Clinical Trials Register

Summary
EudraCT Number:	2019-003775-19
Sponsor's Protocol Code Number:	EFC16461
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2020-05-07
Trial 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

2019-003775-19

A.3

Full title of the trial

Master protocol of two randomized, double-blind, placebo controlled, multi center, parallel-group studies of dupilumab in patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite the use of H1 antihistamine treatment in patients naïve to omalizumab and in patients who are intolerant or incomplete responders to omalizumab

Protocolo maestro de dos estudios aleatorizados, controlados con placebo, doble ciegos, multicéntricos, de grupos paralelos de dupilumab en pacientes con urticaria crónica espontánea (UCE) que siguen siendo sintomáticosa pesar del uso de tratamiento antihistamínico H1 en pacientes sin tratamiento previo con omalizumab y en pacientes intolerantes o con respuesta incompleta a omalizumab.

A.3.1

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

Dupilumab for the treatment of chronic spontaneous urticaria in patients who remain symptomatic despite the use of H1 antihistamine and who are naïve to, intolerant of, or incomplete responders to omalizumab.

Dupilumab para el tratamiento de la urticaria crónica espontánea en pacientes que se mantienen sintomáticos a pesar del uso de antihistamínico H1 y que no hayan tenido un tratamiento previo con omalizumab o que sean intolerantes o presentan una respuesta incompleta al omalizumab.

A.3.2

Name or abbreviated title of the trial where available

CUPID

A.4.1

Sponsor's protocol code number

EFC16461

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	Sanofi-aventis Recherche & Développement
B.1.3.4	Country	France
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	Sanofi-aventis Recherche & Développement
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	sanofi-aventis, s.a.
B.5.2	Functional name of contact point	Unidad de Estudios Clínicos
B.5.3	Address:
B.5.3.1	Street Address	c/ Josep Pla 2, 4ª planta
B.5.3.2	Town/ city	Barcelona
B.5.3.3	Post code	08019
B.5.3.4	Country	Spain
B.5.4	Telephone number	3493485 94 00
B.5.6	E-mail	es-unidadestudiosclinicos@sanofi.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	Dupilumab
D.3.2	Product code	SAR231893
D.3.4	Pharmaceutical form	Solution for injection in pre-filled syringe
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	Dupilumab
D.3.9.2	Current sponsor code	SAR231893
D.3.9.3	Other descriptive name	REGN668
D.3.9.4	EV Substance Code	SUB88511
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
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	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	Dupilumab
D.3.2	Product code	SAR231893
D.3.4	Pharmaceutical form	Solution for injection in pre-filled syringe
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	Dupilumab
D.3.9.2	Current sponsor code	SAR231893
D.3.9.3	Other descriptive name	REGN668
D.3.9.4	EV Substance Code	SUB88511
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	175
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	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	Solution for injection in pre-filled syringe
D.8.4	Route of administration of the placebo	Subcutaneous use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection in pre-filled syringe
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

Chronic spontaneous urticaria

Urticaria crónica espontánea

E.1.1.1

Medical condition in easily understood language

Chronic spontaneous urticaria

Urticaria crónica espontánea

E.1.1.2

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

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	10072757
E.1.2	Term	Chronic spontaneous urticaria
E.1.2	System Organ Class	10040785 - Skin and subcutaneous tissue 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 demonstrate the efficacy of dupilumab in adult and adolescent participants with CSU who remain symptomatic despite the use of H1 antihistamine (Study A: omalizumab naïve; Study B: omalizumab intolerant or incomplete responders)

Demostrar la eficacia de dupilumab en participantes adultos y adolescentes con UCE que siguen sintomáticos a pesar del uso de H1-AH (estudio A: sin tratamiento previo con omalizumab; estudio B: intolerantes a omalizumab o con respuesta incompleta)

E.2.2

Secondary objectives of the trial

- To demonstrate the efficacy of dupilumab on urticaria activity composite endpoint and itch or hives, separately, at various timepoints
- To demonstrate the efficacy of dupilumab on angioedema
- To demonstrate the efficacy of dupilumab on urticaria control
- To demonstrate improvement in health-related quality of life and overall disease status and severity
- To evaluate the ability of dupilumab in reducing the proportion of patients who require treatment with oral corticosteroids (OCS)
- To evaluate safety outcome measures
- To evaluate immunogenicity of dupilumab

