Clinical Trials Register

Summary
EudraCT Number:	2011-004728-36
Sponsor's Protocol Code Number:	747-301
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:	2012-02-22
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

2011-004728-36

A.3

Full title of the trial

A Phase 3, Double Blind, Placebo Controlled Trial and Long Term Safety
Extension of Obeticholic Acid in Patients with Primary Biliary Cirrhosis

Estudio de fase 3, doble ciego, controlado con placebo y extensión de seguridad a largo plazo con ácido obeticólico en pacientes con cirrosis biliar primaria.

A.3.1

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

A study of Obeticholic Acid versus Placebo in Patients with Primary Biliary Cirrhosis plus long term extension study only with Obeticholic Acid

Un estudio con ácido obeticólico comparado con placebo en pacientes con cirrosis biliar primaria más extensión del estudio a largo plazo sólo con ácido obeticólico

A.4.1

Sponsor's protocol code number

747-301

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	Intercept Pharmaceuticals, Inc.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Intercept Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Accelsiors CRO and Consultancy Services
B.5.2	Functional name of contact point	Clinical Trials Info
B.5.3	Address:
B.5.3.1	Street Address	50 Miskolci Utca
B.5.3.2	Town/ city	Budapest
B.5.3.3	Post code	1147
B.5.3.4	Country	Hungary
B.5.4	Telephone number	3612990091
B.5.5	Fax number	3612990096
B.5.6	E-mail	clinicaltrials@accelsiors.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/753
D.3 Description of the IMP
D.3.1	Product name	OCA (INT-747)
D.3.2	Product code	OCA (INT-747)
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	Obeticholic acid
D.3.9.1	CAS number	459789-99-2
D.3.9.2	Current sponsor code	OCA, 6-ECDCA or INT-747
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	5
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/753
D.3 Description of the IMP
D.3.1	Product name	OCA (INT-747)
D.3.2	Product code	OCA (INT-747)
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	Obeticholic acid
D.3.9.1	CAS number	459789-99-2
D.3.9.2	Current sponsor code	OCA, 6-ECDCA or INT-747
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	10
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/753
D.3 Description of the IMP
D.3.1	Product name	OCA (INT-747)
D.3.2	Product code	OCA (INT-747)
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	Obeticholic acid
D.3.9.1	CAS number	459789-99-2
D.3.9.2	Current sponsor code	OCA, 6-ECDCA or INT-747
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	25
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Primary biliary cirrhosis

Cirrosis biliar primaria

E.1.1.1

Medical condition in easily understood language

Primary biliary cirrhosis (PBC) is a rare, chronic, autoimmune liver disease characterized by both liver and biliary tract lesions that progresses to cirrhosis and other complications.

La cirrosis biliar primaria (CBP) es una enfermedad hepática autoinmune crónica y rara que se caracteriza por lesiones hepáticas y biliares que progresan a cirrosis y otras complicaciones

E.1.1.2

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.1
E.1.2	Level	SOC
E.1.2	Classification code	10019805
E.1.2	Term	Hepatobiliary disorders
E.1.2	System Organ Class	10019805 - Hepatobiliary 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

To assess the effects of OCA in patients with PBC on:
-Serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint
-Safety

Evaluar los efectos de ácido obeticólica (AOC) en pacientes con CBP sobre:
- Fosfatasa alcalina (FA) y biliurrina total séricas, conjuntamente como variable compuesta
- Seguridad

E.2.2

Secondary objectives of the trial

To assess the effects of OCA in patients with PBC on:
-Hepatocellular injury and liver function, including histology (inflammatory,
structural [portal, parenchymal] and fibrotic assessments)
-Disease specific symptoms
-Biomarkers and noninvasive assessments of liver fibrosis
-Bile acids (BA)
-Other exploratory evaluations

Evaluar los efectos de OAC en pacientes con CBP sobre:
-Daño hepatocelular y función hepática, incluyendo la histología (evaluaciones inflamatorias, estructurales [portal, parenquimatosa] y fibróticas).
-Síntomas específicos de la enfermedad.
-Biomarcadores y evaluaciones no invasivas de la fibrosis hepática.
-Ácidos biliares (AB).
-Otras evaluaciones exploratorias

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

Optional Genetics Research Study (A Phase 3, Double Blind, Placebo Controlled Trial and Long Term Safety Extension of Obeticholic Acid in Patients with Primary Biliary Cirrhosis)
To study genes that may be involved in the development of Primary Biliary Cirrhosis (PBC) disease (and related conditions).

