ICH GCP - EU Clinical trials Registry - 2011-001033-16 (PL)

Summary
EudraCT Number:	2011-001033-16
Sponsor's Protocol Code Number:	NN7008-3809
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2015-05-07
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

A. Protocol Information

A.1

Member State Concerned

Poland - Office for Medicinal Products

A.2

EudraCT number

2011-001033-16

A.3

Full title of the trial

Safety and Efficacy of turoctocog alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients with Haemophilia A

A.3.1

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

Safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with Haemophilia A

A.3.2

Name or abbreviated title of the trial where available

guardian™4

A.4.1

Sponsor's protocol code number

NN7008-3809

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1119-6116

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/50/2010

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Novo Nordisk A/S
B.1.3.4	Country	Denmark
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	Novo Nordisk A/S
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novo Nordisk A/S
B.5.2	Functional name of contact point	Global Clinical Registry (GCR,1452)
B.5.3	Address:
B.5.3.1	Street Address	Novo Allé
B.5.3.2	Town/ city	Bagsværd
B.5.3.3	Post code	2880
B.5.3.4	Country	Denmark
B.5.6	E-mail	clinicaltrials@novonordisk.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	Yes
D.2.1.1.1	Trade name	NovoEight
D.2.1.1.2	Name of the Marketing Authorisation holder	Novo Nordisk A/S
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	turoctocog alfa 2000 IU/vial
D.3.2	Product code	N8
D.3.4	Pharmaceutical form	Powder and solvent for solution for injection
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	turoctocog alfa
D.3.9.1	CAS number	1192451-26-5
D.3.10	Strength
D.3.10.1	Concentration unit	IU/kg international unit(s)/kilogram
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	2000
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	Yes
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.2.1.1.1	Trade name	NovoEight
D.2.1.1.2	Name of the Marketing Authorisation holder	Novo Nordisk A/S
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	turoctocog alfa 250 IU/vial
D.3.2	Product code	N8 250
D.3.4	Pharmaceutical form	Powder and solvent for solution for injection
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	turoctocog alfa
D.3.9.1	CAS number	1192451-26-5
D.3.10	Strength
D.3.10.1	Concentration unit	IU/kg international unit(s)/kilogram
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	250
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	Yes
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

Haemophilia A

E.1.1.1

Medical condition in easily understood language

Bleeding disorder haemophilia A

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	19.0
E.1.2	Level	LLT
E.1.2	Classification code	10018937
E.1.2	Term	Haemophilia A
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

No

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate safety of turoctocog alfa in paediatric previously untreated patients (PUP) with haemophilia A

E.2.2

Secondary objectives of the trial

- To evaluate efficacy of turoctocog alfa in treatment of bleeds in paediatric PUP with haemophilia A
- To evaluate preventive effect of turoctocog alfa on bleeds in paediatric PUP with haemophilia A

E.2.3

Trial contains a sub-study

No

E.3

Principal inclusion criteria

- Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)
- Male patients diagnosed with congenital severe haemophilia A (FVIII ≤ 1%)
- Age < 6 years
- No prior use of purified clotting FVIII products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma is accepted) including commercially available NovoEight®/Novoeight®

E.4

Principal exclusion criteria

- Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
- Previous participation in this trial defined as withdrawal after administration of trial product
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of FVIII inhibitor
- Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG)), routine systemic corticosteroids)

E.5 End points

E.5.1

Primary end point(s)

Incidence rate of FVIII inhibitors (≥0.6 BU)

E.5.1.1

Timepoint(s) of evaluation of this end point

Will be evaluated for the main phase of the trial

E.5.2

Secondary end point(s)

- Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None
- Annualized bleeding rate
- Incidence rate of FVIII inhibitors (≥0.6 BU)

E.5.2.1

Timepoint(s) of evaluation of this end point

Will be evaluated for the main phase of trial, for the extension phase of trial, and for the combined main and extension phases

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

No

E.6.2

Prophylaxis

No

E.6.3

Therapy

No

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

No

E.6.7

Pharmacodynamic

No

E.6.8

Bioequivalence

No

E.6.9

Dose response

No

E.6.10

Pharmacogenetic

No

E.6.11

Pharmacogenomic

No

E.6.12

Pharmacoeconomic

No

E.6.13

Others

No

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

No

E.7.1.1

First administration to humans

No

E.7.1.2

Bioequivalence study

No

E.7.1.3

Other

No

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

No

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

No

E.8 Design of the trial

E.8.1

Controlled

No

E.8.1.1

Randomised

No

E.8.1.2

Open

No

E.8.1.3

Single blind

No

E.8.1.4

Double blind

No

E.8.1.5

Parallel group

No

E.8.1.6

Cross over

No

E.8.1.7

Other

No

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

No

E.8.2.2

Placebo

No

E.8.2.3

Other

No

E.8.3

The trial involves single site in the Member State concerned

No

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

2

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

10

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

No

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

Algeria

Austria

China

Denmark

Greece

Hong Kong

Hungary

Japan

Poland

Russian Federation

Serbia

Spain

Turkey

United States

E.8.7

Trial has a data monitoring committee

No

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

2

E.8.9.1

In the Member State concerned months

11

E.8.9.1

In the Member State concerned days

16

E.8.9.2

In all countries concerned by the trial years

6

E.8.9.2

In all countries concerned by the trial months

6

E.8.9.2

In all countries concerned by the trial days

0

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

60

F.1.1.1

In Utero

No

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

No

F.1.1.3

Newborns (0-27 days)

Yes

F.1.1.3.1

Number of subjects for this age range:

1

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.4.1

Number of subjects for this age range:

54

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

5

F.1.1.6

Adolescents (12-17 years)

No

F.1.2

Adults (18-64 years)

No

F.1.3

Elderly (>=65 years)

No

F.2 Gender

F.2.1

Female

No

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

No

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

No

F.3.3.1

Women of childbearing potential not using contraception

No

F.3.3.2

Women of child-bearing potential using contraception

No

F.3.3.3

Pregnant women

No

F.3.3.4

Nursing women

No

F.3.3.5

Emergency situation

No

F.3.3.6

Subjects incapable of giving consent personally

No

F.3.3.7

Others

No

F.4 Planned number of subjects to be included

F.4.1

In the member state

2

F.4.2

For a multinational trial

F.4.2.1

In the EEA

15

F.4.2.2

In the whole clinical trial

60

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)

N/A

G. Investigator Networks to be involved in the Trial

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

N.

Competent Authority Decision

Authorised

N.

Date of Competent Authority Decision

2015-07-17

N.

Ethics Committee Opinion of the trial application

Favourable

N.

Ethics Committee Opinion: Reason(s) for unfavourable opinion

N.

Date of Ethics Committee Opinion

2015-05-28

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2018-12-05

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