Myelodysplastic Syndrome (MDS) MedDRA version: 20.0 Level: PT Classification code 10028533 Term: Myelodysplastic syndrome System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: allogeneic HSC
Product Code: allogeneic HSC
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Allogene HSC
O
Sponsors
University Medical Center Hamburg-Eppendorf
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Patients with proven de novo or therapy-related MDS / CMML (WBC 30 %) 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 76 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 34
Exclusion criteria
Exclusion criteria: • Blasts > 30 % in bone marrow at time of diagnosis • Central nervous involvement • Severe irreversible renal, hepatic, pulmonary or cardiac disease, such as • Total bilirubin, SGPT or SGOT = 3 times upper the normal level • Left ventricular ejection fraction < 30 % • Creatinine clearance < 30 ml/min • DLCO < 35 % and/or receiving supplementary continuous oxygen • Pregnant or breastfeeding female subject • Patients with a life-expectancy of less than six months because of another debilitating disease • Serious psychiatric or psychological disorders • Uncontrolled invasive fungal infection at time of registration • Known positive for HIV or acute infectious hepatitis, type A, B or C • Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment until the end of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the overall survival at three years of patients who receive after 4 cycles of 5-azacytidine (Vidaza®) either allogeneic stem cell transplantation or continuous 5-azacytidine (Vidaza®) if no compatible donor is available.; Secondary Objective: Comparison of “overall survival” and “event-free survival” between treatment arm “Allogeneic stem cell transplantation after 5 azacytidine induction” and treatment arm “Continuous 5 azacytidine alone” over the whole study period. To assess the response rate, the event-free survival at three years, toxicity and treatment-related mortality, the impact of the comorbidity-index (HCT-CI) on outcome and the quality of life in both treatment arms. Further to analyse prognostic factors which influence survival in both arms. ;Primary end point(s): Overall survival at three years between both arms.;Timepoint(s) of evaluation of this end point: After three years | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: The endpoints will be evaluated on different timepoints, after 1, 2 and / or 3 years; Secondary end point(s): - Overall survival for all patients assigned to one of the two treatment arms as time to event endpoint. - Event-free survival for all patients assigned to one of the two treatment arms as time to event endpoint. - Comparison of response rate and duration according IWG response criteria in MDS between both treatment arms - The event-free survival will be assessed by three years - The evaluation of toxicity will be performed according to the reporting guidelines as per NCICTCAE - Overall survival and event-free survival adjusted by the comorbidity-index (HCT-CI) according Sorror - Comparison of treatment –related mortality at three years - Comparison of quality of life between both arms according to the life core questionnaire QLQ-C30 and the treatment specific high-dose chemotherapy module QLQ HD-C29 of the EORTC Quality of Life Group | — |
Countries
Germany
Contacts
Public ContactPrincipal Investigator
University Medical Center Hamburg Eppendorf
Outcome results
None listed