Clinical Trials Register

Summary
EudraCT Number:	2018-004211-42
Sponsor's Protocol Code Number:	POL6326-009
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

2018-004211-42

A.3

Full title of the trial

An International, Phase 3, Multicenter, Randomized, Open-Label Trial Comparing Balixafortide in combination with Eribulin versus Eribulin alone in Patients with HER2 negative, Locally Recurrent or Metastatic Breast Cancer

Ensayo de fase 3, internacional, multicéntrico, aleatorizado y abierto para comparar balixafortida en combinación con eribulina frente a eribulina en monoterapia en pacientes con cáncer de mama HER2 negativo, localmente recurrente o metastásico

A.3.1

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

A clinical study to compare treatment of Balixafortide in combination with Eribulin or Eribulin alone, in patients with no protein HER2 present in their cells and had locally repeated or spread breast cancer.

Un estudio clínico para comparar el tratamiento de balixafortida en combinación con eribulina frente a eribulina en monoterapia, en pacientes sin proteína HER2 presente en sus células y tienen cáncer de mama repetido o diseminado localmente

A.4.1

Sponsor's protocol code number

POL6326-009

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03786094

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	Polyphor Ltd.
B.1.3.4	Country	Switzerland
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	Polyphor Ltd.
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	Polyphor Ltd.
B.5.2	Functional name of contact point	Clinical Lead
B.5.3	Address:
B.5.3.1	Street Address	Hegenheimermattweg 125
B.5.3.2	Town/ city	Allschwil
B.5.3.3	Post code	CH-4123
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+34900834223
B.5.6	E-mail	RegistroEspanolDeEstudiosClinicos@druginfo.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	Balixafortide
D.3.2	Product code	POL6326
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Balixafortide
D.3.9.1	CAS number	1051366-32-5
D.3.9.3	Other descriptive name	POL6326
D.3.9.4	EV Substance Code	SUB122614
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	35
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	Comparator
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	Halaven
D.2.1.1.2	Name of the Marketing Authorisation holder	Eisai GmbH
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Halaven
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	ERIBULIN
D.3.9.1	CAS number	253128-41-5
D.3.9.4	EV Substance Code	SUB31134
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	0.44
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

Patients with HER2 negative, Locally Recurrent or Metastatic Breast Cancer

Pacientes con cáncer de mama HER2 negativo, localmente recurrente o metastásico

E.1.1.1

Medical condition in easily understood language

Patients with negative HER2-protein cells, that have locally repeated or spread breast cancer

Pacientes sin proteína HER2 presente en sus células que tienen cáncer de mama repetido o diseminado localmente

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

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	10006198
E.1.2	Term	Breast cancer recurrent
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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	10027475
E.1.2	Term	Metastatic breast cancer
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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 evaluate the efficacy of balixafortide + eribulin versus eribulin monotherapy on (i) PFS in the Overall Population and (ii) PFS and objective response rate (ORR) in the 3rd line + population.

Evaluar la eficacia de balixafortida + eribulina en comparación con eribulina en monoterapia en (i) la SSP en la población total y (ii) la SSP y la tasa de respuesta objetiva (TRO) en la población con tratamiento de tercera línea o posterior.

E.2.2

Secondary objectives of the trial

• To compare the OS between patients in the balixafortide + eribulin treatment arm versus eribulin monotherapy treatment arm.
• To compare measures of tumor response between patients in the balixafortide + eribulin treatment arm versus eribulin monotherapy treatment arm.
• To evaluate the safety and tolerability of balixafortide + eribulin versus eribulin

Comparar la SG entre los pacientes del grupo de tratamiento con balixafortida + eribulina y los del grupo de tratamiento con eribulina en monoterapia.
Comparar las mediciones de la respuesta tumoral entre los pacientes del grupo de tratamiento con balixafortida + eribulina y los del grupo de tratamiento con eribulina en monoterapia.
Evaluar la seguridad y la tolerabilidad de balixafortida + eribulina en comparación con eribulina en monoterapia

