Clinical Trials Register

Summary
EudraCT Number:	2017-002432-17
Sponsor's Protocol Code Number:	ALN-AS1-003
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:	2021-02-02
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

2017-002432-17

A.3

Full title of the trial

ENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias

ENVISION: Studio di fase 3, multicentrico, randomizzato, in doppio cieco, controllato verso placebo, con estensione in aperto volto a valutare l¿efficacia e la sicurezza di givosiran nei pazienti affetti da porfirie epatiche acute

A.3.1

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

Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic Porphyrias

Studio del farmaco sperimentale, Givosiran, per il trattamento delle porfirie epatiche acute

A.3.2

Name or abbreviated title of the trial where available

ENVISION

A.4.1

Sponsor's protocol code number

ALN-AS1-003

A.5.1

ISRCTN (International Standard Randomised Controlled Trial) Number

ISRCTN12345678

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03338816

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1234-1234-1234

A.5.4

Other Identifiers

Name:

IND

Number:

126094

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	ALNYLAM PHARMACEUTICALS, INC.
B.1.3.4	Country	United States
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	Alnylam Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Medpace
B.5.2	Functional name of contact point	Regulatory Submissions
B.5.3	Address:
B.5.3.1	Street Address	5375 Medpace Way
B.5.3.2	Town/ city	Cincinnati, Ohio
B.5.3.3	Post code	45227
B.5.3.4	Country	United States
B.5.4	Telephone number	00498989557180
B.5.5	Fax number	004989895571810
B.5.6	E-mail	regsubmissions@medpace.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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/16/1731
D.3 Description of the IMP
D.3.1	Product name	Givosiran
D.3.2	Product code	ALN-AS1
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	ALN-60519
D.3.9.1	CAS number	1639325-44-2
D.3.9.2	Current sponsor code	ALN-60519
D.3.10	Strength
D.3.10.1	Concentration unit	mg/l milligram(s)/litre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	189
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	No
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

Acute Hepatic Porphyrias (AHP)

Porfiria Epatica Acuta

E.1.1.1

Medical condition in easily understood language

Acute hepatic porphyrias (AHP) represent a sub-group of porphyrias characterized by the occurrence of neuro-visceral attacks with or without cutaneous manifestations

La porfiria epatica acuta (AHP) rappresenta un sottogruppo di porfiria caratterizzato dal verificarsi di attacchi neuro-viscerali con o senza manifestazioni cutanee

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10036182
E.1.2	Term	Porphyria acute
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10036182
E.1.2	Term	Porphyria acute
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

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	10036184
E.1.2	Term	Porphyria hepatic
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10036182
E.1.2	Term	Porphyria acute
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic 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

Evaluate the effect of subcutaneous (SC) givosiran, compared to placebo, on the rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or intravenous (IV) hemin administration at home in patients with acute intermittent porphyria (AIP)

Valutare l¿effetto di givosiran (ALN-AS1) somministrato per via sottocutanea, rispetto al placebo, sul tasso di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti affetti da porfiria intermittente (AIP).

E.2.2

Secondary objectives of the trial

¿ Evaluate the effect of givosiran, compared to placebo, on urinary aminolevulinic acid (ALA) levels
in patients with AIP
¿ Evaluate the effect of givosiran, compared to placebo, on urinary porphobilinogen (PBG) levels in
patients with AIP
¿ Evaluate the effect of givosiran, compared to placebo, on hemin usage in patients with AIP
¿ Evaluate the effect of givosiran, compared to placebo, on the rate of porphyria attacks requiring
hospitalization, urgent healthcare visit, or IV hemin administration at home in patients with any AHP
¿ Evaluate the effect of givosiran, compared to placebo, in patients with AIP on the symptoms of
pain, nausea, and fatigue
¿ Evaluate the effect of givosiran, compared to placebo, in patients with AIP on the Physical
Component Summary (PCS) of the 12-item Short-Form Health Survey (SF-12)
¿ Evaluate the safety and tolerability of givosiran in patients with any AHP

