Clinical Trials Register

Summary
EudraCT Number:	2011-005154-57
Sponsor's Protocol Code Number:	CACZ885D2307E1
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:	2012-01-27
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

2011-005154-57

A.3

Full title of the trial

An open-label extension study to assess efficacy, safety and tolerability
of canakinumab and the efficacy and safety of childhood vaccinations in
patients with Cryopyrin Associated Periodic Syndromes (CAPS)

Estudio de extensión abierto para evaluar la eficacia, seguridad y tolerabilidad de canakinumab y la eficacia y seguridad de las vacunas pediátricas en pacientes con Síndrome Periódico Asociado a Criopirina (CAPS)

A.3.1

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

An extension study to check how safe, beneficial and tolerable the drug
canakinumab, along with childhood vaccines, is for patients with crypoyrin associated periodic syndromes (CAPS).

Estudio de extensión para comprobar cómo de seguro, tolerable y beneficioso es el Canakinumab para pacientes con síndrome asociado a criopirina administrado junto a vacunas infantiles

A.3.2

Name or abbreviated title of the trial where available

D2307E1

A.4.1

Sponsor's protocol code number

CACZ885D2307E1

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/208/2011

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	Inma Bosch
B.5.3	Address:
B.5.3.1	Street Address	Gran Via de las 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	0034933064351
B.5.5	Fax number	0034933064274
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	Yes
D.2.1.1.1	Trade name	ILARIS
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Europharm Limited
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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/3/07/439
D.3 Description of the IMP
D.3.1	Product name	canakinumab
D.3.2	Product code	ACZ885
D.3.4	Pharmaceutical form	Powder for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	CANAKINUMAB
D.3.9.1	CAS number	914613-48-2
D.3.9.2	Current sponsor code	ACZ885
D.3.9.4	EV Substance Code	SUB30137
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	150
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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	anticuerpo monoclonal antihumano de la interleucina 1B

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Powder for solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Cryopyrin Associated Periodic Syndromes (CAPS)

Síndrome Periódico Asociado a Criopirina (CAPS)

E.1.1.1

Medical condition in easily understood language

CAPS is a group of inflammatory disorders (causing redness, swelling, pain and fever) caused by the body making too much of a protein called interleukin 1B (IL-1B)

CAPS es un grupo de trastornos inflamatorios (que produce enrojecimiento, inflamación, dolor y fiebre) causado por la síntesis de una proteína llamada interleuquina 1Beta (IL-1Beta)

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.1
E.1.2	Level	PT
E.1.2	Classification code	10068850
E.1.2	Term	Cryopyrin associated periodic syndrome
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 assess the long-term efficacy of canakinumab with respect to the maintenance of treatment response in CAPS patients who completed the CACZ885D2307 study

Evaluar la eficacia a largo plazo de canakinumab con respecto a la recidiva en pacientes con CAPS que hayan finalizado el estudio CACZ885D2307

E.2.2

Secondary objectives of the trial

- Safety and tolerability as assessed by the overall frequency of adverse events and the number of patients completing the extension study in the overall population
- To assess the presence of protective antibody levels following
immunization with inactivated (killed) vaccines administered during the extension study
- To assess the safety of canakinumab treatment in pediatric patients receiving a concomitant vaccination during the extension study
- To assess the proportion of patients with vaccination-associated
reactions during the extension study
- To assess efficacy with regards to the Physician's Global Assessment of autoinflammatory disease activity and assessment of skin disease
- To evaluate the efficacy of canakinumab with regards to inflammatory markers (C-reactive protein (CRP) or serum amyloid A (SAA)
[...]

- Seguridad y tolerabilidad evaluadas por la frecuencia global de acontecimientos adversos y el número de pacientes que finalicen el estudio de extensión en la población global
- Evaluar la presencia de niveles de anticuerpos protectores después de la inmunización con vacunas inactivadas (muertas) administradas durante el estudio de extensión
- Evaluar la seguridad del tratamiento con canakinumab en pacientes pediátricos que reciban una vacuna concomitante durante el estudio de extensión
- Evaluar la proporción de pacientes con reacciones asociadas a la vacunación durante el estudio de extensión
- Evaluar la eficacia con respecto a la evaluación global del médico de la actividad de la enfermedad autoinflamatoria y la evaluación de la enfermedad cutánea
- Evaluar la eficacia de canakinumab con respecto a marcadores de la inflamación (proteína C reactiva (PCR) o amiloide A sérico (AAS)
[..... ]

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Patients who completed the core CACZ885D2307 study (a patient is defined as having completed the core study if they completed the study up to and including the EOS visit with no major protocol deviations in the core).
2.Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
3.Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.

