CClinicalTrials.gg
CompletedNCT01062490Updated Feb 4, 2010

Treosulfan Based Conditioning Myelodysplastic Syndrome (MDS)

A Phase 2 interventional study of Treosulfan in Myelodysplastic Syndrome, sponsored by medac GmbH. Completed at 1 site in Finland. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2010-02-04.

Sponsored by medac GmbH · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
45
Allocation
Non-randomized
Ages
18 Years to 60 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Myelodysplastic Syndrome

Keywords

  • Treosulfan
  • MDS
  • allogeneic stem cell transplantation
  • leukemia
  • Conditioning
03

In context

Preleukemia

1,317 studies on the registry are indexed under Preleukemia; 57 are open to participants now.

This study's enrollment of 45 is above the median of 36 across 1,060 interventional studies indexed under Preleukemia.

Browse Preleukemia studies →

Lead sponsor

medac GmbH is the lead sponsor of 21 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 60 Years
Sexes eligible
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

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
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
45 participants (actual)

Study arms

  • 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

Interventions

  • DrugTreosulfan

    14 g/m2/d, day -6 to -4

    Also known as: Ovastat

06

What researchers measure

Primary outcomes

  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

07

Study locations

1 site
  • Helsinki University Central Hospital
    Helsinki, 00029, Finland
08

References and documents

Publications

  • 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. PubMed 21659356 ↗
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 4, 2010, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT01062490
Lead sponsor
medac GmbH
First posted
Feb 4, 2010
Start date
Nov 2004
Primary completion
Jul 2008
Completion
Oct 2009
Last update
Feb 4, 2010

Study contacts

Tapani Ruutu, MD
principal investigator · Biomedicum Helsinki 2 C, POB 705, Turkholmankatu 8 C, FIN-00029 HUS Helsinki, Finland

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Feb 2010. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Start the discussion