Clinical Trials Register

Summary
EudraCT Number:	2018-003920-35
Sponsor's Protocol Code Number:	CQAW039B2201
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2019-04-12
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

2018-003920-35

A.3

Full title of the trial

A multicenter, open-label, 8 day treatment study to assess the pharmacokinetics, safety and tolerability of fevipiprant delivered via a once daily chewable tablet in children aged 6 to <12 years with asthma

Estudio multicéntrico, abierto y con un tratamiento de 8 días para evaluar la farmacocinética, seguridad y tolerabilidad de fevipiprant administrando un comprimido masticable una vez al día en niños de 6 a <12 años de edad con asma.

A.3.1

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

A multicenter, open-label, 8 day treatment study to assess the pharmacokinetics, safety and tolerability of fevipiprant once daily tablet in children aged 6 to <12 years with asthma

Farmacocinética, seguridad y tolerabilidad de fevipiprant administrando un comprimido masticable una vez al día en niños de 6 a <12 años de edad con asma.

A.4.1

Sponsor's protocol code number

CQAW039B2201

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03650400

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/124/2018

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Novartis Farmacéutica
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 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	Javier Malpesa
B.5.3	Address:
B.5.3.1	Street Address	Gran Vía 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	+3493 306 44 64
B.5.5	Fax number	----
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.3.1	Product name	Fevipiprant
D.3.2	Product code	QAW039
D.3.4	Pharmaceutical form	Chewable tablet
D.3.4.1	Specific paediatric formulation	Yes
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	Fevipiprant
D.3.9.1	CAS number	872365-14-5
D.3.9.2	Current sponsor code	QAW039
D.3.9.3	Other descriptive name	QAW039-NXA.001
D.3.9.4	EV Substance Code	SUB32073
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	75
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.3.1	Product name	fevipiprant
D.3.2	Product code	QAW039
D.3.4	Pharmaceutical form	Chewable tablet
D.3.4.1	Specific paediatric formulation	Yes
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	fevipiprant
D.3.9.1	CAS number	872365-14-5
D.3.9.2	Current sponsor code	QAW039
D.3.9.3	Other descriptive name	QAW039-NXA.001
D.3.9.4	EV Substance Code	SUB187649
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	225
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

Asthma

Asma

E.1.1.1

Medical condition in easily understood language

Asthma

Asma

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

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	10003553
E.1.2	Term	Asthma
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To determine the key pharmacokinetic parameters of fevipiprant at steady state (ss), after at least four consecutive days of dosing))

Determinar los parámetros farmacocinéticos principales de fevipiprant en estado de equilibrio (ss), después de al menos cuatro días consecutivos con administración de la dosis

E.2.2

Secondary objectives of the trial

- To evaluate additional pharmacokinetic parameters of fevipiprant at steady state
- To evaluate the pharmacokinetics of CCN362, the major metabolite of fevipiprant at steady state
- To evaluate the urinary excretion of fevipiprant and CCN362 at steady state
- To evaluate the safety & tolerability of fevipiprant over treatment period

Objetivo 1: evaluar los parámetros farmacocinéticos adicionales de fevipiprant en estado de equilibrio.
Objetivo 2: evaluar la farmacocinética de CCN362, el metabolito principal de fevipiprant en estado de equilibrio.
Objetivo 3: evaluar la excreción por la orina de fevipiprant y CCN362 en estado estacionario.
Objetivo 4: evaluar la seguridad y tolerabilidad de fevipiprant a lo largo del periodo de tratamiento.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Subjects eligible for inclusion in this study must meet all of the following criteria:
1. Male and female children ≥ 6 years and <12 years.
2. Written informed consent by parent(s)/legal guardian(s) for the pediatric patient and assent by the pediatric patient (depending on local requirements) must be obtained before any study-specific assessment is performed.
3. Confirmed/documented diagnosis of asthma, as defined by national or international asthma guidelines for at least 6 months prior to study enrollment.
4. Subjects using asthma rescue medication (e.g. SABA) without asthma controller therapy or patients receiving daily treatment with a stable dose ICS (with or without additional controller such as long-acting β-agonists (LABA), long-acting muscarinic antagonists (LAMA)) for at least 4 weeks prior to Treatment Visit (Day 1).
5. Subjects must be able to attend study visits as per Study Visit Assessment Schedule (Section 8) which includes 8 to 9 hours in the clinic/home on the day of End of Treatment Visit and have blood draws as scheduled in the study.
6. Parents/legal guardian must be willing and able to attend study visits and assist the child with the procedures outlined in the protocol (e.g. compliance with taking study medication and completing the diary).

1. Niños de ambos sexos >/=6 y <12 años de edad.
2. Se debe obtener el consentimiento informado por escrito de los progenitores/tutores legales y el asentimiento del paciente pediátrico (según los requisitos locales) antes de que se realice cualquier evaluación específica del estudio.
3. Diagnóstico confirmado/documentado de asma según lo definido por las guías internacionales o nacionales de asma durante al menos 6 meses antes de la inclusión en el estudio.
4. Sujetos que hayan tomado medicación de rescate para el asma (p. ej., SABA) sin tratamiento de control del asma o sujetos que hayan recibido tratamiento diario con una dosis estable de CI (con o sin tratamiento de control adicional como agonistas β de acción prolongada [LABA] o antagonistas muscarínicos de acción prolongada [LAMA] durante al menos 4 semanas antes de la visita de tratamiento (día 1).
5. Los sujetos deben poder asistir a las visitas indicadas en el calendario de visitas y evaluaciones del estudio (apartado 8) que incluye de 8 a 9 horas en el centro/domicilio el día de la visita de fin de tratamiento y las extracciones de sangre programadas en el estudio.
6. Los progenitores/tutor legal deben querer y poder asistir a las visitas del estudio y ayudar al niño con los procedimientos descritos en el protocolo (p. ej., con el cumplimiento de la administración de la medicación del estudio y la cumplimentación del diario).

E.4

Principal exclusion criteria

Subjects meeting any of the following criteria are not eligible for inclusion in this study.
1. Use of other investigational drugs within 5 half-lives of enrollment, or (within 30 days (for small molecules)/until the expected pharmacodynamic effect has returned to baseline (for biologics)), whichever is longer.
2. Subject is unable to ingest banana and/or yogurt
3. History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes.
4. History of chronic lung disease other than asthma such as and not limited to, sarcoidosis interstitial lung disease, cystic fibrosis, mycobacterial or other infection (including active tuberculosis or atypical mycobacterial disease).
5. History of active bacterial, viral or fungal infection within 6 weeks of Treatment Visit (Day 1).
6. Subjects who, in the opinion of the investigator, are not able to be compliant with study treatment or who have any medical or mental disorder, situation, or diagnosis, which could interfere with the proper completion of the protocol requirements or risk the subject’s safety while participating in the study.
7. Parent/guardian who, in the opinion of the investigator, has a history of psychiatric disease, intellectual deficiency, substance abuse, or other condition (e.g. inability to read, comprehend and write) which will limit the validity of consent for their child to participate in this study.
8. Hemoglobin levels outside normal ranges at screening.
9. Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardize the subject in case of participation in the study.

1. Uso de cualquier otro fármaco en investigación en un periodo de 5 semividas de la inclusión o (en un periodo de 30 días [para moléculas pequeñas]/hasta que el efecto farmacodinámico previsto vuelva a los valores basales [para productos biológicos]), aquel periodo que sea más largo.
2. Sujetos que no sean capaces de ingerir plátanos ni yogur.
3. Antecedentes de hipersensibilidad a alguno de los fármacos del estudio o sus excipientes o a fármacos de clases químicas similares.
4. Antecedentes de enfermedad pulmonar crónica, salvo asma, y como por ejemplo la enfermedad pulmonar intersticial sarcoidosis, fibrosis quística o infección micobacteriana o de otro tipo (incluida la tuberculosis activa o la enfermedad micobacteriana atípica).
5. Antecedentes de infección bacteriana, vírica o fúngica activa durante las 6 semanas anteriores a la visita de tratamiento (día 1).
6. Sujetos que, según el criterio del investigador, no sean capaces de cumplir
el tratamiento del estudio o que presenten algún trastorno, situación o diagnóstico de carácter médico o mental que pudiera interferir en la cumplimiento adecuado de los requisitos del protocolo o poner en riesgo la seguridad del sujeto mientras esté participando en el estudio.
7. Progenitor/tutor que, según el investigador, tiene antecedentes de enfermedad psiquiátrica, deficiencia intelectual, abuso de sustancias u otra enfermedad (p. ej., incapacidad para leer, comprender y escribir) lo cual limitará la validez del consentimiento para que su hijo participe en este estudio.
8. Niveles de hemoglobina fuera del rango normal en la selección.
9. Cualquier condición médica o quirúrgica que pueda alterar significativamente la absorción, distribución, metabolismo o excreción de los fármacos, o que pueda afectar al sujeto en caso de participar en el estudio.

E.5 End points

E.5.1

Primary end point(s)

Area under the curve (AUC0-24h,ss), maximum plasma concentration (Cmax,ss), and oral clearance (CL/F)

Area bajo la curva (AUC0-24h,ss), concientracion máxima en plasma (Cmax,ss) y aclaramiento oral (CL/F)

E.5.1.1

Timepoint(s) of evaluation of this end point

Day 8

Día 8

E.5.2

Secondary end point(s)

- Tmax,ss, Cmin,ss, (CL/F)/kg of fevipiprant
- Cmax,ss, time of maximum plasma concentration (Tmax,ss), AUC0-24h,ss, minimum plasma concentration (Cmin,ss) of CCN362
- CLr, amount and fraction of dose excreted over the PK collection interval
- Safety laboratory values (including peripheral blood eosinophils), vital signs, electrocardiogram (ECG), adverse events

- Tmax,ss, Cmin,ss, (CL/F)/kg de fevipiprant.
- Cmax,ss, tiempo de concentración plasmática máxima (Tmax,ss), AUC0-24h,ss, concentración plasmática mínima (Cmin,ss) de CCN362.
- CLr, cantidad y fracción de dosis excretada durante el intervalo de recogida para PK.
- Valores de seguridad del laboratorio (que incluyen eosinófilos en sangre periférica), constantes vitales, electrocardiograma (ECG) y acontecimientos adversos.

E.5.2.1

Timepoint(s) of evaluation of this end point

Day 8

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

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

Yes

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

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

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

Spain

United Kingdom

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

LVLS 23-Jul-2020

Última visita del último paciente 23Jul2020

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 months

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)

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

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)

Patient returns to standard of care

Paciente vuelve al tratamiento estandar.

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

2019-06-12

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-05-28

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2019-12-16

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