Clinical Trials Register

Summary
EudraCT Number:	2012-004606-10
Sponsor's Protocol Code Number:	8VWF07
National Competent Authority:	Germany - PEI
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2014-04-07
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

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A. Protocol Information

A.1

Member State Concerned

Germany - PEI

A.2

EudraCT number

2012-004606-10

A.3

Full title of the trial

Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic Optivate® Treatment in Subjects with Severe Haemophilia A.

A.3.1

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

Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic Optivate® Treatment in Subjects with Severe Haemophilia A.

A.4.1

Sponsor's protocol code number

8VWF07

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	BIO PRODUCTS LABORATORY LIMITED (BPL)
B.1.3.4	Country	United Kingdom
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	Bio Products Laboratory Ltd
B.4.2	Country	United Kingdom
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Bio Products Laboratory Limited (BPL)
B.5.2	Functional name of contact point	Medical Department
B.5.3	Address:
B.5.3.1	Street Address	Dagger Lane
B.5.3.2	Town/ city	Elstree
B.5.3.3	Post code	WD6 3BX
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	+44(0)2089572297
B.5.5	Fax number	+44(0)2089572611
B.5.6	E-mail	lisa.wilson@bpl.co.uk

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	Optivate®
D.2.1.1.2	Name of the Marketing Authorisation holder	Bio Products Laboratory Limited
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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.3.4	Pharmaceutical form	Powder for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.11 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	Yes
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

Severe Haemophilia A

E.1.1.1

Medical condition in easily understood language

Severe Haemophilia A

E.1.1.2

Therapeutic area

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

MedDRA Classification

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 post-marketing immunogenicity of Optivate® by monitoring plasma inhibitor levels for at least 100 Exposure Days (EDs) for each subject.

E.2.2

Secondary objectives of the trial

To assess efficacy and tolerability by monitoring factor VIII (FVIII) recovery and Adverse Events (AEs).

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Written informed consent or, if less than 18 years of age written assent (where possible) and their parent/guardian’s has given written informed consent.
Severe haemophilia A (< 1% FVIII:C). Subjects suffering from severe haemophilia A (< 2%) may be enrolled, but only after approval by BPL. Subjects with a Factor VIII of < 2% may not constitute more than 50% of the total patient population. A separate statistical evaluation will be conducted for the < 1% and < 2% populations. Basal FVIII level taken from subject’s lowest level recorded, or the level measured at screening, whichever is lower.
Previously Treated Patients (PTPs) with > 150 exposure days on prior Factor VIII therapy (of which at least the last 50 EDs or 2 years treatment can be confirmed by way of subject records).
Immunocompetent with CD4 count > 200 /μL.
HIV negative or a viral load < 200 particles /μL.

E.4

Principal exclusion criteria

History of inhibitor development to FVIII or a positive result on the Nijmegen-Bethesda at screening (quantitative result of > 0.6 Bethesda Units [BU]) prior to the administration of Optivate®.
Known or suspected hypersensitivity to the Investigational Medicinal Product (IMP) or its excipients.
Clinically significant liver disease (serum Alanine Aminotransferase [ALT] levels greater than three times the upper limit of the normal range), renal disease (serum creatinine > 200mol/L), or coagulopathy other than haemophilia A. History of unreliability or non-cooperation (including not being able to complete the study diary).
Participating in, or have taken part in another trial within the last 30 days.

E.5 End points

E.5.1

Primary end point(s)

Immunogenicity of Optivate® by monitoring plasma inhibitor level for at least 100 EDs for each subject.

E.5.1.1

Timepoint(s) of evaluation of this end point

after 60 weeks

E.5.2

Secondary end point(s)

Recovery with current FVIII (Screening Visit) versus (vs.) 1st dose with Optivate® (V1).
Recovery at 1 ED (V1), 10-15 EDs (V2), 50-75 ED (V3) and 100 EDs (V4).
Number of break-through bleeds including severity, duration, location and cause.
Clinician’s judgement of break-through bleed treatment outcome (excellent, good, moderate, poor). Subject’s judgement of break-through bleed treatment outcome (very helpful, helpful, helped a little, did not help).
Number of exposure days for each subject and per month/subject, per year/subject and overall.
Total dose in IU/kg of Optivate® and average dose per infusion for prophylactic use, to treat a bleed, any additional preventative use and overall use; all analysed on a per subject basis.
Total number of infusions for prophylactic use, to treat a bleed, any additional preventative use and overall use; all analysed on a per subject basis.
Mean dose in IU/kg of Optivate® per subject/month and per subject/year for prophylactic use, to treat a bleed, any additional preventative use and overall use; all analysed on a per subject basis.
Mean number of infusions per subject/month and per subject/year for prophylactic use, to treat a bleed, any additional preventative use and overall use; all analysed on a per subject basis.

E.5.2.1

Timepoint(s) of evaluation of this end point

after 60 weeks

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

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)

E.7.4

Therapeutic use (Phase IV)

Yes

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

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

Colombia

Germany

Poland

United Kingdom

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

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)

Yes

F.1.1.4

Infants and toddlers (28 days-23 months)

Yes

F.1.1.5

Children (2-11years)

Yes

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.2

Adults (18-64 years)

Yes

F.1.3

Elderly (>=65 years)

Yes

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)

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

2014-08-06

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2014-07-17

P. End of Trial
P.	End of Trial Status	Completed

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IMP with orphan designation in the indication
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