¿ Valutare l'effetto di givosiran, rispetto al placebo, sui livelli di acido aminolevulinico (ALA) urinario (ALA) nei pazienti con AIP
¿ Valutare l¿effetto di givosiran, rispetto al placebo, sui livelli di porfobilinogeno (PBG) urinario nei pazienti con AIP
¿ Valutare l¿effetto di givosiran, rispetto al placebo, sull¿uso di emina nei pazienti con AIP
¿ Valutare l¿effetto di givosiran rispetto al placebo, sul tasso di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti affetti da porfiria epatica acuta (AHP)
¿ Valutare l'effetto di givosiran, rispetto al placebo, nei pazienti con AIP sui sintomi del dolore, della nausea e della stanchezza
¿ Valutare l'effetto di givosiran, rispetto al placebo, nei pazienti con AIP sul Physical Component Summary (PCS) del Questionario sullo stato di salute SF-12 [12-item Short-Form Health Survey]
¿ Valutare la sicurezza e la tollerabilit¿ di givosiran nei pazienti con AIP

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Age =12 years
2. Documented diagnosis of AIP, HCP, VP, or ADP based on clinical features (eg, acute
attacks of abdominal, back, chest, extremities, and/or limb pain), at least one documented
urinary or plasma PBG or ALA value =4× upper limit of normal (ULN) within the past
year prior or during Screening, AND one of the following:
¿ Documented genetic evidence of mutation in a porphyria-related gene, defined as
ANY of the following:
¿ AIP: mutation in the hydroxymethylbilane synthase gene (HMBS; also
referred to as the porphobilinogen deaminase [PBGD] gene)
¿ HCP: mutation in the coproporphyrinogen oxidase (CPOX) gene
¿ VP: mutation in the protoporphyrinogen oxidase (PPOX) gene
¿ ADP: mutation in the aminolevulinic acid dehydratase (ALAD) homozygous
or compound heterozygous genes
¿ OR if the results of a patient’s genetic testing do not identify a mutation in a
porphyria-related gene (<5% of cases), a patient may be eligible for the study if
they have both clinical features and diagnostic biochemical criteria consistent with
AHP (Table 10)
3. Have active disease, with at least 2 porphyria attacks requiring hospitalization, urgent
healthcare visit or treatment with IV hemin at home within the 6 months prior to
Screening
4. Willing to discontinue and/or not initiate use of prophylactic hemin at the time of
Screening and for the duration of the study
5. Have adequate venous access for study sample collection as judged by the investigator
6. Be willing to comply with the contraceptive requirements during the study period, as
described in Section 6.4.
7. Be willing and able to comply with the study requirements and to provide written
informed consent as per local and national requirements. In the case of patients under the age of legal consent, legal guardian(s) must provide written informed consent and the patient
should provide assent per
local and national requirements and institutional standards

1. Età = a 12 anni;
2. Diagnosi documentata di AIP, HCP, VP o ADP basata su caratteristiche cliniche (es attacchi acuti di dolore addominale, dolore alla schiena, al petto alle estremità e/o alle gambe), almeno un valore PBG o ALA, urinario o plasmatico, = 4 volte superiore al normale (ULN) nell’anno precedente o durante lo screening e uno dei seguenti:
• AIP: mutazione nel gene dell'idrossimetilbilano-sintasi (HMBS, noto anche come gene porfobilinogeno deaminasi [PBGD])
• HCP: mutazione nel gene della coproporfirinogeno ossidasi (CPOX)
• VP: mutazione nel gene della protoporfirinogeno ossidasi (PPOX)
• ADP: mutazione nei geni omozigoti o eterozigoti composti dell’acido aminolevulinico deidrato (ALAD)
• OR se i risultati dei test genetici di un paziente non identificano una mutazione in un gene correlato alla porfiria (<5% dei casi), un paziente può essere ammesso allo studio se dispongono sia di caratteristiche cliniche che di criteri diagnostici biochimici coerenti con AHP
3. Avere malattia attiva con almeno 2 attacchi che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio prima dello screening
4. Volontà di interrompere e/o non iniziare l’uso profilattico di emina al momento dello screening a per l’intera durata dello studio
5. Avere un'adeguata accessibilità venosa per la raccolta di campioni di studio come giudicata dall'investigatore
6. Essere disposti a rispettare i requisiti contraccettivi durante il periodo di studio, come descritto nella Sezione 6.4
7. Essere di disposti e capaci di rispettare i requisiti dello studio e di fornire consenso scritto secondo i regolamenti locali e nazionali. Nel caso di pazienti minorenni, il tutore(I) legale deve fornire un consenso informato scritto e il paziente dovrebbe dare l'assesnso in accordo ai regolamenti locali e nazionali e gli standard instituzionali.

