Clinical Trials Register

Summary
EudraCT Number:	2016-000750-35
Sponsor's Protocol Code Number:	BP39055
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:	2016-06-16
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

2016-000750-35

A.3

Full title of the trial

A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS.

ESTUDIO DE CONTINUIDAD EN DOS PARTES, MULTICÉNTRICO, ALEATORIZADO, CONTROLADO POR PLACEBO Y CON DOBLE ENMASCARAMIENTO PARA INVESTIGAR LA SEGURIDAD, LA TOLERABILIDAD, LA FARMACOCINÉTICA, LA FARMACODINÁMICA Y LA EFICACIA DE RO7034067 EN PACIENTES CON ATROFIA MUSCULAR ESPINAL DE TIPOS 2 Y 3

A.3.1

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

A study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients

Estudio para investigar la seguridad, tolerabilidad, farmacocinetica, farmacodinamia y eficacia de RO7034067 en pacientes con atrofia muscular espinal tipo 2 y 3.

A.4.1

Sponsor's protocol code number

BP39055

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/284/2017

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	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.5.6	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	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	RO7034067
D.3.2	Product code	RO7034067/F12
D.3.4	Pharmaceutical form	Powder for oral solution
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	n.a
D.3.9.1	CAS number	1825352-65-5
D.3.9.2	Current sponsor code	RO7034067/F06
D.3.9.4	EV Substance Code	SUB179686
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	RO7034067
D.3.2	Product code	RO7034067/F13
D.3.4	Pharmaceutical form	Powder for oral solution
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	n.a
D.3.9.1	CAS number	1825352-65-5
D.3.9.2	Current sponsor code	RO7034067/F07
D.3.9.4	EV Substance Code	SUB179686
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	60
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	Powder for oral solution
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	Powder for oral solution
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

Spinal Muscular Atrophy (SMA)

Atrofia Muscular Espinal (AME)

E.1.1.1

Medical condition in easily understood language

Spinal Muscular Atrophy, a genetic progressive neuromuscular disease characterized by profound weakness and muscle atrophy that is the leading genetic cause of death in babies and toddlers.

Attrofia muscular espinal es una enfermedad genética neuromuscular progresiva caracterizada por profunda debilidad y atrofia muscular que es la causa genética principal de muerte en bebes e infantes

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	PT
E.1.2	Classification code	10041582
E.1.2	Term	Spinal muscular atrophy
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

Part 1:
•To evaluate the safety, tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7034067 in patients with Type 2 and Type 3 (ambulant or non-ambulant) SMA, and to select the dose for Part 2 of the study

Part 2:
•To evaluate efficacy of RO7034067 compared to placebo in terms of motor function in Type 2 and non-ambulant Type 3 SMA patients, as assessed by the change from baseline in the total score of the Motor Function Measure (MFM) at 12 months

Parte 1:
•Evaluar la seguridad, la tolerabilidad, la farmacocinética (FC) y la farmacodinámica (FD) de RO7034067 en pacientes con atrofia muscular espinal (AME) de tipos 2 y 3 (con o sin capacidad deambulatoria) y seleccionar la dosis para la parte 2 del estudio.
Parte 2:
•Evaluar la eficacia de RO7034067 en comparación con el placebo respecto de la función motora en pacientes con AME de tipo 2 y de tipo 3 sin capacidad deambulatoria, mediante la evaluación del cambio respecto del valor basal en la puntuación total de la escala de medición de la función motora (MFM) a los 12 meses.

