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Clinical Trials in Italy / NCT06861257
Recruiting Observational

Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients

NCT06861257 · tracked via the Priya Life Science Italy tracker
Sponsor
Fondazione IRCCS Policlinico San Matteo di Pavia
Phase
Observational
Started
2021-02-24
Last updated
2026-04-24

Condition(s) studied

Pediatric Hematopoietic Stem Cell TransplantationMalignant DisordersNon-malignant Disorders

Study summary

One of the major challenges to improve the outcome of hematopoietic stem cell transplantation (HSCT) is the reduction of toxicity and non-relapse mortality caused by the pre-transplant conditioning regimen, while maintaining efficacy. Treosulfan (TREO) (L-treitol-1,4-bis-methanesulfonate) is a busulfan analogue with a distinct site of alkylation that results in a more favourable toxicity profile in comparison with busulfan and total body irradiation. TREO is the prodrug of L-epoxybutane, a water-soluble bifunctional alkylating agent with remarkable myeloablative and immunosuppressive properties. The use of TREO, in combination with other chemotherapy agents, as part of the conditioning regimen for hematopoietic stem cell transplantation (HSCT) in children has progressively increased during the last decade for both malignant and non-malignant disorders. Data on TREO pharmacokinetics in the pediatric population are still scarce. To date, only a few studies, including small numbers of pediatric patients, have investigated the PK profile of TREO. These studies reported high variability of TREO pharmacokinetics, and the relationship between TREO exposure, toxicity and clinical outcome is still unresolved. Therefore, therapeutic drug monitoring with a personalized approach may be an important tool to optimize outcomes in the pediatric population. The aim of the investigators' study is to characterize TREO PK/PD profiles in children undergoing HSCT and to evaluate the relationship between TREO exposure and early toxicity and clinical outcome.

Eligibility

Sex
ALL
Min age
—
Max age
18 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • percentage of patients in therapeutic range after the first dose of TREO during the pre-transplant conditioning regimen — within 24 hours from the first dose
    proposed cumulative therapeutic target AUC 4800 mg x h /L; range 3840 -6000 mg x h /L

Trial sites (10)

FacilityCityRegionStatus
Policlinico Sant'Orsola Malpighi, Clinica Pediatrica Oncologia Ed Ematologia Pediatrica "Lalla Seràgnoli" Bologna bOLOGNA Recruiting
Ospedali Civili, Presidio Ospedale Dei Bambini, Oncoematologia Pediatrica e TMO Brescia Brescia Recruiting
IRCCS Istituto Giannina Gaslini, U.O.S.D. Centro Trapianto di Midollo Osseo Genova Genova Recruiting
Ospedale San Raffaele, U.O. Immunoematologia Pediatrica Milan Milano Recruiting
Fondazione IRCCS San Gerardo dei Tintori - Clinica Pediatrica Monza Monza-brianza Recruiting
Azienda Ospedaliera di Padova, Oncoematologia Pediatrica Padova Padova Recruiting
Fondazione IRCCS Policlinico San Matteo, S.C. Ematologia 2 - Oncoematologia Pediatrica Pavia Pavia Recruiting
AOU Città della Salute e della Scienza Di Torino, SC Oncoematologia Pediatrica e Centro Trapianti Torino Torino Recruiting
IRCCS Materno Infantile "Burlo Garofolo", SC Oncoematologia Pediatrica e SS Trapianto Di Midollo Trieste Trieste Recruiting
Ospedale Donna Bambino Azienda Ospedaliera Universitaria Integrata, U.O.C. Oncoematologia Pediatrica Verona VR Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06861257 on ClinicalTrials.gov ↗ ← All trials in Italy