E.4

Principal exclusion criteria

1. Any of the following laboratory parameter assessments at Screening:
a. Alanine aminotransferase (ALT) >2×ULN
b. Total bilirubin >1.5× ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2×ULN
c. International normalized ratio (INR) > 1.5 (patients on an anticoagulant [eg, warfarin] with an INR< 3.5 will be allowed)
2. Estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73 m2 using the Modification of Diet in Renal Disease (MDRD) formula
3. On an active liver transplantation waiting list, or anticipated to undergo liver transplantation during the blinded study treatment period
4. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc)
5. History of intolerance to subcutaneous injection(s)
6. Known active HIV infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection
7. Currently enrolled in another investigational device or drug study, or less than 30 days or 5 half-lives (whichever is longer) since ending another investigational device or drug
study(s), or receiving other investigational agent(s)
8. Females who are pregnant, breast-feeding, or planning to become pregnant during the
study
9. Any condition (eg, medical concern or alcohol or substance abuse), which in the opinion of the Investigator, would make the patient unsuitable for dosing or which could interfere with the study compliance, the patient’s safety and/or the patient’s participation in the 6month treatment period of the study. This includes significant active and poorly
controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal or psychiatric disorders
unrelated to porphyria identified by key laboratory abnormalities or medical history.
10. History of recurrent pancreatitis, or acute pancreatitis with disease activity within the past 12 months prior to Screening
11. Has a major surgery planned during the first 6 months of the study
12. History of serious infection within one month prior to Screening
13. Had a malignancy within 5 years prior to Screening, except for basal or squamous cell carcinoma of the skin, cervical in-situ carcinoma, or breast ductal carcinoma, that has been successfully treated

1. Valore di uno qualsiasi dei seguenti parametri di laboratorio al momento dello screening:
a. Alanina aminotransferasi (ALT)> 2 x ULN
b. Bilirubina totale> 1,5 × ULN. I pazienti con valori elevati di bilirubina totale che sono secondari alla sindrome di Gilbert documentata sono ammissibili se la bilirubina totale è < 2 x ULN
c. Rapporto normalizzato internazionale (INR)> 1,5 (i pazienti in terapia anticoagulante [ad esempio warfarin] con un INR <3,5 saranno ammessi)
2. Velocità di filtrazione glomerulare stimata (eGFR) <30 mL / min / 1,73 m2 utilizzando la formula di modifica della dieta nella malattia renale (MDRD)
3. In lista di attesa attiva per il trapianto di fegato o si prevede che il soggetto si sottoponga a trapianto di fegato durante il periodo di trattamento in cieco dello studio
4. Storia di allergie multiple ai farmaci o storia di reazione allergica ad un oligonucleotide o alla N-acetilgalattosamina (GalNAc)
5. Storia di intolleranza alle iniezioni sottocutanee
6. Infezione da HIV attiva nota; o indizi di infezione presente o cronica da parte del virus dell’epatite C (HCV) o dell'epatite B (HBV)
7. Partecipazione ad un altro studio su dispositivo o farmaco sperimentale, o periodo temporale inferiore ai 30 giorni o a 5 emivite (a seconda di quale è il più esteso) dopo aver terminato un altro studio/i su dispositivo o farmaco sperimentale o dopo aver ricevuto uno o più agenti sperimentali
8. Le donne in stato di gravidanza, in allattamento o che intendono rimanere incinte durante lo studio
9. Qualsiasi condizione (ad esempio, controindicazioni mediche o abuso di alcol o sostanze stupefacenti) che a giudizio dello sperimentatore renderebbe il paziente inadatto alla somministrazione o che potrebbe interferire con l’aderenza alle direttive dello studio, con la sicurezza del paziente e / o con la partecipazione del paziente al periodo di trattamento di 6 mesi dello studio. Ciò include disturbi significativi che siano attivi e scarsamente controllati (instabili) a livello cardiovascolare, neurologico, gastrointestinale, endocrino, renale o psichiatrico, che non sono correlati alla porfiria e che sono stati identificati in base ad anomalie di laboratorio o in base all’anamnesi.
10. Storia di pancreatite ricorrente o pancreatite acuta con malattia presente negli ultimi 12 mesi precedenti lo screening
11. Avere un intervento chirurgico maggiore pianificato durante i primi 6 mesi dello studio
12. Storia di infezione grave entro un mese prima dello screening
13. Tumore maligno sviluppato nei 5 anni prima dello screening, ad eccezione del carcinoma cutaneo basale o a cellule squamose, del carcinoma cervicale in situ o del carcinoma duttale al seno che è stato trattato con successo.

E.5 End points

E.5.1

Primary end point(s)

Annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin
administration at home in patients with AIP over the 6-month treatment period

• Tasso annualizzato di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti con AIP durante un periodo di trattamento di 6 mesi.

E.5.1.1

Timepoint(s) of evaluation of this end point

Over the 6-month treatment period.

Durante un periodo di trattamento di 6 mesi.

E.5.2

Secondary end point(s)

Urinary ALA in patients with AIP at 3 months
¿ Urinary ALA in patients with AIP at 6 months
¿ Urinary PBG in patients with AIP at 6 months
¿ Annualized rate of administered hemin doses in patients with AIP over the 6-month treatment
period
¿ Annualized rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin
administration at home in patients with any AHP over the 6-month treatment period
¿ Daily worst pain score as measured by Brief Pain Inventory-Short Form (BPI-SF) numeric rating
scale (NRS) in patients with AIP over the 6-month treatment period
¿ Daily worst nausea score as measured by NRS in patients with AIP over the 6-month treatment
period
¿ Daily worst fatigue score as measured by Brief Fatigue Inventory-Short Form (BFI-SF) NRS in
patients with AIP over the 6-month treatment period
¿ Change from baseline in the PCS of the SF-12 in patients with AIP at 6 months

¿ Acido aminolevulinico (ALA) urinario in pazienti con AIP a 3 mesi
¿ Acido aminolevulinico (ALA) urinario in pazienti con AIP a 6 mesi
¿ Porfobilinogeno (PBG) urinario in pazienti con AIP a 6 mesi
¿ Tasso annualizzato di dosi di emina somministrate in pazienti con AIP durante un periodo di trattamento di 6 mesi.
¿ Tasso annualizzato di attacchi di porfiria che richiedono ricovero, visita medica urgente o somministrazione di emina a domicilio in pazienti con qualsiasi forma di AHP in un periodo di trattamento di 6 mesi
¿ Punteggio giornaliero del dolore peggiore, misurato mediante la scala di valutazione numerica (NRS) del BPI-SF [Brief Pain Inventory-Short Form] in pazienti con AIP in un periodo di trattamento di 6 mesi.
¿ Punteggio giornaliero della nausea peggiore, misurato mediante la scala di valutazione numerica (NRS) in pazienti con AIP in un periodo di trattamento di 6 mesi
¿ Punteggio giornaliero dell¿affaticamento peggiore, misurato mediante la scala di valutazione numerica (NRS) del BPI-SF [Brief Pain Inventory-Short Form] in pazienti con AIP in un periodo di trattamento di 6 mesi
¿ Variazione dal valore basale del Physical Component Summary (PCS) del Questionario sullo stato di salute SF-12 [12-item Short-Form Health Survey] in pazienti con AIP a 6 mesi

E.5.2.1

Timepoint(s) of evaluation of this end point

All secondary endpoints will be measured over the 6-month double-blind
treatment period

Tutti gli endpoint secondari saranno misurati durante il periodo di trattamento in doppio cieco di 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

Lo studio prevede un periodo di 6 mesi in doppio cieco e un successivo periodo in aperto (30 mesi)

The study envisages a double blind 6-month period and a subsequent open-label extension period (30m)

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

Australia

Brazil

Canada

Japan

Korea, Republic of

Mexico

South Africa

Taiwan

United States

Belgium

Bulgaria

Czechia

Denmark

Finland

France

Germany

Italy

Netherlands

Poland

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

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

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)

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

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:

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)

Consistent with pre-study treatment, if any.

Coerente con il trattamento pre-studio, se presente

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

2018-02-19

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-12-12

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

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IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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