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Patients at least 18 years of age.
2. Histologically confirmed BC.
3. Metastatic BC currently of stage IV disease by American Joint Committee on Cancer criteria or unresectable locoregionally recurrent BC.
4. Molecular status and prior therapies:
a) Molecular Status
Eligible patients are, by their patient records and prior therapy, HER2 negative with any ER or PgR status. If a previous record of the HR status is not available, the HR status should be tested locally.
HER2 negative (immunohistochemistry [IHC] 0,1 or fluorescence in situ hybridization [FISH] or chromogenic in situ hybridization [CISH] HER2:CEP17 ratio < 2.0); HER2 2+ patients should be ISH/CISH negative.
b) Prior Therapies Patients with locally recurrent or metastatic BC who have previously received 1−4 chemotherapeutic regimens for the treatment of locally recurrent or metastatic BC. Unless contra-indicated for safety reasons, prior therapy will have included an anthracycline and a taxane in either the adjuvant or metastatic setting.
Patients with HR positive status (ER+ and/or PgR+) must have been treated with at least one line of endocrine therapy and considered by the treating physician not to be a candidate for further endocrine therapy.
5. At least 14 days from the completion of any previous cytotoxic chemotherapy, biological therapy, or any other investigational agent at time of initiation of study medication. Resolution of chemotherapy and radiation therapy related toxicities to Grade 1 or lower severity, except for stable sensory neuropathy Grade 2 or lower and alopecia.
6. Patients must have proved refractory to the most recent chemotherapy, documented by progression on or within six (6) months of therapy.
7. Females of childbearing potential must be willing and able to use highly effective contraception (as described in the protocol) whilst they or their male partners are on this study from randomization until 3 months after the last dose of study medication (Section 5.4.5). Male patients must commit to using a barrier method of contraception whilst on treatment and for 3 months after the last dose of study medication (Section 5.4.5).
8. ECOG performance status of 0-2.
9. Life expectancy of 3 months or more.
10. Adequate organ function defined at Screening as:
a) White blood cell (WBC) ≥3000/mm3.
b) Absolute neutrophil count (ANC) ≥1500/mm3.
c) Platelets ≥75000/mm3.
d) Creatinine Clearance ≥30 mL/minute as calculated by the Cockcroft-Gault equation
or serum creatinine <1.5x institutional upper limit of normal (ULN).
e) Total bilirubin ≤1.5x institutional ULN; aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤3x institutional ULN (for patients with liver metastases, ≤5x ULN).
f) Hemoglobin ≥10 g/dL.
11. Patients may have central nervous system involvement if metastases have been treated and are stable for at least 4 weeks after completion of radiation therapy and/or surgery.
Stable is defined as the absence of the need for dexamethasone or other corticosteroid therapy, and radiographic confirmation of SD.
12. Patients may be receiving bone-modifying agents (BMA [bisphosphonates or denosumab]) if BMA was initiated at least 4 weeks prior to Day 1.
13. Willing and able to comply with the protocol and able to understand and willing to sign an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved written informed consent document.

