Clinical Trials Register

Summary
EudraCT Number:	2017-002338-21
Sponsor's Protocol Code Number:	A4250-005
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:	2018-03-08
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

2017-002338-21

A.3

Full title of the trial

A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1)

Estudio de fase 3 aleatorizado, con doble enmascaramiento, controlado con placebo, para demostrar la eficacia y la seguridad de A4250 en niños con colestasis intrahepática familiar progresiva de tipos 1 y 2 (PEDFIC 1)

A.3.1

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

A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.

Estudio para determinar si A4250 es seguro y puede ser utilizado para tratar a niños con colestasis intrahepática familiar progresiva de tipos 1 y 2.

A.4.1

Sponsor's protocol code number

A4250-005

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/306/2017

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Albireo AB
B.1.3.4	Country	Sweden
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	Albireo AB
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Albireo AB
B.5.2	Functional name of contact point	Clinical Operations Department
B.5.3	Address:
B.5.3.1	Street Address	Arvid Wallgrens backe 20
B.5.3.2	Town/ city	Göteborg
B.5.3.3	Post code	41346
B.5.3.4	Country	Sweden
B.5.4	Telephone number	+34675781547
B.5.5	Fax number	+4631 82 02 23
B.5.6	E-mail	jquintero@vhebron.net

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/3/12/1028
D.3 Description of the IMP
D.3.2	Product code	A4250
D.3.4	Pharmaceutical form	Capsule, hard
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	Not applicable
D.3.9.3	Other descriptive name	A4250 (1200 µg)
D.3.9.4	EV Substance Code	SUB126128
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1200
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/3/12/1028
D.3 Description of the IMP
D.3.2	Product code	A4250
D.3.4	Pharmaceutical form	Capsule, hard
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	Not applicable
D.3.9.3	Other descriptive name	A4250 (600 µg)
D.3.9.4	EV Substance Code	SUB126128
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	600
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/3/12/1028
D.3 Description of the IMP
D.3.2	Product code	A4250
D.3.4	Pharmaceutical form	Capsule, hard
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	Not applicable
D.3.9.3	Other descriptive name	A4250 (400 µg)
D.3.9.4	EV Substance Code	SUB126128
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	400
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/12/1028
D.3 Description of the IMP
D.3.2	Product code	A4250
D.3.4	Pharmaceutical form	Capsule, hard
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	Not applicable
D.3.9.3	Other descriptive name	A4250 (200 µg)
D.3.9.4	EV Substance Code	SUB126128
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	200
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	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	Capsule, hard
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	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

Progressive Familial Intrahepatic Cholestasis Types 1 and 2

Colestasis intrahepática familiar progresiva de tipos 1 y 2.

E.1.1.1

Medical condition in easily understood language

An inherited condition causing reduced bile acid flow and progressive liver disease in children and young people.

Enfermedad hereditaria que provoca una disminución del flujo de ácidos biliares y enfermedad del hígado progresiva en niños y jóvenes.

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10076033
E.1.2	Term	Progressive familial intrahepatic cholestasis
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

To demonstrate the efficacy of repeated daily doses of 40 μg/kg and 120 μg/kg A4250 in children with PFIC Types 1 and 2, as determined by the following:
• The proportion of patients experiencing at least a 70% reduction in fasting s-BA concentration from baseline to end of treatment or a reduction to a specified level, compared to placebo after 24 weeks of treatment
• Change from baseline to Week 24 in pruritus compared to placebo

Demostrar la eficacia de las dosis diarias repetidas de 40 µg/kg/día y 120 µg/kg/día de A4250 en niños con colestasis intrahepática familiar progresiva (CIFP) de los tipos 1 y 2, determinada mediante los siguientes criterios de valoración:
- Proporción de pacientes que presenten como mínimo un 70% de reducción en la concentración de ácido biliar sérico (AB-s) en ayunas desde la situación basal hasta el final del tratamiento o una reducción a un específico nivel en comparación con placebo después de 24 semanas de tratamiento
- Cambio del prurito desde la situación basal hasta la Semana 24 en comparación con placebo

E.2.2

Secondary objectives of the trial

• To evaluate the effect of A4250 on serum ALT concentration
• To evaluate the effect of A4250 on growth
• To evaluate the effect of A4250 on sleep disturbance
• To evaluate the effect of A4250 on the need for surgical treatment (biliary diversion or liver transplantation)
• To assess the safety and tolerability of repeated daily doses of A4250 for 24 weeks

• Evaluar el efecto de A4250 en la concentración de ALT sérica
• Evaluar el efecto de A4250 en el crecimiento
• Evaluar el efecto de A4250 sobre las alteraciones del sueño
• Evaluar el efecto de A4250 sobre la necesidad de tratamiento quirúrgico (derivación biliar o trasplante hepático)
• Evaluar la seguridad y la tolerabilidad de las dosis diarias repetidas de A4250 durante 24 semanas

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. A male or female patient, with clinical diagnosis of PFIC Type 1 or 2, between the ages of ≥6 months and ≤18 years at Visit 1 with a body weight above 5 kg
2. Patient must have genetic confirmation of PFIC-1 or PFIC-2 through identification of biallelic pathogenic variants in either the ATP8B1 or ABCB11 genes
3. Patient must have elevated s-BA concentration prior to randomization
4. Patient must have history of significant pruritus and a caregiver-reported observed scratching in the eDiary in the 2 weeks prior
to randomization
5. Patient and/or legal guardian must sign informed consent (and assent) as appropriate.
Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent in order to remain in the study
6. Patients will be expected to have a consistent caregiver(s) for the duration of the study
7. Caregivers and age-appropriate patients (≥8 years of age) must be willing and able to use an eDiary device as required by the study

1. Pacientes de sexo masculino o femenino con un diagnóstico clínico de CIFP de tipo 1 o 2, con edades comprendidas entre ≥6 meses y ≤18 años en la Visita 1 y un peso corporal superior a 5 kg
2. El paciente deberá tener una confirmación genética de CIFP 1 o 2 mediante la identificación de variantes patogénicas bialélicas de los genes ATP8B1 o ABCB11
3. El paciente deberá presentar una concentración elevada de AB-s antes de la aleatorización
4. El paciente deberá tener antecedentes de prurito significativo y un rascado observado por el cuidador y comunicado por este en el diario electrónico en las 2 semanas previas a la aleatorización
5. El paciente y/o el tutor legal firmarán el consentimiento informado (y el documento de asentimiento) según proceda. Los pacientes que cumplan los 18 años (o la mayoría de edad según el país) durante el estudio deberán firmar un nuevo consentimiento para poder permanecer en el estudio
6. Se espera que los pacientes mantengan el mismo cuidador o cuidadores a lo largo de todo el estudio
7. Los cuidadores, así como los pacientes de edad apropiada (≥8 años) deberán estar dispuestos a utilizar un diario electrónico y ser capaces de usarlo de acuerdo con los requisitos del estudio

E.4

Principal exclusion criteria

1. Patient with pathologic variations of the ABCB11 gene that predict complete absence of the BSEP function
2. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
a) Biliary atresia of any kind
b) Benign recurrent intrahepatic cholestasis
c) Suspected or proven liver cancer or metastasis to the liver on imaging studies
d) Histopathology on liver biopsy is suggestive of alternate non-PFIC related etiology of cholestasis
3. Patient with past medical history of ongoing chronic diarrhea
4. Any patient with suspected or confirmed cancers except for basal cell carcinoma
5. Patient with a past medical history of chronic kidney disease with an impaired renal function and a glomerular filtration rate <70 mL/min/1.73 m2
6. Patient with surgical history of disruption of the enterohepatic circulation excluding those who have undergone a successful reversal procedure that has permanently restored flow of bile acids from the liver to the duodenum
7. Patient has had a liver transplant or listed for liver transplant
8. Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
9. Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC.
10. Patient who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment

1. Pacientes con variantes patológicas del gen ABCB11 que predicen la ausencia completa de la función de la bomba exportadora de sales biliares
2. Pacientes con antecedentes médicos o presencia actual de otros tipos de enfermedades hepáticas, incluyendo, entre otras:
a) Atresia biliar de cualquier tipo
b) Colestasis intrahepática benigna recurrente
c) Sospecha o existencia probada de cáncer hepático, o evidencia de metástasis hepática en los estudios de imagen
d) Biopsia hepática con una histopatología compatible con una etiología alternativa de colestasis no relacionada con la CIFP
3. Pacientes con antecedentes médicos de diarrea crónica persistente
4. Cualquier paciente con sospecha o existencia probada de cáncer, a excepción del carcinoma de células basales
5. Pacientes con antecedentes médicos de enfermedad renal crónica asociada con insuficiencia renal y una tasa de filtración glomerular < 70/ml/1,73 m2
6. Pacientes con antecedentes médicos de interrupción de la circulación enterohepática, excluyendo a aquellos que se hayan sometido en el pasado a un procedimiento de reversión exitoso que haya restablecido de forma permanente el flujo de ácidos biliares desde el hígado hasta el duodeno
7. Pacientes que hayan recibido un trasplante hepático o que estén en lista de espera para recibir un trasplante hepático
8. Enfermedad hepática descompensada, coagulopatía, antecedentes o presencia de ascitis clínicamente significativa, hemorragia por varices y/o encefalopatía
9. Pacientes con un trastorno pruriginoso resistente y descontrolado distinto de la CIFP
10. Pacientes tratados previamente con un inhibidor del transportador de ácido biliar ileal cuyo prurito no haya respondido al tratamiento

