Clinical Trials Register

Summary
EudraCT Number:	2016-000301-37
Sponsor's Protocol Code Number:	EFC13738
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2018-07-06
Trial 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

Spain - AEMPS

A.2

EudraCT number

2016-000301-37

A.3

Full title of the trial

Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3

Estudio abierto, de dos cohortes (con y sin imiglucerasa) y multicéntrico para evaluar la farmacocinética (FC), seguridad y eficacia de eliglustat en pacientes pediátricos con enfermedad de Gaucher (EG) tipo 1 (EG1) y tipo 3 (EG3)

A.3.1

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

Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3

Seguridad y eficacia de eliglustat (con y sin imiglucerasa) en pacientes pediátricos con enfermedad de Gaucher (EG) tipo 1 (EG1) y tipo 3 (EG3)

A.3.2

Name or abbreviated title of the trial where available

ELIKIDS

A.4.1

Sponsor's protocol code number

EFC13738

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1172-2950

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/191/2015

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Genzyme Corporation
B.1.3.4	Country	United States
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	Genzyme Corporation
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	sanofi-aventis, s.a.
B.5.2	Functional name of contact point	Unidad Estudios Clínicos
B.5.3	Address:
B.5.3.1	Street Address	c/ Josep Pla nº2, 4ª planta
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08019
B.5.3.4	Country	Spain
B.5.4	Telephone number	93 485 94 00
B.5.6	E-mail	ES-unidadestudiosclinicos@sanofi.com

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	Cerdelga
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe B.V.
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/07/514
D.3 Description of the IMP
D.3.1	Product name	Eliglustat
D.3.2	Product code	GZ385660
D.3.4	Pharmaceutical form	Capsule, hard
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	Eliglustat
D.3.9.2	Current sponsor code	GZ385660
D.3.9.3	Other descriptive name	ELIGLUSTAT TARTRATE
D.3.9.4	EV Substance Code	SUB180210
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	84
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	Yes
D.3.11.13.1	Other medicinal product type	Eliglustat tartrate is a synthetic small molecule with 2 chiral centers possessing chemical and chiral stability. The drug substance is the 1R,2R isomer.
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Comparator
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	Cerezyme
D.2.1.1.2	Name of the Marketing Authorisation holder	Genzyme Europe B.V.
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	Imiglucerase
D.3.4	Pharmaceutical form	Powder for concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	IMIGLUCERASE
D.3.9.1	CAS number	154248-97-2
D.3.9.3	Other descriptive name	Cerezyme
D.3.9.4	EV Substance Code	SUB08148MIG
D.3.10	Strength
D.3.10.1	Concentration unit	U unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	400
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	Yes
D.3.11.13.1	Other medicinal product type	Recombinant human derived macrophage-targeted beta-Glucocerebrosidase
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/07/514
D.3 Description of the IMP
D.3.1	Product name	Eliglustat
D.3.2	Product code	GZ385660
D.3.4	Pharmaceutical form	Capsule, hard
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	Eliglustat
D.3.9.2	Current sponsor code	GZ385660
D.3.9.3	Other descriptive name	ELIGLUSTAT TARTRATE
D.3.9.4	EV Substance Code	SUB180210
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	21
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	Yes
D.3.11.13.1	Other medicinal product type	Eliglustat tartrate is a synthetic small molecule with 2 chiral centers possessing chemical and chiral stability. The drug substance is the 1R,2R isomer.
D.IMP: 4
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/07/514
D.3 Description of the IMP
D.3.1	Product name	Eliglustat
D.3.2	Product code	GZ385660
D.3.4	Pharmaceutical form	Capsule, hard
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	Eliglustat
D.3.9.2	Current sponsor code	GZ385660
D.3.9.3	Other descriptive name	ELIGLUSTAT TARTRATE
D.3.9.4	EV Substance Code	SUB180210
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	42
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	Yes
D.3.11.13.1	Other medicinal product type	Eliglustat tartrate is a synthetic small molecule with 2 chiral centers possessing chemical and chiral stability. The drug substance is the 1R,2R isomer.

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

Congenital, hereditary and neonatal diseases

Enfermedades congénitas, hereditarias y neonatales

E.1.1.1

Medical condition in easily understood language

Congenital, hereditary and neonatal diseases

Enfermedades congénitas, hereditarias y neonatales

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10075697
E.1.2	Term	Gaucher's disease type I
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10075699
E.1.2	Term	Gaucher's disease type III
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥6 to <18 years old).

Evaluar la seguridad y FC de eliglustat en pacientes pediátricos (≥6 to <18 años)

E.2.2

Secondary objectives of the trial

Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥6 to <18 years old).

Evaluar la seguridad y FC de eliglustat en pacientes pediátricos (≥6 to <18 años)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

-The patient is 6 to <18 years old at the time of informed consent.
-Male and female patients with a clinical diagnosis of Gaucher disease (GD) type 1 or type 3 with documented deficiency of acid beta-glucosidase activity by enzyme assay and glucocerebrosidase (GBA) genotype.
-Postmenarchal female patients must have a documented negative pregnancy test prior to enrollment and throughout the study. Patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use a medically accepted form of contraception throughout the study.

Cohort 1 (Eliglustat monotherapy):
-Patients must have been receiving an enzyme replacement therapy (ERT) for a minimum of 24 months at a monthly dose equivalent to 30 U/kg to 130 U/kg of Cerezyme® (imiglucerase) with treatment ongoing at the time of enrollment. Patients must be at pre-specified treatment goals, as defined by:
-Hemoglobin level for ages 6 to <12 years: ≥11.0 g/dL; for ages 12 to <18 years: ≥11.0 g/dL for females and ≥12.0 g/dL for males;
-Platelet count ≥100,000/mm3;
-Spleen volume <10.0 multiples of normal (MN);
-Liver volume <1.5 MN;
-Absence of GD related pulmonary disease, and severe bone disease, as defined below for Cohort 2.

Cohort 2 (Eliglustat plus imiglucerase):
-Patients must have been receiving an ERT for a minimum of 36 months at a dose equivalent to at least 60 U/kg of imiglucerase every 2 weeks at the time of enrollment with treatment ongoing at the time of enrollment and the dose stable for at least the 6 months preceding enrollment. Patients must have severe clinical manifestations of GD, as defined by the presence of at least of the
following:
-GD related pulmonary disease such as interstitial lung disease (ILD). The diagnosis of ILD must be confirmed by the presence of reticulonodular densities on chest X-ray.
AND/OR
-Symptomatic bone disease characterized by pathological fracture, osteonecrosis,
osteopenia/osteoporosis, or bone crisis occurring in the 12 months prior to enrollment.
AND/OR
-Persistent thrombocytopenia (<80,000/mm3) related to GD.

- El paciente debe tener de 6 a <18 años en el momento de la firma del consentimiento informado.
- Pacientes de ambos sexos con un diagnóstico clínico de EG1 o EG3 con deficiencia de actividad de la betaglucosidasa ácida documentada mediante un análisis enzimático y el genotipo del GBA.
- Postmenarchal female patients must have a documented negative pregnancy test prior to enrollment and throughout the study. In addition, patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use a medically accepted form of contraception throughout the study (barrier method such as condom or diaphragm + spermicide or non-barrier method such as oral, injected, or implanted hormonal contraceptive with ethinylestradiol and
norethindrone or similar active components).
Para la Cohorte 1, los pacientes deben haber estado recibiendo un TSE (aprobado por al menos una autoridad reguladora) durante un mínimo de 24 meses con una dosis mensual equivalente a de 30 U/kg a 130 U/kg de Cerezyme® (imiglucerasa) con el tratamiento en curso en el momento
de la inclusión y los pacientes deben presentar un OT preespecificado para la EG, definido por:
a) Nivel de hemoglobina para las edades de 6 a <12 años: superior o igual 11,0 g/dl; para las edades de 12 a <18 años: superior i gual 11,0 g/dl en niñas y superior o igual 12,0 g/dl en niños, y
b) Recuento de plaquetas superior o igual 100 000/mm3, y
c) Volumen del bazo <10,0 múltiplos del valor normal (MN) y
d) Volumen del hígado <1,5 MN, y
e) Ausencia de enfermedad pulmonar relacionada con la EG y enfermedad ósea sintomática severa, tal y como se define a continuación para la Cohorte 2.
Para la Cohorte 2, los pacientes deben haber estado recibiendo un TSE
(aprobado por al menos una autoridad reguladora) durante un mínimo de 36 meses con una dosis equivalente a por lo menos 60 U/kg de imiglucerasa cada 2 semanas en el momento de
la inclusión con el tratamiento en curso en el momento de la inclusión y la dosis estable durante al menos los 6 meses anteriores a la inclusión y los pacientes deben presentar manifestaciones clínicas severas de EG, que se define por la presencia de al menos uno de los siguientes:
a) Enfermedad pulmonar relacionada con la EG como EPI. El diagnóstico de EPI debe estar confirmado por la presencia de densidades reticulonodulares en la radiografía de tórax.
Y/O
b) Enfermedad ósea sintomática caracterizada por fractura patológica, osteonecrosis, osteopenia/osteoporosis, o crisis ósea en los 12 meses previos a la inclusión.
Y/O
c) Trombocitopenia persistente (<80 000/mm3) relacionada con la EG.

E.4

Principal exclusion criteria

-Substrate reduction therapy for GD within 6 months prior to enrollment
-Partial or total splenectomy if performed within 2 years prior to enrollment
-The patient is transfusion dependent, a history of esophageal varices or liver infarction, elevated liver enzymes, significant congenital cardiac defect, coronary artery disease or left sided heart failure; clinically significant arrhythmias or conduction defect such as Type 2 second degree or third degree atrioventricular (AV) block, complete bundle branch block, prolonged QTc interval, or sustained ventricular tachycardia (VT).
-The patient has any clinically significant disease other than GD.
-The patient has neurological symptoms other than oculomotor apraxia at study entry.
-The patient has received an investigational product within 30 days prior to enrollment.
-The patient is unable to receive treatment with imiglucerase due to a known hypersensitivity or is unwilling to receive imiglucerase treatment every 2 weeks.
-The patient has a known hereditary galactose intolerance, Lapp lactase deficiency or glucose galactose malabsorption, or is a CYP2D6 ultra-rapid metabolizer or indeterminate metabolizer.

- Terapia de reducción de sustrato para la EG durante los 6 meses anteriores a la inclusión.
- Esplenectomía parcial o total si se ha realizado en los 2 años anteriores a la inclusión.
- El paciente depende de transfusiones. Antecedentes de varices esofágicas o infarto de hígado. Enzimas hepáticas (alanina aminotransferasa [ALT], aspartato aminotransferasa [AST]) o bilirrubina
total actualmente >2 veces el límite superior de la normalidad a menos que el paciente tenga un diagnóstico de síndrome de Gilbert. Defectos cardíacos congénitos, arteriopatía coronaria o insuficiencia cardíaca
izquierda clínicamente significativas; arritmias o defecto de conducción clinicamente significativas como bloqueo auriculoventricular (AV) tipo 2 de segundo o tercer grado, bloqueo de rama completo, intervalo QTc prolongado o taquicardia ventricular (TV) sostenida.
- El paciente tiene alguna enfermedad clínicamente significativa, además de la EG, incluida enfermedad cardiovascular, renal, hepática, gastrointestinal, pulmonar, neurológica, endocrina, metabólica (p. ej., hipopotasemia e hipomagnesemia) o psiquiátrica, un resultado positivo
para el virus de la inmunodeficiencia humana (VIH), hepatitis B o hepatitis C, otras enfermedades (p. ej., obesidad mórbida o retraso severo del crecimiento), o enfermedades concomitantes graves que
puedan descartar la participación en el estudio.
- El paciente tiene otros síntomas neurológicos distintos de la apraxia oculomotora al entrar en el estudio.
- El paciente ha recibido un producto en investigación en los 30 días previos a la inclusión.
- Para la Cohorte 1 y 2, el paciente no puede recibir tratamiento con imiglucerasa debido a una hipersensibilidad conocida o no acepta recibir tratamiento con imiglucerasa cada 2 semanas.
- El paciente tiene una intolerancia hereditaria conocida a la galactosa, deficiencia de lactasa de los lapones o malabsorción de glucosa o galactosa.

E.5 End points

E.5.1

Primary end point(s)

1- Maximum concentration (Cmax) of eliglustat in plasma.
2- Area under the plasma eliglustat concentration-time curve (AUC)
3- Number of adverse events in pediatric patients

1- Concentración máxima (Cmax) de eliglustat en plasma.
2- Área debajo de la curva de concentración-tiempo de eliglustat en plasma (AUC)
3- Número de acontecimientos adversos en pacientes pediátricos

E.5.1.1

Timepoint(s) of evaluation of this end point

1- Maximum concentration (Cmax) of eliglustat in plasma : Weeks 2, 13, 26 and 52
2- Area under the plasma eliglustat concentration-time curve (AUC) : Weeks 2 and 52
3- Number of adverse events in pediatric patients : Up to Week 104

1- Concentración máxima (Cmax) de eliglustat en plasma: semanas 2, 13, 26 y 52
2- Área debajo de la curva de concentración-tiempo de eliglustat en plasma (AUC): semanas 2 y 52
3- Número de acontecimientos adversos en pacientes pediátricos: hasta la semana 104

E.5.2

Secondary end point(s)

1- Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients)
2- Percent change from baseline for platelet count (Cohort 1 patients)
3- Percent change from baseline for liver volume (Cohort 1 patients)
4- Percent change from baseline for spleen volume (Cohort 1 patients)
5- Proportion of patients with improvement in pulmonary disease (Cohort 2 patients)
6- Proportion of patients with improvement in bone disease (Cohort 2 patients)
7- Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients)
8- Health-related quality of life will be measured by the Pediatric Quality of Life InventoryTM (PedsQLTM) questionnaires.

1- Cambio absoluto desde el inicio para la hemoglobina (g / dL) (pacientes de la cohorte 1)
2- Cambio porcentual desde el inicio para el recuento de plaquetas (pacientes de la cohorte 1)
3- Cambio porcentual desde el inicio para el volumen del hígado (pacientes de la cohorte 1)
4- Cambio porcentual desde el inicio del estudio para el volumen del bazo (pacientes de la cohorte 1)
5- Proporción de pacientes con mejoría en la enfermedad pulmonar (pacientes de la cohorte 2)
6- Proporción de pacientes con mejoría en la enfermedad ósea (pacientes de la cohorte 2)
7- Proporción de pacientes con mejoría en la trombocitopenia (pacientes de la cohorte 2)
8- La calidad de vida relacionada con la salud se medirá con los cuestionarios Pediatric Quality of Life InventoryTM (PedsQLTM).

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline and Week 52

Basal hasta la semana 52

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

Yes

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

E.6.10

Pharmacogenetic

Yes

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)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

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

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)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

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

Argentina

Canada

Egypt

France

Italy

Netherlands

Russian Federation

Spain

Sweden

Turkey

United Kingdom

E.8.7

Trial has a data monitoring committee

Yes

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

Última visita del último paciente

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

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:

120

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)

F.1.3

Elderly (>=65 years)

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

Pediatric population

F.3.3.7

Others

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

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)

None

Ninguno

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

2018-06-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-05-30

P. End of Trial
P.	End of Trial Status	Ongoing

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Orphan Designation Number:
Results Status:
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