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    Summary
    EudraCT Number:2017-002686-21
    Sponsor's Protocol Code Number:WVE-DMDX51-001
    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: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.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2017-002686-21
    A.3Full title of the trial
    A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy
    Studio di Fase 1 multicentrico, in doppio cieco, controllato con placebo su WVE 210201 somministrato per via endovenosa a pazienti affetti da distrofia muscolare di Duchenne
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the safety and tolerability of different doses of WVE-
    210201 in patients with Duchenne Muscular Dystrophy
    Uno studio per valutare la sicurezza e la tollerabilità di dosi differenti di WVE-210201 in pazienti affetti da distrofia muscolare di Duchenne
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberWVE-DMDX51-001
    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
    Name:-Number:-
    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 USA INC
    B.1.3.4CountryUnited States
    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 pointBruno Rocton
    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 number00441932563375
    B.5.5Fax number00441932563375
    B.5.6E-mailbruno.rocton@ppdi.com
    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 No
    D.2.5.1Orphan drug designation number
    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.3.8INN - Proposed INNWVE-210201
    D.3.9.2Current sponsor codeWVE-210201
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee 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 injection
    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
    Evaluate the safety and tolerability of single ascending doses of WVE-210201 in patients with DMD.
    Valutare la sicurezza e la tollerabilità di singole dosi ascendenti di WVE-210201 in pazienti affetti da DMD.
    E.2.2Secondary objectives of the trial
    Assess the pharmacokinetics (PK) of WVE-210201 in patients with DMD.
    Valutare la farmacocinetica (PK) di WVE-210201 in pazienti affetti da DMD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. 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.
    2. Diagnosis of DMD based on clinical phenotype with increased serum creatine kinase.
    3. Documented mutation in the dystrophin gene associated with DMD
    that is amenable to exon 51 skipping.
    4. Ambulatory or non-ambulatory male.
    5. Age of major or equal to 5 and minor or equal to18 years at randomization
    tests, study restrictions, and all study procedures.
    7. Stable pulmonary and cardiac function, documented within the past year, as measured by:
    a) Reproducible percent predicted forced vital capacity (FVC) major or equal to 50%
    b) Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and >45% in patients major or equal to 10 years of age, as measured (and documented) by echocardiogram.
    8. Sexually mature males must be willing to use contraception for the duration of the study, if the patient is sexually active.
    9.Patients and caregiver must agree not to post any study-related information on social media.
    1.Il paziente e/o genitore o tutore deve avere la capacità ed essere disposto a fornire il consenso informato scritto prima di qualsiasi procedura correlata allo studio.
    2.Diagnosi di DMD basata sul fenotipo clinico con aumento della creatinchinasi sierica.
    3.Mutazione documentata nel gene della distrofina associato alla DMD che è assoggettabile al salto dell’esone 51.
    4.Soggetto di sesso maschile deambulante o non deambulante.
    5.Età compresa tra maggiore o uguale a 5 e minore o uguale a 18 anni alla randomizzazione.
    6.Volontà e capacità di attenersi alle visite programmate, al programma di somministrazione del farmaco, ai test di laboratorio, alle restrizioni previste dallo studio e a tutte le procedure dello studio.
    7.Funzionalità cardiaca e polmonare stabili, documentate entro l’anno passato, come misurato mediante:
    a.Percentuale riproducibile della capacità vitale forzata prevista (FVC) maggiore o uguale a 50%
    b.Frazione di eiezione ventricolare sinistra (LVEF) >55% nei pazienti di età <10 anni e >45% nei pazienti di età magiore o uguale a 10 anni, come misurato (e documentato) da ecocardiogramma.
    8.I soggetti di sesso maschile sessualmente maturi devono essere disposti a fare uso della contraccezione per tutta la durata dello studio, se sessualmente attivi.
    9.Pazienti e caregiver devono accettare di non pubblicare alcuna informazione correlata allo studio sui social media.
    E.4Principal exclusion criteria
    1. 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 unable to complete the study procedures.
    2. Other prior or ongoing medical conditions including:
    a) Acute illness within 28 days of Screening visit;
    b) Abnormal physical findings, other than those associated with musculoskeletal findings attributable to DMD.