E.5 End points

E.5.1

Primary end point(s)

The proportion of patients experiencing at least a 70% reduction in fasting s-BA concentration from baseline to end of treatment or a reduction to a specified level, compared to placebo after 24 weeks of treatment

Proporción de pacientes que presentan una reducción de al menos el 70 % en la concentración de AB-s en ayunas desde la situación basal hasta el final del tratamiento, o bien una disminución hasta un nivel especificado, en comparación con el placebo, tras 24 semanas de tratamiento

E.5.1.1

Timepoint(s) of evaluation of this end point

Randomization (baseline), Week 24, follow-up visit

Aleatorización (situación basal), semana 24, visita de seguimiento

E.5.2

Secondary end point(s)

Secondary efficacy endpoints include the following:
EU and RoW: Change in pruritus, as indexed by caregiver-reported observed scratching, from baseline to Week 24 as measured by the worst scratch of the Albireo ObsRO instrument.
All Regions
All secondary endpoints are compared to placebo:
•Change from baseline to Week 24 in fasting s-BA
•Change from baseline to Week 24 in serum ALT concentration
•Change in growth from baseline to Week 24
•Proportion of patients achieving meaningful reduction in caregiver-reported observed scratching at Week 24
•Change from baseline to Week 24 in sleep parameters measured with the Albireo PRO and ObsRO instruments
•Change from baseline to Week 24 in patient-reported itch severity as measured by the average daily score of the Albireo PRO instrument
•Number of patients undergoing biliary diversion surgery or being listed for liver transplantation

Los criterios secundarios de valoración de la eficacia son:
U.E. y RdM: cambio en el prurito, según el nivel de rascado observado notificado por el cuidador desde la situación basal hasta la Semana 24, en base al peor rascado medido mediante el instrumento ObsRO de Albireo.
Todas las regiones:
Todos los criterios secundarios de valoración se compararán con el placebo:
• Cambio en el nivel de AB-s en ayunas desde la situación basal hasta la Semana 24
• Cambio en la concentración de ALT sérica desde la situación basal hasta la Semana 24
• Cambio en el crecimiento desde la situación basal hasta la Semana 24
• Proporción de pacientes que consiguen una reducción significativa en el nivel de rascado observado notificado por el cuidador en la Semana 24
• Cambio en los parámetros de sueño medidos mediante los instrumentos PRO y ObsRO de Albireo desde la situación basal hasta la Semana 24
• Cambio en la intensidad del picor notificada por el paciente desde la situación basal hasta la Semana 24, determinado por la puntuación diaria promedio del instrumento PRO de Albireo
• Número de pacientes sometidos a cirugía de derivación biliar o en lista de espera para recibir un trasplante hepático

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 24

Semana 24

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

Yes

E.6.13.1

Other scope of the trial description

Quality of Life Assessment (using PedsQL);

Evaluación de calidad de vida (usando PedsQL);

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

Australia

Belgium

Canada

France

Germany

Israel

Italy

Netherlands

Poland

Saudi Arabia

Spain

Sweden

Turkey

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

LSLV

UVUP (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

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

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

Minors unable to speak and read will be enrolled onto this study. Parents' consent will be sought.

Los menores que no puedan hablar y leer serán inscritos en este estudio. Se solicitará el consentimiento de los padres.

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Patients who complete the Treatment Period and Visit 9 (Day 168) in this study will be invited to participate in a 72-week open-label extension study (A4250-008) in which all patients will receive active treatment.

A los pacientes que finalicen el periodo de tratamiento y la visita 9 (día 168) de este estudio se les invitará a participar en un estudio de extensión abierto de 72 semanas de duración (A4250-008) en el que todos los pacientes recibirán tratamiento activo.

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

2018-04-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-04-18

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2020-07-28

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IMP with orphan designation in the indication
Orphan Designation Number:
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