Patients with high-risk hematological diseases, who need a allogeneic stem cell transplantation but who lack a matched unrelated donor MedDRA version: 19.0 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: Ex vivo-expanded umbilical cord blood-derived hematopoietic stem and progenitor cells
Pharmaceutical Form: Solution for infusion in administration system
INN or Proposed INN: Expanded UC
Sponsors
Erasmus MC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Age 18-70 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 1 (=5/6) matched UCB graft with a nuclear cell count > 2,7 x 107/kg (see paragraph 8.2). - Availability of an back-up autograft, harvested and frozen earlier in the course of treatment, (harvest according to local aphersis policies) - WHO performance status 0-2 - 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 9 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: • Bilirubin and/or transaminases > 2.5 x normal value • Creatinine clearance < 40 ml/min • Cardiac dysfunction as defined by: -Reduced left ventricular function with an ejection fraction < 45% as measured by MUGA scan or echocardiogram (another method for measuring cardiac function is acceptable) - 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 |
|---|---|
| Primary end point(s): Feasibility as defined by, and to be achieved in = 80% of (evaluable) patients: 1. SR-1 mediated expansion, resulting in > 20-fold expansion of CD34+ cells, and ;Timepoint(s) of evaluation of this end point: at preparation Expanded UCB-derived HSPCs ;Main Objective: To study the feasibility of single UCBT using one SR-1 expanded unit;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 single unit UCBT, using with one ex-vivo expanded unit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): effective hematopoietic (neutrophils > 0.5 x 109/L) engraftment within 30 days upon transplantation ;Timepoint(s) of evaluation of this end point: within 30 days upon transplantation | — |
Countries
Netherlands
Contacts
Public Contactdept of Hematology
Erasmus MC
Outcome results
None listed