Allogeneic Haematopoietic Stem Cell Transplantation Clinical Trial
Official title:
Evaluation of Treosulfan Pharmacokinetics (PK) in Children Undergoing Allogeneic Haematopoietic Stem Cell Transplantation (HSCT)
Every year around 70 children affected by cancer or life-threatening genetic diseases undergo
haematopoietic cell transplantation (HCT) within the Blood and Marrow Transplant (BMT) unit
at Great Ormond Street Hospital (GOSH).
One of the main goals of the BMT unit over the last decade has been to reduce the morbidity
and mortality related to HCT, and the group has become a world-leader in pioneering less
toxic transplants.
Fixed high doses of chemotherapy drugs are generally used to prepare children for HCT but
several studies have shown a correlation between the concentration of these drugs achieved in
the patient's blood, and the success or failure of the HCT procedure.
Recently a new drug, Treosulfan, has become available for use in patients undergoing HCT, and
GOSH has pioneered its introduction in children undergoing HCT. With promising early results,
Treosulfan has become the pre-HCT drug of choice, however, very little is currently known
about how the drug is metabolised and cleared from the body, particularly in children.
The investigators therefore plan to investigate the pharmacokinetic (PK) profile of
Treosulfan in children undergoing HCT at GOSH and define which parameters affect its
metabolism and clearance, and what blood levels are associated with a favourable outcome
(graft take without toxicity) or a poor result (graft rejection and/or toxicity).
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT00169728 -
Vaccination of Children Following Allogeneic Stem Cell Transplantation
|
Phase 3 | |
| Completed |
NCT00990249 -
Busulfan Plus Clofarabine Followed by Allogeneic Hematopoietic Stem Cell Transplantation
|
Phase 2 |