Clinical Trials Register

Summary
EudraCT Number:	2016-000965-21
Sponsor's Protocol Code Number:	OP-106
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:	2016-08-11
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

2016-000965-21

A.3

Full title of the trial

A Single Arm, Open-Label, Phase 2 Study of Melflufen in Combination with Dexamethasone in Patients with Relapsed Refractory Multiple Myeloma who are Refractory to Pomalidomide and/or Daratumumab.

Estudio de fase II, abierto y de un solo grupo, de melflufen en combinación con dexametasona en pacientes con mieloma múltiple en recaída refractario que presentan resistencia a pomalidomida y/o daratumumab.

A.3.1

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

A Single Arm, Open-Label, Phase 2 Study of Melflufen in Combination with Dexamethasone in Patients with Relapsed Refractory Multiple Myeloma who are Refractory to Pomalidomide and/or Daratumumab.

A.4.1

Sponsor's protocol code number

OP-106

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	Oncopeptides AB
B.1.3.4	Country	Sweden
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	Oncopeptides AB
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Oncopeptides AB
B.5.2	Functional name of contact point	Clinical Operations Director
B.5.3	Address:
B.5.3.1	Street Address	Västra Trädgårdsgatan 15
B.5.3.2	Town/ city	Stockholm
B.5.3.3	Post code	SE-111 53
B.5.3.4	Country	Sweden
B.5.6	E-mail	sara.thuresson@oncopeptides.se

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	Yes
D.2.5.1	Orphan drug designation number	EMA/OD/293/14
D.3 Description of the IMP
D.3.1	Product name	Melflufen
D.3.2	Product code	J1
D.3.4	Pharmaceutical form	Powder for 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	Melflufen
D.3.9.2	Current sponsor code	116362
D.3.9.3	Other descriptive name	J1
D.3.9.4	EV Substance Code	SUB22033
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	20
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	Yes
D.2.1.2	Country which granted the Marketing Authorisation	Spain
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	Dexamethasone
D.3.4	Pharmaceutical form	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	DEXAMETHASONE
D.3.9.1	CAS number	50-02-2
D.3.9.4	EV Substance Code	SUB07017MIG
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	40
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 Relapsed Refractory Multiple Myeloma who are Refractory to Pomalidomide and/or Daratumumab

Pacientes con Mieloma Múltiple en Recaída Refractario que presentan Resistencia a Pomalidomida y/o Daratumumab

E.1.1.1

Medical condition in easily understood language

Patients with Multiple Myeloma treated with at least 2 lines of prior therapy, including an immunomodulator and a proteasone inhibitor, and who are refractory to pomalidomide and/or daratumumab

Pacientes con Mieloma Múltiple tratados como mínimo con 2 líneas previas de tratamiento, incluido un inmunomodulador y un inhibidor de proteasomas y sean resistententes a pomalidomida y/o daratumumab

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	16.1
E.1.2	Level	HLT
E.1.2	Classification code	10028229
E.1.2	Term	Multiple myelomas
E.1.2	System Organ Class	100000004851

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess overall response rate (ORR), i.e. proportion of patients with ≥ Partial response (PR) [stringent CR (sCR), complete response (CR), very good partial response (VGPR), and PR] as best response in efficacy evaluable patients according to IMWG Uniform Response Criteria , for the pomalidomide and daratumumab refractory groups separately.

Evaluar la tasa de respuesta global (TRG); es decir, la proporción de pacientes con ≥ respuesta parcial (RP) [RC rigurosa (RCr), respuesta completa (RC), respuesta parcial muy buena (RPMB) y RP] como la mejor respuesta en pacientes evaluables para eficacia según los criterios de respuesta uniforme del Grupo de Trabajo Internacional del Mieloma (IMWG) para los grupos de pacientes con resistencia a pomalidomida y daratumumab (por separado).

