- Previous AML-induction therapy with more than two courses (e.g. in case of blast excess)
- 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 study
- Non-co-operative behaviour or non-compliance
- Psychiatric diseases or conditions that might impair the ability to give informed consent
Sites / Locations
- Helsinki University Central Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treosulfan
Arm Description
Patients with myelodysplastic syndrome, (MDS) according to WHO classification (< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) indicated for allogeneic transplantation
Outcomes
Primary Outcome Measures
Efficacy: Evaluation of engraftment
Safety: Evaluation of CTC grade 3 and 4 adverse events between Day -6 and Day +28: hyperbilirubinemia and mucositis/stomatitis, veno-occlusive disease, seizures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01062490
Brief Title
Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)
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 Myelodysplastic Syndrome (MDS)
Study Type
Interventional
2. Study Status
Record Verification Date
February 2010
Overall Recruitment Status
Completed
Study Start Date
November 2004 (undefined)
Primary Completion Date
July 2008 (Actual)
Study Completion Date
October 2009 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
medac GmbH
4. Oversight
Data Monitoring Committee
No
5. Study Description
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 MDS.
The aim is to demonstrate a clinical benefit compared to historical data with intravenous busulfan.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndrome
Keywords
Treosulfan, MDS, allogeneic stem cell transplantation, leukemia, Conditioning
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
45 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treosulfan
Arm Type
Experimental
Arm Description
Patients with myelodysplastic syndrome, (MDS) according to WHO classification (< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) indicated for allogeneic transplantation
Intervention Type
Drug
Intervention Name(s)
Treosulfan
Other Intervention Name(s)
Ovastat
Intervention Description
14 g/m2/d, day -6 to -4
Primary Outcome Measure Information:
Title
Efficacy: Evaluation of engraftment
Time Frame
4 years
Title
Safety: Evaluation of CTC grade 3 and 4 adverse events between Day -6 and Day +28: hyperbilirubinemia and mucositis/stomatitis, veno-occlusive disease, seizures
Time Frame
4 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with myelodysplastic syndrome, (MDS) according to WHO classification (< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) 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: HLA-A, -B, -DRB1, DQB1.
Target graft size (unmanipulated) bone marrow: 2 to 10 x 106 CD34+ cells/kg BW recipient or at least 2 x 108 nucleated cells /kg BW or peripheral blood: 4 to 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:
'Secondary' or therapy-related MDS with known history of exposure to cytotoxic alkylating drugs and/or radiation therapy
Previous AML-induction therapy with more than two courses (e.g. in case of blast excess)
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 study
Non-co-operative behaviour or non-compliance
Psychiatric diseases or conditions that might impair the ability to give informed consent
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Tapani Ruutu, MD
Organizational Affiliation
Biomedicum Helsinki 2 C, POB 705, Turkholmankatu 8 C, FIN-00029 HUS Helsinki, Finland
Official's Role
Principal Investigator
Facility Information:
Facility Name
Helsinki University Central Hospital
City
Helsinki
ZIP/Postal Code
00029
Country
Finland
12. IPD Sharing Statement
Citations:
PubMed Identifier
21659356
Citation
Ruutu T, Volin L, Beelen DW, Trenschel R, Finke J, Schnitzler M, Holowiecki J, Giebel S, Markiewicz M, Uharek L, Blau IW, Kienast J, Stelljes M, Larsson K, Zander AR, Gramatzki M, Repp R, Einsele H, Stuhler G, Baumgart J, Mylius HA, Pichlmeier U, Freund M, Casper J. Reduced-toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes: final results of an international prospective phase II trial. Haematologica. 2011 Sep;96(9):1344-50. doi: 10.3324/haematol.2011.043810. Epub 2011 Jun 9.
Results Reference
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Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)
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