Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients

Status: Recruiting
Location: See all (10) locations...
Study Type: Observational
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
Participation Requirements
Sex: All
Maximum Age: 18
Healthy Volunteers: f
View:

• 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.

Locations
Other Locations
Italy
Policlinico Sant'Orsola Malpighi, Clinica Pediatrica Oncologia Ed Ematologia Pediatrica Lalla Seràgnoli
RECRUITING
Bologna
Ospedali Civili, Presidio Ospedale Dei Bambini, Oncoematologia Pediatrica e TMO
RECRUITING
Brescia
IRCCS Istituto Giannina Gaslini, U.O.S.D. Centro Trapianto di Midollo Osseo
RECRUITING
Genova
Ospedale San Raffaele, U.O. Immunoematologia Pediatrica
RECRUITING
Milan
Fondazione IRCCS San Gerardo dei Tintori - Clinica Pediatrica
RECRUITING
Monza
Azienda Ospedaliera di Padova, Oncoematologia Pediatrica
RECRUITING
Padua
Fondazione IRCCS Policlinico San Matteo, S.C. Ematologia 2 - Oncoematologia Pediatrica
RECRUITING
Pavia
AOU Città della Salute e della Scienza Di Torino, SC Oncoematologia Pediatrica e Centro Trapianti
RECRUITING
Torino
IRCCS Materno Infantile Burlo Garofolo, SC Oncoematologia Pediatrica e SS Trapianto Di Midollo
RECRUITING
Trieste
Ospedale Donna Bambino Azienda Ospedaliera Universitaria Integrata, U.O.C. Oncoematologia Pediatrica
RECRUITING
Verona
Contact Information
Primary
Marco Zecca, MD
m.zecca@smatteo.pv.it
+390382502848
Time Frame
Start Date: 2021-02-24
Estimated Completion Date: 2025-12-31
Participants
Target number of participants: 70
Treatments
Pediatric patients with a indication for HSCT and who will will receive TREO
Pediatric patients (aged 0 to 18 years) affected by malignant or non-malignant disorders and with an indication for HSCT and who will will receive TREO as part of the pre-transplant conditioning regimen, in combination with other chemotherapy agents.
Related Therapeutic Areas
Sponsors
Leads: Fondazione IRCCS Policlinico San Matteo di Pavia

This content was sourced from clinicaltrials.gov

Similar Clinical Trials