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Umbilical cord blood transplantation in high-risk hematological patients using stemregenin-1 expanded hematopoietic stem cells.
A feasibility study focusing on engraftment and hematopoietic recovery.



- candidate number26548
- NTR NumberNTR6229
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR26-jan-2017
- Secondary IDsMEC 2016-689 METC Erasmus MC
- Public TitleUmbilical cord blood transplantation in high-risk hematological patients using stemregenin-1 expanded hematopoietic stem cells.
A feasibility study focusing on engraftment and hematopoietic recovery.
- Scientific TitleUmbilical cord blood transplantation in high-risk hematological patients using stemregenin-1 expanded hematopoietic stem cells.
A feasibility study focusing on engraftment and hematopoietic recovery.
- ACRONYMCORDEX
- hypothesis
- Healt Condition(s) or Problem(s) studiedStem cell transplantation , Stem cells
- Inclusion criteria- Age 18-70 years inclusive
- Diagnosis of poor-risk hematological malignancy 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
- 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
- mec approval receivedyes
- multicenter trialyes
- randomisedno
- group[default]
- TypeSingle arm
- Studytypeintervention
- planned startdate 1-mrt-2017
- planned closingdate15-sep-2022
- Target number of participants10
- InterventionsPatients are treated with a reduced-intensity conditioning regimen, irrespective of patient age, followed by single UCBT, using one SR-1 expanded unit. Post grafting immunosuppression is performed by mycophenolate mofetil (30 days) and cyclosporine A (90 days, taper thereafter)1.
- Primary outcomeFeasibility 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
2. effective hematopoietic (neutrophils > 0.5 x 109/L) engraftment within 30 days upon transplantation
- Secondary outcome Cumulative incidence of engraftment
 Cumulative incidence of graft failure
 Time to neutrophil recovery (> 0.5 x 109/L)
 Time to lymphocyte (T-cells + subsets; B-cells; NK-cells) recovery
 Time to platelet recovery ( > 20 x 109/L)
 Time to red blood cell transfusion independence
 Absolute number of CD3+CD4+, CD3+CD8+, CD19+ 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
 Incidence of CTC grade 3-4 adverse events  Progression free survival (PFS, i.e. time from transplantation until progression/relapse or death from any cause, whichever comes first)
 Overall survival (OS) calculated from transplantation. Patients still alive or lost to follow up are censored at the date they were last known to be alive
- Timepoints At entry: within 30 days before start of treatment
 After 1, 2, 3, 6, 12 and 24 months after transplantation and yearly therafter
- Trial web site
- status[default]
- CONTACT FOR PUBLIC QUERIESProf. Dr. J.J. Cornelissen
- CONTACT for SCIENTIFIC QUERIESProf. Dr. J.J. Cornelissen
- Sponsor/Initiator Erasmus Medical Center
- Funding
(Source(s) of Monetary or Material Support)
Erasmus Medical Center
- Publications
- Brief summaryRationale:
Insufficient hematopoietic recovery following UCBT is considered to be primarily due to the low number of hematopoietic stem cells in UCB grafts. In-vitro stem cell expansion can be achieved by SCF, Flt3L, TPO and Stemregenin-1 (SR-1). Transplantation of double UCBT including one SR-1 expanded unit was recently demonstrated feasible and safe. The present study aims to evaluate feasibility, engraftment and recovery following transplantation of one expanded unit.

Study objectives:
 To study the feasibility of single UCBT with one ex-vivo SR-1 expanded unit
 To assess side effects and TRM after single UCBT with one expanded unit
 To assess engraftment and engraftment kinetics; to evaluate immune reconstitution, acute and chronic GVHD, chimerism, toxicity, progression-free survival and overall survival after single UCBT with one expanded unit.

Intervention:
Patients are treated with a reduced-intensity conditioning regimen, irrespective of patient age, followed by single UCBT, using one SR-1 expanded unit. Post grafting immunosuppression is performed by mycophenolate mofetil (30 days) and cyclosporine A (90 days, taper thereafter)

Duration of treatment:
Patients will be treated with a conditioning regimen during 7 days, followed by transplantation. Subsequent immunosuppression may take up to 180 days.
Patients will be followed until 5 years after registration

Expected duration of accrual: 1 year

Main study endpoint:
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
2. effective hematopoietic (neutrophils > 0.5 x 109/L) engraftment within 30 days upon transplantation

Benefit and nature and extent of the burden and risks associated with participation
Benefits for individual patients may include a faster and better hematopoietic recovery, less opportunistic infections after transplantation and less graft versus host disease as compared to double UCBT
Risks of participation include graft failure and autologous recovery
Planned interim analysis and DSMB
An interim analysis will take place after the first 5 patients have been included and are found to be eligible and will be discussed with the DSMB.
- Main changes (audit trail)
- RECORD26-jan-2017 - 12-apr-2017


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