Clinical Trials Register

Summary
EudraCT Number:	2016-001445-61
Sponsor's Protocol Code Number:	FLO-02
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2016-08-17
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2016-001445-61

A.3

Full title of the trial

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.

Studio in aperto, multicentrico, a gruppi paralleli per confrontare la farmacocinetica (PK), la farmacodinamica (PD) e la sicurezza di febuxostat tra pazienti pediatrici (6 ≤ età < 18) e adulti.

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A study to compare the pharmacokinetic (Pharmacokinetic is the effect the body has on the drugs) , pharmacodynamic (pharmacodynamic is the effect that drugs have on the body) and safety of Febuxostat between paediatric patients (≥6<18 years of age) and adults.

Studio per confrontare la farmacocinetica (l’effetto che il corpo ha sui farmaci), la farmacodinamica (l’effetto che i farmaci hanno sul corpo) e la sicurezza di febuxostat tra pazienti pediatrici (6 ≤ età < 18) e adulti.

A.3.2

Name or abbreviated title of the trial where available

FLORET

A.4.1

Sponsor's protocol code number

FLO-02

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/285/2015

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Menarini Ricerche S.p.A.
B.1.3.4	Country	Italy
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Menarini Ricerche S.p.A.
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Menarini Ricerche S.p.A.
B.5.2	Functional name of contact point	Corporate Clin Research Directorate
B.5.3	Address:
B.5.3.1	Street Address	Via Sette Santi 1
B.5.3.2	Town/ city	Florence
B.5.3.3	Post code	50131
B.5.3.4	Country	Italy
B.5.4	Telephone number	+39055 56809900
B.5.5	Fax number	+390555680597
B.5.6	E-mail	ACapriati@menarini-ricerche.it

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Adenuric® 120 mg
D.2.1.1.2	Name of the Marketing Authorisation holder	Menarini International Operations Luxembourg S.A.
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Febuxostat
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	FEBUXOSTAT
D.3.9.1	CAS number	144060-53-7
D.3.9.4	EV Substance Code	SUB25382
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	120
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Adenuric® 80 mg
D.2.1.1.2	Name of the Marketing Authorisation holder	Menarini International Operations Luxembourg S.A.
D.2.1.2	Country which granted the Marketing Authorisation	European Union
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Febuxostat
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	FEBUXOSTAT
D.3.9.1	CAS number	144060-53-7
D.3.9.4	EV Substance Code	SUB25382
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	80
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 3
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Feboxostat 20 mg
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	FEBUXOSTAT
D.3.9.1	CAS number	144060-53-7
D.3.9.4	EV Substance Code	SUB25382
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Hematological malignancies at intermediate to high risk of Tumor Lysis Syndrome ( TLS) prevention.

prevenzione per tumori maligni ematologici a rischio di Sindrome da lisi tumorale da intermedio a elevato

E.1.1.1

Medical condition in easily understood language

Tumore Lysis Syndrome ( TLS) results from the rapid killing of a large quantity of tumor cells which occurs most often after the start of Chemotherapy in patients with haematologic cancer

Sindrome da lisi tumorale derivante dalla rapida lisi di un gran numero di cellule tumorali, osservata molto spesso dopo il trattamento iniziale con chemioterapia in pazienti con tumori ematologici

E.1.1.2

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	19.0
E.1.2	Level	PT
E.1.2	Classification code	10045170
E.1.2	Term	Tumour lysis syndrome
E.1.2	System Organ Class	10027433 - Metabolism and nutrition disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

-To assess the pharmacokinetics (PK) of febuxostat in paediatric patients (≥6<18 years of age) and in adults suffering from hematological malignancies at intermediate to high risk of TLS.
-To compare the febuxostat exposure in pediatric patients (≥6<18 years of age) with the one achieved in adults administered with a dose of 120 mg/QD.

• Valutare la farmacocinetica (PK) di febuxostat nei pazienti pediatrici (6 ≤ età < 18) e negli adulti affetti da tumori ematologici maligni con rischio di TLS da intermedio a elevato.
• Confrontare l'esposizione a febuxostat nei pazienti pediatrici (6 ≤ età < 18) con quella raggiunta negli adulti che hanno ricevuto una dose di 120 mg/QD.

E.2.2

Secondary objectives of the trial

-To compare the pharmacodynamics (PD) of febuxostat between pediatric patients (≥6<18 years of age) and adults.
-To evaluate and compare the PK/PD relationship of febuxostat in pediatric patients (≥6<18 years of age) and adults.
-To evaluate the safety of febuxostat between pediatric patients (≥6<18 years of age) and adults.
-To evaluate the occurrence of Laboratory TLS (LTLS) and Clinical TLS (CTLS) according to Cairo and Bishop Criteria
-To assess the age-appropriate formulation acceptability in children.

• Confrontare la farmacodinamica (PD) di febuxostat tra pazienti pediatrici (6 ≤ età < 18) e adulti.
• Valutare e confrontare la relazione PK/PD di febuxostat nei pazienti pediatrici (6 ≤ età < 18) e negli adulti.
• Valutare la sicurezza di febuxostat tra pazienti pediatrici (6≤ età < 18) e adulti.
• Valutare i casi di TLS di laboratorio (LTLS) e TLS clinica (CTLS) secondo i criteri Cairo-Bishop
• Valutare l'accettabilità della formulazione adeguata all'età nei bambini.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Patients meeting ALL the following criteria will be eligible to enter the study:

1.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:
a.scheduled for first cytotoxic chemotherapy cycle, regardless of the line of treatment, because of hematologic malignancies, and
b.at intermediate or high risk for TLS, and
c.with serum uric acid (sUA) levels < 10 mg/dL at Visit 1 (Day 1), and
d.with no access to rasburicase;

2.Karnofsky performance status (KPS) of 100 to 30 for patients aged 16 years and older; Lansky Play performance status (LPS) of 100 to 30 for patients aged less than 16 years;

3.A female of childbearing potential may be enrolled providing she:
- has a negative pregnancy test during screening period and
- is routinely using a highly effective method of birth control resulting in a failure rate of less than 1% per year when used consistently and correctly (e.g. combined [estrogen and progestogen containing] hormonal contraception associated with inhibition of ovulation [oral, intravaginal, transdermal], progestogen-only containing hormonal contraception associated with inhibition of ovulation [oral, injectable, implantable], intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner or total sexual abstinence) until 6 months from the last study drug administration. ;

4.informed consent before any study-related procedures obtained by patient for adults or by patient’s parents (one or both according to local regulation) or legal guardian for children and adolescents;

5.life expectancy >1 month.

I pazienti che soddisfano TUTTI i seguenti criteri saranno eleggibili per l’ingresso nello studio:
1. bambini/bambine di età compresa tra 6 e meno di 12 anni, adolescenti di età compresa tra 12 e meno di 18 anni e adulti:
a. con primo ciclo di chemioterapia citotossica programmato, a prescindere dalla linea di trattamento, a causa di tumori maligni ematologici, e
b. con rischio di TLS da intermedio a elevato e
c. con livelli di acido urico nel siero (sUA) < 10 mg/dL alla Visita 1 (Giorno 1) e
d. senza accesso a rasburicase;
2. stato di performance di Karnofsky (KPS) da 100 a 30 per i pazienti di età pari o superiore a 16 anni; stato di performance di Lansky (LPS) relativo alla capacità di gioco da 100 a 30 per pazienti di età inferiore a 16 anni;
3. Le pazienti in età fertile potranno essere arruolate a condizione che:
- presentino un test di gravidanza negativo durante il periodo di screening e
- utilizzino di routine un metodo contraccettivo altamente efficace con un tasso di insuccesso inferiore a 1% all'anno se impiegato in modo costante e corretto (ad es. contraccettivo ormonale combinato [contenente estrogeni e progestinici] associato a inibizione dell'ovulazione [orale, intravaginale, transdermico], contraccettivo ormonale a base di soli progestinici associato a inibizione dell'ovulazione [orale, iniettabile, impiantabile], dispositivo intrauterino, sistema intrauterino a rilascio di ormoni, occlusione bilaterale delle tube, partner vasectomizzato o astinenza totale) fino a 6 mesi dopo l'ultima somministrazione di farmaco in studio;
4. consenso informato prima di qualsiasi procedura correlata allo studio ottenuto dal paziente per gli adulti o dai genitori del paziente (uno o entrambi in funzione delle normative locali) o dal tutore legale per i bambini e gli adolescenti;
5. aspettativa di vita > 1 mese.

E.4

Principal exclusion criteria

Patients will not be eligible to participate in the study if they meet ANY of the following exclusion criteria:

1.patients known to be hypersensitive to febuxostat or to any components of the formulation;
2.patients with hereditary problems of galactose intolerance, the lapp lactase deficiency, or glucose-galactase malabsorption;
3.pregnant or breastfeeding women;
4.patients receiving febuxostat or any other urate-lowering therapy (e.g. allopurinol, rasburicase, probenecid) within 30 days prior to Visit 1 (Day 1);
5.patients receiving mercaptopurine and azathioprine within 14 days prior to Visit 1 (Day 1);
6.patients with severe renal insufficiency;
7.patients with severe hepatic insufficiency;
8.patients with diagnosis of Laboratory TLS (LTLS) or Clinical TLS (CTLS) at Visit 1 (Day 1).
9.Patients receiving any other investigational agent within 30 days prior to study Visit 1 (Day 1).

I pazienti non saranno eleggibili alla partecipazione allo studio se soddisfano uno QUALSIASI dei seguenti criteri di esclusione:
1. pazienti con ipersensibilità nota a febuxostat o a qualsiasi componente della formulazione;
2. pazienti con problemi ereditari di intolleranza al galattosio, deficit di Lapp lattasi o malassorbimento di glucosio-galattosio;
3. donne in gravidanza o allattamento;
4. pazienti che ricevono febuxostat o qualsiasi altra terapia per l'abbassamento dell'urato (ad es. allopurinolo, rasburicase, probenecid) nei 30 giorni precedenti la Visita 1 (Giorno 1);
5. pazienti che ricevono mercaptopurina e azatioprina nei 14 giorni precedenti la Visita 1 (Giorno 1);
6. pazienti con grave insufficienza renale;
7. pazienti con grave insufficienza epatica;
8. pazienti con diagnosi di TLS di laboratorio (LTLS) e TLS clinica (CTLS) alla Visita 1 (Giorno 1).
9. Pazienti che ricevono qualsiasi altro agente sperimentale nei 30 giorni precedenti la Visita 1 dello studio (Giorno 1).

E.5 End points

E.5.1

Primary end point(s)

Pharmacokinetic endpoints derived from the study will include:
 Primary PK parameters: CL/F, Vd/F and Ka.
 Derived PK parameters: AUC and Cmax.

Gli endpoint di farmacocinetica ottenuti dallo studio comprenderanno:
 Parametri PK primari: CL/F, Vd/F e Ka.
 Parametri PK derivati: AUC e Cmax.

E.5.1.1

Timepoint(s) of evaluation of this end point

Blood samples will be collected on Visits 2, 3, 4, 5, 6, 7 and 8 (i.e., after the first treatment administration and on each following visit until the Evaluation Visit).
Four additional blood samples are required for each patient on Visit 7 (Day 7) at the selected sampling intervals: 0.5-2 hours, 2-4 hours, 4-6 hours and 6-8 hours post study drug intake.

I campioni di sangue saranno raccolti alle visite 2, 3, 4, 5, 6, 7 e 8 (cioè dopo la prima somministrazione del farmaco e ad ogni visita successiva fino alla visita di Valutazione).
Alla visita 7 (7 ° giorno) quattro campioni di sangue addizionali sono necessari per ogni paziente con la seguente frequenza di campionamento: 0,5-2 ore, 2-4 ore, 4-6
ore e 6-8 ore dopo l'assunzione del farmaco in studio.

E.5.2

Secondary end point(s)

Pharmacodynamic endpoint:
1. Area under the curve of sUA from baseline (Visit 1, Day 1) to the Evaluation Visit (Visit 8, Day 8) (AUC sUA 1-8) based on central laboratory results.

PK/PD endpoint:
2.The PK/PD relationship will be evaluated based on sUA levels and febuxostat exposure.

Clinical endpoints:
3. Incidence of Laboratory TLS (LTLS) from Start of Chemotherapy (Visit 3, Day 3) to the Evaluation Visit (Visit 8, Day 8), based on local laboratory results.
-Incidence and grading of Clinical TLS (CTLS) from Start of Chemotherapy (Visit 3, Day 3) to the Evaluation Visit (Visit 8, Day 8) based on local laboratory results.

Safety endpoints:
4.Incidence, severity (both through Mild/Moderate/Severe scale and NCI-CTCAE grade), seriousness and treatment causality of Treatment-Emergent Signs and Symptoms (TESS).
-Frequency of clinically significant abnormalities in physical examination, safety laboratory tests, vital signs and 12 Lead ECG.
-Change in PS according to Karnofsky/Lansky scales.

5. Acceptability assessment of the age-appropriate formulation only for children.

1. Endpoint di farmacodinamica:
 Area sotto la curva di sUA dalla baseline (Visita 1, Giorno 1) alla Visita di valutazione (Visita 8, Giorno 8) (AUC sUA 1-8) sulla base dei risultati del laboratorio centrale.
2. Endpoint PK/PD:
 La relazione PK/PD sarà valutata sulla base dei livelli di sUA e dell'esposizione a febuxostat.
3. Endopoint clinici:
 Incidenza della TLS di laboratorio (LTLS) dall'Inizio della chemioterapia (Visita 3, Giorno 3) alla Visita di valutazione (Visita 8, Giorno 8), sulla base dei risultati del laboratorio locale.
 Incidenza e classificazione della TLS clinica (CTLS) dall'Inizio della chemioterapia (Visita 3, Giorno 3) alla Visita di valutazione (Visita 8, Giorno 8), sulla base dei risultati del laboratorio locale.
4. Endpoint di sicurezza:
 Incidenza, gravità (sia secondo la scala lieve/moderato/grave sia secondo il grado NCI-CTCAE), serietà e relazione di causalità con il trattamento dei Segni e sintomi emergenti dal trattamento (TESS).
 Frequenza delle anomalie clinicamente significative rilevate mediante esame obiettivo, analisi di laboratorio di sicurezza, funzioni vitali e ECG a 12 derivazioni.
 Variazioni dello stato di performance (PS) secondo le scale di Karnofsky/Lansky.
5. Valutazione dell'accettabilità della formulazione adeguata all'età (solo per i bambini).

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Blood samples for sUA analysis will be performed from Visits 1 to 8 (i.e., before 1st drug intake, on each following visit until the Evaluation Visit).
2. PK/PD timepoints are the same of both primary endpoint and the pharmacodynamic timepoints.
3. From start of treatment (day 1) to the end of study visit 10 (day 14±2)
4. From start of chemotherapy (day 3) to evaluation visit (day 8)
5. From start of treatment (day 1) to visit 7 (day 7 ) or 9 (day 9) based on investigator's judgment.

1. I campioni di sangue per l'analisi sUA saranno prelevati dalla Visita 1 alla 8
(Cioè prima della 1° assunzione del farmaco, e ad ogni successiva visita fino alla Visita di Valutazione).
2. I timepoints PK/PD sono gli stessi dell’endpoint primario e dell’analisi farmacodinamica.
3. dall'inizio del trattamento (giorno 1) alla fine dello studio visita 10 (giorno 14 ± 2)
4. Dall'inizio della chemioterapia (giorno 3) alla visita di valutazione (giorno 8)
5. Dall’inizio del trattamento (giorno 1) alla visita 7 (giorno 7) o 9 (giorno 9) in base al giudizio dello sperimentatore.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

Yes

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

To assess the PK and PD of febuxostat in pediatric patients (≥6<18 years of age).

valutare la PK e PD di febuxostat in pazienti pediatrici (6 ≤ età < 18)

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Bulgaria

Croatia

Czech Republic

Hungary

Italy

Russian Federation

Spain

United Kingdom

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Paediatric population from 6 to less than 18 years of age.

popolazione pediatrica da 6 a meno di 18 anni

F.3.3.7

Others

Information not present in EudraCT

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

100

F.4.2.2

In the whole clinical trial

120

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

After the completetion of the trial participation, a standard TLS prevention treatment could be applied during the following
chemotherapy cycles, as per standard local clinical practice.

Dopo il completamento della partecipazione allo studio, secondo la pratica clinica standard locale, un trattamento standard di prevenzione TLS può essere utilizzato durante i seguenti cicli di chemioterapia.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2016-09-12

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-07-06

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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Orphan Designation Number:
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