Patients with high-risk hematological diseases, who need a allogeneic stem cell transplantation but who lack a matched unrelated donor MedDRA version: 14.1 Level: PT Classification code 10061187 Term: Haematopoietic neoplasm System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: Cord blood unit
Pharmaceutical Form: Solution for infusion in administration system
Other descriptive name: hematopoietic stem cells
Sponsors
HOVON Foundation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Age 18-65 years inclusive •Diagnosis of poor-risk hematological malignancy or (V)SAA relapsing after or failing immunosuppressive therapy and meeting the criteria for a MUD allo SCT •Lacking a sufficiently matched volunteer unrelated donor or lacking such a donor within the required time period of = 2 months in case of urgently needed alloSCT •Availability of 2 (=4/6) matched UCB grafts with a total nuclear cell count > 4 x 107/kg 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 68 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: •Bilirubin and/or transaminases > 2.5 x normal value •Creatinine clearance < 40 ml/min •Cardiac dysfunction as defined by:ejection fraction < 45%,unstable angina, unstable cardiac arrhythmias • Pulmonary function test with VC, FEV1 and/ or DCO < 50% •Active, uncontrolled infection •History of high dose (= 8 Gy) total body irradiation •Pregnant or lactating females •HIV positivity
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: at 1, 2, 3, 6 and 12 months:;Main Objective: To study the presence of an alloreactive immune response of CD4+ T-cells of the predominant CBU, directed against the non-engrafting CBU as a causative mechanism in CBU predominance.;Secondary Objective: To assess engraftment and engraftment kinetics; to evaluate immune reconstitution, acute and chronic GVHD, chimerism, toxicity, progression-free survival and overall survival after double unit UCBT;Primary end point(s): The proportion of patients with activated class II-specific T-cells (aTCs), defined as: the number of patients with aTCs, divided by the number of patients with class II mismatches for which there are tests available (defined as evaluable patients). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Cumulative incidence of engraftment •Cumulative incidence of graft failure •Time to neutrophil recovery •Time to lymphocyte recovery •Time to platelet recovery •Time to red blood cell transfusion independence •Count of total CD3+, CD4+,CD8+ and CD19+ cells and CD3-CD16/56+ cells at 1,2, 3, 6, 12 and 24 months after UCBT •Incidence and grade of acute GVHD •Incidence of chronic GVHD •Incidence of infections •Transplant related mortality •Progression free survival calculated from transplantation. •Overall survival (OS) calculated from transplantation. ;Timepoint(s) of evaluation of this end point: last visit last patient, 5 years after transplantation last patient | — |
Countries
Netherlands
Contacts
Public ContactHOVON Data Center
HOVON
Outcome results
None listed