Clinical Trials Register

Summary
EudraCT Number:	2018-004009-22
Sponsor's Protocol Code Number:	WVE-DMDX51-003
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2020-11-05
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2018-004009-22

A.3

Full title of the trial

A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy

Studio randomizzato, in doppio cieco, controllato con placebo, per valutare l'efficacia e la sicurezza di WVE-210201 in pazienti deambulanti affetti da distrofia muscolare di Duchenne

A.3.1

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

Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy

Studio di WVE-210201 in pazienti (in grado di camminare in maniera indipendente) con distrofia muscolare di Duchenne

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

WVE-DMDX51-003

A.5.1

ISRCTN (International Standard Randomised Controlled Trial) Number

ISRCTN00000000

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT00000000

A.5.3

WHO Universal Trial Reference Number (UTRN)

U0000-0000-0000

A.5.4

Other Identifiers

Name:

Number:

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	Wave Life Sciences UK Limited
B.1.3.4	Country	United Kingdom
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	Wave Life Sciences Ltd.
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	PPD
B.5.2	Functional name of contact point	Andrew Murphy
B.5.3	Address:
B.5.3.1	Street Address	1-2 Crown Walk, Jewry Street
B.5.3.2	Town/ city	Winchester, Hampshire
B.5.3.3	Post code	SO23 8BB
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	00441698575291
B.5.5	Fax number	00441698575291
B.5.6	E-mail	Andrew.Murphy@ppdi.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/3/18/2051 - EMA/OD/032/18
D.3 Description of the IMP
D.3.1	Product name	WVE-210201
D.3.2	Product code	[WVE-210201]
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.3.9.1	CAS number	2142024-01-7
D.3.9.2	Current sponsor code	WVE-210201
D.3.9.3	Other descriptive name	suvodirsen
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
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 infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Duchenne muscular dystrophy

Distrofia muscolare di Duchenne

E.1.1.1

Medical condition in easily understood language

Duchenne muscular dystrophy

Distrofia muscolare di Duchenne

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	PT
E.1.2	Classification code	10013801
E.1.2	Term	Duchenne muscular dystrophy
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

-To evaluate the efficacy of WVE-210201 by assessing changes in dystrophin levels (United States/Other regions, as applicable)
-To evaluate the efficacy of WVE-210201 by assessing changes in motor function by North Star Ambulatory Assessment (NSAA) (European Union [EU]/Japan)

-Valutare l'efficacia di WVE-210201 valutando le variazioni dei livelli di distrofina (Stati Uniti/altre regioni, come applicabile)
-Valutare l'efficacia di WVE-210201 valutando le variazioni della funzionalità motoria mediante Valutazione della deambulazione North Star (NSAA) (Unione Europea [UE]/Giappone)

E.2.2

Secondary objectives of the trial

-To evaluate the efficacy of WVE-210201 by assessing changes in upper limb proximal strength
-To evaluate the efficacy of WVE-210201 by assessing changes in lower limb motor function
-To evaluate the efficacy of WVE-210201 by assessing changes in respiratory function
-To evaluate the efficacy of WVE-210201 by assessing changes in stride velocity
-To evaluate the safety of WVE-210201

-Valutare l'efficacia di WVE-210201 valutando le variazioni della forza prossimale degli arti superiori
-Valutare l'efficacia di WVE-210201 valutando le variazioni della funzionalità motoria degli arti inferiori
-Valutare l'efficacia di WVE-210201 valutando le variazioni della funzionalità respiratoria
-Valutare l'efficacia di WVE-210201 valutando le variazioni della velocità del passo
-Valutare la sicurezza di WVE-210201

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

-Patient and/or parent or legal guardian must have the ability and be willing to provide written informed consent prior to any study-related procedures.
-Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase.
-Documented mutation in the Dystrophin gene associated with DMD that is amenable to exon 51 skipping.
-Ambulatory male, able to walk independently for at least 10 meters in 20 seconds or less at the time of Screening visit
-Age of =5 and =12 years at time of randomization
-Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures, including undergoing the biopsy procedures.
-Stable pulmonary and cardiac function, as measured by:
a. Reproducible percent predicted forced vital capacity (FVC) =50%
b. Left ventricular ejection fraction (LVEF) >55% in patients <10 years
of age and >45% in patients =10 years of age, as measured (and
documented) by echocardiogram.
-Currently on a stable corticosteroid therapy regimen, defined as: initiation of systemic corticosteroid therapy occurred =6 months prior to Screening, and no changes in dose =3 months prior to Screening visit.
-Adequate deltoid muscle at baseline to perform open muscle biopsies
-Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active.
-Patient and caregivers must agree not to post any study-related information on social media.

-Il paziente e/o il genitore o il tutore deve essere in grado e disposto a fornire il consenso informato scritto prima di qualsiasi procedura correlata allo studio.
-Diagnosi di DMD basata sul fenotipo clinico con aumento della creatinchinasi sierica.
-Mutazione documentata nel gene della distrofina associato alla DMD, riconducibile allo skipping dell’esone.
-Soggetti di sesso maschile deambulanti, in grado di camminare autonomamente per almeno 10 metri in 20 secondi o meno al momento della Visita di screening
-Età compresa tra =5 e =12 anni alla randomizzazione
-Volontà e capacità di attenersi alle visite programmate, al programma di somministrazione del farmaco, agli esami di laboratorio, alle restrizioni dello studio e a tutte le procedure previste dallo studio, inclusa l'esecuzione di procedure bioptiche.
-Funzionalità polmonare e cardiaca stabili, come misurato mediante:
a. Percentuale riproducibile della capacità vitale forzata prevista (FVC) =50%
b. Frazione di eiezione ventricolare sinistra (LVEF) >55% nei pazienti di età <10 anni e >45% nei pazienti di età =10 anni, come misurato (e documentato) con ecocardiogramma.
-Attualmente in regime di terapia corticosteroidea stabile, ovvero inizio della terapia corticosteroidea sistemica avvenuto =6 mesi prima dello Screening e nessuna modifica della dose =3 mesi prima della Visita di screening.
-Muscolo deltoide alla baseline idoneo allo svolgimento di biopsie muscolari a cielo aperto
-I soggetti di sesso maschile sessualmente maturi devono essere disposti a fare uso della contraccezione per tutta la durata dello studio, se sessualmente attivi.
-Pazienti e caregiver devono accettare di non pubblicare alcuna informazione correlata allo studio sui social media.

E.4

Principal exclusion criteria

-Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures.
-Other prior or ongoing medical conditions including:
a. Acute illness within 4 weeks of the initial Screening visit;
b. Abnormal physical findings, other than those associated with musculoskeletal findings attributable to DMD
-Laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results. These include, but are not limited to:
a. Renal insufficiency;
b. Impaired hepatic function glutamate dehydrogenase (GLDH) = 2.5x upper limit of normal (ULN) and bilirubin = 2x ULN (or International Normalized Ratio [INR] = 1.5x ULN);
c. Activated partial thromboplastin time (aPTT) values above ULN;
d. Platelet count < lower limit of normal (LLN).
-Documented positive hepatitis B surface antigen or hepatitis C antibody test.
-Known to be positive for human immunodeficiency virus (HIV).
-Severe mental retardation and/or behavioral problems that, in the opinion of the Investigator, could prohibit participation in this study.
-Cardiac insufficiency:
a. Severe cardiomyopathy that, in the opinion of the Investigator, prohibits participation in this study; however, cardiomyopathy that is managed by ACE inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criterion
b. Any other evidence of clinically significant structural or functional heart abnormality
c. A cardiac troponin I value > 0.2 ng/mL
-Need for daytime mechanical or non-invasive ventilation OR anticipated need for daytime mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator. Nighttime noninvasive ventilation is permitted.
-Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify (dose or regimen) during the study.
-Currently on anticoagulants or drugs that are known to significantly increase the risk of bleeding, such as NSAIDs and heparin.
-Received prior treatment with drisapersen or with an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO).
-Received prior treatment with WVE-210201.
-Received prior treatment with gene therapy for DMD
-Received treatment with ataluren or eteplirsen within the 14 weeks prior to the planned Baseline biopsy collection.
-Received any investigational drug within 3 months or 5 half-lives, whichever is longer prior to the planned baseline biopsy collection.
-Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction such as changes in pulse, blood pressure,
breathing function, etc.
-Parent or legal guardian is directly or indirectly involved in the conduct and administration of this study as an Investigator, subinvestigator,
study coordinator, or other study staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the study.

-Risultato medico clinicamente significativo all’esame obiettivo, diverso dalla DMD, che a giudizio dello sperimentatore renderà il paziente inadatto per la partecipazione e/o per il completamento delle procedure dello studio.
-Altre condizioni mediche precedenti o in corso tra cui:
a. Malattia acuta entro 4 settimane dalla Visita di screening iniziale;
b. Risultati dell'esame obiettivo anomali, diversi da quelli associati a esiti muscolo-scheletrici attribuibili alla DMD
-Anomalie di laboratorio che, a giudizio dello sperimentatore, potrebbero pregiudicare la sicurezza del paziente, rendere improbabile che possa essere completato il corso del trattamento o il follow-up o compromettere la valutazione dei risultati dello studio. Queste comprendono, a titolo esemplificativo ma non esaustivo:
a. Insufficienza renale;
b. Funzionalità epatica compromessa rilevata da glutammato deidrogenasi (GLDH) = 2,5 volte il limite superiore della norma (ULN) e bilirubina = 2x ULN (o Rapporto normalizzato internazionale [INR] = 1,5 x ULN);
c. Valori del tempo di tromboplastina parziale attivata (aPTT) al di sopra dell’ULN;
d. Conta piastrinica <limite inferiore della norma (LLN).
-Risultati documentati positivi al test per antigene di superficie dell’epatite B o anticorpi dell’epatite C.
-Nota positività al virus dell'immunodeficienza umana (HIV).
-Ritardo mentale grave e/o problemi comportamentali che, secondo il parere dello sperimentatore, potrebbero vietare la partecipazione a questo studio.
-Insufficienza cardiaca:
a. Cardiomiopatia grave che, secondo il parere dello Sperimentatore, vieta la partecipazione a questo studio; tuttavia, la cardiomiopatia controllata con ACE-inibitori o beta-bloccanti è accettabile a condizione che il paziente soddisfi il criterio di inclusione della LVEF
b. Qualsiasi altra evidenza di anomalia cardiaca strutturale o funzionale clinicamente significativa
c. Valore della troponina I cardiaca > 0,2 ng/ml
-Necessità di ventilazione diurna meccanica o non invasiva OPPURE prevista necessità di ventilazione diurna meccanica o non invasiva entro l’anno seguente, secondo il parere dello sperimentatore. La ventilazione notturna non invasiva è consentita.
-Cambiamenti negli integratori alimentari o erboristici o farmaci concomitanti entro 1 mese prima della Visita di screening o piani per modificare (dose o regime) durante lo studio.
-Attualmente in terapia con anticoagulanti o farmaci noti per aumentare significativamente il rischio di sanguinamento, come i FANS e l'eparina.
-Precedente trattamento con drisapersen o con un oligomero morfolino fosforodiamidato sperimentale coniugato con peptidi (PPMO).
Precedente trattamento con WVE-210201.
-Precedente trattamento con terapia genica per DMD
-Precedente trattamento con ataluren o eteplirsen entro 14 settimane prima del prelievo della biopsia basale programmata.
-Precedente trattamento con qualsiasi farmaco sperimentale entro 3 mesi o 5 emivite, a seconda di quale sia il periodo più lungo, prima del prelievo della biopsia basale programmata.
-Nota ipersensibilità a qualsiasi oligonucleotide, come dimostrato da una reazione allergica sistemica come cambiamenti a livello di pulsazioni, pressione sanguigna, funzione respiratoria, ecc.
-Genitore o tutore legale direttamente o indirettamente coinvolto nella conduzione e gestione del presente studio in qualità di sperimentatore, assistente sperimentatore, coordinatore dello studio o altro membro dello staff di ricerca oppure il paziente è un parente di primo grado, partner, o parente residente presso una delle suddette persone coinvolte direttamente o indirettamente nello studio.

E.5 End points

E.5.1

Primary end point(s)

1. Change from baseline in dystrophin level (% normal dystrophin) assessed by Western blot of muscle tissue (United States/Other regions, as applicable)
2. Change from baseline in NSAA at 48 weeks (EU/Japan)

1. Variazione rispetto al baseline del livello di distrofina (% di distrofina normale) valutata mediante Western blot su tessuto muscolare (Stati Uniti/altre regioni, come applicabile)
2. Variazione rispetto al baseline della NSAA fino a 48 settimane (UE/Giappone)

E.5.1.1

Timepoint(s) of evaluation of this end point

1. 1 of 3 timepoints, week 12, week 22 or week 46
2. 48 weeks

1. 1 di 3 timepoints, settimana 12, settimana 22 o settimana 46
2. 48 settimane

E.5.2

Secondary end point(s)

1. Key secondary endpoint - Change from baseline in NSAA at 48 weeks (United States/Other regions as applicable)
2. Key secondary endpoint - Change from baseline dystrophin level (% normal dystrophin) assessed by Western blot of muscle tissue (EU/Japan)
3. Change from baseline in upper limb proximal strength assessed by hand held myometry at 48 weeks
4. Change from baseline in 4-stair climb (4SC) at 48 weeks
5. Change from baseline in the 10-meter walk/run test at 48 weeks
6. Change from baseline in FVC (% predicted) at 48 weeks
7. Change from baseline in the 95th percentile of stride velocity at 48 weeks

1. Endpoint secondario principale - Variazione rispetto al baseline della NSAA fino a 48 settimane (Stati Uniti/altre regioni, come applicabile)
2. Endpoint secondario principale - Variazione rispetto al baseline del livello di distrofina (% di distrofina normale) valutata mediante Western blot su tessuto muscolare (UE/Giappone)
3. Variazione rispetto al baseline della forza prossimale degli arti superiori valutata mediante miometria manuale fino a 48 settimane
4. Variazione rispetto al baseline del tempo per salire 4 gradini (4SC) fino a 48 settimane
5. Variazione rispetto al baseline del test di corsa/camminata per 10 metri fino a 48 settimane
6. Variazione rispetto al baseline della FVC (% prevista) fino a 48 settimane
7. Variazione rispetto al baseline del 95° percentile della velocità del passo fino a 48 settimane

E.5.2.1

Timepoint(s) of evaluation of this end point

1. 48 weeks
2. 1 of 3 timepoints, week 12, week 22 or week 46
3. 48 weeks
4. 48 weeks
5. 48 weeks
6. 48 weeks
7. 48 weeks

1. 48 settimane
2. 1 di 3 timepoints, settimana 12, settimana 22 o settimana 46
3. 48 settimane
4. 48 settimane
5. 48 settimane
6. 48 settimane
7. 48 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

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)

Yes

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

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

Belgium

Canada

Czechia

France

Germany

Ireland

Italy

Japan

Netherlands

Poland

Spain

Sweden

Turkey

United Kingdom

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

LSLV

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

125

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)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

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

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Children aged =5 and =12 years at time of randomization.

Bambini di età =5 e =12 al momento della randomizzazione.

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

150

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

After the 48-week treatment period, all patients will have the opportunity to take part in an OLE study of WVE-210201. If a patient does not wish to participate in the OLE, a follow-up call approximately 2 weeks after the Week 48 visit will be required, to ensure that the patient does not have any new AE/SAE.

Dopo 48 settimane di trattamento, tutti i pazienti avranno l'opportunità di partecipare allo studio OLE WVE-210201. Se il soggetto non desiderasse partecipare nell'OLE, sarà richiesto un Follow-up telefonico circa 2 settimane dopo la settimana 48, al fine di assicurarsi che il soggetto non ha registrato EA/SAE.

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

2019-07-09

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-04-15

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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