Clinical Trials Register

Summary
EudraCT Number:	2007-001425-10
Sponsor's Protocol Code Number:	747-202
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:	2008-07-23
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Spain - AEMPS

A.2

EudraCT number

2007-001425-10

A.3

Full title of the trial

Estudio del INT-747 (6-ECDCA) en combinación con el ácido ursodesoxicólico (URSO®, UDCA) en pacientes con cirrosis biliar primaria

A Study of INT-747 (6-ECDCA) in Combination with Ursodeoxycholic Acid (URSO®, UDCA) in Patients with Primary Biliary Cirrhosis

A.4.1

Sponsor's protocol code number

747-202

A.7

Trial is part of a Paediatric Investigation Plan

Information not present in EudraCT

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Intercept Pharmaceuticals
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
B.4.2	Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation
B.5.2	Functional name of contact point

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	INT-747
D.3.2	Product code	INT-747
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	Information not present in EudraCT
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	3α,7α-dihydroxy-6α-ethyl-5βcholan-24-oic acid
D.3.9.1	CAS number	459789-99-2
D.3.9.2	Current sponsor code	6-ECDCA or INT-747
D.3.9.3	Other descriptive name	6α-ethylchenodeoxycholic acid, 6-ethylchenodeoxycholic acid
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	50
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)	Information not present in EudraCT
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	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	Information not present in EudraCT
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	Information not present in EudraCT
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	Yes
D.3.11.13.1	Other medicinal product type	sintético químico
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	INT-747
D.3.2	Product code	INT-747
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	Information not present in EudraCT
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	3α,7α-dihydroxy-6α-ethyl-5βcholan-24-oic acid
D.3.9.1	CAS number	459789-99-2
D.3.9.2	Current sponsor code	6-ECDCA or INT-747
D.3.9.3	Other descriptive name	6α-ethylchenodeoxycholic acid , 6-ethylchenodeoxycholic acid
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)	Information not present in EudraCT
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	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	Information not present in EudraCT
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	Information not present in EudraCT
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	Yes
D.3.11.13.1	Other medicinal product type	sintético químico
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	INT-747
D.3.2	Product code	INT-747
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	Information not present in EudraCT
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	3α,7α-dihydroxy-6α-ethyl-5βcholan-24-oic acid
D.3.9.1	CAS number	459789-99-2
D.3.9.2	Current sponsor code	6-ECDCA or INT-747
D.3.9.3	Other descriptive name	6α-ethylchenodeoxycholic acid , 6-ethylchenodeoxycholic acid
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)	Information not present in EudraCT
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	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	Information not present in EudraCT
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	Information not present in EudraCT
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	Yes
D.3.11.13.1	Other medicinal product type	sintético químico

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	Capsule, hard
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

cirrosis biliar primaria

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	9.1
E.1.2	Level	PT
E.1.2	Classification code	10004661
E.1.2	Term	Biliary cirrhosis primary

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

En pacientes con cirrosis biliar primaria (PBC), tomando UDCA, para evaluar los efectos de INT-747 en:
- Niveles de fosfatasa alcalina (AP)
- Seguridad

E.2.2

Secondary objectives of the trial

En pacientes con cirrosis biliar primaria (PBC), tomando UDCA, para evaluar los efectos de INT-747 en:
- Lesión hepatocelular y función del hígado
- Síntomas generales de salud y específicos de la enfermedad
- Bioindicadores de inflamación hepática y fibrosis
- Concentration en plasma del INT-747 y sus metabolitos principales conocidos

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Se requiere que los pacientes cumplan con el siguientes criterios para poder ser incluídos en el estudio:
· Hombre o mujer en edades comprendidas entre los 18 y 70 años de edad.
· Una dosis estable de ácido ursodesoxicólico (URSO®, UDCA) por lo menos 6 meses antes de la visita de selección.
· Las pacientes femeninas deberán ser quirúrgicamente estériles, estar en la post menopausia o, en caso de estar en edad fértil, deben estar dispuestas a utilizar un método de contracepción fiable con todas sus parejas sexuales durante la duración del estudio y hasta 14 días después de la última toma de la medicación en estudio. Métodos fiables de contracepción son:
° Dispositivos de tipo barrera, por ej. (a) preservativo –femenino o masculino-, o
(b) diafragma con espermicida
° Hormonales (por ej. La píldora anticonceptiva, implantables); o
° Dispositivos Intrauterinos; o
° Vasectomía (de la pareja)
· Los pacientes masculinos deberán estar dispuestos a usar 1 método de contraceptivo con todas sus parejas sexuales durante la duración del estudio a menos que hayan tenido una vasectomía previa.
· PBC comprobada o posible, demostrada por el paciente, quien presenta por lo menos 2 de los 3 siguientes factores de diagnóstico:
o Historial de incremento en los niveles de AP por lo menos 6 meses antes del Día 0
o Título de AMA positivo (Título>1:40 en inmunofluorescencia or M2 positivo en ELISA) o anticuerpos antinucleares específicos de PBC (punto antinuclear y borde nuclear positivo)
o Biopsia de hígado consistente con PBC
· Valor de selección de AP entre 1.5 and 10 × ULN.
· Dispuesto y capacitado para dar un consentimiento informado por escrito.

E.4

Principal exclusion criteria

Los pacientes con las siguientes características serán excluídos del estudio:
· Administración de los siguientes medicamentos en cualquier momento durante los 3 meses anteriores a la selección del estudio: colchicine, methotrexate, azathioprine, o corticoesteroides sistémicos.
· Valores basales de bilirrubina conjugada (directa) >2 x ULN .
· Valores basales de ALT or AST >5 × ULN
· Valores basales de creatinina en suero >1.5 mg/dL (133 µmol/L)
· Historial o presencia de decompensación hepática (p.ej. sangrados varicosos, encefalopatía o ascitis deficientemente controlada.)
· Historial o presencia de otras enfermedades del hígado concomitantes, incluyendo hepatitis debido a la infección del virus de hepatitis B o C (HCV, HBV) colangitis esclerosante primaria (PSC), enfermedad del hígado alcohólico, enfermedad definitiva hepática autoinmune o biopsia que prueba esteatohepatitis no alcohólica (NASH).
· Historial conocido de infección del virus de inmunodeficiencia humana (HIV) .
· Historial o presencia de cualquier otra enfermedad o condición, la cual se sabe que interfiere con la absorción, distribución, metabolismo o excreción de medicamentos incluyendo el metabolismo de sales biliares en el intestino grueso (por ejemplo, enfermedad inflamatoria del intestino.)
· Otras condiciones médicas clínicamente significantes, incluyendo la insuficiencia renal.
· Otras condiciones médicas que no están bien controladas o para las cuales se anticipe que habrá cambio en las necesidades de medicamentos durante el estudio. Los medicamentos concomitantes deberán ser estables durante 14 días anteriores a la primera dosis de medicamento del estudio, y deberá esperarse que se mantengan estables durante el curso del estudio.
· Historial del abuso de alcohol (definido como consumo de más de 210 mL de alcohol por semana o el equivalente de 14 copas de vino de 4 onzas, o 14 latas o botellas de cerveza o vino mezclado con jugo (wine coolers) de 12 onzas u otro abuso de substancia durante el año anterior.
· Participación en otro estudio de medicamento experimental, biológico o de un dispositivo médico dentro de los 30 días antes del Día 0.
· Un historial de falta de cumplimiento con respecto a regímenes médicos, o pacientes los cuales son considerados como posiblemente no confiables .
· Donación de sangre o plasma durante los 30 días anteriores a la dosificación.
· Inestabilidad o incompetencia mental de tal manera que la validez del consentimiento informado o el cumplimiento con el estudio sean inciertos.
· Embarazo, lactancia o resultado positivo del test de embarazo en suero o en orina

E.5 End points

E.5.1

Primary end point(s)

La valorización final de la eficacia primaria es el efecto de INT-747 sobre los niveles séricos de AP.

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)

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.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

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

Information not present in EudraCT

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

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

Como final del ensayo se considerá la última visita del último paciente.

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects
F.1 Age Range
F.1.1	Trial has subjects under 18	No
F.1.1.1	In Utero	Information not present in EudraCT
F.1.1.2	Preterm newborn infants (up to gestational age < 37 weeks)	Information not present in EudraCT
F.1.1.3	Newborns (0-27 days)	Information not present in EudraCT
F.1.1.4	Infants and toddlers (28 days-23 months)	Information not present in EudraCT
F.1.1.5	Children (2-11years)	Information not present in EudraCT
F.1.1.6	Adolescents (12-17 years)	Information not present in EudraCT
F.1.2	Adults (18-64 years)	Yes
F.1.3	Elderly (>=65 years)	Yes
F.2 Gender
F.2.1	Female	Yes
F.2.2	Male	Yes
F.3 Group of trial subjects
F.3.1	Healthy volunteers	No
F.3.2	Patients	Yes
F.3.3	Specific vulnerable populations	Yes
F.3.3.1	Women of childbearing potential not using contraception	No
F.3.3.2	Women of child-bearing potential using contraception	Yes
F.3.3.3	Pregnant women	No
F.3.3.4	Nursing women	No
F.3.3.5	Emergency situation	No
F.3.3.6	Subjects incapable of giving consent personally	No
F.3.3.7	Others	No
F.4 Planned number of subjects to be included
F.4.1	In the member state	20
F.4.2	For a multinational trial
F.4.2.1	In the EEA	50
F.4.2.2	In the whole clinical trial	140

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

2008-09-24

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2008-09-19

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2010-11-29

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials