Clinical Trials Register

Summary
EudraCT Number:	2011-003448-28
Sponsor's Protocol Code Number:	IgPro20_3003
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-03-29
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2011-003448-28

A.3

Full title of the trial

Randomized, multicenter, double-blind, placebo-controlled, parallelgroup phase III study to investigate the efficacy, safety, and tolerability of 2 different doses of IgPro20 (subcutaneous immunoglobulin) for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) – the PATH study

Studio di fase 3, multicentrico, randomizzato, in doppio cieco, controllato verso placebo, a gruppi paralleli teso a valutare l'ÃƒÂƒÃ‚Â‚ÃƒÂ‚Ã‚Â™efficacia, la sicurezza e la tollerabilita' di 2 diverse dosi di IgPro20 (immunoglobulina per via sottocutanea [SC]) per il trattamento della polineuropatia demielinizzante infiammatoria cronica (CIDP) ' Studio PATH

A.3.1

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

Chronic inflammatory demyelinating polyneuropathy (CIDP) and treatment with subcutaneous immunoglobulin (IgPro20)

IgPro20 (immunoglobulina per via sottocutanea [SC]) per il trattamento della polineuropatia demielinizzante infiammatoria cronica (CIDP)

A.3.2

Name or abbreviated title of the trial where available

PATH

A.4.1

Sponsor's protocol code number

IgPro20_3003

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	CSL BEHRING GMBH
B.1.3.4	Country	Germany
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	CSL Behring GmbH
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	CSL Behring GmbH
B.5.2	Functional name of contact point	Trial Disclosure Manager
B.5.3	Address:
B.5.3.1	Street Address	Emil-von-Behring-Strasse 76
B.5.3.2	Town/ city	Marburg
B.5.3.3	Post code	35041
B.5.3.4	Country	Germany
B.5.4	Telephone number	---
B.5.5	Fax number	---
B.5.6	E-mail	clinicaltrials@cslbehring.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	Hizentra®
D.2.1.1.2	Name of the Marketing Authorisation holder	CSL Behring GmbH
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	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	HUMAN NORMAL IMMUNOGLOBULIN
D.3.9.1	CAS number	0
D.3.9.2	Current sponsor code	IgPro10-SOL
D.3.9.4	EV Substance Code	SUB14196MIG
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	200
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
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Polineuropatia demielinizzante infiammatoria cronica (CIDP)

E.1.1.1

Medical condition in easily understood language

Inflammatory disease of peripheral nervous system

Malattia infiammatoria del sistema nervoso periferico

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.1
E.1.2	Level	PT
E.1.2	Classification code	10061811
E.1.2	Term	Demyelinating polyneuropathy
E.1.2	System Organ Class	10029205 - Nervous system disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To determine the efficacy of 2 different doses of IgPro20 (0.2 g/kg bw and/or 0.4 g/kg bw) in the maintenance treatment of CIDP in comparison to placebo.

Determinare l’efficacia di 2 dosi diverse di IgPro20 (0,2 g/kg di peso corporeo e/o 0,4 g/kg di peso corporeo) nel trattamento di mantenimento della CIDP rispetto a placebo.

E.2.2

Secondary objectives of the trial

- To investigate the efficacy of IgPro20 with additional clinical outcome measures in comparison to placebo. - To investigate the safety and tolerability of IgPro20 in comparison to placebo. - To investigate health-related quality of life (HRQL) following treatment with IgPro20.

• valutare l’efficacia di IgPro20 rispetto a placebo in altre misure di esito clinico; • valutare la sicurezza e la tollerabilità di IgPro20 rispetto a placebo; • valutare la qualità di vita correlata alla salute (HRQL) dopo il trattamento con IgPro20.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Definite or probable CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria 2010. 2. Repeated treatment with IVIG (≥ 4 infusions) within the last 9 months prior to enrollment. 3. An IVIG treatment during the last 8 weeks prior to enrollment. 4. Age ≥18 years. 5. Written informed consent for study participation obtained before undergoing any study-specific procedures.

1. CIDP confermata o sospetta in base ai criteri EFNS/PNS 2010. 2. Trattamento ripetuto con IVIg (≥ 4 infusioni) negli ultimi 9 mesi prima dell’arruolamento. 3. Trattamento con IVIg nelle ultime 8 settimane prima dell’arruolamento. 4. Età ≥ 18 anni. 5. Consenso informato scritto alla partecipazione allo studio ottenuto prima di sottoporre il soggetto a qualsiasi procedura specifica dello studio.

E.4

Principal exclusion criteria

1. Any polyneuropathy of other causes 2. Any other disease (mainly neurological or chronic orthopedic) that has caused neurological symptoms or may interfere with treatment oroutcome assessments 3. Severe diseases and conditions that are likely to interfere with evaluation of the study product or satisfactory conduct of the study 4. History of thrombotic episodes within the 2 years prior to enrolment 5. Known allergic or other severe reactions to blood products including intolerability to previous IVIG

1. Qualsiasi polineuropatia per altre cause;2. Eventuale altra patologia (principalmente di origine neurologica o ortopedica cronica) che abbia provocato sintomi neurologici o che possa interferire con le valutazioni del trattamento o degli esiti;3. Malattie e affezioni gravi che probabilmente interferirebbero con la valutazione del prodotto in studio o con la conduzione soddisfacente dello studio;4. Storia di episodi trombotici nei 2 anni precedenti l’arruolamento;5. Reazioni di tipo allergico o altre reazioni gravi note a prodotti ematici, inclusa l’intolleranza alla precedente IVIG.

E.5 End points

E.5.1

Primary end point(s)

Percentage (%) of subjects who relapse during the SC treatment period

Percentuale (%) di soggetti che manifestano recidiva* durante il trattamento SC.

E.5.1.1

Timepoint(s) of evaluation of this end point

24 weeks

24 settimane

E.5.2

Secondary end point(s)

1. Change in mean Inflammatory Neuropathy Cause and Treatment (INCAT) scores during the SC treatment period 2. Change in mean maximum grip strength scores during the SC treatment period 3. Change in mean Medical Research Council (MRC) sum scores during the SC treatment period 4. Change in mean Rasch-built Overall Disability Scale (R-ODS) scores during the SC treatment period 5. Time to CIDP relapse 6. Rate of adverse events per SC infusion 7. Number of subjects with adverse events during the SC Treatment Period 8. Percentage of subjects with adverse events during the SC Treatment Period

Variazioni nelle medie del punteggio INCAT durante il Periodo di trattamento SC 2-Variazioni nelle medie del punteggio forza di presa massima (mano dominante/non-dominante) durante il Periodo di trattamento SC ; 3-Variazioni nelle medie del punteggio MRC complessivo durante il Periodo di trattamento SC ; 4- Variazioni nelle medie del punteggio del questionario R-ODS durante il Periodo di trattamento SC. Differenza nel “tempo alla recidiva della CIDP” Tasso di AE per infusione durante il Periodo di trattamento SC Numero di soggetti con AE durante il Periodo di trattamento SC 8- Percentuale di soggetti con AE durante il Periodo di trattamento

E.5.2.1

Timepoint(s) of evaluation of this end point

1. - 4. SC week 1, SC week 25 5. up to 24 weeks 6. - 8. 24 weeks

1. - 4. SC alla settimana 1, SC alla settimana 25 5. Fino a 24 settimane, 6.-8. 24 settimane

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

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

Yes

E.6.13.1

Other scope of the trial description

Health-related quality of life (HRQL)

Qualità di vita correlata alla salute (HRQL)

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

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

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

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

Argentina

Australia

Canada

Korea, Republic of

Switzerland

United States

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''

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

Number of subjects for this age range:

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:

225

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

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

147

F.4.2.2

In the whole clinical trial

250

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)

Standard care for CIDP

Terapia standard per CIDP

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

2012-08-23

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-03-12

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

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