Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)

This study has been completed.
Sponsor:
Information provided by:
medac GmbH
ClinicalTrials.gov Identifier:
NCT01062490
First received: February 3, 2010
Last updated: NA
Last verified: February 2010
History: No changes posted
  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 MDS.

The aim is to demonstrate a clinical benefit compared to historical data with intravenous busulfan.


Condition Intervention Phase
Myelodysplastic Syndrome
Drug: Treosulfan
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:


Further study details as provided by medac GmbH:

Primary Outcome Measures:
  • Efficacy: Evaluation of engraftment [ Time Frame: 4 years ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]

Enrollment: 45
Study Start Date: November 2004
Study Completion Date: October 2009
Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

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

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
  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: NCT01062490

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

No publications provided by medac GmbH

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Joachim Baumgart, PhD, medac Gesellschaft für klinische Spezialpraeparate mbH
ClinicalTrials.gov Identifier: NCT01062490     History of Changes
Other Study ID Numbers: MC-FludT.8/MDS
Study First Received: February 3, 2010
Last Updated: February 3, 2010
Health Authority: Finland: Finnish Medicines Agency

Keywords provided by medac GmbH:
Treosulfan
MDS
allogeneic stem cell transplantation
leukemia
Conditioning

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Syndrome
Bone Marrow Diseases
Disease
Hematologic Diseases
Neoplasms
Pathologic Processes
Precancerous Conditions
Treosulfan
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on October 23, 2014