Clinical Trials Register

Summary
EudraCT Number:	2018-004774-97
Sponsor's Protocol Code Number:	C-935788-057
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:	2019-06-11
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

2018-004774-97

A.3

Full title of the trial

A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Estudio de fase III, multicéntrico, aleatorizado, doble ciego y controlado con placebo del fostamatinib disódico en el tratamiento de la anemia hemolítica autoinmunitaria por anticuerpos calientes

A.3.1

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

This is a Phase 3 multi-center, randomized, double-blind, placebo-controlled, parallel group study to investigate the efficacy of 24 weeks of treatment with fostamatinib (R788) vs. placebo in increasing hemoglobin in subjects with warm antibody autoimmune hemolytic anemia (wAIHA) who have failed at least one prior treatment regimen.

Se plantea un ensayo clínico de fase III, multicéntrico, aleatorizado, doble ciego, controlado con placebo y en grupos paralelos, para estudiar la eficacia de 24 semanas de tratamiento con fostamatinib (R788), en comparación con un placebo, a fin de incrementar la hemoglobina de los sujetos con anemia hemolítica autoinmunitaria por anticuerpos calientes (AHAIc) que no han respondido al menos a una pauta de tratamiento con anterioridad.

A.4.1

Sponsor's protocol code number

C-935788-057

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	Rigel Pharmaceuticals, Inc.
B.1.3.4	Country	United States
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	Rigel Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Pharm-Olam International (Spain) S.L.U
B.5.2	Functional name of contact point	Regulatory Department
B.5.3	Address:
B.5.3.1	Street Address	C/Antracita, 7 Planta 1 A Nave 6
B.5.3.2	Town/ city	Madrid
B.5.3.3	Post code	28045
B.5.3.4	Country	Spain
B.5.4	Telephone number	(+) 34 91-145 91 10
B.5.5	Fax number	(+) 34 91-434-27 73
B.5.6	E-mail	regulatory.spain@pharm-olam.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	Fostamatinib Disodium
D.3.2	Product code	R935788
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	Fostamatinib
D.3.9.1	CAS number	914295-16-2
D.3.9.2	Current sponsor code	Fostamatinib Disodium R935788
D.3.9.3	Other descriptive name	R788, fostamatinib disodium hexahydrate (in USPI/FPI)
D.3.9.4	EV Substance Code	SUB32625
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	100
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	Fostamatinib Disodium
D.3.2	Product code	R935788
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	Fostamatinib
D.3.9.1	CAS number	914295-16-2
D.3.9.2	Current sponsor code	Fostamatinib Disodium R935788
D.3.9.3	Other descriptive name	R788, fostamatinib disodium hexahydrate (in USPI/FPI)
D.3.9.4	EV Substance Code	SUB32625
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	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	Film-coated tablet
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	Film-coated tablet
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

Warm antibody autoimmune hemolytic anemia (wAIHA)

Anemia hemolítica autoinmunitaria por anticuerpos calientes (AHAIc)

E.1.1.1

Medical condition in easily understood language

Autoimmune hemolytic anemia (AIHA) is an acquired disorder manifested by autoantibody- mediated red blood cell (RBC) destruction.

La anemia hemolítica autoinmunitaria (AHAI) es un trastorno adquirido que se manifiesta por la destrucción de los glóbulos rojos mediada por autoanticuerpos.

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	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10003825
E.1.2	Term	Autoimmune hemolytic anemia
E.1.2	System Organ Class	100000004851

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).

El objetivo principal de este estudio es evaluar la eficacia del fostamatinib en pacientes con anemia hemolítica autoinmunitaria por anticuerpos calientes (AHAIc).

E.2.2

Secondary objectives of the trial

The secondary objective of this study is to assess the safety of fostamatinib in subjects with wAIHA.

El objetivo secundario de este estudio es evaluar la seguridad del fostamatinib en pacientes con AHAIc.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject must be willing and able to give written informed consent by signing an IRB approved Informed Consent Form prior to undergoing any study-specific procedures.
2. Subject must have a diagnosis of primary or secondary wAIHA as documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-Ig A. Eligibility may be based on a historical DAT obtained within 12 months of the screening visit from a local laboratory, provided that specific IgG positivity is documented; otherwise, this assay will be done at screening by a central laboratory.
3. Has failed or not tolerated at least one prior wAIHA treatment, e.g., steroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil (MMF), danazol, vincristine, ESA or splenectomy (folate, iron or other supplements do not fulfill this criterion).
4. Has haptoglobin <LLN or total bilirubin >ULN or lactate dehydrogenase (LDH) >ULN.
5. At screening, subject’s hemoglobin level must be ≤9 g/dL
OR
If the hemoglobin value is >9 g/dL and <10 g/dL, subject must be on an allowed wAIHA treatment (see Allowed AIHA Therapy table) AND the subject must have documented symptoms related to anemia (e.g., weakness, dizziness, fatigue, shortness of breath, chest pain).
6. Male or female at least 18 years of age at screening.
7. Karnofsky performance status (KPS) ≥70.
8. Subject’s concurrent treatment for wAIHA may consist of no more than two of any of the following agents: azathioprine, steroids, ESAs, mycophenolate mofetil, dapsone or danazol at a stable dose, as defined in the Allowed AIHA Therapies table.
9. Female subjects must be either post-menopausal for at least 1 year or surgically sterile; or, if of childbearing potential, must not be pregnant or lactating and must agree to use a highly effective method of birth control throughout the duration of the trial and for 30 days following the last dose. Acceptable methods of birth control are defined as: hormonal contraception (pill, injection or implant) used consistently for at least 30 days prior to screening, an intrauterine device (IUD), or intrauterine hormone-releasing system (IUS), or true abstinence (i.e. abstinence is in
line with the preferred and usual lifestyle of the subject).
10. In the investigator’s opinion, the subject has the ability to understand the nature of the study and any hazards of participation and to communicate satisfactorily with the investigator.