1. Edad mínima de 18 años.
2. CM confirmado histológicamente.
3. CM metastásico actualmente en estadio IV según los criterios del American Joint Committee on Cancer o CM con recidiva locorregional irresecable.
4. Estado molecular y tratamientos previos:
a) Estado molecular
Podrán participar pacientes que, según sus registros y tratamientos previos, sean HER2 negativos, con cualquier estado de RE o RPg. Si no se dispone de un registro previo del estado de RH, se tendrá que determinar el estado de RH mediante análisis en un laboratorio local.
Negatividad para el HER2 (inmunohistoquímica [IHQ] 0,1 o hibridación in situ con fluorescencia [FISH] o hibridación in situ cromógena [CISH] cociente HER2:CEP17 < 2,0); los pacientes HER2 2+ deberán obtener un resultado negativo en la FISH/CISH.
b) Tratamientos previos
Pacientes con CM localmente recurrente o metastásico que hayan recibido anteriormente 1-4 pautas de quimioterapia para el tratamiento de un CM localmente recurrente o metastásico. A menos que esté contraindicado por motivos de seguridad, el tratamiento previo habrá incluido una antraciclina y un taxano como tratamiento adyuvante o para la enfermedad metastásica.
Los pacientes con estado positivo para los RH (RE+ o RPg+) deberán haber recibido al menos una línea de tratamiento endocrino y el médico responsable de su tratamiento deberá considerar que no son candidatos para recibir tratamiento endocrino adicional.
5. Al menos 14 días después de la finalización de cualquier tratamiento previo con quimioterapia citotóxica, un fármaco biológico o algún otro fármaco en investigación en el momento de iniciar la medicación del estudio. Resolución de las toxicidades relacionadas con la quimioterapia y la radioterapia hasta un grado 1 o inferior de intensidad, salvo neuropatía sensitiva estable de grado 2 o inferior y alopecia.
6. Los pacientes deberán haber mostrado resistencia a la quimioterapia más reciente, documentada por progresión durante el tratamiento o en los seis (6) meses siguientes.
7. Las mujeres en edad fértil deberán tener voluntad y capacidad para utilizar un método anticonceptivo muy eficaz (según se describe en el protocolo) durante su participación o la de sus parejas masculinas en el estudio, desde la aleatorización hasta 3 meses después de la última dosis de la medicación del estudio (sección 5.4.5). Los varones deberán comprometerse a utilizar un método anticonceptivo de barrera durante el tratamiento y hasta 3 meses después de recibir la última dosis de la medicación del estudio (sección 5.4.5).
8. Estado funcional del ECOG de 0-2.
9. Esperanza de vida de 3 meses o más.
10. Función orgánica adecuada, definida en la selección como:
a) Recuento de leucocitos ≥ 3000/mm3.
b) Recuento absoluto de neutrófilos (RAN) ≥ 1500/mm3.
c) Recuento de plaquetas ≥75000/mm3.
d) Aclaramiento de creatinina ≥ 30 ml/minuto calculado mediante la ecuación de Cockcroft-Gault o creatinina sérica <1,5 veces el límite superior de la normalidad (LSN) del centro.
e) Bilirrubina total ≤ 1,5 veces el LSN del centro; aspartato aminotransferasa (AST) y alanina aminotransferasa (ALT) ≤ 3 veces el LSN del centro (en los pacientes con metástasis hepáticas, ≤ 5 veces el LSN).
f) Hemoglobina ≥ 10 g/dl.
11. Los pacientes podrán presentar afectación del sistema nervioso central si las metástasis se han tratado y se mantienen estables durante al menos 4 semanas después de finalizar la radioterapia o la cirugía. Estable se define como la ausencia de necesidad de dexametasona u otros corticosteroides y la confirmación radiológica de EE.
12. Los pacientes podrán estar recibiendo fármacos modificadores del hueso (bisfosfonatos o denosumab) si su administración se inició al menos 4 semanas antes del día 1.
13. Disposición y capacidad de cumplir el protocolo y de comprender y estar dispuestos a firmar un documento de consentimiento informado por escrito aprobado por un comité ético de investigación clínica (CEIC) independiente

E.4

Principal exclusion criteria

1. Previously received eribulin.
2. Peripheral neuropathy Grade ≥3.
3. Receipt of prior CXCR4 therapy.
4. Receipt of colony stimulating factors (CSFs) filgrastim, pegfilgrastim, or sargramostim within 14 days prior to study Day 1.
5. Radiation therapy within 14 days prior to study Day 1.
6. Severe concurrent illness or psycho-social situation that would limit compliance with study requirements or that, in the Investigator’s opinion, would preclude enrolment.
7. History of allergic reactions attributed to compounds of similar chemical or biologic composition to balixafortide or eribulin or other agents used in the study.
8. Breast feeding or pregnant, as determined by a serum pregnancy test beta human chorionic gonadotrophin (β-HCG) at Screening, prior to the administration of study medication.
9. Patients with congestive heart failure, electrolyte abnormalities, bradyarrhythmias, known congenital long QT syndrome, QT interval corrected with Fridericia's formula (QTcF) ≥470 msec at baseline in the absence of bundle branch block, or currently taking drugs at known risk of prolonging the QT interval or causing torsades de pointes (including Class Ia and III anti-arrhythmic drugs; see also Appendix 1 Prohibited Medications).
Patients with hypokalemia or hypomagnesemia should not receive eribulin until the hypokalemia or hypomagnesemia is corrected.

