Clinical trial NCT03605212: Febuxostat for Tumor Lysis Syndrome Prevention in Hematological Malignancies of Paediatric Patients and Adults
Official title: Open Label, Multi-centre, Parallel Group Study to Compare the Pharmacokinetics (PK), Pharmacodynamics (PD) and Safety of Febuxostat Between Pediatric Patients (≥6<18 Years of Age) and Adults
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Tumor Lysis Syndrome
Keywords: TLS
FLO-02
Hematological Malignancies
Lead sponsor: Menarini Group
Sponsor class: INDUSTRY
First posted: 2018-07-30
Enrollment: 30
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: The purpose of this study is to compare the exposure of febuxostat in pediatric patients (≥6\<18 years of age) and in adults suffering from hematological malignancies at intermediate to high risk of TLS and to compare the effect in terms of serum uric acid levels.

Detailed description: In the FLORET study it is planned to enroll 3 groups of patients in order to receive oral administration (film-coated tablets) of two different dose levels of febuxostat: children (from 6 to less than 12 years of age) will receive two different dose levels respectively; adolescents (from 12 to less than 18 years of age) will receive 80 and 120 mg/day respectively and adults (equal or major than 18 years of age) will receive 120 mg/day. The two dose levels for children and adolescents groups were to be sequentially administered, whereas the groups that will receive the first dose levels will simultaneously start the treatment at the study beginning. The individual treatment duration will be of 7 to 9 days, according to chemotherapy duration, as per Investigator's judgement.

Eligibility criteria: Inclusion Criteria:

male and female children of 6 to less than 12 years of age, adolescents of 12 to less than 18 years of age and adults from 18 years:

* scheduled for first cytotoxic chemotherapy cycle because of hematologic malignancies
* and at intermediate or high risk of TLS
* and with no access to rasburicase

Exclusion Criteria:

* patients with contraindications as per febuxostat summary of product characteristics
* patients with severe renal insufficiency
* patients with severe hepatic insufficiency
* patients with diagnosis of Laboratory TLS (LTLS) or Clinical TLS (CTLS)

Primary outcomes:
measure: Pharmacokinetic (PK) Parameter: Apparent Clearance (CL/F)
timeFrame: 7 days
measure: PK Parameter: Apparent Volume of Distribution (Vd/F)
timeFrame: 7 days
measure: PK Parameter: Absorption Rate Constant (Ka)
timeFrame: 7 days
measure: PK Parameter: Area Under Curve (AUC)
timeFrame: 7 days
measure: PK Parameter: Maximum Plasma Concentration (Cmax)
timeFrame: 7 days
measure: PK Parameter: Tmax
timeFrame: 7 days
Secondary outcomes:
measure: Pharmacodynamic (PD) Parameter: Area Under the Curve of Serum Uric Acid (sUA)
timeFrame: 8 days
measure: Assessment of Laboratory Tumor Lysis Syndrome (LTLS)
timeFrame: 7 days
measure: Assessment of Clinical Tumor Lysis Syndrome (CTLS)
timeFrame: 7 days
measure: Assessment of Treatment Emergent Signs and Symptoms (TESS)
timeFrame: Estimated maximum time frame: 27 days
measure: Assessment of Participants Affected by Treatment Emergent Signs and Symptoms (TESS)
timeFrame: Estimated maximum time frame: 27 days
measure: Performance Status (PS) Evaluation
timeFrame: Estimated maximum time frame: 27 days
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: University Hospital Tsaritsa Yoanna
city: Budapest
country: Hungary
facility: Semmelweis Egyetem (paediatric)
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Központ
city: Aviano
country: Italy
facility: SOC Oncologia Medica A - Centro di Riferimento Oncologico
state: Pordenone
city: Bologna
country: Italy
facility: Policlinico S. Orsola Malpighi
city: Firenze
country: Italy
facility: A.O.U.C. Azienda Ospedaliero - Universitaria Careggi
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Meyer
city: Genova
country: Italy
facility: Istituto G Gaslini Ospedale Pediatrico IRCCS
city: Pisa
country: Italy
facility: Azienda Ospedaliero-Universitaria Pisana
city: Rome
country: Italy
facility: IRCCS Ospedale Pediatrico Bambino Gesù
city: Torino
country: Italy
facility: Ospedale Infantile Regina Margherita
city: Verona
country: Italy
facility: Azienda Ospedaliera Universitaria Integrata di Verona
city: Caceres
country: Spain
facility: Hospital de San Pedro de Alcantara
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Maranon
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Valencia
country: Spain
facility: Hospital Universitari i Politècnic La Fe
Study officials:
affiliation: IRCCS Ospedale Pediatrico Bambino Gesù, Piazza Sant'Onofrio, 4, 00165 Rome, IT
name: Franco Locatelli, Prof, MD
role: STUDY_CHAIR
References: