Clinical Trials Register

Summary
EudraCT Number:	2013-001263-23
Sponsor's Protocol Code Number:	BP27832
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:	2014-01-09
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

2013-001263-23

A.3

Full title of the trial

A MULTI-CENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 2 STUDY OF THE EFFICACY, SAFETY AND TOLERABILITY OF RO5186582 IN ADULTS AND ADOLESCENTS WITH DOWN SYNDROME (CLEMATIS)

ESTUDIO MULTICÉNTRICO, ALEATORIZADO, DOBLE CIEGO, CONTROLADO CON PLACEBO, FASE 2 PARA EVALUAR LA EFICACIA, SEGURIDAD Y TOLERANCIA DE RO5186582 EN ADULTOS Y ADOLESCENTES CON SÍNDROME DE DOWN (CLEMATIS)

A.3.1

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

Safety Efficacy Study in 12-30y old Down Syndrome subjects

Estudio de eficacia y seguridad en pacientes con sindrome de Down de 12 a 30 años de edad

A.3.2

Name or abbreviated title of the trial where available

CLEMATIS

A.4.1

Sponsor's protocol code number

BP27832

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Roche Farma, S.A. en nombre de 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.5.6	E-mail	global.rochegenentechtrials@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	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	GABAA ?5 negative allosteric modulator
D.3.2	Product code	RO5186582/F07
D.3.4	Pharmaceutical form	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	GABAA ?5 negative allosteric modulator
D.3.9.2	Current sponsor code	RO5186582
D.3.9.3	Other descriptive name	RO5186582
D.3.9.4	EV Substance Code	SUB31464
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.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	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	GABAA ?5 negative allosteric modulator
D.3.2	Product code	RO5186582/F08
D.3.4	Pharmaceutical form	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	GABAA ?5 negative allosteric modulator
D.3.9.2	Current sponsor code	RO5186582
D.3.9.3	Other descriptive name	RO5186582
D.3.9.4	EV Substance Code	SUB31464
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	40
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	GABAA ?5 negative allosteric modulator
D.3.2	Product code	RO5186582/F09
D.3.4	Pharmaceutical form	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	GABAA ?5 negative allosteric modulator
D.3.9.2	Current sponsor code	RO5186582
D.3.9.3	Other descriptive name	RO5186582
D.3.9.4	EV Substance Code	SUB31464
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: 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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	GABAA ?5 negative allosteric modulator
D.3.2	Product code	RO5186582/F11
D.3.4	Pharmaceutical form	Granules
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	GABAA ?5 negative allosteric modulator
D.3.9.2	Current sponsor code	RO5186582
D.3.9.3	Other descriptive name	RO5186582
D.3.9.4	EV Substance Code	SUB31464
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.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	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	GABAA ?5 negative allosteric modulator
D.3.2	Product code	RO5186582/F13
D.3.4	Pharmaceutical form	Granules
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	GABAA ?5 negative allosteric modulator
D.3.9.2	Current sponsor code	RO5186582
D.3.9.3	Other descriptive name	RO5186582
D.3.9.4	EV Substance Code	SUB31464
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	40
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: 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	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	GABAA ?5 negative allosteric modulator
D.3.2	Product code	RO5186582/F15
D.3.4	Pharmaceutical form	Granules
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	GABAA ?5 negative allosteric modulator
D.3.9.2	Current sponsor code	RO5186582
D.3.9.3	Other descriptive name	RO5186582
D.3.9.4	EV Substance Code	SUB31464
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.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Granules
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Down Syndrome

Sindrome de Down

E.1.1.1

Medical condition in easily understood language

Down Syndrome

Sindrome de Down

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	16.1
E.1.2	Level	PT
E.1.2	Classification code	10044688
E.1.2	Term	Trisomy 21
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic 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

The primary objective of this study is as follows:
? To evaluate the efficacy of 26 weeks of treatment with RO5186582 on a composite endpoint derived from clinically meaningful responses in working memory and on the level of independent functioning/adaptive behavior or global improvement as compared to placebo.

Evaluar la eficacia de 26 semanas de tratamiento con RO5186582 sobre un criterio de valoración compuesto derivado de respuestas clínicamente significativas en la memoria de trabajo y el nivel de funcionamiento
independiente/conducta adaptativa o mejora global en comparación con el placebo.

E.2.2

Secondary objectives of the trial

Safety Objective:
? Evaluate the tolerability and safety of 26 weeks treatment of RO5186582 and 4?6 weeks after the end of the treatment period
Efficacy Objectives:
? Evaluate the effect of 26 weeks treatment of RO5186582 compared to placebo on working memory
? Evaluate the effect of 26 weeks treatment of RO5186582 compared to placebo on the level of independent functioning and adaptive behavior
? Assess the effect of 26 weeks treatment of RO5186582 compared to placebo on global clinical condition
? Evaluate the effect of 26 weeks treatment of RO518652 compared to placebo on executive function
? Evaluate the effect of 26 weeks treatment of RO518652 compared to placebo on language function
? Evaluate the effect of 26 weeks treatment of RO518652 compared to placebo on everyday memory function
? Evaluate the effect of 26 weeks treatment of RO518652 compared to placebo on subject?s cognition and health-related quality of life as reported by the parents

Seguridad
· Evaluar la tolerancia y seguridad de 26 semanas de tratamiento con RO5186582 y 4-6 semanas después de finalizar el tratamiento
Eficacia
· Evaluar el efecto de 26 semanas de tratamiento con RO5186582 en comparación con el placebo sobre la memoria de trabajo
· Evaluar el efecto de 26 semanas de tratamiento con RO5186582 en comparación con el placebo sobre el nivel de funcionamiento independiente y conducta adaptativa
· Valorar el efecto de 26 semanas de tratamiento con RO5186582 comparado con el placebo en condiciones clínicas globales

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Individuals aged 12?30 years of age inclusive.

2. Clinical diagnosis of Down syndrome (trisomy 21) confirmed by chromosomal analysis (karyotyping). Subjects may have standard trisomy 21, Robertsonian translocation, isochromosome 21 (so called 21q21q Robertsonian) translocation), Down syndrome with reciprocal translocation or mosaicism.

3. Males, or nonpregnant, nonlactating females. For females of childbearing potential, strict contraceptive prevention is required: continuation of hormonal contraception, or intra uterine device. True abstinence is acceptable provided that participants are under the supervision of a caregiver attesting that participants are not sexually active and the results of pregnancy tests are negative before onset of treatment.

4. Body-mass Index (BMI) 18?42 and 15?30 kg/m2 inclusive for adults and adolescents respectively

5. Ability to complete the Clinical Evaluation of Language Fundamentals (CELF)?preschool 2 word classes task (i.e., ? 7 in the expressive raw score).

1. Individuos de 12-30 años, ambas edades incluidas.
2. Diagnóstico clínico del síndrome de Down (trisomía 21)
confirmado por análisis cromosómico (cariotipado). Los sujetos deben tener una trisomía 21 estándar, translocación
Robertsoniana, isocromosoma 21, síndrome de Down con
translocación recíproca o en mosaico.
3. Hombres o mujeres no embarazadas ni lactantes. En el caso
de mujeres en edad fértil, se requiere una prevención
contraceptiva estricta: contracepción hormonal continua o
mediante dispositivo intrauterino. La abstinencia real es
aceptable siempre y cuando los participantes estén bajo la
supervisión de un cuidador que asegure que los participantes
no son sexualmente activos y el resultado de las pruebas de
embarazo sea negativo antes de iniciar el tratamiento.
4. Índice de Masa Corporal (IMC) 18-42 y 15-30 kg/m2 incluidos para adultos y adolescentes respectivamente
5. Capacidad para completar la versión preescolar de la
Evaluación Clínica de los Fundamentos del Lenguaje (CELF)
utilizando clases de dos palabras (p. ej. ³7 en la nota de
expresión).

E.4

Principal exclusion criteria

1. Subjects with a current Diagnostic and Statistical Manual of Mental Disorders (DSM 5) diagnosis of any primary psychiatric diagnosis (including autism spectrum disorder or major depressive disorder). Diagnoses that are secondary, such as intellectual disability, attention deficit hyperactivity disorder, depression and conduct disorder are allowed as long as they are considered to not interfere with study conduct and the subject is on stable treatment for 3 months preceding enrollment.

2. Subjects with a history of infantile spasms, of West syndrome, Lennox-Gastaut syndrome, Early Infantile Epileptic Encephalopathy or any treatment-refractory epilepsy associated with cognitive or developmental regression, of severe head trauma or CNS infections (e.g. meningitis).

3. Subjects with a known or suspected clinical seizure event of any type within 24 months prior to screening.

1. Sujetos con un diagnóstico principal de algún trastorno psiquiátrico en la actualidad (incluido trastorno de espectro autista o trastorno depresivo mayor) de acuerdo con el Manual Diagnóstico y Estadístico de los Trastornos Mentales (DSM 5). Los diagnósticos secundarios, como discapacidad intelectual, trastorno por déficit de atención con hiperactividad, depresión y trastorno de conducta están permitidos siempre y cuando no se considere que interfieran en el estudio y el sujeto esté bajo tratamiento estable durante 3 meses antes de su inclusión en
el estudio.
2. Sujetos con un historial de espasmos infantiles, síndrome de West, síndrome de Lennox-Gastaut, encefalopatía epiléptica infantil temprana o epilepsia refractaria al tratamiento asociada a regresión cognitiva o de desarrollo, traumatismo craneal o infecciones del sistema nervioso central (p. ej. meningitis).
3. Sujetos con una crisis epiléptica conocida o sospecha de cualquier tipo en los 24 meses previos a la selección.

E.5 End points

E.5.1

Primary end point(s)

?Cognition as assessed by the Repeatable Battery for the Assessment of ?Neuropsychological Status (RBANS) sub-tests
?Clinical global impression as assessed by Clinician Rated Global Improvement (CGI-I) scale

Cognición según las sub-pruebas de la Batería Repetible para la Evaluación del Estado Neuropsicológico (RBANS)
Conducta adaptativa según las puntuaciones estándar de las Escalas de Conducta Adaptativa de Vineland II (VABSII)
Impresión clínica global según la Mejoría Global de la Escala de Impresión Clínica Global (CGI-I)

E.5.1.1

Timepoint(s) of evaluation of this end point

? Base Line (except for CGI-I) 12 weeks and 26 Weeks (see protocol for details)

Linea de base (excepto para CGI-I) de 12 semanas y 26 semanas (ver protocolo para más detalle)

E.5.2

Secondary end point(s)

? Everyday working memory as assessed by the Rivermead Behavioral Memory Test for Children (RBMT-C) sub-tests
? Executive function as assessed by the Behavior Rating Inventory of Executive Function-Preschool (BRIEF-P)
? Daily functional memory as assessed by the Observer Memory Questionnaire-Parent Form (OMQ-PF)
? Language function as assessed by the Clinical Evaluation of Language Fundamentals (CELF-4)
? Health-related quality of life as assessed by the Pediatric Quality of Life (PedsQL) Generic Core Module v4.0
? Cognition as assessed by the PedsQL Cognitive Functioning Scale

-Memoria cotidiana según las sub-pruebas del Test Conductual de Memoria Rivermead para Niños (RBMT-C).
-Función ejecutiva según el Inventario de Evaluación Conductual de la Función Ejecutiva para Preescolares (BRIEF-P).
-Memoria diaria funcional según el Cuestionario de Memoria del Observador - Formulario para Padres (OMQ-PF).
-Función del lenguaje según la Evaluación Clínica de Fundamentos del Lenguaje (CELF-4)
-Calidad de vida relacionada con la salud según el cuestionario de Calidad de Vida Pediátrica (PedsQL), módulo general, versión 4.0
- Cognición según la Escala de Funcionamiento Cognitivo PedsQL

E.5.2.1

Timepoint(s) of evaluation of this end point

? 12 weeks and 26 Weeks

12 semanas y 26 semanas

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

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Tolerability

Tolerabilidad

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

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

Yes

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

Canada

France

Italy

New Zealand

Argentina

Germany

Spain

Mexico

Singapore

United Kingdom

United States

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

Ultima visita del ultimo 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

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)

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)

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

?Parent or legal guardian/representative willing to give written informed consent.
?Subject willing and consenting or assenting to participate.

Padre/madre o guardian/representate legal dispuesto a dar consentimiento informado por escrito.
Paciente dispuesto a consentir o asentir a participar

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

180

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)

The Sponsor does not intend to provide RO5186582 or other study interventions to subjects after conclusion of the study or any earlier subject withdrawal. However, any potential extensions may be evaluated at the end of the study and will be subject to a protocol amendment.

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

2014-04-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2014-02-11

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2016-05-06

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Orphan Designation Number:
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