RecruitingACTRN12615000038594

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.

Sponsor

The Children's Hospital at Westmead

Enrollment

60 participants

Start Date

Oct 5, 2013

Study Type

Observational

Conditions

mucopolysaccharidosis infantile malignant osteoporosis CD40 ligand deficiency

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.

Eligibility

Sex: Both males and femalesMax Age: 18 Yearss

Plain Language Summary

Simplified for easier understanding

This study is measuring the blood levels of a chemotherapy medication called treosulfan in children receiving bone marrow or stem cell transplants. Treosulfan is given before the transplant to prepare the body to accept the new cells. Getting the dose right is very important — too much can cause serious side effects, and too little may allow the disease to return. By measuring how each child's body processes the drug, researchers can develop better guidelines for personalising doses in children. You may be eligible if: - You are a child or adolescent under 18 years old - You have any diagnosis (cancer or genetic/non-malignant conditions) that requires a blood or marrow transplant - You are scheduled to receive treosulfan as part of your transplant preparation - A parent or guardian can provide informed consent You may NOT be eligible if: - Parental informed consent cannot be obtained Talk to your doctor about whether this trial might be right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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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.

Locations(3)

The Children's Hospital at Westmead - Westmead

NSW,QLD, Australia

Sydney Children's Hospital - Randwick

NSW,QLD, Australia

Royal Children's Hospital - Herston

NSW,QLD, Australia

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ACTRN12615000038594

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