- Demostrar la eficacia de dupilumab con respecto al criterio de valoración compuesto de la actividad de la urticaria y el picor o las ronchas, por separado, en diversos momentos.
- Demostrar la eficacia del dupilumab en el angioedema
- Demostrar la eficacia del dupilumab para controlar la urticaria
- Demostrar una mejoría en la calidad de vida relacionada con la salud y en el estado general de la enfermedad y la intensidad
- Evaluar la capacidad de dupilumab para reducir la proporción de pacientes que requieren tratamiento con corticosteroides orales (OCS)
- Evaluar las medidas de los resultados de seguridad
- Evaluar la inmunogenia del dupilumab

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Must be 12 to 80 years of age, at the time of signing the informed consent
- Participants who have a diagnosis of CSU refractory to H1 antihistamines (H1-AH) at the time of randomization defined by
• Diagnosis of CSU>6 months prior to screening visit
• Presence of itch and hives for >6 consecutive weeks at any time prior to screening visit despite the use of H1-AH during this time period
• Using a study defined H1-antihistamine for CSU treatment
• During the 7 days before randomization:
UAS7=>16
ISS7=> 8
• In-clinic UAS> 4 prior to randomization
• Study A: omalizumab naïve, Study B; intolerant or incomplete responder to omalizumab
- Participants must be willing and able to complete a daily symptom eDiary for the duration of the study

- El participante debe tener ≥ 12 años hasta 80 años de edad al momento de firmar el consentimiento informado.
- Participantes que tienen un diagnóstico de UCE refractario a H1-AH en el momento de la aleatorización, según lo definido por todo lo siguiente:
• Diagnóstico de UCE > 6 meses antes de la visita de selección.
• Presencia de picor y ronchas durante > 6 semanas consecutivas en cualquier momento antes de la visita de selección a pesar del uso de H1-AH durante este período de tiempo.
• Los participantes que utilizan un H1-AH definido en el estudio para el tratamiento de la UCE.
• Durante los 7 días previos a la aleatorización:
- UAS7 >16
- ISS7 >8
• UAS en la clínica > 4 antes de la aleatorización (en V1 o V2).
• Estudio A : sin tratamiento previo con omalizumab; Estudio B: intolerantes al omalizumab o con respuesta incompleta
• Los participantes deben estar dispuestos y ser capaces de completar un diario electrónico de síntomas diarios durante el estudio.

E.4

Principal exclusion criteria

Participants are excluded from any of the studies if any of the following criteria apply:
- Weight is less than 30 kg
- Clearly defined underlying etiology for chronic urticarias other than CSU
- Presence of skin morbidities other than CSU that may interfere with the assessment of the study outcomes
- Active atopic dermatitis
- Severe concomitant illness(es) that, in the investigator’s judgment, would adversely affect the patient’s participation in the study
- Active tuberculosis or non-tuberculous mycobacterial infection, or a history of incompletely treated tuberculosis
- Diagnosed active endoparasitic infections; suspected or high risk of endoparasitic infection
- Active chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiprotozoals, or antifungals within 2 weeks before the screening visit
- Known or suspected immunodeficiency
- Active malignancy or history of malignancy within 5 years before the baseline visit, except completely treated in situ carcinoma of the cervix, completely treated and resolved non-metastatic squamous or basal cell carcinoma of the skin.
- History of systemic hypersensitivity or anaphylaxis to omalizumab or any biologic therapy, including any excipients

Los participantes quedan excluidos del estudio si procede alguno de los siguientes criterios:
- Peso inferior a 30 kg.
- Etiología subyacente claramente definida para UC distinta a la UCE.
- Presencia de morbilidades cutáneas distintas de la UCE que pueden interferir en la evaluación de los resultados del estudio.
- Pacientes con dermatitis atópica activa.
- Enfermedad(es) concomitante(s) grave(s) que, a criterio del investigador, pudiera(n) afectar negativamente la participación del paciente en el estudio.
- Los pacientes con tuberculosis (TB) activa, infección micobacteriana no tuberculosa o antecedentes de tuberculosis tratada de forma incompleta.
- Diagnóstico de infección endoparasitaria activa; riesgo alto o sospecha de infección endoparasitaria.
- Infección crónica o aguda activa que requiere tratamiento con antibióticos sistémicos, antivirales, antiprotozoarios o antifúngicos en un plazo de 2 semanas antes de la visita de selección.
- Informe o sospecha de inmunodeficiencia.
- Neoplasia maligna activa o antecedentes de neoplasia maligna en los 5 años previos a la visita de selección, excepto carcinoma del cuello uterino in situ completamente tratado, carcinoma de células basales o de células escamosas no metastásico en la piel completamente tratado y resuelto.
- Antecedentes de hipersensibilidad sistémica o de anafilaxia a omalizumab o a cualquier tratamiento biológico, incluyendo cualquier excipiente.

