Clinical Trials Register

Summary
EudraCT Number:	2011-005968-24
Sponsor's Protocol Code Number:	Vx-001-201
National Competent Authority:	Germany - PEI
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2012-02-15
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

Germany - PEI

A.2

EudraCT number

2011-005968-24

A.3

Full title of the trial

A multicenter, randomized, double-blind, placebo-controlled Phase IIb
Efficacy Study of Vx-001, a peptide-based cancer vaccine aimed to
maintain disease control after first line treatment in HLA-A*0201
positive patients with TERT positive NSCLC (stage IV or recurrent
stage I-III)

Eine multizentrische, randomisierte, doppel-blinde, Placebo-kontrollierte Phase IIb-Studie zur Wirksamkeit von Vx-001, einem Peptid-basierten Tumorimpfstoff zur Response-Erhaltung nach first line Chemotherapie bei HLA-A*0201 - positiven Patienten mit TERT - positivem, nicht-kleinzelligem Lungenkrebs NSCLC (Stadium IV oder rezidivierendem Stadium I-III)

A.3.1

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

A.3.2

Name or abbreviated title of the trial where available

VaxLung

Vaxlung

A.4.1

Sponsor's protocol code number

Vx-001-201

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	Vaxon Biotech
B.1.3.4	Country	France
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Vaxon Biotech
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Vaxon Biotech
B.5.2	Functional name of contact point	Kostas Kosmatopoulos
B.5.3	Address:
B.5.3.1	Street Address	3 Rue de l' Arrivee
B.5.3.2	Town/ city	Paris
B.5.3.3	Post code	75015
B.5.3.4	Country	France
B.5.4	Telephone number	0033 145886762
B.5.5	Fax number	0033145886780
B.5.6	E-mail	kkosmatopoulos@vaxon-biotech.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	Yes
D.2.5.1	Orphan drug designation number	C(2007)6738
D.3 Description of the IMP
D.3.1	Product name	CRYPTIC PEPTIDE
D.3.2	Product code	ARG-Vx001
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.2	Current sponsor code	Vx-001
D.3.9.3	Other descriptive name	OLIGOPEPTIDE
D.3.9.4	EV Substance Code	SUB14950MIG
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	4
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	Yes
D.2.5.1	Orphan drug designation number	C(2007)6738
D.3 Description of the IMP
D.3.1	Product name	OPTIMIZED PEPTIDE
D.3.2	Product code	TYR-Vx001
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.2	Current sponsor code	Vx-001
D.3.9.3	Other descriptive name	OLIGOPEPTIDE
D.3.9.4	EV Substance Code	SUB14950MIG
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	4
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	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.2.1.1.1	Trade name	Montanide ISA 51 VG
D.2.1.1.2	Name of the Marketing Authorisation holder	SEPPIC
D.2.1.2	Country which granted the Marketing Authorisation	France
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	Montanide
D.3.2	Product code	ISA 51 VG
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N/A
D.3.9.1	CAS number	N/A
D.3.9.2	Current sponsor code	Vx-001
D.3.9.3	Other descriptive name	MINERAL OIL
D.3.9.4	EV Substance Code	SUB12534MIG
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.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Non-small cell lung cancer

E.1.1.1

Medical condition in easily understood language

Lung cancer

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	17.1
E.1.2	Level	PT
E.1.2	Classification code	10061873
E.1.2	Term	Non-small cell lung 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

The primary objective is to compare survival rate at 12 months in Vx-001 treated vs placebo treated patients.

E.2.2

Secondary objectives of the trial

The secondary objectives are:
• Time-to-event comparison of overall survival (OS) in Vx-001 treated vs placebo treated patients,
• Comparison of Time to Treatment Failure in Vx-001 treated vs placebo treated patients

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or female >/=18 years of age;
2. Documented stage IV NSCLC as defined by IASLC Lung Cancer Staging Project (7th edition) or recurrent stage I-III disease at least 6 months after resection or after the end of adjuvant chemotherapy or after standard locoregional treatment as defined by the American College of Chest Physicians;
3. Patients treated with 4 cycles platinum based 1st line chemotherapy as defined by the American College of Chest Physicians (i.e. radiotherapy are not allowed except palliative radiotherapy of bone metastasis);
4. Documented HLA-A*0201 positivity, as determined by a local laboratory;
5. TERT-positive NSCLC, as assessed by a central laboratory; for this, availability of adequate tissue biopsy from the primary tumor, lymph nodes or distant metastases is a prerequisite;
6. CR, PR, or SD according to RECIST criteria after the completion of platinum-based first-line chemotherapy;
7. First vaccination have to be administered no more than 4 weeks after the last administration of platinum-based 1st line chemotherapy;
8. ECOG performance status 0, 1;
9. Patients must have adequate renal and hepatic function as assessed by standard laboratory criteria;
10. Patients must have adequate haematological function:
• Platelet count >/= 100 x 109/L;
• WBC count >/= 2.5 x 109/L;
• Hemoglobin >/= 90g /L;
11. Female patients must be of non-child-bearing potential (i.e., women with functioning ovaries who have a documented tubal ligation or hysterectomy, ovariectomy or women who are post-menopausal). Women of child-bearing potential must have a negative urine pregnancy test at baseline and agree to practice adequate contraception for 30 days prior to administration of investigational product, throughout the study treatment period and 30 days after completion of injections;
12. In the investigator’s opinion, the patient is capable and willing to comply with the requirements of the study;
13. Willing and able to sign a written informed consent.

E.4

Principal exclusion criteria

1. Mixed small cell and NSCLC histologies;
2. Patients with stage IV or recurrent NSCLC who have been previously treated with therapy other than platinum-based first-line chemotherapy;
3. Prior treatment with cancer vaccines;
4. Prior treatment with immunotherapy (eg interferons, interleukins, TNF, or biological response modifiers, such as GM-CSF etc) within four weeks prior to randomization;
5. Prior treatment with hormone (including corticosteroids) within 2 weeks prior to randomization;
6. Prior treatment with any investigational drugs, within 4 weeks prior to randomization;
7. Patients with brain metastases;
8. Past and current history of neoplasm other than NSCLC, except for curatively treated non-melanoma skin cancer, in situ carcinoma of the cervix, or other cancer curatively treated with no evidence of disease for at least five years;
9. Autoimmune or immunodeficiency disease that in the opinion of the investigator may compromise the safety of the patient in the study;
10. Any pre-existing medical condition requiring concomitant systemic corticosteroid or immunosuppressive therapy. The use of inhaled corticosteroids for COPD or topical steroids is allowed;
11. Known hepatitis B and/or C infection, testing not required;
12. Known HIV-positivity, testing not required;
13. Clinically significant hepatic dysfunction (ALT>2.5 times normal upper limits [ULN], AST>2.5 times ULN, bilirubin>1.5 times ULN);
14. Clinically significant renal dysfunction (serum creatinine>1.5 time ULN);
15. Uncontrolled congestive heart failure or hypertension, unstable heart disease (coronary artery disease with unstable angina or myocardial infarction within 6 months of randomization) or uncontrolled ventricular arrhythmias at the time of enrolment in the study (atrial fibrillation or flutter is acceptable);
16. Splenectomy or splenic irradiation;
17. Any infectious condition that, in the opinion of the investigator, could compromise the patient’s ability to develop an immune response;
18. Pregnant or lactating females (female patients of child-bearing potential will undertake pregnancy testing at screening and during study completion/withdrawal visits);
19. Alcohol or drug use or dependence;
20. Requirement of concurrent treatment with prohibited medication (investigational product, other anti-cancer treatments including chemotherapy, non-palliative radiotherapy, biological agents and immunomodulating agents, systemic immunosuppressive agents, including systemic corticosteroids);
21. The investigator considers the patient unfit for the study as a result of the medical interview, physical examination, or screening investigations.

E.5 End points

E.5.1

Primary end point(s)

The primary objective is to compare survival rate in Vx-001 treated vs placebo treated patients.

E.5.1.1

Timepoint(s) of evaluation of this end point

12 months from start of therapy with IMP

E.5.2

Secondary end point(s)

• Time-to-event comparison of overall survival (OS) in Vx-001 treated vs placebo treated patients,
• Comparison of Time to Treatment Failure in Vx-001 treated vs placebo treated patients

E.5.2.1

Timepoint(s) of evaluation of this end point

•Death of patient
•Progression of disease - onset of 2nd line therapy

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

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

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

Yes

E.8.1.7.1

Other trial design description

multi-centre

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

E.8.6.2

Trial being conducted completely outside of the EEA

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

Disease progression - onset of 2nd line therapy

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:

110

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

110

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

220

F.4.2.2

In the whole clinical trial

220

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)

if patients progress, then IMP treatment shall stop and will be succeeded by 2nd line treatment

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2013-01-23

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-06-11

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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