Clinical Trials Register

Summary
EudraCT Number:	2012-000802-32
Sponsor's Protocol Code Number:	CLCQ908B2305
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:	2012-08-09
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Spain - AEMPS

A.2

EudraCT number

2012-000802-32

A.3

Full title of the trial

An open label, 52-week, safety and tolerability extension to a randomized, double-blind, placebo controlled study of LCQ908 in subjects with Familial Chylomicronemia Syndrome

Ensayo clínico abierto, de extensión de 52 semanas de duración, sobre seguridad y tolerabilidad de un ensayo clínico randomizado, doble ciego, controlado con placebo, para evaluar la eficacia, la seguridad y la tolerabilidad de LCQ908 en pacientes con síndrome de hiperquilomicronemia familiar

A.3.1

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

Extension to a study of LCQ908 in subjects with Familial Chylomicronemia Syndrome

Extensión del estudio de LCQ908 en pacientes con Síndrome de Hiperquilomicronemia Familiar

A.4.1

Sponsor's protocol code number

CLCQ908B2305

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01589237

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	Novartis Farmacéutica, S.A.
B.1.3.4	Country	Spain
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	Novartis Pharma Services AG
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	Novartis Farmacéutica, S.A.
B.5.2	Functional name of contact point	Inmaculada Bosch Tirau
B.5.3	Address:
B.5.3.1	Street Address	Gran Via de les Corts Catalanes, 764
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08013
B.5.3.4	Country	Spain
B.5.4	Telephone number	+3493306 4342_
B.5.5	Fax number	+3493306 4290_
B.5.6	E-mail	eecc.novartis@novartis.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.3.2	Product code	LCQ908
D.3.4	Pharmaceutical form	Film-coated 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	Not assigned
D.3.9.1	CAS number	Not assigned
D.3.9.2	Current sponsor code	LCQ908
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: 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.3.2	Product code	LCQ908
D.3.4	Pharmaceutical form	Film-coated 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	Not assigned
D.3.9.1	CAS number	Not assigned
D.3.9.2	Current sponsor code	LCQ908
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: 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.3.2	Product code	LCQ908
D.3.4	Pharmaceutical form	Film-coated 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	Not assigned
D.3.9.1	CAS number	Not assigned
D.3.9.2	Current sponsor code	LCQ908
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	40
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Familial Chylomicronemia Syndrome (FCS) (HLP Type I)

Síndrome de Hiperquilomicronemia Familiar (FCS) (HLP Type I)

E.1.1.1

Medical condition in easily understood language

Familial Chylomicronemia Syndrome

Síndrome de Hiperquilomicronemia Familiar

E.1.1.2

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.1
E.1.2	Level	LLT
E.1.2	Classification code	10020607
E.1.2	Term	Hyperchylomicronemia
E.1.2	System Organ Class	10027433 - Metabolism and nutrition 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 determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I)

Determinacion de seguridad, tolerabilidad y eficacia continuada a largo plazo de LCQ908 en la dismisnucion de triglicéridos en pacientes con Síndrome de Hiperquilomicronemia Familiar (FCS) (HLP type I).

E.2.2

Secondary objectives of the trial

To assess changes in lipid and lipoprotein profiles

Evaluacion de cambios en el perfil de lipidos y lipoproteinas

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Written informed consent must be obtained before any assessment is performed
- Subjects that either discontinue prematurely or complete the CLCQ908B2302 study after 52 weeks or FCS subjects who have previously completed study CLCQ908A2212

1. Antes de realizar cualquier evaluación se deberá obtener el consentimiento informado.
2. Pacientes que hayan abandonado de forma prematura el ensayo LCQ908B2302 o que hayan completado sus 52 semanas de duración, o que hayan completado el ensayo LCQ908A2212.

E.4

Principal exclusion criteria

-Subjects discontinued from the CLCQ908B2302 study for serious, potentially study drug related adverse events
-Subjects from the CLCQ908B2302 study who have developed any other contraindication to participation (for example, renal failure)
- History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
- Preganant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test
- Subjects with type 1 diabetus mellitus or type 2 diabetes mellitus if HbA1c is >=8.5%
- Treatment with fish oil preparations within 4 weeks prior to randomization
- Treatment with bile acid binding resins (i.e. colesevelam etc) within 4 weeks prior to randomization
- Treatment with fibrates within 8 weeks prior to randomization. Washout may occur following screening if required
- Glybera [alipogene tiparvovec (AAV1-LPLS447X)] gene therapy exposure within two years prior to screening
- eGFR < 45 ml/min/1.73m2 or history of chronic renal disease

1. Pacientes que hayan abandonado el ensayo CLCQ908B2302 debido a acontecimientos adversos graves y potencialmente relacionados con el fármaco del estudio.
2. Pacientes del ensayo CLCQ908B2302 que hayan presentado cualquier otra contraindicación de la participación (por ejemplo, insuficiencia renal)
3. Uso de otros fármacos en investigación en las 5 semividas previas a la inclusión, o en los 30 días previos [hasta que el efecto FD previsto haya recuperado el valor basal], lo que suponga más tiempo.
4. Antecedentes de hipersensibilidad a cualquier fármaco del estudio o a cualquier fármaco perteneciente a una clase similar de fármacos.
5. Antecedentes clínicos de neoplasias malignas en cualquier sistema u órgano (distintas al carcinoma basocelular localizado), tratadas o no, durante los 5 últimos años, independientemente de si existen pruebas de recidivas locales o de metástasis.
6. Mujeres embarazadas o lactantes (que estén amamantando); por embarazo se entiende el estado en que se encuentra la mujer tras la concepción y hasta el término de la gestación, confirmado mediante el análisis clínico de la HCG (gonadotropina coriónica) con resultado positivo.
7. Mujeres en edad fértil, definidas como todas las mujeres con capacidad fisiológica para quedarse embarazadas, a menos que utilicen métodos anticonceptivos de gran eficacia durante la administración y durante los 100 días posteriores a la interrupción del fármaco del estudio.
8. Pacientes con diabetes de tipo 1 o tipo 2, si la HbA1c ? 8,5 %.
9. Tratamiento con preparados que contengan aceite de pescado en las 4 semanas previas a la randomización.
10. Tratamiento con resinas fijadoras de los ácidos biliares (es decir, colesevelam, etc.) en las 4 semanas previas a la randomización.
11. Tratamiento con fibratos en las 8 semanas previas a la randomización. El período de reposo farmacológico se podrá realizar después que el screening, en caso de ser necesario.
12. Haber estado sometido a terapia génica con Glybera (alipogen tiparvovec [AAV1-LPLS447X]) en los dos años previos al screening.
13. Cualquier trastornos médico o quirúrgico, enfermedad aguda o crónica inestable que pudiera, según el criterio del Investigador, poner en peligro la vida del paciente en el caso de que este participe en el estudio o pueda alterar de forma significativa la absorción, la distribución, el metabolismo o la excreción del fármaco del estudio. Antecedentes médicos de drogodependencia o alcoholismo en los 12 meses previos a la randomización, o indicios de dichas dependencias durante el screening. Signos indicativos de hepatopatía o daño hepático, indicados por anomalías en las pruebas de función hepática, como la ASAT, la ALAT o la bilirrubina sérica. El Investigador deberá guiarse por los siguientes criterios:
? Cualquier aumento de los niveles de ASAT y ALAT superior al triple del límite superior de la normalidad (LSN) en el Screening, confirmado mediante la repetición de la determinación en el plazo de una semana.
? Bilirrubina total superior al doble del LSN o bilirrubina directa superior al LSN en el Screening, confirmado mediante la repetición de la determinación realizada en el plazo de una semana (No se excluirá a los pacientes con diagnóstico confirmado de enfermedad de Gilbert).
14. FGe < 45 ml/min/1,73 m2 o antecedentes clínicos de nefropatía crónica.

E.5 End points

E.5.1

Primary end point(s)

Number of patients with adverse and serious adverse events

Número de pacientes con acontecimientos adversos graves y no graves

E.5.1.1

Timepoint(s) of evaluation of this end point

up to 52 weeks

hasta 52 semanas

E.5.2

Secondary end point(s)

- changes in lipid and lipoprotein profiles from baseline up to 52 weeks
- changes from baseline in triglyceride levels up to 52 weeks

- cambios el los perfiles de lípidos y lipoproteinas desde el estadio basal hasta la semana 52
- cambios en el nivel de triglicéridos desde el estadio basal hasta la semana 52

E.5.2.1

Timepoint(s) of evaluation of this end point

- baseline, week 12, week 24 and 52

- estado basal, semana 12, semana 24 y 52

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

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

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)

Information not present in EudraCT

E.8.2.2

Placebo

Information not present in EudraCT

E.8.2.3

Other

Information not present in EudraCT

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

South Africa

United States

E.8.7

Trial has a data monitoring committee

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

LPLS

Ultima 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

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:

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

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)

None

Ninguno

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

2013-01-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-12-20

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2015-05-20

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