Clinical Trials Register

Summary
EudraCT Number:	2016-004779-39
Sponsor's Protocol Code Number:	NH19708
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-12-21
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

Spain - AEMPS

A.2

EudraCT number

2016-004779-39

A.3

Full title of the trial

AN OPEN-LABEL, SINGLE-ARM, MULTICENTER STUDY TO ASCERTAIN THE OPTIMAL STARTING DOSE OF MIRCERA® GIVEN SUBCUTANEOUSLY FOR THE MAINTENANCE TREATMENT OF ANEMIA IN PEDIATRIC PATIENTS WITH CHRONIC KIDNEY DISEASE ON DIALYSIS OR NOT YET ON DIALYSIS.

ESTUDIO MULTICÉNTRICO, SIN ENMASCARAMIENTO Y DE UN SOLO GRUPO PARA DETERMINAR LA DOSIS INICIAL ÓPTIMA DE MIRCERA® ADMINISTRADO POR VÍA SUBCUTÁNEA PARA EL TRATAMIENTO DE MANTENIMIENTO DE LA ANEMIA EN PACIENTES PEDIÁTRICOS CON INSUFICIENCIA RENAL CRÓNICA SOMETIDOS O AÚN NO A DIÁLISIS

A.3.1

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

A Study to Ascertain the Optimal Starting Dose of Mircera Given Subcutaneously For the Maintenance Treatment of Anemia in Pediatric Patients with Chronic Kidney Disease on Dialysis or Not yet on Dialysis

Un estudio para determinar la dosis inicial óptima de Mircera administrada por vía subcutánea para el tratamiento de mantenimiento de la anemia en pacientes pediátricos con enfermedad renal crónica en diálisis o que aún no se encuentran en diálisis.

A.4.1

Sponsor's protocol code number

NH19708

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/317/2017

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Roche Farma S.A(Soc unipersonal)que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche LTD.
B.1.3.4	Country	Switzerland
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	F. Hoffmann-La Roche LTD.
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	F. Hoffmann-La Roche LTD.
B.5.2	Functional name of contact point	Trial Information Support Line-TISL
B.5.3	Address:
B.5.3.1	Street Address	Grenzacherstrasse 124
B.5.3.2	Town/ city	Basel
B.5.3.3	Post code	4070
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	34913257300
B.5.5	Fax number	34913248195
B.5.6	E-mail	spain.start_up_unit@roche.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	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	30
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	Yes
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	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
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	Yes
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	Yes
D.2.1.1.1	Trade name	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	75
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	Yes
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: 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	Yes
D.2.1.1.1	Trade name	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
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	Yes
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: 5
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	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
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	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	Yes
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: 6
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	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	150
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	Yes
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: 7
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	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	200
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	Yes
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: 8
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	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	250
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	Yes
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: 9
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	Mircera®
D.2.1.1.2	Name of the Marketing Authorisation holder	Roche Registration Limited
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.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.1	CAS number	677324-53-7
D.3.9.2	Current sponsor code	RO0503821
D.3.9.3	Other descriptive name	METHOXY POLYETHYLENE GLYCOL-EPOETIN BETA
D.3.9.4	EV Substance Code	SUB26425
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	360
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	Yes
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

Chronic renal anemia

INSUFICIENCIA RENAL CRÓNICA

E.1.1.1

Medical condition in easily understood language

Anemia is a deficiency of red cells or of hemoglobin in the blood commonly occurs in people with chronic kidney disease (CKD) leads to the permanent, partial loss of kidney function

La anemia es una deficiencia de glóbrojos o hemoglobina en la sangre se produce comúnmente en personas con enfermedad renal crónica (ERC) conduce a la pérdida parcial permanente de la función renal

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10058132
E.1.2	Term	Renal anemia
E.1.2	System Organ Class	100000004851

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10072870
E.1.2	Term	Chronic anemia
E.1.2	System Organ Class	100000004851

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 ascertain the starting dose of Mircera given subcutaneously in pediatric patients CKD on dialysis or not yet on dialysis when switching from stable SC maintenance treatment with epoetin alfa, epoetin beta, or darbepoetin alfa

Determinar la dosis inicial de Mircera administrado por vía subcutánea en pacientes pediátricos con IRC sometidos o aún no a diálisis que previamente
recibían tratamiento de mantenimiento estable con epoetina alfa, epoetina beta o darbepoetina alfa por vía subcutánea.

E.2.2

Secondary objectives of the trial

• To assess the safety and tolerability of multiple doses of Mircera given subcutaneously in pediatric patients
• To evaluate the pharmacokinetics(PK) and the pharmacodynamics(PD) of Mircera in patients on dialysis or not yet on dialysis who receive the study medication by the Subcutaneous(SC) route of administration

- Evaluar la seguridad y la tolerabilidad de dosis múltiples de Mircera, administrado por vía subcutánea, en pacientes pediátricos
- Evaluar la FC y la FD de Mircera en pacientes sometidos o aún no a diálisis que reciben la medicación del estudio por vía subcutánea

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Written informed consent from parent/legal guardian and willingness of parent/legal guardian to abide by the requirements of the study
- Written informed consent or assent from child where appropriate If required by national legislation, patients < 18 years of age at screening who are legally considered to be adults according to national legislation must consent in their own right
- Pediatric patients 3 months−17 years of age
- CKD with estimated glomerular filtration rate (eGFR) of < 45 mL/min/1.73 m2
- For patients on PD: a weekly Kt/V >= 1.8
- For patients on hemodialysis (HD): adequate HD, urea reduction ratio (URR) > 65% or Kt/V > 1.2 for patients on HD three times per week
- Baseline hemoglobin (Hb) concentration 10.0−12.0 g/dL determined from the mean of two Hb values measured at Visit 1 and Visit 2
- Stable SC maintenance treatment with epoetin alfa, epoetin beta, or darbepoetin alfa with the same dosing interval for at least 6 weeks before the first dose of Mircera
- Stable dose of epoetin alfa, epoetin beta, or darbepoetin alfa treatment with no weekly dose change > 25% (increase or decrease) for at least 4 weeks before the first dose of Mircera
- Adequate iron status
- For post-pubertal female patients of childbearing potential: agreement to remain abstinent or to use acceptable contraceptive methods during the study and for 90 days after the last dose of Mircera

-Consentimiento informado por escrito de un progenitor o tutor legal y voluntad del progenitor o tutor legal de cumplir los requisitos del estudio
-Consentimiento informado por escrito o asentimiento del niño, cuando proceda.
Si la legislación nacional así lo exige, los pacientes menores de 18 años en el momento de la selección que se consideren legalmente adultos conforme a la legislación nacional deberán otorgar el consentimiento por sí mismos.
-Pacientes pediátricos de 3 meses a 17 años de edad
-IRC con una tasa de filtración glomerular estimada (TFGe) < 45 ml/min/1,73 m2
-En pacientes con diálisis peritoneal (DP) : Kt/V semanal ≥ 1,8
-En pacientes con hemodiálisis: HD adecuada, tasa de reducción de la urea (TRU) > 65% o Kt/V > 1,2 para los pacientes con tres sesiones semanales de HD
-Concentración basal de Hb de 10,0 a -12,0 g/dl, calculada a partir de la media de dos valores de Hb determinados en las visitas 1 y 2
-Tratamiento de mantenimiento estable con epoetina alfa, epoetina beta o darbepoetina alfa por vía subcutánea con el mismo intervalo de administración durante al menos 6 semanasantes de la primera dosis de Mircera.
-Tratamiento con epoetina alfa, epoetina beta o darbepoetina alfa en dosis estables sin cambios (aumento o disminución) superiores al 25% durante las 4 semanas previas a laprimera dosis de Mircera
- Niveles de hierro adecuados
-Las pacientes femeninas pospúberes en edad fértil deben comprometerse a practicar abstinencia o utilizar métodos anticonceptivos aceptables durante el estudio y hasta 90 días después de la última dosis de Mircera.

E.4

Principal exclusion criteria

- Overt gastrointestinal bleeding within 8 weeks before screening or during the screening period
- RBC transfusions within 8 weeks before screening or during the screening period
- Hemoglobinopathies
- Hemolytic anemia
- Active malignant disease
- PD subjects with an episode of peritonitis within the past 30 days prior to screening and/or during the screening period
- Uncontrolled or symptomatic inflammatory disease
- Uncontrolled hypertension as assessed by the investigator
- Epileptic seizures within 3 months prior to screening and during the screening period
- Administration of any investigational drug within 4 weeks prior to screening or planned during the study
- Severe hyperparathyroidism or biopsy-proven bone marrow fibrosis
- Known hypersensitivity to recombinant human erythropoietin(EPO), polyethylene glycol, or any constituent of the study drug formulation
- Anti-EPO antibody (AEAB)-mediated pure red cell aplasia (PRCA) or history of AEAB-mediated PRCA or positive AEAB test result in the absence of PRCA
- High likelihood of early withdrawal or interruption of the study
- Planned elective surgery during the entire study period
- Females who are pregnant or breastfeeding or who intend to become pregnant during the study or within 90 days after the last dose of Mircera
- Patients of childbearing potential must have a negative serum pregnancy test result within 21 days prior to initiation of study drug

-Hemorragia intestinal manifiesta en las 8 semanas anteriores a la selección o durante el período de selección.
-Transfusión de eritrocitos en las 8 semanas anteriores a la selección o durante el períodode selección
-Hemoglobinopatías
-Anemia hemolítica
-Neoplasia maligna activa
-Pacientes en diálisis peritoneal con un episodio de peritonitis en los 30 días previos a la selección o durante la misma.
-Enfermedad inflamatoria no controlada o sintomática
-Hipertensión arterial no controlada según lo determinado por el investigador
-Crisis epilépticas en los 3 meses previos a la selección y durante la misma.
-Administración de un fármaco en investigación en las 4 semanas previas a la selección o prevista durante el estudio
-Hiperparatiroidismo severo o mielofibrosis confirmada mediante biopsia
-Hipersensibilidad confirmada a la eritropoyetina (EPO) humana recombinante, al
polietilenglicol o a cualquier constituyente de la formulación del fármaco del estudio
-Aplasia eritrocitaria pura (AEP) mediada por anticuerpos antieritropoyetina (AcAE),antecedentes de AEP mediada por AcAE o resultado positivo para AcAE en ausencia de AEP
-Alta probabilidad de retirada o interrupción prematura del estudio
-Intervención quirúrgica programada prevista para el período de duración del estudio

E.5 End points

E.5.1

Primary end point(s)

Change in Hb concentration (g/dL) between the baseline and the evaluation period for each patient

El criterio de valoración principal será la variación de la concentración de Hb (g/dl) entre los períodos basal y de evaluación de cada paciente

E.5.1.1

Timepoint(s) of evaluation of this end point

Up to Week 45

Hasta la semana 45

E.5.2

Secondary end point(s)

1. Number of patients with an average Hb concentration during the evaluation period within ± 1 g/dL of their baseline Hb or above, within or below the range of 10−12 g/dL
2. Change in Mircera dose over time, including the change between the starting dose and the evaluation period
3. Occurrence and severity of adverse events
4. Change from baseline in targeted vital signs
5. Change from baseline in targeted clinical laboratory test results
6. Hb concentrations
7. Serum concentrations of Mircera

1. Número de pacientes con una concentración promedio de Hb durante el período de evaluación dentro de ± 1 g / dL de su Hb basal o superior, dentro o por debajo del rango de 10-12 g / dL
2. Cambio en la dosis de Mircera a lo largo del tiempo, incluido el cambio entre la dosis inicial y el período de evaluación
3. Ocurrencia y gravedad de los eventos adversos
4. Cambio desde la línea de base en los signos vitales dirigidos
5. Cambio desde la línea de base en resultados de pruebas de laboratorio clínico
6. Concentraciones de Hb
7. Concentraciones séricas de Mircera

E.5.2.1

Timepoint(s) of evaluation of this end point

1-6. Up to Week 45
7. Week 1, Week 3, Week 9, Week 17, Week 19

-6. Hasta la semana 45
7. Semana 1, Semana 3, Semana 9, Semana 17, Semana 19
7. Concentraciones séricas de Mircera

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

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

tolerability and maintenance treatment

tolerabilidad y tratamiento de mantenimiento

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)

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

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.8.3

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

France

Germany

Hungary

Italy

Lithuania

Poland

Spain

United States

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2017-12-21.

Yes

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

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

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)

Currently, the Sponsor does not have any plans to provide the Roche IMP (Mircera) or any other study treatments or interventions to patients who have completed the study. The Roche Global Policy on Continued Access to Investigational Medicinal Product is available at the following website: http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf

Actualmente, el Sponsor no tiene nunguna intención para proporcionar el IMP de Roche (Mircera) ni ningún otro tratamiento o intervención del estudio a los pacientes que hayan completado el estudio. La Política global de Roche sobre el acceso continuo a los medicamentos de investigación está disponible en el siguiente sitio web: http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf

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

2018-02-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-02-16

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-07-19

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Orphan Designation Number:
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