Estudio de investigación genética opcional (Estudio de fase 3, doble ciego, controlado con placebo y extensión de seguridad a largo plazo con ácido obeticólico en pacientes con cirrosis biliar primaria) para el estudio de genes que pueden estar involucrados en el desarrollo de la cirrosis biliar primaria y afecciones relacionadas.

E.3

Principal inclusion criteria

1. Definite or probable PBC diagnosis (consistent with AASLD and EASL Practice Guidelines; [Lindor 2009; EASL 2009]), as demonstrated by the presence of ? 2 of the following 3 diagnostic factors:
-History of elevated ALP levels for at least 6 months prior to Day 0
-Positive AMA titer or PBC specific antibodies
-Liver biopsy consistent with PBC
2. At least 1 of the following qualifying biochemistry values:
- ALP?1.67x ULN
-Total bilirubin > ULN but < 2x ULN
3. Age ?18 years
4. Taking UDCA for at least 12 months (stable dose for ? 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ? 3 months) prior to Day 0
5. Contraception: Female patients of childbearing potential must use ?1 effective (?1% failure rate) method of contraception during the trial and until the End of Treatment (EOT) visit.
6. Must provide written informed consent and agree to comply with the trial protocol.

1.Diagnóstico confirmado o probable de CBP (compatible con las directrices de práctica de la AASLD y AEEH; [Lindor 2009; AEEH 2009]), demostrado por la presencia de al menos 2 de los siguientes 3 factores diagnósticos:
-Antecedentes de niveles elevados de FA durante un mínimo de 6 meses antes del día 0.
-Valor positivo de AAM o de anticuerpos específicos de la CBP.
-Biopsia hepática compatible con CBP.
2.Al menos uno de los siguientes valores bioquímicos habilitantes:
-FA ? 1,67x LSN.
-Bilirrubina total > LSN pero < 2x LSN.
3.Edad ? 18 años.
4.Tratamiento con AUDC durante al menos 12 meses (con una dosis estable durante ? al menos 3 meses) antes del día 0, o incapacidad de tolerar el AUDC (ausencia de AUDC durante ? al menos 3 meses) antes del día 0.
5.Anticoncepción: las pacientes en edad fértil deben utilizar como mínimo un método anticonceptivo efectivo (?1 % de tasa de fracaso) durante el ensayo y hasta la consulta visita de fin de tratamientofinal del tratamiento (FDT).
6.Haber otorgadodo su consentimiento informado por escrito y aceptar cumplir el protocolo del ensayo.

E.4

Principal exclusion criteria

Patients will be excluded from the trial if they meet any of the following:
1. History or presence of other concomitant liver diseases including:
-Hepatitis B or C virus (HCV, HBV) infection
-Primary sclerosing cholangitis (PSC)
-Alcoholic liver disease
-Definite autoimmune liver disease or overlap hepatitis
-Nonalcoholic steatohepatitis (NASH)
-Gilbert"s Syndrome (exclusion due to interpretability of bilirubin levels)
2. Presence of clinical complications of PBC or clinically significant hepatic
decompensation, including:
-History of liver transplantation, current placement on a liver transplant list or current MELD score ?15
-Portal hypertention and complications, including: known esophageal varices, poorly controlled or diuretic resistant ascites, history of variceal bleeds or related interventions (e.g., insertion of variceal bands or transjugular intrahepatic portosystemic shunt [TIPS]), hepatic encephalopathy
-Cirrhosis with complications, including history or presence of: spontaneous
-Hepatorenal syndrome (type I or II) or Screening serum creatinine > 2 mg/dL (178 ?mol/L) bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2x ULN
3. Patients with a history of severe pruritus requiring current or prior systemic treatment (e.g., with bile acid sequestrants [BAS] or rifampicin)
4. Administration of the following medications is prohibited as specified below:
-Prohibited 6 months prior to Day 0 and throughout the trial (i.e., to last dose and/or EOT): azathioprine, colchicine, cyclosporine, methotrexate,
mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates;
budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including ?-methyl-dopa, sodium valproic acid, isoniazide, or nitrofurantoin)
-Prohibited 12 months prior to Day 0 and throughout the trial (i.e., to last dose and/or EOT): antibodies or immunotherapy directed against interleukins or other cytokines or chemokines
5. Patients who have previously participated in a clinical trial of OCA will not be allowed to participate.
6. History or presence of clinically concerning cardiac arrhythmias, or prolongation of Screening (pretreatment) QT or QTc interval of > 500 milliseconds (msec).
7. If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating

Se excluirán del estudio aquellos pacientes que cumplan cualquiera de los siguientes criterios:
1.Antecedentes o presencia de otras enfermedades hepáticas concurrentes, entre ellas:
-Infección por el virus de la hepatitis B o C (VHB, VHC).
-Colangitis esclerosante primaria (CEP).
-Hepatitis alcohólica.
-Enfermedad hepática autoinmune confirmada o síndrome de sobreposición
-Esteatohepatitis no alcohólica (ENA).
-Síndrome de Gilbert (exclusión debida a la interpretación de los niveles de bilirrubina).
2.Presencia de complicaciones clínicas de la CBP o descompensación hepática clínicamente significativa, incluyendo:
-Antecedentes de trasplante hepático, inclusión vigente en una lista de trasplante hepático o puntuación actual ?15 en la escala MELD.
-Hipertensión portal y complicaciones portales, incluyendo: varices esofágicas conocidas, ascitis mal controlada o resistente a diuréticos, antecedentes de hemorragias de varices esofágicas o intervenciones relacionadas (p. ej., inserción de bandas varicosas o derivación intrahepática portosistémica transyugular [DIPST]), encefalopatía hepática.
-Cirrosis con complicaciones, incluidos los antecedentes o la presencia de: peritonitis bacteriana espontánea, carcinoma hepatocelular, bilirrubina >2x LSN.
-Síndrome hepatorrenal (tipo I o II) o creatinina sérica en el momento de la selección >2 mg/dl (178 ?mol/l).
3.Pacientes con antecedentes de prurito intenso que requiere o ha requerido tratamiento polisistémico actual o anterior (p. ej., con secuestradores de ácidos biliares [SAB] o rifampicina).
4.La administración de los siguientes medicamentos está prohibida en la medida indicada a continuación:
-Medicación prohibida en los 6 meses anteriores al día 0 y durante todo el ensayo (hasta la última dosis y/o FDT): azatioprina, colchicina, ciclosporina, metotrexato, mofetil micofenolato, pentoxifilina, fenofibrato u otros fibratos, budesonida y otros corticoides sistémicos, fármacos potencialmente hepatotóxicos (entre ellos ?-metil-dopa, valproato sódico, isoniacida o nitrofurantoína).
-Medicación prohibida en los 12 meses anteriores al día 0 y durante todo el ensayo (hasta la última dosis y/o FDT): anticuerpos o inmunoterapia dirigida contra interleucinas u otras citocinas o quimiocinas.
5.No se permitirá la participación de los pacientes que hayan participado anteriormente en un ensayo clínico sobre el AOC.
6.Antecedentes o presencia de arritmias cardíacas de interés clínico, o prolongación del intervalo QT o QTc >500 milisegundos (ms) en el momento de la selección (antes del tratamiento).
7.En las mujeres: embarazo conocido, o prueba de embarazo positiva en orina (confirmada por una prueba de embarazo positiva en suero), o lactancia.

E.5 End points

E.5.1

Primary end point(s)

Primary Endpoint (evaluated as a responder analysis):
ALP < 1.67x ULN and total bilirubin within normal limits (WNL), and
ALP decrease of ? 15% (to exclude clinically insignificant ALP changes)

Variable principal (evaluado como análisis respondedor):
FA<1.67x LSN y bilurribina total dentro de los límites de normalidad (DLN), y
Disminución de FA ? 15% (para excluir cambios en FA clínicamente no significativos)

E.5.1.1

Timepoint(s) of evaluation of this end point

Double blind phase : Screening(? -1 to-8 Wks), Day 0, W2, M3, M6, M9, M12/LTSE D1, End of Treatment (if applicable), follow-up

Long term safety extension: M3, M6, M9, M12, End of Treatment, follow up

Fase doble ciego: selección ((? -1 a -8 semanas), Día 0, Semana 2, Mes 3, Mes 6, Mes 9, Mes 12/Día 1 de extensión a largo plazo, Fin de tratamiento (si aplica), seguimiento (si aplica)

Extensión de seguridad a larga plazo: mes 3, Mes 6, Mes 9, Mes 12 (durante 5 años), Fin de tratamiento, seguimiento

E.5.2

Secondary end point(s)

Analyses regarding secondary endpoints will be specified in the SAP and conducted for the following parameters. It is anticipated that additional disease prognostic algorithms will be published during the course of the trial. Where appropriate, secondary analyses will be conducted using such algorithms. At the blinded data review a determination will be made as to which algorithms will be evaluated statistically, as it is likely that there will be small numbers of patients for some algorithms and formal statistical analysis will not therefore be appropriate.

- ALP response rates of 10%, 20% and 40% change
- Disease Prognostic Risk (criteria in relevant patients):
-ALP ? 3x ULN and AST ? 2x ULN and normal bilirubin
- ALP ? 1.5x ULN and AST ? 1.5x ULN and bilirubin within normal limits
- ALP ? 1.67x ULN and normal bilirubin
- Normal bilirubin and normal albumin
- Clinical laboratory values:
- GGT, ALT, AST, total and conjugated bilirubin
- Albumin, prothrombin time and INR
-Liver biopsy/histology: Inflammatory, structural (portal, parenchymal) and fibrotic assessments
- Disease Specific Symptoms:
- PBC-40
- 5-D Pruritus Questionnaire
- Pruritus VAS
- Biomarkers and non-invasive assessments of liver fibrosis
- Fibrosis biomarkers (ELF)
- TE (at selected trial sites)
- Other analytes: TNF-?, TGF-?, IL-6, CK-18 and lysophosphatidic acid
-Bile acids
- Plasma OCA, other bile acids and conjugate concentrations
- Bile and feces concentrations (at selected trial sites)

Los análisis a realizar en relación a las variables secundarias se especificarán en el plan de análisis estadístico y se realizarán para los parámetros que se describen posteriormente. Se cree que durante el curso del estudio se publicarán nuevos algoritmos de pronóstico de la enfermedad, y donde sea apropiado, se realizarán análisis secundarios utilizando dichos algoritmos. En la revisión ciega de los datos se tomará la decisión de qué algoritmos se evaluarán estadísticamente ya que es probable que haya un pequeño número de pacientes para algunos algoritmos y entonces no estaría indicado un análisis estadístico formal.
-Tasas de respuesta de la FA con un cambio de 10 %, 20 %, 40 % de cambio.
-Riesgo pronóstico de enfermedad (criterios en pacientes relevantes):
-FA ? 3x LSN y ASAT ? 2x LSN y bilirrubina normal
-FA ? 1,5x LSN y ASAT? 1,5x LSN y bilirrubina dentro de los límites normales
-FA ? 1,67x LSN y bilirrubina normal.
-Bilirrubina normal y albúmina normal
-Valores analíticos:
-GT, ALAT, ASAT, bilirrubina total y conjugada.
-Albúmina, tiempo de protrombina y CIN.
-Biopsia/histología hepática: evaluaciones inflamatorias, estructurales (portal, parenquimatosa) y fibróticas
-Síntomas específicos de la enfermedad:
-Cuestionario 5-D
-EAV del prurito
-CBP-40.
-Biomarcadores y evaluaciones no invasivas de fibrosis hepática:
-Biomarcadores de fibrosis (FHM).
-Elastografía transitoria (ET; en los centros del ensayo donde esté disponible).
-Otros parámetros analíticos: FNT-?, FCT-?, IL-6, CC-18 y ácido lisofosfatídico.
-Ácidos biliares
-AOC plasmático, otros ácidos biliares y conjugados.
-Concentraciones de ácidos biliares en la bilis y en las heces (en algunos centros del ensayo).

E.5.2.1

Timepoint(s) of evaluation of this end point

At all study visits.

En todas las visitas del estudio.

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

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

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

Austria

Belgium

Canada

France

Germany

Italy

Netherlands

Poland

Spain

Sweden

Switzerland

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

Last visit of the last subject

Última visita del último sujeto

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

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)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

150

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

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

150

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)

Follow up visit occurring 4 weeks (± 7 days) after receiving the last dose of study medication.

Visita de seguimiento a las 4 semanas ((± 7 días) tras recibir la última dosis de medicación del estudio

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

2012-02-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-02-13

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2018-12-17

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Select Date Range: to
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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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