Clinical Trials Register

Summary
EudraCT Number:	2014-002480-15
Sponsor's Protocol Code Number:	M14REP
National Competent Authority:	Netherlands - Competent Authority
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2014-10-15
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

Netherlands - Competent Authority

A.2

EudraCT number

2014-002480-15

A.3

Full title of the trial

A Phase II, Open-Label, Multicenter Study of Vemurafenib plus Cobimetinib (GDC-0973) in Unresectable Stage IIIc or Metastatic Melanoma; Response Monitoring and Resistance Prediction with Positron Emission Tomography and Tumor Characteristics.

Een fase II, open-label, multicenter onderzoek bij patiënten met irresectabel stadium IIIc of gemetastaseerd melanoom die behandeld worden met vemurafenib in combinatie met cobimetinib (GDC-0973); het meten van respons en het voorspellen van resistentie met behulp van Positron Emissie Tomografie en weefselanalyse.

A.3.1

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

A Multicenter Study of Vemurafenib plus Cobimetinib (GDC-0973) in Unresectable Stage IIIc or Metastatic Melanoma; Response Monitoring and Resistance Prediction with Positron Emission Tomography and Tumor Characteristics.

Een multicenter onderzoek bij patiënten met uitgezaaid melanoom die behandeld worden met vemurafenib in combinatie met cobimetinib (GDC-0973); het meten van respons en het voorspellen van resistentie met behulp van Positron Emissie Tomografie en weefselanalyse.

A.3.2

Name or abbreviated title of the trial where available

REPOSIT

A.4.1

Sponsor's protocol code number

M14REP

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	The Dutch Working Group on Immunotherapy of Oncology (WIN-O)
B.1.3.4	Country	Netherlands
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Roche Medical B.V.
B.4.2	Country	Netherlands
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	The Netherlands Cancer Institute - Antoni van Leeuwenhoek Hospital
B.5.2	Functional name of contact point	B. van der Hiel
B.5.3	Address:
B.5.3.1	Street Address	Plesmanlaan 121
B.5.3.2	Town/ city	Amsterdam
B.5.3.3	Post code	1066CX
B.5.3.4	Country	Netherlands
B.5.4	Telephone number	003120512 2285
B.5.5	Fax number	003120512 2290
B.5.6	E-mail	bvdhiel@nki.nl

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	Cobimetinib
D.3.2	Product code	GDC-0973
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	Cobimetinib
D.3.9.1	CAS number	934660-93-2
D.3.9.2	Current sponsor code	RO5514041
D.3.9.3	Other descriptive name	COBIMETINIB
D.3.9.4	EV Substance Code	SUB122319
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	60
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	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	18F-FLT
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous bolus use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	18F-FLT
D.3.9.1	CAS number	25526-93-6
D.3.9.2	Current sponsor code	18F-FLT
D.3.9.3	Other descriptive name	3-DEOXY-3-FLUOROTHYMIDINE
D.3.9.4	EV Substance Code	SUB33340
D.3.10	Strength
D.3.10.1	Concentration unit	MBq/ml megabecquerel(s)/millilitre
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	37 to 1250
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: 3
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	18F-FDG
D.2.1.1.2	Name of the Marketing Authorisation holder	IBA PHARMA S.A. 3, Chemin du Cyclotron 1348 OTTIGNIES LOUVAIN-LA-NEUVE BELGIUM
D.2.1.2	Country which granted the Marketing Authorisation	Netherlands
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	18F-FDG
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous bolus use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	FLUORINE (18F) FLUDEOXYGLUCOSE
D.3.9.1	CAS number	105851-17-0
D.3.9.4	EV Substance Code	SUB07680MIG
D.3.10	Strength
D.3.10.1	Concentration unit	MBq/ml megabecquerel(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	185
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: 4
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	Vemurafenib
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	Vemurafenib
D.3.9.1	CAS number	918504-65-1
D.3.9.2	Current sponsor code	RO5185426
D.3.9.3	Other descriptive name	VEMURAFENIB
D.3.9.4	EV Substance Code	SUB32161
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	960
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

BRAFV600-mutation positive patients with unresectable stage IIIc or IV melanoma.

Irresectabel stadium IIIc of IV BRAFV600 positieve melanoompatienten

E.1.1.1

Medical condition in easily understood language

BRAFV600-mutation (gene-alteration) positive patients with unresectable metastatic melanoma.

Niet operabele of uitgezaaid melanoompatienten die een mutatie (verandering) in het BRAFV600 gen hebben.

