Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)

• Status: Completed
• Trial end date: 2009-10-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: medac GmbH
• Treatments: Busulfan; Treosulfan

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