Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients

Launched by FONDAZIONE IRCCS POLICLINICO SAN MATTEO DI PAVIA · Feb 28, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Treosulfan (TREO) to see how it can help children undergoing hematopoietic stem cell transplantation (HSCT) for various medical conditions, both cancerous and non-cancerous. HSCT is a treatment that involves replacing damaged or diseased bone marrow with healthy stem cells. The researchers want to learn more about how TREO behaves in children's bodies and how it might affect their recovery and potential side effects. By closely monitoring the drug levels in the patients, the goal is to find the right dosage that minimizes risks while still being effective.

To be eligible for this trial, children aged 0 to 18 years with a life expectancy of more than 12 weeks and a diagnosis that requires HSCT can participate, as long as their overall health is stable. Parents or guardians will need to provide written consent for their child to take part. Participants in this study can expect to undergo regular check-ups and monitoring while receiving TREO as part of their treatment, helping researchers understand how to improve care for future patients. It's important to note that children with certain health issues, like severe infections or significant organ dysfunction, will not be able to participate to ensure their safety.

Gender

ALL

Eligibility criteria

• 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.