1. Voluntad y capacidad de prestar el consentimiento informado por escrito, mediante la firma del correspondiente documento aprobado por el comité de ética, antes de proceder a las actividades específicas del ensayo.
2. Diagnóstico de AHAIc primaria o secundaria, documentado por un positivo en una prueba de antiglobulina directa (PAD) específica contra la IgG o la Ig A. La PAD puede haberse realizado en los 12 meses anteriores a la visita de selección y en un laboratorio local, siempre que se haya documentado la positividad específica de IgG; si no, se realizará en el momento de la selección a través del laboratorio centralizado.
3. Ausencia de respuesta o de tolerancia al menos a un tratamiento anterior contra la AHAIc, p. ej., corticoesteroides, rituximab, azatioprina, ciclofosfamida, ciclosporina, micofenolato de mofetilo (MMF), danazol, vincristina, estimulantes de la eritropoyesis o esplenectomía (no se incluyen en este criterio los folatos, el hierro y otros suplementos).
4. Haptoglobina < LIN o bilirrubina total > LSN o lactato-deshidrogenasa (LDH) > LSN.
5. En la selección, nivel de hemoglobina ≤ 9 g/dl
o bien,
si el nivel de hemoglobina es > 9 g/dl e < 10 g/dl, el sujeto debe estar siguiendo un tratamiento permitido contra la AHAIc (véase la tabla de tratamientos permitidos) y, ADEMÁS, debe presentar sintomatología documentada en relación con la anemia (p. ej., debilidad, mareos, cansancio, disnea, dolor torácico, etc.).
6. Sexo masculino o femenino y edad ≥ 18 años en el momento de la selección.
7. Estado funcional ≥ 70 en la escala de Karnofsky.
8. El tratamiento concurrente contra la AHAIc debe comprender, como máximo, dos de los siguientes productos: azatioprina, corticoesteroides, estimulantes de la eritropoyesis, micofenolato de mofetilo, dapsona o danazol, en dosis estables, según se define en la tabla de tratamientos permitidos contra la AHAIc.
9. Las mujeres deben ser posmenopáusicas desde al menos un año antes o haberse sometido a una esterilización quirúrgica; de lo contrario, si tienen capacidad de concebir, no deben estar embarazadas ni en período de lactancia y deben comprometerse a utilizar métodos anticonceptivos de gran eficacia durante todo el ensayo y durante los 30 días siguientes a la última dosis. Se aceptan los métodos siguientes: anticonceptivos hormonales (píldora, inyecciones o implantes) empleados de forma constante durante como mínimo los 30 días antes de la selección; dispositivo intrauterino (DIU) o sistema intrauterino (SIU) de liberación hormonal; o abstinencia verdadera (es decir, la abstinencia sexual es conforme con los hábitos y preferencias personales del sujeto).
10. En opinión del investigador, capacidad para comprender la naturaleza del estudio y los riesgos de la participación y de comunicarse satisfactoriamente con el investigador.

E.4

Principal exclusion criteria

1. Subject with other types of AIHA (e.g., cold antibody AIHA, cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria).
2. Subject has AIHA secondary to autoimmune disease, including systemic lupus erythematosus (SLE), or lymphoid malignancy if the underlying disease is not stable or is not well-controlled on current therapy, per investigator medical judgment.
3. Subject has a history of or active, clinically significant, cardiovascular, respiratory, gastrointestinal, renal, hepatic, neurological, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorder that, in the investigator’s opinion, could affect the conduct of the study or the absorption, metabolism or excretion of the study drug.
4. Subject has uncontrolled or poorly controlled hypertension, defined as systolic blood pressure ≥135 mmHg or diastolic blood pressure ≥85 mmHg, whether or not the subject is receiving anti-hypertensive treatment.
5. Subject has one or more of the following laboratory abnormalities at screening: neutrophil count of <1,000/µL or platelet count of <30,000/μL, unless due to Evans syndrome; transaminase levels (i.e., alanine aminotransferase [ALT] or aspartate aminotransferase [AST]) >1.5 x ULN.
6. Has documented active hepatitis B or hepatitis C infection or HIV infection.
7. Subject is currently enrolled in an investigational drug or device study or has used an investigational drug or device within 30 days or 5 half-lives (whichever is longer) of Day 1.
8. In the judgment of the investigator, the subject may not be able to fully comply with study requirements.
9. Subject has been treated with fostamatinib previously for any indication.
10. Subject has a known allergy and/or sensitivity to the test article or its components.
11. Subject has had a splenectomy within the past 4 weeks.

1. Otros tipos de AHAI (p. ej., AHAI por crioanticuerpos, síndrome de crioaglutininas, AHAI de tipo mixto o hemoglobinuria paroxística por frío).
2. AHAI secundaria a enfermedad autoinmunitaria, como el lupus eritematoso sistémico (LES), o neoplasia linfática maligna, en el supuesto de que la enfermedad subyacente no esté estabilizada o controlada correctamente con el tratamiento actual, según el criterio médico del investigador.
3. Antecedentes o presencia de trastornos cardiovasculares, respiratorios, digestivos, renales, hepáticos, neurológicos, psiquiátricos, osteomusculares, genitourinarios, dermatológicos o de otra índole que tengan relevancia clínica y que, a juicio del investigador, puedan afectar a la realización del ensayo o a la absorción, el metabolismo o la excreción del fármaco en estudio.
4. Hipertensión arterial mal controlada o incontrolada, definida como una tensión sistólica ≥ 135 mmHg o diastólica ≥ 85 mmHg, independientemente de que el sujeto siga tratamiento antihipertensor.
5. Presencia de al menos una de las alteraciones analíticas siguientes en el momento de la selección: cifra de neutrófilos < 1.000 /µl, o de plaquetas < 30.000 /μl, salvo que se deba a un síndrome de Evans; transaminasas (alanina-aminotransferasa [ALAT] o aspartato-aminotransferasa [ASAT]) > 1,5 × LSN.
6. Infección activa documentada por el virus de la hepatitis B o la hepatitis C o infección por el VIH.
7. Participación simultánea en un estudio con un fármaco o un producto sanitario en investigación o utilización de un fármaco o producto sanitario en investigación en los 30 días o 5 semividas antes del día 1 (lo que resulte más prolongado).
8. A juicio del investigador, incapacidad para cumplir adecuadamente los requisitos del estudio.
9. Antecedentes de tratamiento con fostamatinib, por cualquier indicación.
10. Alergia o sensibilidad conocidas al producto experimental o a cualquiera de los ingredientes.
11. Esplenectomía en las últimas 4 semanas.

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy endpoint is the proportion of subjects who achieve a durable response.
A hemoglobin response is defined as a hemoglobin level of >10 g/dL and ≥2 g/dL higher than the baseline (Day 1) value if, during the previous 4 weeks, the steroid dose was maintained at the baseline level and rescue medication was not administered.
A durable response is defined as a hemoglobin response on at least 3 scheduled visits during the 24-week evaluation period.

El criterio principal de valoración de la eficacia es la proporción de sujetos que alcanzan una respuesta duradera.
Por respuesta hemoglobínica se entenderá un nivel de hemoglobina > 10 g/dl y ≥ 2 g/dl más alto que el valor basal (día 1) siempre que, durante las últimas cuatro semanas, se haya mantenido la dosis basal del corticoesteroide y no se haya administrado medicación de rescate.
Por respuesta duradera se entenderá una respuesta hemoglobínica en al menos tres visitas programadas durante el período de evaluación de 24 semanas.

E.5.1.1

Timepoint(s) of evaluation of this end point

24 week evaluation

Evaluación de 24 semanas

E.5.2

Secondary end point(s)

• Proportion of subjects with a hemoglobin response by Week 24
• Proportion of subjects requiring wAIHA rescue therapy
• Average number of weeks with hemoglobin response

Safety Endpoints:
• Incidence of adverse events
• Incidence of abnormal changes from baseline in laboratory values per
CTCAE criteria V 5.0 (e.g., hematology, chemistry)
• Incidence of changes in blood pressure compared to baseline

- Proporción de sujetos con respuesta hemoglobínica a la semana 24.
- Proporción de sujetos que necesitan tratamiento de rescate contra la AHAIc.
- Promedio de semanas con respuesta hemoglobínica.

Criterios de valoración de la seguridad:
•Incidencia de los acontecimientos adversos.
•Incidencia de las anomalías analíticas respecto a los valores basales, con arreglo a la versión 5.0 de los criterios CTCAE (p. ej., hematimetría, bioquímica).
•Incidencia de las alteraciones en la tensión arterial, respecto a los valores basales.

E.5.2.1

Timepoint(s) of evaluation of this end point

24 week evaluation

Evaluación de 24 semanas

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

Yes

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

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

Austria

Belarus

Belgium

Bulgaria

Canada

Czech Republic

Denmark

France

Georgia

Germany

Hungary

Italy

Netherlands

Norway

Romania

Russian Federation

Serbia

Spain

Ukraine

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

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

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)

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)

Extended treatment will be available to eligible subjects under a separate a protocol. All other subjects will return to standard treatment.

El tratamiento extendido estará disponible para los pacientes elegibles en un protocolo diferente. Todos los demás pacientes volverán al tratamiento estándar.

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

2019-09-09

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-07-19

P. End of Trial
P.	End of Trial Status	Completed

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