Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01062490
Recruitment Status : Completed
First Posted : February 4, 2010
Last Update Posted : February 4, 2010
Information provided by:
medac GmbH

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.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Drug: Treosulfan Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : November 2004
Actual Primary Completion Date : July 2008
Actual Study Completion Date : October 2009

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Treosulfan
Patients with myelodysplastic syndrome, (MDS) according to WHO classification (< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) indicated for allogeneic transplantation
Drug: Treosulfan
14 g/m2/d, day -6 to -4
Other Name: Ovastat

Primary Outcome Measures :
  1. Efficacy: Evaluation of engraftment [ Time Frame: 4 years ]
  2. 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 ]

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

Inclusion Criteria:

  1. Patients with myelodysplastic syndrome, (MDS) according to WHO classification (< 20 % myeloblasts in peripheral blood or bone marrow at initial diagnosis) indicated for allogeneic transplantation
  2. 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.
  3. 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
  4. Age > 18 and < 60 years
  5. Karnofsky Index > 80 %
  6. Adequate contraception in female patients of child-bearing potential
  7. Written informed consent

Exclusion Criteria:

  1. 'Secondary' or therapy-related MDS with known history of exposure to cytotoxic alkylating drugs and/or radiation therapy
  2. Previous AML-induction therapy with more than two courses (e.g. in case of blast excess)
  3. Previous allogeneic transplantation
  4. Severe concomitant illnesses / medical conditions (e.g. impaired respiratory and/or cardiac function)
  5. Known and manifested malignant involvement of the CNS
  6. Active infectious disease
  7. HIV- positivity or active hepatitis infection
  8. Impaired liver function (Bilirubin > upper normal limit; Transaminases > 3.0 x upper normal limit)
  9. Impaired renal function (Creatinine-clearance < 60 ml/min; Serum Creatinine > 1.5 x upper normal limit).
  10. Pleural effusion or ascites > 1.0 L
  11. Pregnancy or lactation
  12. Known hypersensitivity to treosulfan and/or fludarabine
  13. Participation in another experimental drug trial within 4 weeks before study
  14. Non-co-operative behaviour or non-compliance
  15. Psychiatric diseases or conditions that might impair the ability to give informed consent

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01062490

Helsinki University Central Hospital
Helsinki, Finland, 00029
Sponsors and Collaborators
medac GmbH
Principal Investigator: Tapani Ruutu, MD Biomedicum Helsinki 2 C, POB 705, Turkholmankatu 8 C, FIN-00029 HUS Helsinki, Finland

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Joachim Baumgart, PhD, medac Gesellschaft für klinische Spezialpraeparate mbH Identifier: NCT01062490     History of Changes
Other Study ID Numbers: MC-FludT.8/MDS
First Posted: February 4, 2010    Key Record Dates
Last Update Posted: February 4, 2010
Last Verified: February 2010

Keywords provided by medac GmbH:
allogeneic stem cell transplantation

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Myeloablative Agonists