A Phase II Multicentre Open-label Study on Allogeneic Stem Cell Transplantation From Unrelated, Cord-blood and Family Haploidentical Donors in Patients With Active Acute Leukemia
The experimental treatment consists in the application of a therapeutic strategy of
allogeneic transplantation using either a Marrow Unrelated Donor (MUD) or a Cord Blood (CB)
unit or a family Haploidentical (Haplo) donor as a potential curative procedure in a
population of patients with chemoresistant acute leukemias for increase the overall survival
in this patients.
Therapeutic intervention, namely the conditioning regimen as well as GVHD prophylaxis, are
based on regimens currently in standard use in the context of allogeneic transplantation.
The Data Safety Monitoring Board (DSMB) in collaboration with the Steering Committee (SC)
will make periodic monitoring to ensure the safety of patients enrolled in to the study. In
particular, DSMB will check the periodic safety reports of serious adverse events, the
primary or secondary graft failure and treated related mortality (TRM) data generated by the
Data Management Center. A safety report will be generated every 30 enrolled patients
completed 100 days of follow-up.
The population for analysis in the trial will be the Intention to Treat (ITT) population.
All patients enrolled in the study will be included in the ITT analysis.
This study will explore the feasibility, safety and efficacy of allogeneic stem cell
transplantation from unrelated, cord-blood and haploidentical donor in patients with an
active leukemia. Due to the lack of detailed information from literature and the absence of
alternative curative options in this patient population, criteria for sample size assessment
do not refer to a formal statistical power calculation. Therefore, GITMO will promote
enrollment of all patients with active leukemia eligible to allogeneic SCT in all Italian
centres with the aim to collect outcome variables in ITT in the widest and most
representative cohort of this specific patient population.
The choice of 80 patients transplanted is based on feasibility reasons and the expected
patient population with these characteristics referred to the main Italian Transplant
Centres in two year. GITMO survey data on transplant activity points to an estimated accrual
of 40 patients per year over a 24 months enrolment period. Criteria for defining sample size
do not follow statistical power estimates in order to demonstrate difference between the
alternative donor options.
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall Survival (OS)
The overall survival at 2 years (from time of enrolment) of all patients enrolled in to the study (either transplanted or not). This is the simplest outcome, defined as the probability of survival irrespective of disease state at any point in time. Patients alive at their last follow-up are censored. It is analyzed by the Kaplan-Meier method, Log-Rank Test and parametric or semiparametric survival models.
2 years from time of enrolment
Fabio Ciceri, MD
Ospedale San Raffaele
Italy: Ethics Committee