patients with haematologic maliignancies prior to allogenic haematopoietic stem cell MedDRA version: 6.1 Level: HLGT Classification code 10024324
Conditions
Interventions
Trade Name: Treosulfano
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Treosulfan
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
Trade Name
Sponsors
OSPEDALE S. RAFFAELE
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Patients with haematological malignancies acute myeloid leukaemia-AML, acute lymphoblast leukemia-ALL, chronic myeloid leukemia-CML, myelodysplastic syndrome-MDS, diffuse large cell lymphoma-DLCL, Hodgkin lymphoma, chronic lymphocytic leukemia-CLL, multiple myeloma-MM or other heamatological malignancies 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 A, B, DRB1, DQB1. 3. Target graft size unmanipulated - bone marrow 2 - 10 x 106 CD34 cells/kg BW recipient or 2 x 108 nucleated cells/kg BW recipient or - peripheral blood 4 - 10 x 106 CD34 cells/kg BW recipient4. Age 18 and 70 years 65 for MUD only 5. Karnofsky Index 80 6. Adequate contraception in female patients of child-bearing potential 7. Written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Secondary malignancies2. Previous allogeneic transplantation3. Severe concomitant illnesses / medical conditions e.g. impaired respiratory and/or cardiac function 4. Known and manifested malignant involvement of the CNS 5. Active infectious disease 6. HIV- positivity or active hepatitis infection7. Impaired liver function Bilirubin upper normal limit; Transaminases 3.0 x upper normal limit 8. Impaired renal function Creatinine-clearance 60 ml/min; Serum Creatinine 1.5 x upper normal limit .9. Pleural effusion or ascites 1.0 L 10. Pregnancy or lactation11. Known hypersensitivity to treosulfan and/or fludarabine12. Participation in another experimental drug trial within 4 weeks before day -613. Non-co-operative behaviour or non-compliance 14. Psychiatric diseases or conditions that might impair the ability to give informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): Efficacy- Evaluation of engraftment Safety- Evaluation of the incidence of CTC grade 3 and 4 adverse events between day -6 and day 28;Main Objective: Evaluation of safety and efficacy of treosulfan based conditioning prior to allogenic haematopoietic stem cell transplantation.;Secondary Objective: Efficacy- Evaluation of disease free survival DFS - Evaluation of overall survival OS - Evaluation of relapse incidence RI - Documentation of donor chimerism on day 28, 56 and 100Safety- Evaluation of incidence of non-relapse mortality NRM on day 28 and day 100 and the cumulative incidence of NRM- Evaluation of cumulative incidence and severity of acute and chronic graft vs. host disease GvHD .End of total follow up for OS, DFS, RI, NRM, cGvHD 365 days after transplantation of the last patient included | — |
Countries
Italy
Outcome results
None listed