MedPath

Dose-range Finding Treosulfan-based Conditioning

Phase 1
Completed
Conditions
Hematological Malignancies
Interventions
Registration Number
NCT01063647
Lead Sponsor
medac GmbH
Brief Summary

Evaluation of the safety and efficacy of 3 x 10, 3 x 12 or 3 x 14 g/m² Treosulfan resp., combined with 5 x 30 mg/m² fludarabine prior to allogeneic, hematopoietic stem cell transplantation of patients with hematological malignancies, but non-eligible to standard conditioning treatment.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
56
Inclusion Criteria
  1. Patients with a haematological chemosensitive malignancy indicated for an allogeneic transplantation, but presenting an increased toxicity risk for classical (high-dose busulfan or standard-dose total body irradiation) conditioning therapies (remission criteria ref. to Appendix L):

    • CML in first or subsequent chronic phase
    • NHL in 2nd CR/PR, chemosensitive PR after autologous transplantation ; CLL in 2nd or subsequent CR/PR
    • Relapsed Morbus Hodgkin (MH) after autologous transplantation
    • Multiple Myeloma (MM) stage II and III according to Durie and Salmon
    • AML in 2nd CR/PR or high-risk AML in 1st CR/PR

    High-risk defined for example by the following:

    • Cytogenetics: -5/5q, -7/7q, del(5q), abnormalities of 3q, complex karyotype (> 3 abnormalities), or
    • PR after 1 cycle of induction therapy
    • ALL in 2nd CR/PR or high-risk ALL in 1st CR/PR

    High-risk defined as follows:

    • Leukocytes > 3000/µl (B-Linage) or > 100000/µl (T-Linage);
    • Pro-B-ALL, pre-T-ALL
    • Cytogenetics: t(9;22)/BCR-ABL; t(4;11)/ALL1-AF
    • MDS (patients without prior chemotherapy may be included)
  2. Availability of an HLA-identical sibling donor (MRD) or HLA-identical unrelated donor (MUD) or one mismatch (out of the 6 standard markers) sibling donor (1 misMRD):

    • HLA-identity defined by the following markers: A, B, DRB1. DQB1 must be recorded.

  3. Age > 18 years

  4. Karnofsky Index > 80 %

  5. Adequate contraception in female patients of child-bearing potential

  6. Co-operative behavior of individual patients

  7. Written informed consent

Exclusion Criteria
  1. Completely chemotherapy-resistant disease
  2. Severe cardiac insufficiency, severe cardio-vascular or other severe concomitant diseases
  3. Symptomatic malignant involvement of the CNS
  4. Active infectious disease
  5. HIV-positive or active hepatitis infection
  6. Impaired liver function (Bilirubin > 1.5 x upper normal limit; Transaminases > 3.0 x upper normal limit)
  7. Impaired renal function (Creatinine-clearance < 60 ml/min; Serum Creatinine > 1.5 x upper normal limit).
  8. Pleural effusion or ascites > 1.0 L
  9. Pregnancy or lactation
  10. Known hypersensitivity to fludarabine and/or treosulfan
  11. Parallel participation in another experimental drug trial

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
2TreosulfanTreosulfan:12 g/m² i.v. on 3 consecutive days (day -6 to -4)
1TreosulfanTreosulfan: 10 g/m² i.v. on 3 consecutive days (day -6 to -4)
3TreosulfanTreosulfan: 14 g/m² i.v. on 3 consecutive days (day -6 to -4)
Primary Outcome Measures
NameTimeMethod
Safety - Evaluation of feasibility and tolerability of 3 x 10, 12 or 14 g/m² Treosulfan combined with 5 x 30 mg/m² fludarabine prior to allogeneic stem cell transplantation • frequency and severity of TRM until 6 months after transplantation6 months
Secondary Outcome Measures
NameTimeMethod
Efficacy - Evaluation of the proportion of relapse- and/or progression free patients six months after transplantation (using standard remission criteria)6 months

Trial Locations

Locations (7)

Silesian Medical University

🇵🇱

Katowice, Poland

Helsinki University Central Hospital

🇫🇮

Helsinki, Finland

Charité University Hospital Berlin

🇩🇪

Berlin, Germany

5th Medical Clinic, Clinic North

🇩🇪

Nuremberg, Germany

University Hospital Hamburg Eppendorf

🇩🇪

Hamburg, Germany

Karolinska University Hospital & Karolinska Institute

🇸🇪

Stockholm, Sweden

University Hospital Rostock

🇩🇪

Rostock, Germany

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