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Trial registered on ANZCTR

Registration number
Ethics application status
Date submitted
Date registered
Date last updated
Type of registration
Retrospectively registered

Titles & IDs
Public title
A multi-centre study investigating the pharmacokinetics of treosulfan in children undergoing stem cell transplantation.
Scientific title
Using blood treosulfan concentrations to reduce toxicity and improve outcome in children undergoing stem cell transplantation.
Secondary ID [1] 285421 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
immunodeficiency 293258 0
CD40 ligand deficiency 293259 0
mucopolysaccharidosis 293260 0
infantile malignant osteoporosis 293261 0
Condition category
Condition code
Human Genetics and Inherited Disorders 293449 293449 0 0
Other human genetics and inherited disorders
Cancer 293560 293560 0 0
Children's - Other

Study type
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
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.
Intervention code [1] 290339 0
Not applicable
Comparator / control treatment
No control group
Control group

Primary outcome [1] 293266 0
We will establish the pharmacokinetics of treosulfan in a large population of 60 children. Total exposure to treosulfan and metabolites (area-under-the-concentration-versus-time curve, AUC) and other pharmacokinetic parameters will be determined for each individual using the blood concentrations.
Timepoint [1] 293266 0
Measured after a single dose.
The time points for children less than 20 kg are prior to the infusion start, then at 5 min, 30min, 3h, 5h and 7h after the infusion end. The time points for children greater than 20kg are prior to the infusion then at 5 min, 30 min, 1h, 2h, 4h, 6h, 8h and 10 h after the infusion end.
Primary outcome [2] 293267 0
Data about the patient (e.g. age, weight, height, surface area, renal function, liver function, laboratory values, concomitant medications, diagnosis) will be collected to allow an assessment of the factors that contribute to the variability in treosulfan pharmacokinetics.
Timepoint [2] 293267 0
Collected prior to the treosulfan dose, on study enrolment.
Primary outcome [3] 293268 0
We will examine the association between exposure to treosulfan (AUC) and the incidence of Grade 3+ transplant-related toxicities.
Timepoint [3] 293268 0
Toxicities (e.g. renal dysfunction, liver dysfunction, gastrointestinal toxicity, mucositis, haematological toxicity), Graft versus Host Disease) will be followed up to 100 days post transplant.
Secondary outcome [1] 310696 0
Primary outcome 4. We will examine the association between exposure to treosulfan (AUC) and transplant outcome, including survival, relapse and engraftment.
Timepoint [1] 310696 0
Survival, engraftment and disease response will be followed until five years post transplant.

Key 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.
Minimum age
No limit
Maximum age
18 Years
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
Unable to obtain parental informed consent.

Study design
Natural history
Defined population
Statistical methods / analysis

Recruitment status
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 3029 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 3030 0
Sydney Children's Hospital - Randwick
Recruitment hospital [3] 3031 0
Royal Children's Hospital - Herston

Funding & Sponsors
Funding source category [1] 290025 0
Government body
Name [1] 290025 0
Kids Cancer Alliance (Cancer Institute NSW)
Address [1] 290025 0
Australian Technology Park,
Level 9, 8 Central Ave
Eveleigh, NSW, 2015,
Country [1] 290025 0
Primary sponsor type
The Children's Hospital at Westmead
Hawkesbury Rd., Westmead, NSW, 2145
Secondary sponsor category [1] 288776 0
Name [1] 288776 0
Address [1] 288776 0
Country [1] 288776 0

Ethics approval
Ethics application status
Ethics committee name [1] 291734 0
Sydney Children's Hospital Human Research Ethics Committee
Ethics committee address [1] 291734 0
Research and Development Office,
Ethics and Governance,
Locked Bag 4001,
Westmead NSW, 2145.
Ethics committee country [1] 291734 0
Date submitted for ethics approval [1] 291734 0
Approval date [1] 291734 0
Ethics approval number [1] 291734 0

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.

Trial website
Trial related presentations / publications
SR. Koyyalamudi, U Kuzhiumparambil, CE Nath, J A. Byrne, CJ. Fraser, TA O’Brien, JW Earl, PJ Shaw. Development and Validation of High pressure liquid chromatography-UV Method for the Determination of Treosulfan and its Epoxy Metabolites in Human Plasma and its Application in Pharmacokinetic Studies. J. Chromatogr.Sci. 2016 54: 326 – 333.
Public notes

Principal investigator
Name 51790 0
Dr Christa Nath
Address 51790 0
Department of Biochemistry,
The Children's Hospital at Westmead,
Locked Bag 4001,
Westmead, NSW, 2145
Country 51790 0
Phone 51790 0
Fax 51790 0
Email 51790 0
Contact person for public queries
Name 51791 0
Dr Christa Nath
Address 51791 0
Department of Biochemistry,
The Children's Hospital at Westmead,
Locked Bag 4001,
Westmead, NSW, 2145
Country 51791 0
Phone 51791 0
Fax 51791 0
Email 51791 0
Contact person for scientific queries
Name 51792 0
Dr Christa Nath
Address 51792 0
Department of Biochemistry,
The Children's Hospital at Westmead,
Locked Bag 4001,
Westmead, NSW, 2145
Country 51792 0
Phone 51792 0
Fax 51792 0
Email 51792 0

No information has been provided regarding IPD availability
Summary results
No Results