E.2.2

Secondary objectives of the trial

Part 2:To investigate
•the PK/PD relationship of RO7034067 by PK/PD modeling
•the efficacy of 12 month treatment with RO7034067 in terms of motor function as assessed by the Hammersmith Functional Motor Scale Expanded (HFMSE), the revised upper limb module (RULM) and responder analyses of the MFM
•the efficacy of 12 month treatment with RO7034067 in terms of respiratory function as assessed by Sniff nasal inspiratory pressure (SNIP) and, in patients aged 6 years and older,by Maximal Inspiratory Pressure (MIP),Maximal Expiratory Pressure (MEP),Forced vital capacity (FVC),Forced expiratory volume (FEV1) and Peak cough flow (PCF)
•proportion of patients who experience a pre-specified disease related adverse event by Month 12
•The efficacy of 12 month treatment with RO7034067 in terms of global health status as assessed by the Clinical Global Impression of Change (CGI-C) and independence, as measured by the SMA Independence Scale (SMAIS)
•the safety and tolerability of RO7034067

Parte 2: Investigar
•La relación FC/FD de RO7034067 mediante la generación de modelos FC/FD
•La eficacia del tratamiento durante 12 meses con RO7034067 respecto de la función motora mediante la evaluación en función de HFMSE, de la RULM y los análisis de respuesta de la MFM.
•La eficacia del tratamiento durante 12 meses con RO7034067 respecto de la función respiratoria mediante la SNIP y, en pacientes con edad igual o superior a 6 años, de la presión PIM, la presión espiratoria máxima (PEM), la CVF, el volumen espiratorio forzado en 1 segundo (VEF1) y el flujo pico de tos (FPT).
•Proporción de pacientes que sufran un acontecimiento adverso predeterminado relacionado con la enfermedad hasta el mes 12
•Eficacia del tratamiento durante 12 meses con RO7034067 respecto del estado general de salud mediante la evaluación en función de la Escala CGI-C Escala de independencia SMA (SMAIS).
•Investigar la seguridad y la tolerabilidad del tratamiento con RO7034067.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Males and females 2 to 25 years of age inclusive
- For Part 1: Type 2 or 3 SMA ambulant or non-ambulant. For Part 2: Type 2 or 3 SMA non-ambulant.
Non-ambulant is defined as not having the ability to walk unassisted for 10 m or more
- Confirmed diagnosis of 5q-autosomal recessive SMA
- Negative blood pregnancy test at screening and agreement to comply with measures to prevent pregnancy and restrictions on sperm donation

-Varones y mujeres de 2 a 25 años inclusive
-Para la parte 1: AME de tipos 2 o 3 con o sin capacidad deambulatoria.
Para la parte 2: AME de tipo 2 o AME de tipo 3 sin capacidad deambulatoria. La ausencia de capacidad deambulatoria se define como la incapacidad de andar sin ayuda a lo largo de 10 m o más.
-Diagnóstico confirmado de AME con cromosoma 5q autosómico recesivo, lo que abarca:
-Prueba de embarazo en sangre negativa en la selección y compromiso de cumplir las medidas para evitar el embarazo y las restricciones sobre la donación de semen

E.4

Principal exclusion criteria

- Concomitant or previous participation in any investigational drug or device study within 90 days
prior to screening, or 5 half-lives of the drug, whichever is longer
- Concomitant or previous administration of a SMN2 targeting antisense oligonucleotide, SMN2 splicing modifier or gene therapy either in a clinical study or as part of medical care.
- Any history of cell therapy
- Hospitalization for a pulmonary event within the last 2 months or planned at time of screening
- Surgery for scoliosis or hip fixation in the one year preceding screening or planned within the next 18 months
- Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator
- Presence of clinically significant ECG abnormalities before study drug administration from average of triplicate measurement or cardiovascular disease indicating a safety risk for patients as determined by the Investigator
- History of malignancy if not considered cured
- Significant risk for suicidal behavior, in the opinion of the Investigator as assessed by the C-SSRS (>6 years of age)
- Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration
- Any Organic cation transporter (OCT)-2 and multidrug and toxin extrusion (MATE) substrates within 2 weeks before dosing
- Use of the following medications within 90 days prior to randomization: riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, agents anticipated to increase or decrease muscle strength, agents with known or presumed histone deacetylase (HDAC) inhibitory effect, and medications with known phototoxicity liabilities
- Recently initiated treatment (within < 6 months prior to randomization) with oral salbutamol or another 2-adrenergic agonist taken orally
- Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine, is not allowed.
- Clinically significant abnormalities in laboratory test results
- Donation or loss of blood >= 10% of blood volume within three months prior to screening
- Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7034067 or to the constituents of its formulation
- Recent history (less than one year) of ophthalmological diseases
- Patients requiring invasive ventilation or tracheostomy

-Participación previa o concomitante en cualquier estudio con un fármaco o dispositivo experimental en un plazo de 90 días antes de la selección o 5 semividas del fármaco, lo que sea más largo.
-Cualquier antecedente de terapia celular.
-Hospitalización a causa de un acontecimiento pulmonar en los últimos 2 meses o que esté prevista en el momento de la selección.
-Intervención quirúrgica para tratar la escoliosis o de fijación de cadera en el año anterior a la selección o que esté prevista en los 18 meses siguientes.
-Afecciones gastrointestinales, renales, hepáticas, endocrinas o cardiovasculares inestables que el investigador considere significativas desde el punto de vista clínico.
-Presencia de anomalías en el ECG significativas desde el punto de vista clínico antes de la administración del fármaco con respecto al promedio de la medición por triplicado o enfermedad cardiovascular que pongan de manifiesto un riesgo para la seguridad de los pacientes, según determine el investigador.
-Antecedentes de neoplasia maligna que no se considere curada.
-Riesgo significativo de conducta suicida, en opinión del investigador, evaluada mediante el cuestionario C-SSR (>6 años).
-Cualquier enfermedad importante un mes antes de las pruebas de la selección o cualquier enfermedad febril en el plazo de una semana antes de la selección y hasta la administración de la primera dosis.
-Cualquier sustrato de OCT-2 y MATE en un plazo de 2 semanas antes de la administración.
-El uso de los siguientes medicamentos en un plazo de 90 días antes de la aleatorización: riluzol, ácido valproico, hidroxiurea, fenilbutirato de sodio, derivados del butirato, creatina, carnitina, hormona del crecimiento, esteroides anabólicos, probenecid, fármacos que se prevé que aumenten o disminuyan la fuerza muscular, fármacos con efectos conocidos o supuestos de inhibición de la histona deacetilasa (HDAC) y fármacos con efectos fototóxicos conocidos.
-El tratamiento iniciado de forma reciente (en un plazo 6 meses antes de la aleatorización) con salbutamol oral u otro agonista adrenérgico β2 por vía oral
-Cualquier uso previo de cloroquina, hidroxicloroquina, retigabina, vigabatrina o tioridazina no está permitido.
-Anomalías significativas desde el punto de vista clínico en los resultados de pruebas analíticas
-Donación de sangre o pérdida >=10 % del volumen de sangre en un plazo de tres meses antes de la selección.
-Hipersensibilidad conocida o presunta (por ejemplo, reacción anafiláctica) a RO7034067 o a los componentes de su formulación
-Enfermedades o afecciones concomitantes o tratamientos que pudieran interferir con la realización del estudio o que, en opinión del investigador, supongan un riesgo inaceptable para el paciente en este estudio.
-Antecedentes recientes (menos de un año) de enfermedades oftalmológicas
-Pacientes que precisen de ventilación invasiva o de una traqueotomía.

E.5 End points

E.5.1

Primary end point(s)

Part 1
1. Incidence of adverse events and serious adverse events
2. Maximum plasma concentration (Cmax) of RO7034067
3. Area under the curve (AUC) of RO7034067
4. Concentration at the end of a dosing interval (Ctrough) of RO7034067

Part 2
5. Change from baseline in the total MFM 32 score at Month 12

Parte 1
1. Incidencia de acontecimientos adversos y acontecimientos adversos graves
2. Concentración máxima plasmática (Cmax) de RO7034067
3. Area bajo la curva (AUC) de RO7034067
4. Concentración al final de un intervalo de dosificiacion (Ctrough) de RO7034067

Parte 2
5. Cambio desde basal en la puntuación MFM total 32 en el mes 12

E.5.1.1

Timepoint(s) of evaluation of this end point

1. Up to 24 months
2-4. Days 1, 7, 14, 28, 56, 120, 246, 365, 490, 609, and 729
5. Baseline (Day -1) and Month 12

1. Hasta un máximo de 24 meses
2-4 Dias 1,7, 14, 28, 56, 120, 246, 365, 490, 609 y 729
5. Basal (dia -1) y mes 12.

E.5.2

Secondary end point(s)

Part 2
1.SMN2 mRNA in blood
2.SMN protein levels in blood
3.Change from baseline in total score of HFMSE at Month 12
4.Change from baseline in the total score of the revised upper limb module (RULM) at Month 12
5.Proportion of patients who achieve stabilization or improvement (i.e., a change from baseline >= 0) on the total MFM score at Month 12
6.Proportion of patients who achieve an improvement of at least one standard error of measurement (SEM calculated at baseline) on the total MFM score at Month 12.
7.Change from baseline in the MFM domain scores of D1, D2, D3 and the total combined score of (D1 + D2) at Month 12.
8.Change from baseline in the best SNIP (expressed as a percentage of the predicted value) at Month 12.
9.Change from baseline in the best MIP at Month 12.
10.Change from baseline in the best MEP at Month 12
11.Change from baseline in FEV1 in patients aged 6 to 25 years at Month 12
12.Change from baseline in FVC in patients aged 6 to 25 years at Month 12.
13.Change from baseline in the peak cough flow (PCF) in patients aged 6 to 25 years at Month 12.
14.Change from baseline in the Total Score of the caregiver-reported SMA independence Scale (SMAIS) at Month 12.
15.Change from baseline in the Total score of the patient-reported SMA independence scale (SMAIS) at Month 12
16.Proportion of patients rated by clinicians as no change or improved in the Clinical Global Impression of Change (CGI-C) Scale at Month 12 (Part 2 only).
17.Proportion of patients rated by clinicians as improved in the Clinical Global Impression of Change (CGI-C) Scale at Month 12.
18.Proportion of patients who experience at least one disease related adverse event by Month 12
19.Number of disease-related adverse events per patient-year at Month 12
20.PedsQL 4.0 Generic Core scale(Part 1 only)
21.PedsQL 3.0 Neuromuscular module (Part 1 only)

Parte 2
1. SMNA 2 mRNA en sangre
2. Niveles de proteina de SMN en sangre
3. Cambio desde basal en la puntuación local de HFMSE en el mes 12
4.Cambio desde basal en la puntuacion total del RULM en el mes 12
5.Proporcion de pacientes que alcanzan la estabilización o mejora (es decir, cambio desde basal >= 0) en la puntuacion total del MFM en el mes 12.
6.Proporcion de pacientes que alcanzan una mejoria de al menos una medida standart de error (SEM calculada en basal) en la puntuacion total de MFM en el mes 12.
7.Cambio desde basal en los dominios D1, D2 y D3 de la puntuación del MFM y la puntuacion total combinada de (D1+ D2) en el mes 12.
8.Cambio desde basal en el mejor SNIP (eexpresado como porcentaje del valor predictivo) en el mes 12.
9.CCambio desde basal en el mejor MIP en el mes 12.
10.Change from baseline in the best MEP at Month 12
11.Cambio desde basal en el FEV1 en pacientes entre 6 y 25 años en el mes 12.
12.Cambio desde basal en el FVC de pacientes entre 6 y 25 años en el mes 12.
13.Cambio desde basal en el PCF en pacientes entre 6 y 25 años en el mes 12.
14.Cambio desde basal en la puntuacion total del SMAIS del cuidador en el mes 12
15.Cambio desde basal en la puntuacion total del SMAIS del paciente en el mes 12
16.Proporcion de pacientes clasificados por el medico como no cambio o mejora en la escala Clinical Global Impression of Change (CGI-C) en el mes Mes 12 (Solo Parte 2).
17. Proporcion de pacientes clasificados por el medico como mejora en la escala Clinical Global Impression of Change (CGI-C) en el mes 12.
18.Proporcion de pacientes que experimentan al menos una enfermedad relacionada con acontecimiento adverso hasta el mes 12.
19.Proporcion de acontecimientos adversos relacionados con la enfermedad por paciente en el mes 12.
20.Escala PedsQL 4.0 Generic Core (Solo Parte 1)
21.Modulo PedsQL 3.0 Neuromuscular (Solo Parte 1)

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Part 1: Days -1, 1, 7, 14, 28, 120, 246, 365, 729; Part 2: Days -1, 1, 7, 28, 120, 246, 365, 729
2. Part 1: Days -1, 7, 14, 28, 120, 246, 365, 729; Part 2: Days -1, 7, 28, 120, 246, 365, 729
3-17. Baseline (Day-1) and Month 12
18-19. Up to Month 12
20-21. Week 35, Week 62, at early withdrawal

1. Parte 1: Dias -1, 1, 7, 14, 28, 120, 246, 365, 729; Parte 2: Dias -1, -1, 7, 28, 120, 246, 365, 729
2. Parte 1: Dias -1, 7, 14, 28, 120, 246, 365, 729; Parte 2: Dias -1, 7, 28, 120, 246, 365, 729
3-17. Basal (dia -1) y mes 12
18-19. Hasta el mes 12 como máximo
20-21. Semana 35, 62 y retirada prematurura

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

genetic testing

testing genetico

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

Yes

E.8.1.7.1

Other trial design description

2 part design

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

Argentina

Belgium

Brazil

Bulgaria

Canada

China

Croatia

France

Germany

Hungary

Italy

Japan

Mexico

Poland

Romania

Russian Federation

Serbia

Spain

Switzerland

Turkey

Ukraine

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

Since this study includes an OLE phase for all enrolled patients, it will continue until RO7034067 is commercially available in the patient’s country, or as per local regulation, or per the Sponsor’s decision to terminate RO7034067 development. In any case the study will not exceed 4 years (or less as per country specific requirements) after the last patient is enrolled in the study.
The end of this study is defined as the date when the last patient last visit (LPLV) occurs.

Como el ensayo incluye una Fase OLE para todos los pacientes incluidos, continuará hasta que RO7034067 este disponible comercialmente en el país del paciente, o según legislación local o por decisión del promotor de terminar el desarrollo de RO7034067. En cualquier caso, el estudio no excederá 4 años (o menos por requerimientos de cada país) después de que el ultimo paciente sea incluido
El final del estudio se define como la fecha cuando la ultima visita del ultimo paciente (UVUP) ocurra

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

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:

185

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:

123

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

Pediatric patients may provide according to local regulation informed assent
informed consent for study participation will be obtained from parents or legal guardian

Los pacientes pediátricos pueden proporcionar asentimiento informado de acuerdo a su regulación local. El consentimiento informado para la participación en el estudio será obtenida de los padres o tutores legales

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

150

F.4.2.2

In the whole clinical trial

219

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)

A patient will be eligible to receive study drug after completing the study if all of the following conditions are met: The patient has a life-threatening or severe medical condition and requires continued study drug treatment for his or her well-being/There are no appropriate alternative treatments available to the patient/The patient and his/her doctor comply with and satisfy any legal or regulatory requirements that apply to them. See furher details in section 4.4.4 of the protocol.

Un paciente será elegible para recibir fármaco del estudio después de completarlo si todas las condiciones siguientes se cumplen: El paciente tiene una enfermedad severa o amenazante para la vida que requiere tratamiento continuo con fármaco del estudio para su bienestar/No hay alternativas de tratamiento apropiadas disponibles para el paciente/El paciente y su medico cumplen y satisfacen cualquier requerimiento legal o regulatorio que les aplica. Ver detalles en la sección 4.4.4 del protocolo

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

2018-03-02

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	Ongoing

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