Clinical Trials Register

Summary
EudraCT Number:	2014-005496-87
Sponsor's Protocol Code Number:	161403
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:	2021-01-22
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2014-005496-87

A.3

Full title of the trial

A Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of
Immune Globulin Infusion 10% (Human) with Recombinant Human
Hyaluronidase (HYQVIA/HyQvia) and Immune Globulin Infusion (Human),
10% (GAMMAGARD LIQUID/KIOVIG) for the Treatment of Chronic
Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Studio di fase III per valutare l¿efficacia, la sicurezza e la tollerabilit¿ di un¿infusione di immunoglobulina (umana) al 10% con ialuronidasi umana ricombinante (HYQVIA/HyQvia) e di un¿infusione di immunoglobulina (umana) al 10% (LIQUIDO DI GAMMAGARD/KIOVIG) per il trattamento della poliradiculoneuropatia demielinizzante infiammatoria cronica (CIDP)

A.3.1

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

Study to asses if the immunoglobulin product for subcutaneous use in
combination with Hyaluronidase has a positive effect on your disease, and
if it safe to use. The study will also assess if the intravenous
immunoglobulin product has a positive effect and is safe to use.

Studio finalizzato alla valutazione dell'efficacia e sicurezza di immunoglobuline somministrate per via sottocutanea in combinazione con ialuronidasi nel trattamento della CIDP. Lo studio tester¿ anche l'efficacia e la sicurezza delle immunoglobuline somministrate per endovena.

A.3.2

Name or abbreviated title of the trial where available

Phase III Efficacy, Safety, and Tolerability Study of HyQvia and KIOVIG in CIDP

Studio di fase 3 per testare l'efficacia la sicurezza e la tollerabilit¿ di HyQvia e di Kiovig nella

A.4.1

Sponsor's protocol code number

161403

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02549170

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	BAXTER S.P.A.
B.1.3.4	Country	Austria
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	Baxalta Innovations GmbH
B.4.2	Country	Austria
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Baxalta Innovation GmbH
B.5.2	Functional name of contact point	Andras Nagy
B.5.3	Address:
B.5.3.1	Street Address	Industriestrasse 67
B.5.3.2	Town/ city	Vienna
B.5.3.3	Post code	1221
B.5.3.4	Country	Austria
B.5.4	Telephone number	431201002476725
B.5.5	Fax number	000000000
B.5.6	E-mail	andras.nagy@shire.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	HYQVIA - 100 MG/ML - SOLUZIONE PER INFUSIONE - USO SOTTOCUTANEO - FLACONCINO (VETRO) 300 ML E FLACONCINO (VETRO) 15 ML - 1 FLACONCINO + 1 FLACONCINO
D.2.1.1.2	Name of the Marketing Authorisation holder	BAXTER INNOVATIONS GMBH
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	HyQvia
D.3.2	Product code	[non fornito]
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	Immunoglobulina normale umana
D.3.9.2	Current sponsor code	non fornito
D.3.9.3	Other descriptive name	Himan Normal Immunoglobulin
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	100
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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.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	KIOVIG - 100 MG/ML SOLUZIONE PER INFUSIONE USO INTRAVENOSO FLACONE VETRO 1 G/10 ML 1 FLACONE
D.2.1.1.2	Name of the Marketing Authorisation holder	BAXTER AG
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	Kiovig
D.3.2	Product code	[non fornito]
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	Immunoglobulina normale umana
D.3.9.2	Current sponsor code	non fornito
D.3.9.3	Other descriptive name	Human normal immunoglobulin
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	100
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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
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 infusion
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 polyradiculoneuropathy

Poliradicolopatia acuta infiammatoria demielinizzante

E.1.1.1

Medical condition in easily understood language

Chronic Inflammatory Demyelinating Polyneuropathy is a rare disorder
of the peripheral nerves (nerves outside the central nervous system)
caused by damage to the covering of the nerves, called myelin.

La Poliradicolopatia acuta infiammatoria demielinizzante è un raro disturbo dei nervi periferici (nervi fuori dal sistema nervoso centrale) per un deterioramento della mielina che ricopre i nervi.

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	20.0
E.1.2	Level	SOC
E.1.2	Classification code	10029205
E.1.2	Term	Nervous system disorders
E.1.2	System Organ Class	10029205 - Nervous system disorders

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

Epoch 1:SC Treatment Period
1.To evaluate the efficacy of HyQvia as a maintenance therapy for CIDP
to prevent relapse of neuromuscular disability and impairment.
Epoch 2: IV Treatment Period
1.To evaluate the efficacy of KIOVIG for the treatment of CIDP to
improve neuromuscular disability and impairment.

Fase 1: valutare l¿efficacia di HYQVIA/HyQvia come terapia di mantenimento per la CIDP intesa a prevenire la recidiva di invalidit¿ e insufficienza neuromuscolare.
fase 2. Valutare l¿efficacia del LIQUIDO di GAMMAGARD/KIOVIG nel trattamento della CIDP per migliorare l¿invalidit¿ e l¿insufficienza neuromuscolare.

E.2.2

Secondary objectives of the trial

Epoch 1:SC Treatment Period
1. To assess the time to CIDP relapse during maintenance therapy with
HyQvia, compared to placebo.
2. To assess the effect of HyQvia on activities of daily living (ADL).
3. To assess the safety and tolerability of HyQvia.
4. To monitor for the presence of binding and neutralizing anti-rHuPH20
antibodies following HyQvia administration.
Epoch 2: IV Treatment Period
1. To assess the safety and tolerability of KIOVIG.

Fase 1: Valutare il tempo prima di incorrere in una recidiva di CIDP durante la terapia di mantenimento con HYQVIA/HyQvia, rispetto al placebo.
Valutare l¿effetto di HYQVIA/HyQvia sulle attivit¿ della vita quotidiana (Activities of Daily Living, ADL).
Valutare la sicurezza e la tollerabilit¿ di HYQVIA/HyQvia.
Monitorare la presenza di anticorpi leganti e neutralizzanti anti-rHuPH20 a seguito della somministrazione di HYQVIA/HyQvia.
Fase 2: Valutare la sicurezza e la tollerabilit¿ del LIQUIDO di GAMMAGARD/KIOVIG.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male and female subjects of age = 18 years at the time of screening.
2. The subject has a documented diagnosis of certain or probable CIDP (atypical CIDP and atypical sensory CIDP will be excluded)
3.The subject has responded to a previous treatment of IgG (partial or complete resolution of neurologic symptoms and deficits) and must currently be treated with stable doses of IGIV treatment included in the dosage interval equivalent to a monthly cumulative dose of 0, 5 and 2.4 g / kg (inclusive) of BW
administered intravenously for at least 12 weeks prior to screening. The IGIV treatment administration interval should be between 2 and 6 weeks (inclusive).
4.The INCAT disability score must be between 0 and 7 (inclusive). For subjects with INCAT scores of 0, 1 (arising from the upper or lower limbs) or 2 (if at least 1 point is derived from an upper limb), screening and / or baseline will be required to present a history of significant disability according to the definition determined by an INCAT disability score equal to 2 (exclusively deriving from the lower limbs) or higher in the documented in the medical record. Subjects will be eligible if one of the eligibility criteria below is met:
a. IncAT disability score at screening or baseline between 3 and 7 (inclusive).
b. IncAT score for baseline and / or baseline disability of 2 (both scores refer to lower extremities)
c. IncAT score for baseline and / or baseline disability of 2 (both scores are not derived from the lower extremities). It shows at least a score equal to or greater than 2 documented in the medical record before screening. If a score was greater than 2 documented in the medical record before screening, at least 2 points must derive from the lower extremities.
d. IncAT score for baseline and / or basal disability of 0 or 1 And at least a score of 2 or higher (both from the lower extremities) documented in the medical record before screening, at least 2 points must be derived from the lower extremities.

5.For females of child bearing age, the subject must present a pregnancy test with negative screening results and agree to adopt a highly effective contraceptive method during the study and for at least 30 days after the last administration of the product experimental.
6.The subject intends and is able to sign an Informed Consent form
7. The subject intends and is able to comply with the requirements set by the protocol.

1.Soggetti di sesso maschile e femminile di età = 18 anni al momento dello screening.
2.Il soggetto presenta una diagnosi documentata di CIDP certa o probabile (sarà esclusa la CIDP atipica e la CIDP pura sensoriale atipica)
3.Il soggetto ha risposto a un pregresso trattamento di IgG (risoluzione parziale o completa di sintomi e deficit neurologici) e deve essere attualmente trattato con dosi stabili di trattamento IGIV comprese nell’intervallo di dosaggio equivalente a una dose cumulativa mensile compresa tra 0,5 e 2,4 g/kg (inclusi) di BW
somministrata per via endovenosa per almeno 12 settimane prima dello screening. L’intervallo di somministrazione del trattamento IGIV deve essere compreso tra 2 e 6 settimane (incluse).
4.Il punteggio di invalidità INCAT deve essere compreso tra 0 e 7 (inclusi). Ai soggetti con punteggi INCAT pari a 0, 1 (derivanti dagli arti inferiori o superiori) o 2 (se almeno 1 punto deriva da un arto superiore) allo screening e/o al basale sarà richiesto di
presentare un’anamnesi di invalidità rilevante secondo la definizione determinata da un punteggio di invalidità INCAT pari a 2 (esclusivamente derivante dagli arti inferiori) o superiore nei documentato nella cartella clinica. I soggetti saranno idonei se viene soddisfatto uno dei criteri di idoneità qui sotto:
a. Punteggio di invalidità INCAT allo screening o al basale compreso tra 3 e 7 (inclusi).
b. Punteggio di invalidità INCAT allo screening e/o al basale pari a 2 (entrambi i punteggi riguardano le estremità inferiori)
c. Punteggio di invalidità INCAT allo screening e/o al basale pari a 2 (entrambi i punteggi non derivano dalle estremità inferiori) E mostra almeno un punteggio pari o superiore a 2 documentato nella cartella clinica prima dello screening. Se un punteggio era superiore a 2 documentato nella cartella clinica prima dello screening, almeno 2 punti devono derivare dalle estremità inferiori.
d. Punteggio di invalidità INCAT allo screening e/o al basale pari a 0 o 1 E almeno un punteggio di 2 o superiore (entrambi dalle estremità inferiori) documentato nella cartella clinica prima dello screening, almeno 2 punti devono derivare dalle estremità inferiori.

5.Per i soggetti di sesso femminile in età fertile, il soggetto deve presentare un test di gravidanza con esito negativo allo screening e accettare di adottare un metodo contraccettivo altamente efficace nel corso dello studio e per almeno 30 giorni dopo l’ultima somministrazione del prodotto sperimentale.
6.Il soggetto intende ed è in grado di firmare un modulo di Consenso Informato
7.Il soggetto intende ed è in grado di attenersi ai requisiti previsti dal protocollo.

E.4

Principal exclusion criteria

1. Subjects with atypical focal CIDP or atypical sensory CIDP.
2. Any neuropathy due to other causes, including:
a. Hereditary demyelinating neuropathies, such as hereditary sensory and motor neuropathy (HSMN) (Charcot-Marie-Tooth disease [CMT])]), acquired multifocal sensorimotor demyelinating neuropathy (multifocal acquires demyelinating sensory and motor neuropathy, MADSAM) and sensory neuropathies and autonomic hereditary (HSAN)
b. Neuropathies secondary to systemic infections, disorders or diseases such as infection with Borrelia burgdorferi (Lyme disease), diphtheria, systemic lupus erythematosus, POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, M-protein and skin changes), osteosclerotic myeloma, radial-plexus neuropathy - diabetic and nondiabetic lumbosacral, lymphoma and amyloidosis
c. Multifocal motor neuropathy (MMN)
d. Peripheral neuropathy induced by drugs, biological substances, chemotherapy or toxins
3.Map-immunoglobulin immunoglobulin M (IgM), including monoclonal IgM gammopathy with antibodies with a high titer of glycoprotein associated with myelin
4. Presence of prominent sphincter disorder.
5. Any central demyelinating disorder, including multiple sclerosis.
6. Any chronic or debilitating disease or central nervous system disorder that causes neurological symptoms or may interfere with CIDP assessment or outcome measures, including (without limitation) arthritis, stroke, Parkinson's disease and diabetic peripheral neuropathy
7. The subject has received or is receiving treatment with immunomodulatory / immunosuppressive agents within 6 months prior to screening.
8. Subject has received or is receiving corticosteroid treatment within 3 months prior to screening. The following exceptions are allowed for prednisolone or its equivalents: stable doses of low-dose systemic corticosteroids (= 10 mg prednisolone / day or its equivalent) and non-systemic corticosteroids (eg topical glucocorticoids, ophthalmic or inhaled). In addition and for the purpose of treating EAs or non-CIDP intercurrent disease, a single dose of corticosteroids> 10 mg prednisolone or a single short cycle = 7 days (eg a dose of methylprednisolone) is allowed within 3 months prior to screening.
9. The subject has undergone plasma exchange within 3 months prior to screening.
10. The subject presents any pathology or condition which, in the opinion of the investigator, may hinder the participation of the subject in the study, constitute a greater risk for the subject or confuse the results of the study.
11. The subject breastfeeds or intends to start lactation during the course of the study

1.Soggetti con CIDP atipica focale o CIDP atipica sensoriale pura.
2.Qualsiasi neuropatia riconducibile ad altre cause, tra cui:
a. Neuropatie ereditarie demielinizzanti, come neuropatia ereditaria sensoriale e motoria (HSMN) (malattia di Charcot-Marie-Tooth [CMT]) ]), neuropatia multifocale demielinizzante sensitivo-motoria acquisita (multifocal acquires demyelinating sensory and motor neuropathy, MADSAM) e neuropatie sensoriali e autonomiche ereditarie (HSAN)
b. Neuropatie secondarie a infezioni, disturbi o malattie sistemiche come infezione da Borrelia burgdorferi (malattia di Lyme), difterite, lupus eritematoso sistemico, sindrome di POEMS (polineuropatia, organomegalia, endocrinopatia, proteina M e alterazioni cutanee), mieloma osteosclerotico, neuropatia radicolo-plesso-lombosacrale diabetica e non diabetica, linfoma e amiloidosi
c. Neuropatia motoria multifocale (MMN)
d. Neuropatia periferica indotta da farmaci, sostanze biologiche, chemioterapia o tossine
3.Paraproteinemia dell’immunoglobulina M (IgM), tra cui gammopatia monoclonale di tipo IgM con anticorpi ad alto titolo di glicoproteina associata alla mielina
4.Presenza di disturbo da sfintere prominente.
5.Qualsiasi disturbo demielinizzante centrale, tra cui sclerosi multipla.
6.Qualsiasi malattia cronica o debilitante o disturbo del sistema nervoso centrale che causi sintomi neurologici o possa interferire con la valutazione della CIDP o le misure degli esiti, tra cui (senza limitazioni) artrite, ictus, malattia di Parkinson e neuropatia periferica diabetica
7. Il soggetto ha ricevuto o sta ricevendo un trattamento con agenti immunomodulatori/immunosoppressori entro 6 mesi prima dello screening.
8. Il soggetto ha ricevuto o sta ricevendo un trattamento con corticosteroidi entro 3 mesi prima dello screening. Sono ammesse le seguenti eccezioni per il prednisolone o suoi equivalenti: dosaggi stabili di corticosteroidi sistemici a basso dosaggio (= 10 mg di prednisolone/giorno o il suo equivalente) e corticosteroidi non sistemici (ad esempio, glucocorticoidi topici, oftalmici o inalatori). Oltre e al fine di trattare gli EA o la malattia intercorrente non CIDP, è consentita una dose singola di corticosteroidi > 10 mg di prednisolone o un singolo ciclo breve = 7 giorni (ad esempio una confezione di dose di metilprednisolone) entro 3 mesi prima dello screening.
9. Il soggetto ha subito uno scambio di plasma entro 3 mesi prima dello screening.
10. Il soggetto presenta qualsiasi patologia o condizione che, a giudizio dello sperimentatore, possa ostacolare la partecipazione del soggetto allo studio, costituire un maggiore rischio per il soggetto o confondere i risultati dello studio.
11. Il soggetto allatta o intende iniziare l’allattamento durante il corso dello studio

E.5 End points

E.5.1

Primary end point(s)

Epoch 1: SC Treatment Period
Relapse rate
Epoch 2: IV Treatment Period
Responder rate

Fase 1: Trattamento SC: tasso di recidiva
Fase 2:Trattamento EV: tasso di risposta

E.5.1.1

Timepoint(s) of evaluation of this end point

Epoch 1: SC Treatment Period
up to 6 months
Epoch 2: IV Treatment Period
up to 6 months

Fase 1: Trattamento SC: fino a 6 mesi
Fase 2: Trattamento EV: fino a 6 mesi

E.5.2

Secondary end point(s)

Phase I, subcutaneous treatment:
Effectiveness
1. Percentage of subjects experiencing worsening of functional disability defined as an increase of = 1 point from baseline score prior to SC treatment in 2 consecutive adjusted scores of INCAT O disability that show a worsening of CIDP (defined as a decrease = 8 kPa in the grip force of the hand in the most affected hand) OR a decrease = 4 points in the R-ODS compared to the baseline score before the SC treatment at 2 consecutive time points (at the time of withdrawal from the SC treatment period)
2. Time to relapse
3. Change from baseline R-ODS score prior to SC treatment)
Safety
Nr (%) of subjects with EA / Nr (%) of subjects with EA associated with infusions / Nr (%) of reductions, interruptions, discontinuations due to EA / Systemic and local EA rates
Phase II Intravenous treatment:
Safety
Nr (%) of subjects with EA / Nr (%) of subjects with EA associated with infusions / Nr (%) of reductions, interruptions, discontinuations due to EA / Incidence of development of binding and / or neutralizing antibodies to rHuPH20 / Rates of EA systemic and local
Effectiveness
1. Percentage of subjects with clinically significant improvement in functional capacity, defined as a decrease of = 1 point in the adjusted INCAT O disability score showing an improvement in CIDP (defined as an increase of = 8 kPa in the hand holding force most affected OR an increase of = 4 points in the R-ODS) at the end of the EV treatment period [6 months] or at the last study visit of the EV treatment period, compared to the baseline score preceding the EV treatment

E.5.2 End point secondario (ripetere se necessario):
Fase I, Trattamento sottocutaneo:
Efficacia
1. Percentuale di soggetti che manifesta un peggioramento dell’invalidità funzionale definito come un aumento = 1 punto rispetto al punteggio basale precedente al trattamento SC in 2 punteggi aggiustati consecutivi dell’invalidità INCAT O che presentano un peggioramento della CIDP (definito come una diminuzione = 8 kPa nella forza di presa della mano nella mano maggiormente interessata) O una diminuzione = 4 punti nell’R-ODS rispetto al punteggio basale precedente al trattamento SC a 2 punti temporali consecutivi (al momento del ritiro dal periodo di trattamento SC)
2. Tempo alla recidiva
3. Variazione dal punteggio R-ODS basale precedente al trattamento SC)
Sicurezza
Nr (%) di soggetti con EA / Nr (%) di soggetti con EA associati alle infusioni / Nr (%) di riduzioni, interruzioni, discontinuazioni dovute ad EA / Tassi di EA sistemici e locali
Fase II Trattamento endovena:
Sicurezza
Nr (%) di soggetti con EA / Nr (%) di soggetti con EA associati alle infusioni / Nr (%) di riduzioni, interruzioni, discontinuazioni dovute ad EA / Incidenza di sviluppo di anticorpi leganti e/o neutralizzanti a rHuPH20 / Tassi di EA sistemici e locali
Efficacia
1. Percentuale di soggetti con miglioramento clinicamente significativo nella capacità funzionale, definita come una diminuzione di = 1 punto nel punteggio aggiustato di invalidità INCAT O che presentano un miglioramento della CIDP (definito come un incremento = 8 kPa nella forza di presa della mano nella mano maggiormente interessata O un incremento = 4 punti nell’R-ODS) al termine del periodo di trattamento EV [6 mesi] o all’ultima visita dello studio del periodo di trattamento EV, rispetto al punteggio basale precedente al trattamento EV

E.5.2.1

Timepoint(s) of evaluation of this end point

up to 6 months

fino a 6 mesi

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

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

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

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

fase 1: doppio cieco- fase 2 in aperto

Epoch 1: Double blind. Epoch 2: open label

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

Information not present in EudraCT

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

Brazil

Canada

Colombia

Israel

Mexico

Russian Federation

Serbia

Turkey

United States

Austria

Czechia

Denmark

France

Germany

Greece

Italy

Norway

Poland

Portugal

Slovakia

Spain

Sweden

Switzerland

United Kingdom

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

study completion is last subject last visit.

la fine dello studio ¿ la data della visita dell'ultimo paziente

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:

197

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

164

F.4.2.2

In the whole clinical trial

232

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 of treatment

Trattamento standard

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

2018-06-14

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-09-13

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

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Orphan Designation Number:
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