A multi-centre study investigating the pharmacokinetics of treosulfan in children undergoing stem cell transplantation.

Using blood treosulfan concentrations to reduce toxicity and improve outcome in children undergoing stem cell transplantation.

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ANZCTR

Registry ID

ACTRN12615000038594

Enrollment

Registered

2015-01-19

Start date

2013-10-05

Completion date

2018-10-16

Last updated

2020-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

This study involves measuring the blood levels of the medication, treosulfan, to improve the way we adjust the dose in children. Who is it for? Children and adolescents with either malignant or non malignant (genetic) diseases can join this study if they are receiving the medication, treosulfan, prior to undergoing blood or marrow transplantation. Trial details A series of blood levels will be collected after the treosulfan dose to see how well their body breaks down and gets rid of the medication. This study does not replace other studies, but simply investigates key medication widely used in the treatment of children's cancer. Participants will be studied for several years after the study to monitor their health. This study will define the best way to adjust the dose of the drug, treosulfan, a key medication in blood or marrow transplantation conditioning regimens. Too much causes toxicity and too little risks the disease being more likely to return.

Interventions

This is a multi-centre prospective evaluation of the pharmacokinetics of treosulfan and metabolites after intravenous administration prior to blood or marrow transplantation. The exact dose, dosing schedule and infusion duration of treosulfan will be as specified in the protocol by which the patient is being treated. This is an observational study with no control group. Patients will be followed for up to 5 years post transplant.

Eligibility

Sex/Gender

All

Age

No minimum to 18 Years

Healthy volunteers

Inclusion criteria

1. Children and adolescents with any diagnosis who are scheduled to receive tresosulfan-based conditioning prior to blood or marrow transplantation. 2. Parental informed consent.

Exclusion criteria

Unable to obtain parental informed consent.

Outcome results