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Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients

Recruitingn/aAges any - 18 YearsAllFondazione IRCCS Policlinico San Matteo di Pavia
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In plain words

What the trial is, and whether it could suit you

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 th

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📋 Eligibility rules (as published)
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.
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What it tests

  • Not listed
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Where

10 sites
Policlinico Sant'Orsola Malpighi, Clinica Pediatrica Oncologia Ed Ematologia Pediatrica "Lalla Seràgnoli"
Bologna, Italy
Ospedali Civili, Presidio Ospedale Dei Bambini, Oncoematologia Pediatrica e TMO
Brescia, Italy
IRCCS Istituto Giannina Gaslini, U.O.S.D. Centro Trapianto di Midollo Osseo
Genova, Italy
Ospedale San Raffaele, U.O. Immunoematologia Pediatrica
Milan, Italy
Fondazione IRCCS San Gerardo dei Tintori - Clinica Pediatrica
Monza, Italy
Azienda Ospedaliera di Padova, Oncoematologia Pediatrica
Padova, Italy
Fondazione IRCCS Policlinico San Matteo, S.C. Ematologia 2 - Oncoematologia Pediatrica
Pavia, Italy
AOU Città della Salute e della Scienza Di Torino, SC Oncoematologia Pediatrica e Centro Trapianti
Torino, Italy

…and 2 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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