1. Tratamiento previo con eribulina.
2. Neuropatía periférica de grado ≥ 3
3. Tratamiento previo con CXCR4.
4. Tratamiento previo con los factores estimuladores de colonias (CSF) filgrastim, pegfilgrastim o sargramostim en los 14 días previos al día 1 del estudio.
5. Radioterapia en los 14 días previos al día 1 del estudio.
6. Enfermedad concurrente grave o situación psicosocial que limitaría el cumplimiento de los requisitos del estudio o que, en opinión del investigador, impediría el reclutamiento.
7. Antecedentes de reacciones alérgicas atribuidas a compuestos de composición química o biológica similar a la balixafortida, la eribulina u otros fármacos utilizados en el estudio.
8. Lactancia o embarazo, según lo determinado mediante una prueba de embarazo en suero con gonadotropina coriónica humana beta (β-HCG) en la selección, antes de la administración de la medicación del estudio.
9. Pacientes con insuficiencia cardíaca congestiva, anomalías electrolíticas, bradiarritmias, síndrome del QT largo congénito conocido, intervalo QT corregido con la fórmula de Fridericia (QTcF) ≥ 470 ms en el momento basal en ausencia de bloqueo de rama, o tratamiento actual con fármacos que tengan un riesgo conocido de prolongar el intervalo QT o causar taquicardia helicoidal (como los antiarrítmicos de clase Ia y III; véase también el Apéndice 1 Medicamentos prohibidos).
Los pacientes con hipopotasemia o hipomagnesemia no deberán recibir eribulina hasta que se corrija la hipopotasemia o la hipomagnesemia

E.5 End points

E.5.1

Primary end point(s)

PFS time (defined as the time from randomization to the first progression or death, as judged by an IRC) is the primary endpoint in the Overall Population. PFS will be analyzed via Cox regression modelling in the intent-to-treat (ITT) population. Patients free from progression and death will be censored at their last follow-up visit. The analysis will be stratified for randomization stratification factors and a fixed effect term will be included for randomized treatment. The hazard ratio will be estimated from the model along with the associated confidence interval (CI) and 2-sided p value. The data will also be displayed using Kaplan- Meier curves and median PFS times will be estimated.

El tiempo de SSP (definido como el tiempo transcurrido desde la aleatorización hasta la primera progresión o la muerte, según el criterio de un CRI) es el criterio de valoración principal en la población total. La SSP se analizará utilizando un modelo de regresión de Cox en la población por intención de tratar (IT). Los pacientes sin progresión ni muerte se censurarán en su última visita de seguimiento. El análisis se estratificará en función de los factores de estratificación de la aleatorización y se incluirá el tratamiento aleatorizado como un término de efecto fijo. El modelo permitirá calcular la razón de riesgos instantáneos, junto con el intervalo de confianza (IC) asociado y el valor p bilateral. Los datos se presentarán también utilizando curvas de Kaplan-Meier y se calculará la mediana de los tiempos de SSP

E.5.1.1

Timepoint(s) of evaluation of this end point

Every 6 weeks (±7 days) during the first year, and then every 12 weeks (±7 days), thereafter, until Progressed Disease (PD) is documented

Cada 6 semanas (± 7 días) durante el primer año, y luego cada 12 semanas (± 7 días), a partir de entonces, hasta que se documente la enfermedad progresiva (EP)

E.5.2

Secondary end point(s)

OS will be the key secondary efficacy endpoint in the Overall Population. OS will be analyzed in a fashion similar to that described for PFS.

La SG será el criterio de valoración secundario fundamental de la eficacia en la población total. La SG se analizará de forma similar a la descrita para la SSP.

E.5.2.1

Timepoint(s) of evaluation of this end point

every 6 weeks (±7 days) during the first year, and then every 12 weeks (±7 days), thereafter, until PD is documented

Cada 6 semanas (± 7 días) durante el primer año, y luego cada 12 semanas (± 7 días), a partir de entonces, hasta que se documente la EP

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

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

Biomarker testing in Blood, Plasma and tumor tissue

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

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

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)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Eribulin

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

Belgium

Brazil

Czech Republic

France

Germany

Italy

Korea, Republic of

Russian Federation

Spain

Taiwan

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

The end of the study (study completion) is defined as the date on which all patients have completed the 30-day Safety Follow-up and when the final OS analysis is completed, this is expected to be 2 years after the last patient is enrolled on study.

El final del estudio (finalización del estudio) se define como la fecha en la que todos los pacientes han completado el Seguimiento de seguridad de 30 días y cuando se completa el análisis final de la SG, se espera que esto ocurra 2 años después de que el último paciente sea incluido en el estudio.

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:

357

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

100

F.4.2.2

In the whole clinical trial

384

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)

Standard of care

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

2019-05-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-04-26

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2021-08-04

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