Clinical Trials Register

Summary
EudraCT Number:	2021-000740-21
Sponsor's Protocol Code Number:	LEX-210
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-12-30
Trial 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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-000740-21

A.3

Full title of the trial

Study of four-factor prothrombin complex concentrate, OCTAPLEX, in patients with acute major bleeding on direct oral anticoagulant (DOAC) therapy with factor Xa inhibitor.

Studio del concentrato di complesso protrombinico a 4 fattori, OCTAPLEX, in pazienti con sanguinamento acuto maggiore che assumono una terapia anticoagulante orale ad azione diretta (DOAC) con un inibitore del fattore Xa

A.3.1

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

Study to demonstrate the efficacy and safety of OCTAPLEX in patients with acute major bleeding that are taking direct oral anticoagulant.

Studio per dimostrare l'efficacia e la sicurezza di OCTAPLEX in pazienti con sanguinamento acuto maggiore che assumono anticoagulanti orali diretti.

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

LEX-210

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04867837

A.5.4

Other Identifiers

Name:

IND

Number:

18149

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	Clinical Project Manager
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	8853
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+41554512177
B.5.6	E-mail	corlex-210@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	Octaplex
D.2.1.1.2	Name of the Marketing Authorisation holder	Octapharma Pharmazeutika Produktionsges.m.b.H
D.2.1.2	Country which granted the Marketing Authorisation	Austria
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	Octaplex
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Powder and solvent for concentrate for 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	COMPLESSO PROTROMBINICO UMANO
D.3.9.1	CAS number	37224-63-8
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	PROTHROMBIN COMPLEX CONCENTRATES
D.3.9.4	EV Substance Code	SUB33011
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	500
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)	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.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	Octaplex
D.2.1.1.2	Name of the Marketing Authorisation holder	Octapharma Pharmazeutika Produktionsges.m.b.H
D.2.1.2	Country which granted the Marketing Authorisation	Austria
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	Octaplex
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Powder and solvent for concentrate for 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	COMPLESSO PROTROMBINICO UMANO
D.3.9.1	CAS number	37224-63-8
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	PROTHROMBIN COMPLEX CONCENTRATES
D.3.9.4	EV Substance Code	SUB33011
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	500
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)	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.IMP: 3
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	Octaplex
D.2.1.1.2	Name of the Marketing Authorisation holder	Octapharma Pharmazeutika Produktionsges.m.b.H
D.2.1.2	Country which granted the Marketing Authorisation	Austria
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	Octaplex
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Powder and solvent for concentrate for 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	COMPLESSO PROTROMBINICO UMANO
D.3.9.1	CAS number	37224-63-8
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	PROTHROMBIN COMPLEX CONCENTRATES
D.3.9.4	EV Substance Code	SUB33011
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1000
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)	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.IMP: 4
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	Octaplex
D.2.1.1.2	Name of the Marketing Authorisation holder	Octapharma Pharmazeutika Produktionsges.m.b.H
D.2.1.2	Country which granted the Marketing Authorisation	Austria
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	Octaplex
D.3.2	Product code	[NA]
D.3.4	Pharmaceutical form	Powder and solvent for concentrate for 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	COMPLESSO PROTROMBINICO UMANO
D.3.9.1	CAS number	37224-63-8
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	PROTHROMBIN COMPLEX CONCENTRATES
D.3.9.4	EV Substance Code	SUB33011
D.3.10	Strength
D.3.10.1	Concentration unit	IU international unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1000
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)	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

Acute major bleeding in patients receiving DOAC therapy with factor Xa inhibitor

Sanguinamento acuto maggiore in pazienti che assumono una terapia DOAC con un inibitore del fattore Xa

E.1.1.1

Medical condition in easily understood language

Acute major bleeding in patients on oral anticoagulants

Sanguinamento acuto maggiore in pazienti che assumono anticoagulanti orali

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	10009731
E.1.2	Term	Coagulation disorder
E.1.2	System Organ Class	100000004851

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 demonstrate superior haemostatic effectiveness of OCTAPLEX dosed at 50 IU/Kg body weight vs. 15 IU/Kg body weight for emergency reversal of the anticoagulant effect of DOACs in patients with major bleeding associated with factor Xa inhibition.

Dimostrare la superiorità dell’efficacia emostatica di OCTAPLEX con una dose di 50 IU/kg di peso corporeo rispetto a 15 IU/kg di peso corporeo per l’inversione di emergenza dell’effetto anticoagulante dei DOAC in pazienti con sanguinamento maggiore associato all’inibizione del fattore Xa

E.2.2

Secondary objectives of the trial

To evaluate the effect of OCTAPLEX on thrombin generation in patients with acute major bleeding on DOAC therapy with factor Xa inhibitor.

To evaluate the safety of OCTAPLEX in patients with acute major bleeding on DOAC therapy with factor Xa inhibitor.