E.5 End points

E.5.1

Primary end point(s)

Change from baseline in weekly itch severity score ; Change from baseline in weekly itch severity score (ISS7) at Week 24

Cambio con respecto al momento inicial en la puntuación semanal de la intensidad del picor (ISS7) en la semana 24.

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline to Week 24

Momento inicial a semana 24

E.5.2

Secondary end point(s)

1 - 2 - Change from baseline in weekly itch severity score (ISS7) at Week 12 and 24 / Change from baseline in weekly hives severity score (HSS7) at Week 12 and Week 24 ; The hives severity score will be recorded daily in the patient eDiary, on a scale of 0 (none) to 3 (severe). The baseline weekly hive score is the sum of the daily hives scores over the 7 days prior to randomization (Day 1 visit), and the weekly ihives score at Weekk12/ Week 24 is the sum of daily hives scores over the 7 days prior to the Week 12/ Week 24 visit.
3 - Time to ISS7 minimally important (MID) (ISS7 ≥5) response ; The itch severity score will be recorded daily in the patient eDiary, on a scale of 0 (none) to 3 (severe).The weekly itch score is the sum of the daily itch scores over 7 days
4 - Proportion of ISS7 MID (≥5 points) responders at Week 12 and Week 24 ; The itch severity score will be recorded daily in the patient eDiary, on a scale of 0 (none) to 3 (severe).The weekly itch score is the sum of the daily itch scores over 7 days.
5 - Change from baseline in ISS7 at all time points (onset of action is assessed by the first p<0.05 that remains significant at subsequent measures until Week 24 ; The itch severity score will be recorded daily in the patient eDiary, on a scale of 0 (none) to 3 (severe).The weekly itch score is the sum of the daily itch scores over 7 days
6 - Change from baseline in UAS7 at Week 12 ; The UAS is a composite eDiary-recorded score with numeric severity intensity ratings on a scale of 0-3 (0=none to 3=intense/severe) for 1) the number of wheals (hives); and 2) the intensity of the itch measured daily. UAS7 is the sum of the daily UAS scores over 7 days.
7 - 8 - Proportion of patients with UAS7 ≤6 at Week 12 and Week 24 / Proportion of patients with UAS7=0 at Week 12 and Week 24 ; The UAS is a composite eDiary-recorded score with numeric severity intensity ratings on a scale of 0-3 (0=none to 3=intense/severe) for 1) the number of wheals (hives); and 2) the intensity of the itch measured daily. UAS7 is the sum of the daily UAS scores over 7 days.
9 - Change from baseline in angioedema activity score over 7 days (AAS7) at Week 12 and Week 24 ; Angioedema activity score (AAS) is recorded once daily in the Diary by the participants. This validated tool assesses episodes of angioedema including their duration, severity and impact on daily functioning and appearance.
10 - Change from baseline in urticaria control test (UCT) at Week 12 and Week 24 ; The urticarial control test (UCT) is instrument that works in all types of chronic urticaria (not only for CSU), and it can also be used in urticaria patients suffering from recurrent angioedema.otal score ranges 0 to 16; higher scores indicate better disease control.
11 - Proportion of well controlled patients (UCT ≥12) at Week 12 and Week 24 ; The urticarial control test (UCT) is instrument that works in all types of chronic urticaria (not only for CSU), and it can also be used in urticaria patients suffering from recurrent angioedema.otal score ranges 0 to 16; higher scores indicate better disease control. Scores ≥12 are consistent with well-controlled disease.
12 - Change from baseline in health-related quality-of-life as measured by Dermatology Life Quality Index (DLQI) in patients 16 years old and above, and in Children’s Dermatology Life Quality Index (CDLQI) in patients 12 to 16 years old at Week 12 and Week 24 ; The Dermatology Life Quality Index (DLQI) is a 10-item dermatology-specific health-related quality of life measure. Overall scoring ranges from 0 to 30, with a high score indicative of a poor HRQOL.
The Children’s Dermatology Quality Life Quality Index (CDLQI) is a validated questionnaire designed to measure the impact of skin disease on children’s HRQOL. Overall scoring ranges from 0 to 30. The higher the score, the greater the impact is on the child’s HRQOL.
13 - Change from baseline in Patient Global Impression of Severity (PGIS) of CSU at Week 12 and Week 24; The Patient Global Impression of Severity (PGIS◦) is a one-item questionnaire that asks participants to provide the overall self-assessment of their participant’ disease severity on a 4-point scale for the past week.
14 - Patient Global Assessment of Change (PGIC) of CSU at Week 12 and Week 24; The Patient Global Impression of Change (PGIC) is a one-item questionnaire that asks the participant to provide the overall self-assessment of change in their CSU on a 7-point scale, compared to just before participant started taking the study treatment
15 - Proportion and time to event of patients receiving OCS for CSU during the planned treatment period ;
16 - Safety; Percentage of participants experiencing treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs)
Immunogenicity; Incidence of treatment-emergent ADA against dupilumab over time

1 -2 - Cambio con respecto al momento inicial en la puntuación semanal de la actividad de la urticaria (UAS7 compuesto, el paciente informó puntuación del picor y las ronchas) en la semana 12 y la semana 24. Cambio con respecto al momento inicial en la ISS7 en la semana 12. Cambio con respecto al momento inicial en la puntuación semanal de la intensidad de la urticaria (HSS7) en la semana 12 y en la semana 24
3 - Tiempo hasta la respuesta ISST mínimamente importante (MID) (ISS ≥ 5)
4 - Proporción de pacientes con respuesta ISS7 MID (≥5 puntos) en la semana 12 y en la semana 24
5 - Cambio con respecto al momento inicial de la ISS7 en todos los momentos (el inicio de la acción se evalúa en el primer p < 0,05 que sigue siendo significativo en las medidas posteriores hasta la semana 24)
6 - Cambio con respecto al momento inicial de la UAS7 en la semana 12
7 - 8 Proporción de pacientes con UAS7 ≤6 en la semana 12 y la semana 24; Proporción de pacientes con UAS7 = 0 en la semana 12 y en la semana 24
9 - Cambio con respecto al momento inicial en la puntuación de la actividad del angioedema durante 7 días (AAS7) en la semana 12 y en la semana 24
10 - Cambio con respecto al momento inicial en la prueba de control de la urticaria (UCT) en la semana 12 y en la semana 24
11 - Proporción de pacientes bien controlados (UCT ≥12) en la semana 12 y en la semana 24
12 - Cambio con respecto al momento inicial en la calidad de vida relacionada con la salud (CVRS), medida con el índice de calidad de vida en dermatología (DLQI) en pacientes de ≥16 años, y con el índice de calidad de vida en dermatología infantil (CDLQI) en pacientes de ≥12 a <16 años en la semana 12 y en la semana 24
13 - Impresión global del paciente con respecto a los cambios (IGPC) de la UCE en la semana 12 y en la semana 24
14 - Cambio con respecto al momento inicial en la impresión global del paciente con respecto la intensidad (IGPI) de la UCE en la semana 12 y en la semana 24
15 - Tiempo hasta la presentación del acontecimiento y proporción de pacientes que reciben OCS para UCE durante el período de tratamiento programado
16 - Porcentaje de los participantes que experimentan acontecimientos adversos derivados del tratamiento (AADT) y acontecimientos adversos graves (AAG)
Incidencia de los anticuerpos antifármaco (AAF) derivados del tratamiento contra dupilumab con el paso del tiempo

E.5.2.1

Timepoint(s) of evaluation of this end point

1, 2 : Baseline to Week 12 and Week 24
3 : Baseline over time until Week 24
4 : Week 12 and Week 24
5 : Baseline to Week 24
6 : Baseline to Week 12
7, 8 : Week 12 and Week 24
9, 10 : Baseline to Week 12 and Week 24
11 : Week 12 and Week 24
12, 13 : Baseline to Week 12 and Week 24
14 : Week 12 and Week 24
15 : Baseline over time to Week 24
16 : Baseline to Week 24

1, 2 : Momento inicial a Semana 12 y Semana 24
3 : Momento inicial con el paso del tiempo a Semana 24
4 : Semana 12 y Semana 24
5 : Momento inicial a Semana 24
6 : Baseline to Semana 12
7, 8 : Semana 12 y Semana 24
9, 10 : Momento inicial a Semana 12 y Semana 24
11 : Semana 12 y Semana 24
12, 13 : Momento inicial a Semana 12 y Semana 24
14 : Semana 12 y Semana 24
15 : Momento inicial con el paso del tiempo a Semana 24
16 : Momento inicial a 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

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

Yes

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

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

Argentina

Canada

China

France

Germany

Hungary

Japan

Russian Federation

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

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

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)

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:

204

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

230

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

2020-04-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-03-31

P. End of Trial
P.	End of Trial Status	Ongoing

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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