Monitoring Treosulfan in Children Undergoing Stem Cell Transplant

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study focuses on the use of Treosulfan as part of the pre-transplant conditioning regimen for pediatric patients undergoing hematopoietic stem cell transplantation (HSCT). It aims to gather data on the pharmacokinetics of Treosulfan in children, addressing the challenges of reducing toxicity and non-relapse mortality while maintaining treatment efficacy. The study includes children aged 0 to 18 years with various malignant and non-malignant disorders who are indicated for HSCT. By monitoring the drug's effects, the study seeks to optimize treatment protocols for better patient outcomes.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age range 0 - 18 years.
* Life expectancy \> 12 weeks.
* Diagnosis of malignant or non-malignant disorder.
* Pre-HSCT Lansky / Karnofsky score ≥ 40%.
* Indication to allogeneic or autologous HSCT with TREO as part of the pre-transplant conditioning regimen.
* Negativity of pregnancy test for female patients.
* Written informed consent signed by the parents or guardians.

Exclusion Criteria:

* Absence of written informed consent signed by the parents or guardians.
* Current clinically active infectious disease (including positive HIV serology or viral RNA).
* Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \<40%).
* Liver dysfunction (AST/ALT ≥ 3 times institutional upper limit normal value -ULN- or bilirubin \> 3 times ULN).
* Renal dysfunction: serum creatinine \> 1.5 times ULN or calculated creatinine clearance \< 60 ml/min/1.73 m2
* End stage irreversible multi-system organ failure.
* Pregnant or breast feeding female patient.

Where it's running

• Policlinico Sant'Orsola Malpighi, Clinica Pediatrica Oncologia Ed Ematologia Pediatrica "Lalla Seràgnoli"
  Bologna, bOLOGNA, Italy Recruiting
• Ospedali Civili, Presidio Ospedale Dei Bambini, Oncoematologia Pediatrica e TMO
  Brescia, Brescia, Italy Recruiting
• IRCCS Istituto Giannina Gaslini, U.O.S.D. Centro Trapianto di Midollo Osseo
  Genova, Genova, Italy Recruiting
• Ospedale San Raffaele, U.O. Immunoematologia Pediatrica
  Milan, Milano, Italy Recruiting
• Fondazione IRCCS San Gerardo dei Tintori - Clinica Pediatrica
  Monza, Monza-brianza, Italy Recruiting
• Azienda Ospedaliera di Padova, Oncoematologia Pediatrica
  Padova, Padova, Italy Recruiting

Study contacts

• Study coordinator: Marco Zecca, MD
• Email: m.zecca@smatteo.pv.it
• Phone: +390382502848

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.