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	17.0
E.1.2	Level	PT
E.1.2	Classification code	10025671
E.1.2	Term	Malignant melanoma stage IV
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	17.0
E.1.2	Level	PT
E.1.2	Classification code	10025670
E.1.2	Term	Malignant melanoma stage III
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 study whether 18F-FDG or 18F-FLT PET can be used for early detection of response to treatment with vemurafenib plus cobimetinib (GDC-0973) compared to standard response assessment with CT according to RECIST 1.1 at 7 weeks.
• To study whether 18F-FDG or 18F-FLT PET can predict Progression Free Survival (PFS).

• onderzoeken of er een relatie is tussen vroege metabole veranderingen op 18F-FDG of 18F-FLT PET (na 2 weken) en standaard respons evaluatie volgens RECIST1.1 op CT (in de 7e week van behandeling),
• onderzoeken of 18F-FDG of 18F-FLT PET progressieve vrije overleving kan voorspellen,

E.2.2

Secondary objectives of the trial

• To study whether level of target inhibition can predict the type (CR/PR, SD or PD) and duration of response.
• To study the correlation between PET imaging and several key tumor characteristics at different levels
• To elucidate molecular mechanisms of resistance by integrating protein phosphorylation, genomic and cancer mutation data.
• To study the correlation between the pharmacokinetics of vemurafenib/ cobimetinib (GDC-0973) and PET imaging and therapy response.
• To study various quantitative measures of radiotracer uptake from baseline upon PET imaging for predicting the type (CR/PR, SD or PD) and Progression Free Survival and Overall Survival (PFS and OS).

• onderzoeken of de mate van metabole tumor respons het type (CR/PR, SD of PD) en responsduur kan voorspellen,
• de correlatie onderzoeken tussen PET imaging en verscheidene tumoreigenschappen in weefsel,
• onderzoeken van resistentie-mechanismen door het integreren van data verkregen uit next-generation sequencing, immunohistochemie, genexpressie analyse en phosphoproteomics,
• de correlatie onderzoeken tussen PET imaging en therapierespons en farmacokinetiek van vemurafenib en cobimetinib (GDC-0973),

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Patients with histologically confirmed melanoma, either unresectable stage IIIc or stage IV metastatic melanoma, as defined by AJCC 7th edition.
• Patients must be naïve to treatment for locally advanced unresectable or metastatic disease. Prior adjuvant immunotherapy (including ipilimumab) is allowed.
• Documentation of BRAFV600 mutation-positive status in melanoma tumor tissue (archival or newly obtained tumor samples).
• Measurable disease per RECIST v1.1 (50), which are accessible to biopsies.
• Biopsy lesion is within scan reach of diagnostic CT and PET-CT (thorax- abdomen-pelvis)
• ECOG performance status of 0 or 1.
• Male or female patient aged ≥ 18 years.
• Life expectancy ≥ 12 weeks.
• Adequate hematologic and end organ function within 14 days prior to first dose of study drug treatment.

• Patiënten met BRAFV600E of BRAFV600K gemuteerd irresectabel stadium IIIc of gemetastaseerd melanoom
• Geen systemische therapie voor melanoom gehad, met uitzondering van immunotherapie
• Bewezen BRAFV600 mutatie uit tumorweefselanalyse (uit opgeslagen of nieuw verkregen tumormateriaal)
• Meetbare ziekte volgens RECIST 1.1 én toegankelijk voor biopsie
• Te biopteren lesie ligt binnen het scanbereik van PET en CT (thorax-abdomen-pelvis)
• ECOG performance status 0 of 1
• Leeftijd ≥18 jaar
• Levensverwachting ≥ 12 weken
• Adequate hematologische en orgaanfunctie parameters 14 dagen voorafgaand aan start studiemedicatie

E.4

Principal exclusion criteria

• History of prior RAF or MEK pathway inhibitor treatment.
• Palliative radiotherapy, major surgery or traumatic injury within 14 days prior to the first dose of study treatment.
• Active malignancy within the past 3 years other than melanoma that could potentially interfere with the interpretation of efficacy measures.
• History of or evidence of retinal pathology, clinically significant cardiac dysfunction, patients with active CNS lesions, renal or liver dysfunction as described in main protocol (REPOSIT NL48639.031.14).
• Pregnant, lactating, or breast-feeding.
• Unwillingness or inability to comply with study and follow-up procedures (i.e. severe anxiety disorder preventing PET/CT imaging.

• Eerder behandeling met RAF- of MEK-pathway remmers gehad.
• Palliatieve radiotherapie, trauma of grote chirurgische ingreep binnen 14 dagen voor aanvang van de studiemedicatie
• Andere maligniteit < 3 jaar geleden dat mogelijk interfereert met de interpretatie van uitkomstmaten, m.u.v. basaalcelcarcinoom
• Retinopathie of glaucoom, nierfunctiestoornissen (GFR < 40 ml/min), leverfunctiestoornissen, hartfalen (LVEF < 50%) of symptomatische CZS betrokkenheid
• Zwangerschap of het geven van borstvoeding
• Onwelwillendheid of onmogelijkheid zich te kunnen houden aan het studieprotocol en follow-up procedures (bijvoorbeeld ernstige claustrofobie, waardoor PET/CT scanning niet mogelijk is).

E.5 End points

E.5.1

Primary end point(s)

• Progression Free survival (PFS)
• Correlation between changes of metabolic tracer uptake on PET and of size on diagnostic CT according to RECIST 1.1 from baseline, Day 14/15 Cycle 1, Day 21 Cycle 2 and at the time of progression.
• Diagnostic accuracy and best cut-off values of PET at Day 14/15 Cycle 1 and Day 21 Cycle 2 for distinguishing responders from non-responders.
• Investigation of the continuous parameters of PET in association with Progression Free Survival.

• Progressieve vrije overleving (PFS)
• Correlatie tussen metabole veranderingen op de PET en tumorgrootte op diagnostische CT volgens RECIST 1.1 bij baseline, Dag 14/15 Cycle 1, Dag 21 Cycle 2 en op het moment van progressie.
• Diagnostische nauwkeurigheid / voorspellende waarde van PET bij 2 en 7 weken voor het onderscheiden van responders en non-responders.
• Correlatie tussen diverse PET-parameters en progressie vrije overleving.

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline, Day 14/15 Cycle 1, Day 21 Cycle 2 and at the time of progression.

Baseline, Dag 14/15 Cycle 1, Dag 21 Cycle 2 en op het moment van progressie.

E.5.2

Secondary end point(s)

• Correlation of PET-imaging with 18F-FDG PET and 18F-FLT PET between baseline and Day 14/15 Cycle 1 by means of Standardized Uptake Value.
• Correlation of PET-imaging with 18F-FDG PET and 18F-FLT PET at the time of progression by means of Standard Uptake Value.
• Correlation between 18F-FDG/FLT uptake and immunohistochemical analysis in responders and non responders early after the initiation of therapy.
• Correlation between 18F-FDG/FLT uptake and resistance by means of diagnostic CT and PFS.
• Best quantification of metabolic imaging. Various quantitative measures of radiotracer uptake determined in RECIST 1.1 target lesions, correlated to PFS and OS.
• Correlation between genetic analysis and resistance in terms of RECIST1.1 criteria on diagnostic CT and PFS.
• Correlation between phosphoprotein profiles and resistance in terms of RECIST1.1 criteria on diagnostic CT and PFS.
• Overall Survival (OS)
• Drug level monitoring of vemurafenib and cobimetinib (GDC-0973) at Day 15 Cycle 1 compared to tumor response (RECIST1.1 criteria) and PFS.
• Drug level monitoring of vemurafenib and cobimetinib (GDC-0973) at Day 15 Cycle 1 compared to 18F-FDG/FLT uptake.
• ECOG Performance Status

• Correlatie tussen SUV-waarden van 18F-FDG en 18F-FLT PET imaging van baseline en 2 weken na start van de therapie.
• Correlatie tussen SUV-waarden van 18F-FDG en 18F-FLT PET imaging op het moment van progressie.
• Correlatie tussen 18F-FDG/FLT uptake en immunohistochemische analyses in responders en non-responders.
• Correlatie tussen 18F-FDG/FLT uptake en resistentie (o.b.v. RECIST1.1 en PFS)
• Onderzoek naar de beste kwantificatie voor metabole imaging; verscheidene kwantitatieve analyses van tracer uptake in RECIST 1.1 target laesies.
• Correlatie tussen genetische analyse en resistentie.
• Correlatie tussen phosphoproteomics en resistentie.
• Overall Survival.
• Drug level monitoring van vemurafenib en cobimetinib (GDC-0973) bij 2 weken vergeleken met 18F-FDG/FLT uptake.
• Drug level monitoring van vemurafenib en cobimetinib (GDC-0973) bij 2 weken vergeleken met tumor respons (RECIST1.1) en PFS.
• ECOG Performance Status

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline, Day 14/15 Cycle 1, Day 21 Cycle 2 and at the time of progression.

Baseline, Dag 14/15 Cycle 1, Dag 21 Cycle 2 en op het moment van progressie.

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

E.6.5

Efficacy

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.3

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

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

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 study will end when all patients enrolled have been followed for at least three years, withdrawal of consent, lost to follow-up, or the Sponsor decides to end the trial, whichever occurs first.

De studie eindigt wanneer alle patienten die geincludeerd zijn in de studie voor ten minste 3 jaar gevolgd zijn, zich hebben teruggetrokken of lost to follow-up of wanneer de Sponsor heeft besloten de studie terug te trekken (conform WMO), wat zich het eerst voordoet.

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

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

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

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.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

Geen

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

2014-10-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2014-10-14

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

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IMP with orphan designation in the indication
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