A Pilot Study of Clofarabine Pre-conditioning Prior to Full or Reduced Intensity Allogeneic Transplantation in the Treatment of High Risk Acute Myeloid Leukaemia and Myelodysplasia.
This is a pilot study. Twenty patients in total will be treated in two cohorts of 10
- Cohort One: Patients suitable for full intensity conditioning will receive Clofarabine
pre-conditioning followed by a Cyclophosphamide and Total Body Irradiation conditioned
allogeneic stem cell transplant.
- Cohort Two: Patients not suitable for a full intensity TBI-based transplant due to age
or co-morbidity will receive Clofarabine pre-conditioning followed by a reduced
intensity allogeneic stem cell transplant using Fludarabine, intravenous Busulphan and
The cohorts will be recruited concurrently. It is anticipated that recruitment of the 20
subjects will be achieved in 18 months to two years.
The study will be conducted at a single centre (Southampton, UK) in the first instance.
This design allows the use of a full intensity, TBI-based transplant conditioning schedule,
for younger patients able to tolerate this approach but also the use of a reduced intensity
transplant conditioning schedule in older or less fit patients who may still benefit from
pre-conditioning with Clofarabine followed by an allogeneic stem cell transplant. This
design, therefore, does not restrict potential recruitment to the study on age or
performance status alone (within the limits set by ability to tolerate intensive
chemotherapy and a transplant procedure).
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Treatment related mortality (TRM)
Treatment related mortality (TRM) measured at day 100 and 1 year post transplant and cause of mortality
day 100 and 1 year post transplant
Deborah S Richardson, MA MB BChir MD FRCP FRCPath
University Hospital Southampton NHS Foundation Trust.
United Kingdom: Medicines and Healthcare Products Regulatory Agency