Valutare l’effetto di OCTAPLEX sulla produzione di trombina in pazienti con sanguinamento acuto maggiore durante la terapia con DOAC con un inibitore del fattore Xa.

Valutare la sicurezza di OCTAPLEX in pazienti con sanguinamento acuto maggiore durante la terapia con DOAC con un inibitore del fattore Xa.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Patients who received or who are believed by the investigator to have received a dose of oral factor Xa inhibitor
• Aged =18 years
• Patients who have given written informed consent or for whom written informed consent has been obtained from the patient's legally authorised representative on their behalf
• Patients who have acute major bleeding defined as follows:
- Bleeding that is potentially life-threatening or uncontrolled, e.g., with signs or symptoms of haemodynamic compromise, such as severe hypotension, poor skin perfusion, or low cardiac output that cannot be otherwise explained
OR
- Symptomatic bleeding in critical organs (intracranial, intraspinal, intraocular, retroperitoneal, intra-articular, pericardial, or intramuscular with compartment syndrome)
OR
- Acute overt bleeding associated with a fall in haemoglobin (Hgb) level of =2 g/dL, OR a Hgb level =8 g/dL if no baseline Hgb level is available, OR in the opinion of the investigator that the patient’s Hgb level will fall to =8 g/dL with resuscitation
• Patients with baseline anti-factor Xa activity equivalent to at least 100 ng/mL according to the available test (e.g., chromogenic assay)

• Pazienti che hanno ricevuto o che, secondo il parere dello sperimentatore, hanno ricevuto una dose di inibitore orale del fattore Xa
• Età = 18 anni
• Pazienti che hanno dato il consenso informato scritto o per i quali è stato ottenuto il consenso informato scritto da parte del rappresentante legalmente autorizzato del paziente
• Pazienti con sanguinamento acuto maggiore, definito come segue:
- Sanguinamento potenzialmente fatale o incontrollato, ovvero con segni e sintomi di compromissione emodinamica, come ipotensione grave, scarsa perfusione cutanea o bassa portata cardiaca che non può essere altrimenti giustificata
OPPURE
- Sanguinamento sintomatico in organi critici (intracranica, intraspinale, intraoculare, retroperitoneale, intrarticolare, pericardica, o intramuscolare con sindrome compartimentale)
OPPURE
- Sanguinamento acuto manifesto associato a diminuzione dell’emoglobina (Hgb) di =2 g/dl, OPPURE Hgb =8 g/dl se non è disponibile una misurazione dell’Hgb basale OPPURE se, secondo il parere dello sperimentatore, l’Hgb del paziente diminuirà a =8 g/dl con la rianimazione
• Pazienti con attività basale anti-fattore Xa equivalente ad almeno 100 ng/ml secondo il test disponibile (ad es. saggio cromogenico)

E.4

Principal exclusion criteria

• Patients with bleeding that is immediately life-threatening
• Patients with ‘Do not resuscitate’ (DNR) orders
• Patients with acute trauma for which reversal of DOAC therapy with factor Xa inhibitor alone would not be expected to control the bleeding event
• Hgb decrease without accompanying evidence of source of bleeding
• Acute coronary syndrome, ischaemic stroke or venous thromboembolism (VTE) within the preceding 3 months
• Patients with a history, within the last 3 months, of disseminated intravascular coagulation (DIC) or hyperfibrinolysis
• Patients with a known congenital coagulation disorder
• Known inhibitors to coagulation factors II, VII, IX, or X; or heparin-induced, type II thrombocytopenia
• Known hypersensitivity to plasma-derived products
• Patients who received haemostatic agents, including plasma, platelets, PCC, activated PCC (aPCC), recombinant factor VIIa, or recombinant factor Xa inactivated-zhzo (andexanet alfa), for the current bleeding event prior to enrolment (antifibrinolytic drugs and local haemostatic agents are allowed)
• Patients who received clopidogrel within 5 days or prasugrel within 7 days or ticagrelor within the 48 hours preceding the bleeding event
• Patients on enoxaparin therapy for thromboembolic prophylaxis
• A score of less than 7 on the Glasgow Coma Scale or an estimated intracerebral haematoma volume of more than 60 mL
• Patients with expected survival of less than 3 days
• Patients scheduled to undergo surgery in less than 12 hours, with the exception of some minor/invasive procedures that are allowed for diagnostic or therapeutic reasons (see Section 4.1.2)
• Patients who are pregnant or breastfeeding at the time of enrolment
• Patients previously enrolled in this study
• Patients participating in another interventional clinical treatment study currently or during the past 1 month prior to study inclusion