E.2.2

Secondary objectives of the trial

Key Secondary Objectives
- To assess PFS according to IMWG Criteria
- To assess duration of response (DOR) in efficacy evaluable patients with ≥ PR (sCR, CR, VGPR, PR) as best response according to IMWG Uniform Response Criteria
- To assess Overall Survival (OS)

Other Secondary Objectives
- To assess clinical benefit rate (CBR, i.e. proportion of patients with ≥ MR) as best response in efficacy evaluable patients, time to response (TTR), time to progression (TTP)
- To assess the safety and tolerability

Objetivos secundarios clave
- Evaluar la SSP según los criterios del IMWG
- Evaluar la duración de la respuesta (DR) en pacientes evaluables para eficacia con ≥ RP (RCr, RC, RPMB, RP) como la mejor respuesta según los criterios de respuesta uniforme del IMWG
- Evaluar la supervivencia global (SG)

Otros objetivos secundarios
- Evaluar la tasa de beneficio clínico (TBC) (es decir, la proporción depacientes con ≥ RM) como la mejor respuesta en pacientes evaluables para eficacia, el tiempo transcurrido hasta la respuesta (TTR) y el tiempo transcurrido hasta la progresión (TTP)
- Evaluar la seguridad y la tolerabilidad

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or female, age 18 years or older.
2 A prior diagnosis of multiple myeloma with documented disease progression in need of treatment at time of screening.
3. Measurable disease defined as any of the following:
 Serum monoclonal protein ≥ 0.5 g/dL (≥ 5 g/L) by protein electrophoresis (SPEP)
 ≥ 200 mg/24 hours of monoclonal protein in the urine on 24-hour urine electrophoresis (UPEP)
 Serum immunoglobulin free light chain ≥10 mg/dL (≥ 100 mg/L) AND abnormal serum immunoglobulin kappa to lambda free light chain ratio
4. A minimum of 2 prior lines of therapy, including an IMiD and a PI, and is refractory to pomalidomide and/or daratumumab. (Refractory status includes patients who relapse while on therapy or within 60 days of last dose).
5. Life expectancy of ≥ 6 months.
6. ECOG performance status ≤ 2. (Patients with worse performance status based solely on bone pain secondary to multiple myeloma will be eligible).
7. Female of child bearing potential (FCBP)* and non-vasectomized male agree to practice appropriate methods of birth control (See Section 7.6.1).
8. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information.
9. 12-lead ECG with QTcF interval of ≤ 470 msec (Appendix H).
10. The following laboratory results must be met during screening (within 21 days) and also prior to study drug administration on Cycle 1 Day 1:
 Absolute neutrophil count (ANC) ≥ 1,000 cells/mm3 (1.0 x 109/L) (Growth factors cannot be used within 10 days before first drug administration)
 Platelet count ≥ 75,000 cells/mm3 (75 x 109/L) (without transfusions required during the 10 days prior to initiation of therapy)
 Hemoglobin ≥ 8.0 g/dl (RBC transfusions are permitted)
 Total Bilirubin ≤ 1.5 x upper limit of normal (ULN) or patients diagnosed with Gilberts syndrome, have been reviewed and approved by the medical monitor).
 AST (SGOT) and ALT (SGPT) ≤ 3.0 x ULN
 Renal function: Estimated creatinine clearance by Cockcroft-Gault formula ≥ 45 mL/min. (Appendix G)
11. Must have, or accept to have, an acceptable central catheter for infusion of melflufen (Port a cath, PICC line, or central venous catheter).
*(FCBP) is any sexually mature female who: 1) has not undergone a hysterectomy or bilateral oophorectomy or 2) has not been naturally postmenopausal (not having menstrual cycles due to cancer therapy does not rule out childbearing potential) for at least 24 consecutive months.

1. Hombre o mujer de 18 años o más.
2. Diagnóstico previo de mieloma múltiple con enfermedad progresiva documentada que precisa tratamiento en el momento de la selección.
3. Enfermedad medible que viene determinada por cualquiera de los siguientes criterios:
 Proteína monoclonal sérica ≥0,5 g/dl (≥5 g/l) en la electroforesis de proteínas en suero (EFPS).
 Proteína monoclonal urinaria ≥200 mg/24 horas en la electroforesis de proteínas en orina (EFPU) de 24 horas.
 Cadena ligera libre de inmunoglobulinas en suero ≥10 mg/dl (≥100 mg/l) Y relación anómala de las cadenas ligeras libres de las inmunoglobulinas kappa y lambda en suero.
4. Un mínimo de dos líneas previas de tratamiento, incluido un IM y un IP, y mostrar resistencia a pomalidomida y/o daratumumab (el término “resistencia” hace referencia a los pacientes con recidivas durante el tratamiento o en los 60 días siguientes a la administración de la última dosis de pomalidomida y /o daratumumab, en cualquier línea de tratamiento y con independencia de la respuesta obtenida).
5. Esperanza de vida ≥6 meses.
6. Puntuación ≤2 en la escala de estado funcional del Grupo Oncológico Cooperativo de la Costa Este (ECOG). (Los pacientes que obtengan una peor puntuación en el estado funcional basado exclusivamente en el dolor óseo secundario a mieloma múltiple serán aptos para participar).
7. Mujeres en edad fértil (MEF)* y hombres no vasectomizados que acepten utilizar métodos anticonceptivos adecuados (Sección 7.6.1).
8. Capacidad de entender la finalidad y los riesgos del estudio, firmar y fechar un consentimiento informado y permitir el uso de la información médica protegida.
9. ECG de 12 derivaciones con intervalo QTc calculado por la fórmula de Fridericia (QTcF) de ≤470 ms (Anexo H).
10. Los resultados de laboratorio que se indican a continuación deben alcanzarse durante la selección (en un plazo de 21 días) y también antes de la administración del fármaco del estudio en el día 1 del ciclo 1.
 Recuento absoluto de neutrófilos (RAN) ≥1000 células/mm3 (1,0 x 109/l) (no pueden utilizarse factores de crecimiento en los 10 días previos a la primera administración del fármaco).
 Recuento de plaquetas ≥75 000 células/mm3 (75 x 109/l) (no está permitido realizar transfusiones en los 10 días previos al inicio del tratamiento).
 Hemoglobina ≥8,0 g/dl (está permitido realizar transfusiones de eritrocitos).
 Bilirrubina total ≤1,5 x límite superior de la normalidad (LSN) o pacientes diagnosticados de síndrome de Gilbert, que han sido analizados y aprobados por el supervisor médico.
 Aspartato-aminotransferasa (ASAT) y alanina-aminotransferasa (ALAT) ≤3,0 x LSN.
 Función renal: Eliminación estimada de la creatinina según la fórmula de Cockcroft-Gault de ≥45 ml/min. (Anexo G)
11. Debe tener o aceptar tener un catéter central aceptable para la perfusión de melflufen antes de la administración de la primera dosis. (Catéter implantable, catéter central de inserción periférica [CCIP] o catéter venoso central).
*(MEF) es una mujer sexualmente madura que: (1) no se ha sometido a una histerectomía u ooforectomía bilateral o (2) no ha tenido la menopausia de forma natural (no tener los ciclos menstruales debido a un tratamiento oncológico no excluye la posibilidad de quedarse embarazada) durante al menos 24 meses consecutivos.

E.4

Principal exclusion criteria

1. Evidence of mucosal or internal bleeding and/or is platelet transfusion refractory (i.e., unable to maintain a platelet count ≥ 75,000 cells/mm3)
2. Any medical conditions that, in the Investigator’s opinion, would impose excessive risk to the patient or would adversely affect his/her participating in this study. Examples of such conditions are: a significant history of cardiovascular disease (e.g., myocardial infarction (MI), significant conduction system abnormalities, uncontrolled hypertension, ≥ grade 3 thromboembolic event in the last 6 months).
3. Known active infection requiring parenteral or oral anti-infective treatment within 14 days of initiation of treatment.
4. Primary refractory (never responded (≥ MR) to any prior therapy)
5. Other malignancy diagnosed or requiring treatment within the past 3 years with the exception of adequately treated basal cell carcinoma, squamous cell skin cancer, carcinoma in-situ of the cervix or breast.
6. Pregnant or breast-feeding females
7. Serious psychiatric illness, active alcoholism, or drug addiction that may hinder or confuse compliance or follow-up evaluation
8. Known HIV or active hepatitis B or C viral infection
9. Concurrent symptomatic amyloidosis or plasma cell leukemia
10. POEMS syndrome [plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein (M-protein) and skin changes]
11. Previous cytotoxic therapies, including cytotoxic investigational agents, for multiple myeloma within 3 weeks (6 weeks for nitrosoureas) prior to start of study treatment. IMiDs, PIs and or corticosteroids within 2 weeks prior to start of study treatment. Investigational therapies and monoclonal antibodies within 4 weeks of start of study therapy. Prednisone up to but no more than 10 mg orally q.d. or its equivalent for symptom management of comorbid conditions is permitted but dose should be stable for at least 7 days prior to study treatment.
12. Residual side effects to previous therapy > grade 1 prior to initiation of therapy (Alopecia any grade and/or neuropathy grade 2 without pain are permitted)
13. Prior autologous or allogeneic stem cell transplant within 12 weeks of initiation of therapy
14. Prior allogeneic stem cell transplant with active graft-versus-host- disease (GVHD).
15. Prior major surgical procedure or radiation therapy within 4 weeks of the first dose of study treatment (this does not include limited course of radiation used for management of bone pain within 7 days of first dose of study therapy).
16. Known intolerance to steroid therapy

1. Indicios de hemorragia interna o mucosa y/o resistencia a la transfusión de plaquetas (es decir, imposibilidad de mantener un recuento de plaquetas ≥75 000 células/mm3).
2. Presentar cualquier enfermedad que —según el investigador— supondría un riesgo excesivo para el paciente o podría afectar negativamente a su participación en el estudio. Algunos ejemplos de estas enfermedades son: antecedentes significativos de enfermedad cardiovascular (p. ej., infarto de miocardio, anomalías significativas en el sistema de conducción cardíaca, hipertensión no controlada, acontecimiento tromboembólico de grado 3 o superior en los últimos 6 meses).
3. Infección activa conocida que precisa tratamiento antiinfeccioso oral o parenteral en los 14 días siguientes al inicio del tratamiento.
4. Enfermedad con resistencia primaria al tratamiento (es decir, nunca respondió [≥ RM] a ningún tratamiento previo).
5. Otros tumores malignos diagnosticados o que precisaron tratamiento en los 3 últimos años, salvo el carcinoma basocelular, el carcinoma epidermoide de piel o el carcinoma localizado en el cuello uterino o mama que hayan sido tratados adecuadamente.
6. Mujeres embarazadas o en periodo de lactancia.
7. Enfermedades psiquiátricas graves, alcoholismo activo o toxicomanía que puedan complicar o dificultar la evaluación de seguimiento o el cumplimiento del tratamiento.
8. VIH conocido o infección activa por el virus de la hepatitis B o C.
9. Leucemia de células plasmáticas o amiloidosis sintomáticas simultáneas.
10. Síndrome POEMS (discrasia de células plasmáticas con polineuropatía, visceromegalia, endocrinopatía, proteína monoclonal [proteína M] y anomalías cutáneas).
11. Tratamientos citotóxicos previos —incluidos los fármacos citotóxicos en fase de investigación— para el mieloma múltiple en las 3 semanas (6 semanas para nitrosureas) previas al inicio del tratamiento del estudio. IM, IP y/o corticoesteroides en las 2 semanas previas al inicio del tratamiento del estudio. Tratamientos en fase de investigación o anticuerpos monoclonales en las 4 semanas previas al inicio del tratamiento del estudio. Se permite administrar un máximo de 10 mg diarios de prednisona por vía oral o un fármaco equivalente para tratar los síntomas de las enfermedades concomitantes, aunque la dosis debe ser estable durante un mínimo de 7 días antes del inicio del tratamiento del estudio.
12. Efectos secundarios residuales del tratamiento anterior > grado 1 antes de iniciar el tratamiento (salvo la alopecia de cualquier grado o la neuropatía de grado II sin dolor).
13. Trasplante hematopoyético autólogo o alogénico en las 12 semanas previas al inicio del tratamiento.
14. Trasplante hematopoyético alogénico con enfermedad de injerto contra huésped (EICH) previo.
15. Intervención quirúrgica importante o radioterapia en las 4 semanas previas a la administración de la primera dosis del tratamiento del estudio (este tratamiento no incluye un ciclo limitado de radiación empleado para el tratamiento del dolor óseo en los 7 días siguientes a la administración de la primera dosis del fármaco del estudio).
16. Intolerancia conocida al tratamiento con corticoesteroides.

E.5 End points

E.5.1

Primary end point(s)

ORR (Overall Response Rate)

TRG (Tasa de Respuesta Global)

E.5.1.1

Timepoint(s) of evaluation of this end point

During the course of the study

A lo largo del estudio

E.5.2

Secondary end point(s)

- PFS
- DOR
- OS
- CBR
- Time to response (TTR)
- Time to progression (TTP)
- Duration of SD
- Frequency and grade of AEs

- SSP
- DR
- SG
- TBC
- TTR
- TTP
- Duración de la enfermedad estable (EE)
- Frecuencia y clasificación de los acontecimientos adversos

E.5.2.1

Timepoint(s) of evaluation of this end point

During the course of the study

A lo largo del estudio

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

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

Italy

Spain

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

LPLV

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

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)

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

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

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

2016-10-27

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-09-30

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2021-11-11

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IMP with orphan designation in the indication
Orphan Designation Number:
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