1. Pacientes que finalizaron el estudio principal CACZ885D2307 (se define que un paciente ha finalizado el estudio principal si ha finalizado el estudio hasta e incluida la visita EOS sin
desviaciones importantes del protocolo en el estudio principal).
2. Pacientes hombres y mujeres que tengan ? 1 año de edad en el momento de la visita de inclusión.
3. Deberá obtenerse el consentimiento informado por escrito del padre/madre o tutor legal antes de realizar ninguna evaluación en el estudio de extensión CACZ885D2307E1.

E.4

Principal exclusion criteria

1.Patients for whom continued treatment in the CACZ885D2307E1
extension study is not considered appropriate by the treating physician.
2.Patients who discontinued from the core CACZ885D2307 study.

1. Pacientes para los que el tratamiento continuado en el estudio de extensión CACZ885D2307E1 no se considere apropiado a juicio del médico tratante.
2. Pacientes que fueron retirados del estudio principal CACZ885D2307.
Para asegurar que la población del estudio será representativa de todos los pacientes elegibles, el investigador no podrá aplicar ninguna exclusión adicional.

E.5 End points

E.5.1

Primary end point(s)

To assess the long-term efficacy of canakinumab with respect to the maintenance of treatment response in CAPS patients who completed the CACZ885D2307 study

Evaluar la eficacia a largo plazo de canakinumab con respecto al mantenimiento de la respuesta al tratamiento en pacientes con CAPS que completaron el estudio CACZ885D2307

E.5.1.1

Timepoint(s) of evaluation of this end point

A minimum of 6 months and maximum of 24 months

un mínimo de 6 meses y un máximo de 24 meses

E.5.2

Secondary end point(s)

- Safety and tolerability as assessed by the overall frequency of adverse events and the number of patients completing the extension study in the overall population
- To assess the presence of protective antibody levels following
immunization with inactivated (killed) vaccines (see Section 6.1 of
protocol) administered during the extension study
- To assess the safety of canakinumab treatment in pediatric patients receiving a concomitant vaccination during the extension study
- To evaluate the proportion of patients with vaccinated-associated
reactions
- To assess efficacy with regards to the Physician's Global Assessment of autoinflammatory disease activity and assessment of skin disease
- To assess the reduction of inflammation marker (C-reactive protein (CRP) or serum amyloid A (SAA) after treatment initiation

E.5.2.1

Timepoint(s) of evaluation of this end point

A minimum of 6 months and maximum of 24 months

un mínimo de 6 meses y un máximo de 24 meses

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

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

Assessment of Immunogenicity; Assessment of antibody titers against vaccine antigen

Evaluación de la inmunogenicidad, evaluación de los títulos de anticuerpos contra el antígeno de la vacuna

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

E.8.1.2

Open

Yes

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)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Sólo hay una rama de tratamiento Canakinumab activo. Abierto

There is only 1 treatment arm: Canakinumab. Open

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

Belgium

Canada

France

Germany

Israel

Spain

United Kingdom

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

LVLS

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)

F.1.2

Adults (18-64 years)

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

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

paediatric patients

pacientes pediátricos

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)

Upon availability of the approved and marketed drug for the pediatric CAPS population, patients will be
offered to complete the study and roll-over into an open label registry study (CACZ885D2401), which
will allow for the long-term assessment of safety and disease-modification.

Cuando se disponga del fármaco aprobado y comercializado para la población pediátrica con CAPS, a los pacientes se les ofrecerá finalizar el estudio y ser reclutados en un estudio de registro abierto (CACZ885D2401), lo que permitirá la evaluación a largo plazo de la seguridad y la
modificación de la enfermedad.

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

2012-03-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-03-06

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2015-10-13

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