• Pazienti con sanguinamento potenzialmente fatale nell’immediato
• Pazienti per i quali è stato espresso l’ordine di non rianimare
• Pazienti con trauma acuto per i quali non ci si attende che la sola inversione della terapia con DOAC con inibitore del fattore Xa possa controllare l’evento emorragico
• Diminuzione dell’Hgb in assenza di evidenza correlata in merito all’origine del sanguinamento
• Sindrome coronarica acuta, ictus ischemico o tromboembolismo venoso (TEV) nei 3 mesi precedenti
• Pazienti con anamnesi, nei 3 mesi precedenti, di coagulazione intravascolare disseminata (CID) o iperfibrinolisi
• Pazienti con una patologia congenita della coagulazione nota
• Inibitori noti dei fattori della coagulazione II, VII, IX o X; oppure trombocitopenia di tipo II indotta da eparina
• Ipersensibilità nota ai plasmaderivati
• Pazienti che hanno ricevuto agenti emostatici, compresi plasma, piastrine, PCC, PCC attivato (aPCC), fattore ricombinante VIIa o fattore ricombinante Xa zhzo-inattivato (andexanet alfa), per l’evento emorragico attuale prima dell’arruolamento (sono consentiti farmaci antifibrinolitici e agenti emostatici locali)
• Pazienti che hanno ricevuto clopidogrel nei 5 giorni precedenti o prasugrel nei 7 giorni precedenti o ticagrelor nelle 48 ore precedenti l’evento emorragico
• Pazienti in terapia con enoxaparina per la profilassi tromboembolica
• Punteggio inferiore a 7 sulla Glasgow Coma Scale o un volume stimato dell’ematoma intracerebrale superiore a 60 ml
• Pazienti con sopravvivenza stimata inferiore a 3 giorni
• Pazienti con un intervento chirurgico programmato in meno di 12 ore, ad eccezione di procedure minori/invasive che sono consentite a scopo diagnostico o terapeutico (consultare la Sezione 4.1.2 )
• Pazienti in stato di gravidanza o che allattano al seno al momento dell’arruolamento
• Pazienti precedentemente arruolati in questo studio
• Pazienti che partecipano a un altro studio clinico interventistico che prevede un trattamento al momento o nel mese precedente l’inclusione nello studio

E.5 End points

E.5.1

Primary end point(s)

The proportion of patients in whom OCTAPLEX demonstrates haemostatic effectiveness, i.e., binary outcome of effective (rating of excellent or good) or non-effective (rating of poor/none) in management of major bleeding events.

La percentuale di pazienti per i quali OCTAPLEX dimostra un’efficacia emostatica, ovvero esito binario di efficacia (valutazione eccellente o buona) o non efficacia (valutazione scarsa/nessuna) nella gestione di eventi emorragici maggiori

E.5.1.1

Timepoint(s) of evaluation of this end point

Within 24 hours after the start of initial management, as assessed by the Independent Data Monitoring and Endpoint Adjudication Committee (IDMEAC) according to predefined criteria (Section 7.2 of the protocol).

Entro 24 ore dall’inizio della gestione iniziale, secondo la valutazione del Comitato indipendente per il monitoraggio dei dati e l’assegnazione degli endpoint (IDMEAC) in base a criteri predefiniti (Sezione 7.2 del protocollo)

E.5.2

Secondary end point(s)

• Change in endogenous thrombin potential (ETP) as measured by thrombin generation assay (TGA) from baseline to 1 hour after OCTAPLEX administration
• 30-day event rate of thromboembolic events (TEEs) and all-cause mortality
• Occurrence of adverse events (AEs) during a 48-hours follow up period after OCTAPLEX administration
• Vital signs and laboratory parameters during a 48-hours follow up period after OCTAPLEX administration

• Variazione del potenziale endogeno di trombina (ETP), misurato dal test di generazione della trombina (TGA) dal basale a 1 ora dopo la somministrazione di OCTAPLEX
• Tasso di eventi tromboembolici (TEE) e mortalità per qualsiasi causa a 30 giorni
• Manifestazione di eventi avversi (AE) durante un periodo di follow-up di 48 ore dopo la somministrazione di OCTAPLEX
• Segni vitali e parametri di laboratorio durante un periodo di follow-up di 48 ore dopo la somministrazione di OCTAPLEX

E.5.2.1

Timepoint(s) of evaluation of this end point

From baseline to 30 days after infusion.

Dalla visita basale fino a 30 giorni dopo l'infusione.

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

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

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Stesso farmaco in studio con dose differente

Same study drug with different dose

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

Austria

Belgium

Canada

Germany

Italy

Netherlands

Poland

Spain

Ukraine

United Kingdom

United States

France

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

Last visit last subject

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:

120

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

Yes

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Potentially unconscious subjects.

Soggetti potenzialmente inconsci.

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

100

F.4.2.2

In the whole clinical trial

200

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)

Medical treatment according to local standards.

Cure mediche secondo gli standard locali.

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

2022-03-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-02-08

P. End of Trial
P.	End of Trial Status	Ongoing

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials