Clinical Trials Register

Summary
EudraCT Number:	2012-000796-16
Sponsor's Protocol Code Number:	GAMr-30
National Competent Authority:	Bulgarian Drug Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-05-22
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

Bulgarian Drug Agency

A.2

EudraCT number

2012-000796-16

A.3

Full title of the trial

PROSPECTIVE, OPEN-LABEL, NON-CONTROLLED, MULTICENTER, PHASE III CLINICAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF OCTAGAM 10% IN PRIMARY IMMUNE THROMBOCYTOPENIA

Проспективно, открито, неконтролирано, многоцентрово, фаза III клинично изпитване за оценка ефикасността и безопасността на Octagam 10% при Първична Имунна Тробоцитопения

A.3.1

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

Study to evaluate the efficacy and safety of human immune
globulin in patients with primary immune thrombocytopenia

Проучване за оценка ефикасността и безопасността на човешки имуноглобулин при пациенти с Първична Имунна Тробоцитопения

A.4.1

Sponsor's protocol code number

GAMr-30

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 Pharmazeutika Produktionsgesellschaft m.b.H.
B.5.2	Functional name of contact point	Clinical Research Department
B.5.3	Address:
B.5.3.1	Street Address	Oberlaaer Straße 235
B.5.3.2	Town/ city	Vienna
B.5.3.3	Post code	1100
B.5.3.4	Country	Austria
B.5.4	Telephone number	+43 1 610321295
B.5.5	Fax number	+43 1 61032 9249
B.5.6	E-mail	clinical.department@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	Octagam 10%
D.2.1.1.2	Name of the Marketing Authorisation holder	Octapharma (IP) Limited
D.2.1.2	Country which granted the Marketing Authorisation	Bulgaria
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	Octagam 10%
D.3.4	Pharmaceutical form	Solution for infusion
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	Human normal immunoglobulin
D.3.9.1	CAS number	308067-58-5
D.3.9.2	Current sponsor code	Octagam 10%
D.3.9.3	Other descriptive name	IMMUNOGLOBULIN G
D.3.9.4	EV Substance Code	SUB20618
D.3.10	Strength
D.3.10.1	Concentration unit	% percent
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
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)	Yes
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

Primary Immune Thrombocytopenia (ITP)

Първична Имунна Тромбоцитопения

E.1.1.1

Medical condition in easily understood language

immune mediated disorder characterized by increased platelet
destruction

имунно заболяване, характеризиращо се с повишено разрушаване на тромбоцитите

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	15.1
E.1.2	Level	LLT
E.1.2	Classification code	10023095
E.1.2	Term	ITP
E.1.2	System Organ Class	100000004851

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 the efficacy of Octagam 10% in correcting the platelet count (PC).

Да се оцени ефикасността на Octagam 10% при коригиране на броя тромбоцити

E.2.2

Secondary objectives of the trial

To evaluate the safety of Octagam 10%.

Да се оцени безопасността на Octagam 10%

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Age of >=18 and <= 65 years
• Confirmed diagnosis of chronic primary ITP of at least 12 months duration and fulfilling the following criteria...
• Platelet count of <30x10^9/L with or without bleeding manifestations.
• Freely given written informed consent from patient.
• Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropine [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study

• Възраст >= 18 и ≤ 65години
• Потвърдена диагноза за хронична първична ITP с продължителност поне 12 месеца и покрити следните критерии.....
• Брой на тромбоцитите <30x10^9/L със или без прояви на кръвотечение.
• Доброволно предоставено писмено информирано съгласие от пациента.
• Жените с детероден потенциал трябва да имат отрицателен резултат от теста за бременност (изследване на базата на човешки хорионгонадотропин [ЧХГ] и трябва да използват средство за предпазване от забременяване с доказана надеждност през цялото времетраене на проучването.

E.4

Principal exclusion criteria

• Thrombocytopenia secondary to other diseases or drug-related thrombocytopenia.
• Administration of intravenous immunoglobulin (IVIG), anti-D or thrombopoetin receptor agonists or other platelet enhancing drugs (incl. immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrolment, except for:
a) long-term corticosteroid therapy
b) long-term azathioprine, cyclophosphamide or attenuated androgen therapy
• Unresponsive to previous treatment with IVIG or anti-D immunoglobulin.
• Experimental treatment (e.g. Rituximab) within 3 months before enrolment.
• Splenectomy in the previous 4 weeks or planned splenectomy throughout the study period.
• Patient with Evans syndrome
• Known or suspected human immunodeficiency virus (HIV) or hepatitis C virus (HCV) infection.
• Live viral vaccination within the last 2 months before study entry.
• Emergency operation.
• Severe liver or kidney disease
• Congestive heart failure New York Heart Association (NYHA) class III or IV.
• Non-controlled arterial hypertension
• History of hypersensitivity to blood or plasma derived products, or any component of the investigational product.
• Known immunoglobulin A (IgA) deficiency and antibodies against IgA.
• History of, or suspected alcohol or drug abuse.
• Pregnant or nursing women.
• Unable to consent or not capable to understand the nature, significance and implications of the clinical study, or unable or unwilling to comply with the study procedures
• Vulnerable patients (e.g. kept in detention or institutionalised)
• Participating in another interventional clinical study or planned participation in another trial for the duration of this study.
• Receiving any investigational medicinal product (IMP) within 3 months before study entry.
• Patients with risk factors for thromboembolic events in whom the risks outweigh the potential benefit of Octagam treatment.

• Тромбоцитопения, която е вторична при други заболявания или лекарствено свързана тромбоцитопения.
• Интравенозно прилагане на имуноглобулини (IVIG), анти-D или агонисти на тромбопоетиновия рецептор или други медикаменти за усилване на тромбоцитите (вкл. имуносупресори или други имуномодулаторни медикаменти) до 3 седмици преди включване в проучването, с изключение на:
a) продължително лечение с кортикостероиди
b) продължително лечение с азатиоприн, циклофосфамид или атенюирана андрогенна терапия.
• Липса на повлияване от предшестващо лечение с IVIG или анти-D имуноглобулин.
• Експериментално лечение (като напр. Rituximab) до 3 месеца преди включването.
• Спленектомия през последните 4 седмици или планирана спленектомия в периода на проучването.
• Пациенти със синдрома на Evans
• Установена или подозирана инфекция от човешки имунодефицитен вирус (HIV) или от вируса на хепатит C (HCV).
• Ваксинация с жива вирусна ваксина през последните 2 месеца преди включване в проучването.
• Спешна операция.
• Тежко чернодробно или бъбречно заболяване
• Застойна сърдечна недостатъчност от Клас ІІІ или ІV по New York Heart Association (NYHA).
• Неконтролирана артериална хипертония .
• Данни за свръхчувствителност в миналото към произведени от кръв или плазма продукти или към някой от компонентите на изпитвания продукт.
• Известен дефицит на имуноглобулин A (IgA) и антитела срещу IgA.
• Анамнеза или подозрение за злоупотреба с алкохол или наркотици.
• Бременни и кърмещи жени.
• Неспособност да даде информирано съгласие или неспособност да разбере същността, значението и обхвата на клиничното проучване или неспособност или нежелание за спазване на процедурите по проучването
• Уязвими пациенти, т.е. затворници или институционализирани
• Участие в друго интервенционално клинично проучване или планирано участие в друго клинично проучване по време на това проучване.
• Прием на изпитвания медицински продукт (IMP) през последните 3 месеца преди включване в проучването.
• Пациенти с рискови фактори за тромбоемболични събития, при които рисковете превишават вероятната полза от лечението с Octagam.

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy measure is the PC and the increase in platelets to – and the maintenance of – specific thresholds.

Основната мярка за ефикасност е броят на тромбоцитите и увеличението на тромбоцитите до – и подържането на – специфични прагове.

E.5.1.1

Timepoint(s) of evaluation of this end point

continuously, see protocol

постоянно, вж. протокола по проучването

E.5.2

Secondary end point(s)

Secondary Efficacy Endpoints:
• All platelet measurements will be listed and presented in standard summary statistics for each category of response and in total.
• Number and proportion of responders with platelets reaching normal levels
• Maximum PC.
• Regression of haemorrhages.
• Relationship of any new haemorrhages to PC.
Secondary Safety Endpoints:
• Vital signs.
• Physical examinations.
• AEs.
• Laboratory parameters

Вторични крайни точки за оценка на ефикасност:
• Всички измервания на тромбоцитите ще бъдат описани и представени в стандартни статистически кратки описания по отделно за всяка категория на повлияване, както и обобщени.
• Брой и пропорция (относителен дял) на респондерите с достигане на нормално ниво на тромбоцитите
• Максимален брой тромбоцити.
• Регресия на кръвоизливите.
• Връзка на всяка нова хеморагия с броя на тромбоцитите
Вторични крайни точки за оценка на безопасност:
• Жизнени показатели.
• Медицински прегледи.
• НС.
• Лабораторни показатели.

E.5.2.1

Timepoint(s) of evaluation of this end point

continuously, see protocol

постоянно, вж. протокола по проучването

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

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)

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

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

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

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

LVLS

Последно посещение на последен пациент

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)

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

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

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 end of study participation, the patient will receive the most suitable therapy currently available for his condition.

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

2012-06-28

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-07-18

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2013-03-29

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