Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7263   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools

    < Back to search results

    Print Download

    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
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-004009-22
    A.3Full 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.3.1Title 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.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberWVE-DMDX51-003
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN00000000
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00000000
    A.5.3WHO Universal Trial Reference Number (UTRN)U0000-0000-0000
    A.5.4Other Identifiers
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorWave Life Sciences UK Limited
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportWave Life Sciences Ltd.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPPD
    B.5.2Functional name of contact pointAndrew Murphy
    B.5.3 Address:
    B.5.3.1Street Address1-2 Crown Walk, Jewry Street
    B.5.3.2Town/ cityWinchester, Hampshire
    B.5.3.3Post codeSO23 8BB
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number00441698575291
    B.5.5Fax number00441698575291
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2051 - EMA/OD/032/18
    D.3 Description of the IMP
    D.3.1Product nameWVE-210201
    D.3.2Product code [WVE-210201]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 2142024-01-7
    D.3.9.2Current sponsor codeWVE-210201
    D.3.9.3Other descriptive namesuvodirsen
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne muscular dystrophy
    Distrofia muscolare di Duchenne
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy
    Distrofia muscolare di Duchenne
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main 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.2.2Secondary 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.2.3Trial contains a sub-study No
    E.3Principal 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.4Principal 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.5.1Primary 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.5.1.1Timepoint(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.5.2Secondary 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.5.2.1Timepoint(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.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    United States
    E.8.7Trial 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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 125
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 25
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. 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.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 85
    F.4.2.2In 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
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-07-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands