Clinical Trials Register

Summary
EudraCT Number:	2014-002986-30
Sponsor's Protocol Code Number:	GENA-21b
National Competent Authority:	Slovenia - JAZMP
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-05-12
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

Slovenia - JAZMP

A.2

EudraCT number

2014-002986-30

A.3

Full title of the trial

Prospective, open-label, multicentre phase 3b study to assess the efficacy and safety of personalized prophylaxis with Human-cl rhFVIII in previously treated adult patients with severe haemophilia A

Prospektivna, odprta, multicentrična raziskava faze 3b za oceno učinkovitosti in varnosti personalizirane profilakse z zdravilom Human-cl rhFVIII pri predhodno zdravljenih odraslih bolnikih s hudo hemofilijo A

A.3.1

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

Efficacy and safety of personalized prophylaxis treatment with Human-cl rhFVIII in previously treated adult patients, affected by inherited gender-related coagulation disorder, in which affected males do not produce functional coagulation factor VIII (FVIII) in sufficient quantities to achieve satisfactory blood clotting.

A.4.1

Sponsor's protocol code number

GENA-21b

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02256917

A.5.4

Other Identifiers

Name:

IND Number

Number:

BB-IND 13722

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	OCTAPHARMA AG
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	OCTAPHARMA AG
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	Octapharma AG
B.5.2	Functional name of contact point	Johann Bichler
B.5.3	Address:
B.5.3.1	Street Address	Seidenstrasse 2
B.5.3.2	Town/ city	Lachen
B.5.3.3	Post code	CH-8853
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+4155451 21 77
B.5.5	Fax number	+4155451 21 51
B.5.6	E-mail	johann.bichler@octapharma.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	Nuwiq
D.2.1.1.2	Name of the Marketing Authorisation holder	Octapharma AB, Sweden
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	Human cell line recombinant factor VIII
D.3.2	Product code	Human-cl rhFVIII
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	Simoctocog alfa
D.3.9.1	CAS number	N.A
D.3.9.2	Current sponsor code	140
D.3.9.3	Other descriptive name	Human Coagulation Factor VIII
D.3.9.4	EV Substance Code	SUB13815MIG
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	250 to 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.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

Severe Haemophilia A

E.1.1.1

Medical condition in easily understood language

An inherited gender-related coagulation disorder in which affected males do not produce functional coagulation factor VIII (FVIII) in sufficient quantities to achieve satisfactory blood clotting.

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

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	10018938
E.1.2	Term	Haemophilia A (Factor VIII)
E.1.2	System Organ Class	100000004850

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 compare the annualised total bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-cl rhFVIII from study GENA-01

E.2.2

Secondary objectives of the trial

1.To compare the annualised spontaneous bleeding rate of individually tailored prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-clrhFVIII
2.To compare the annualised total bleeding rate in patients with 2x/week(or less) prophylaxis with the historical bleeding rate observed in patients having received on-demand treatment with Human-clrhFVIII
3.To assess the median prophylactic dosing interval
4.To assess the PK of Human-clrhFVIII in terms of FVIII:C
5.To assess the safety of Human-clrhFVIII
Additional Objectives
1.To assess the clinical efficacy of Human-clrhFVIII in the treatment of breakthrough bleeding episodes (BEs)
2.To assess the clinical efficacy of Human-clrhFVIII in surgical prophylaxis
3.To assess the correlation of VWF antigen concentration and half-life of Human-clrhFVIII
4.To assess the association between ABO blood type and half-life of Human-cl rhFVIII
5.To assess Human-clrhFVIII consumption data

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

(a) Severe haemophilia A (FVIII:C < 1%) according to medical history
(b) Male patients ≥ 18 years of age
(c) Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 EDs
(d) Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start
(e) Immunocompetence (CD4+ count > 200/μL)
(f) Freely given written informed consent

E.4

Principal exclusion criteria

(a) Any coagulation disorder other than haemophilia A
(b) Present or past FVIII inhibitor activity (≥ 0.6 BU) according to medical history
(c) Severe liver or kidney disease (ALT and AST levels > 5 times of upper limit of normal, creatinine > 120 μmol/L)
(d) Treatment with any investigational medicinal product (IMP) except FVIII IMP within 14 days prior to the screening visit

E.5 End points

E.5.1

Primary end point(s)

Reduction of the annualised total bleeding rate observed in the GENA-01 study (58.1 total bleeding episodes per patient per year) by 50% during individually tailored prophylaxis

E.5.1.1

Timepoint(s) of evaluation of this end point

During individually tailored prophylaxis phase

E.5.2

Secondary end point(s)

1.Reduction of the annualised spontaneous bleeding rate observed in the GENA-01 study (38.5 spontaneous bleeding episodes per patient per year) by 50% during individually tailored prophylaxis
2. Reduction of the annualised bleeding rate observed in GENA-01 by 50% in patients with 2x/week prophylaxis or less
3. Median prophylactic dosing interval during individually tailored prophylaxis
4. Human-cl rhFVIII consumption data (FVIII IU/kg per month per patient) during individually tailored prophylaxis
5. Safety and tolerability of Human-cl rhFVIII by monitoring adverse events (AEs) throughout the study

E.5.2.1

Timepoint(s) of evaluation of this end point

Secondary End Points 1-4 - During individually tailored prophylaxis phase
Secondary End Point 5 - Throughout the study

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

Yes

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

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

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

Yes

E.8.4

The trial involves multiple sites in the 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

Canada

Croatia

Finland

France

Japan

Macedonia, the former Yugoslav Republic of

Netherlands

Slovenia

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

LVLS

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:

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

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.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)

After the study patients will have the opportunity to continue treatment with commercially available Human-cl rhFVIII.

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

2017-05-23

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-05-17

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2018-09-05

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Orphan Designation Number:
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