NCT02048800

Treosulfan Pharmacokinetics in Children Undergoing Allogeneic HSCT

Evaluation of Treosulfan Pharmacokinetics (PK) in Children Undergoing Allogeneic Haematopoietic Stem Cell Transplantation (HSCT)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 61 (actual)
• Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust · Academic / Other
• Sex: All
• Age: 28 Days – 18 Years
• Healthy volunteers: Not accepted

Summary

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).

Conditions

• Allogeneic Haematopoietic Stem Cell Transplantation

Interventions

Type	Name	Description
DRUG	Treosulfan	Treosulfan will be administered over 3 days prior to HSCT at the following dose: 10 g/m2 (children aged \< 3months) or 12 g/m2 (children aged 3/12 months) or 14 g/m2 (children aged \> 12 months)

Timeline

• Start date: 2014-03-01
• Primary completion: 2017-07-31
• Completion: 2017-07-31
• First posted: 2014-01-29
• Last updated: 2019-09-04

Locations

2 sites across 1 country: United Kingdom