Monitoring Treosulfan in Children Undergoing Stem Cell Transplant

Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients

This study is testing how Treosulfan works in children getting a stem cell transplant to see if it can help them have better outcomes with fewer side effects.

Quick facts

• Study type: Observational
• Enrollment: 70 (estimated)
• Ages: N/A to 18 Years
• Sex: All
• Sponsor: Fondazione IRCCS Policlinico San Matteo di Pavia (other)
• Drugs / interventions: chemotherapy
• Locations: 10 sites (Bologna, bOLOGNA and 9 other locations)
• Trial ID: NCT06861257 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are children aged 0 to 18 years diagnosed with malignant or non-malignant disorders who require HSCT with Treosulfan as part of their treatment.

Not a fit: Patients with severe cardiovascular, liver, or renal dysfunction, or those with active infectious diseases may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved safety and efficacy of HSCT in pediatric patients.

How similar studies have performed: While there is limited data on Treosulfan in the pediatric population, the increasing use of this drug in HSCT suggests potential for success, although this specific approach may be novel.

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.

Where this trial is running

Bologna, bOLOGNA and 9 other locations

• 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)
• Fondazione IRCCS Policlinico San Matteo, S.C. Ematologia 2 - Oncoematologia Pediatrica — Pavia, Pavia, Italy (RECRUITING)
• AOU Città della Salute e della Scienza Di Torino, SC Oncoematologia Pediatrica e Centro Trapianti — Torino, Torino, Italy (RECRUITING)
• IRCCS Materno Infantile "Burlo Garofolo", SC Oncoematologia Pediatrica e SS Trapianto Di Midollo — Trieste, Trieste, Italy (RECRUITING)
• Ospedale Donna Bambino Azienda Ospedaliera Universitaria Integrata, U.O.C. Oncoematologia Pediatrica — Verona, VR, Italy (RECRUITING)

Study contacts

• Study coordinator: Marco Zecca, MD

Conditions: Pediatric Hematopoietic Stem Cell Transplantation, Malignant Disorders, Non-malignant Disorders