    3. 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 (ALT and AST elevations inconsistent with age and creatine kinase [CK] level, and elevated direct or indirect bilirubin);
    c) aPTT values above the upper limit of normal (ULN);
    d) Platelet count <lower limit of normal (LLN).
    4. Positive hepatitis B surface antigen or hepatitis C antibody test.
    5. Known to be positive for human immunodeficiency virus (HIV).
    6. Severe mental retardation and/or behavioral problems that, in the opinion of the Investigator, could prohibit participation in this study.
    7. Severe cardiomyopathy that, in the opinion of the Investigator, prohibits participation in this study. Cardiomyopathy that is managed by angiotensin-converting enzyme (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF inclusion criteria.
    8. Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or non-invasive ventilation within the next year, in the opinion of the Investigator.
    9. 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.
    10. Currently on anticoagulants or antithrombotics.
    11. Received prior treatment with drisapersen.
    12. Received treatment with eteplirsen or ataluren within the past 14 weeks.
    13. Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.
    14. Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction such as changes in pulse, blood pressure, breathing function, etc.
    15. Parent or legal guardian is directly or indirectly involved in the conduct and administration of this study as an Investigator, sub investigator, 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.
    1. Risultato medico clinicamente significativo all’esame obiettivo, diverso dalla DMD, che a giudizio dello sperimentatore renderà il paziente inadatto per alla partecipazione e/o non in grado di completare le procedure dello studio.
    2. Altre condizioni mediche precedenti o in corso tra cui:
    a. Malattia acuta entro 28 giorni dalla Visita di screening;
    b. Risultati fisici anomali, diversi da quelli associati a risultati muscoloscheletrici attribuibili alla DMD.
    3. 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 (aumento dell’alanina aminotransferasi
    c. [ALT] e aspartato aminotransferasi [AST] incoerenti con l'età e i livelli di creatinchinasi [CK] e aumento della bilirubina diretta o indiretta);
    d. Valori del tempo di tromboplastina parziale attivata [aPTT] al di sopra del limite superiore della norma [ULN];
    e. Conta delle piastrine < limite inferiore della norma (LLN).
    4. Risultati positivi al test per antigene di superficie dell’epatite B o anticorpi dell’epatite C.
    5. Nota positività al virus dell'immunodeficienza umana (HIV).
    6. Ritardo mentale grave e/o problemi comportamentali che, secondo il parere dello sperimentatore, potrebbero vietare la partecipazione a questo studio.
    7. Cardiomiopatia grave che, secondo il parere dello sperimentatore, vieta la partecipazione a questo studio. La cardiomiopatia gestita da inibitori dell'enzima di conversione dell'angiotensina (ACE) o beta-bloccanti è accettabile a condizione che il paziente soddisfi i criteri di inclusione della LVEF.
    8. Necessità di ventilazione meccanica o non invasiva OPPURE prevista necessità di ventilazione meccanica o non invasiva entro l’anno seguente, secondo il parere dello sperimentatore.
    9. 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.
    10. Attuale terapia con anticoagulanti o antitrombotici.
    11. Ha ricevuto un precedente trattamento con drisapersen.
    12. Ha ricevuto un trattamento con eteplirsen o ataluren entro le ultime 14 settimane.
    13. Ha ricevuto qualsiasi farmaco sperimentale entro gli ultimi 3 mesi o 5 emivite, a seconda del periodo più lungo.
    14. Nota ipersensibilità a qualsiasi oligonucleotide, come dimostrato da una reazione allergica sistemica come cambiamenti a livello di pulsazioni, pressione sanguigna, funzione respiratoria, ecc.
    15. Genitore o tutore legale direttamente o indirettamente coinvolto nella conduzione e gestione del presente studio come sperimentatore, assistente sperimentatore, coordinatore dello studio, o altro membro dello staff di ricerca oppure il paziente è membro della famiglia di primo grado, partner, o parente residente con una delle suddette persone coinvolte direttamente o indirettamente nello studio.

    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the safety and tolerability of WVE-210201, as compared with placebo, as assessed by the number of patients with adverse events (AEs), severity of AEs, number of patients with serious AEs (SAEs), and the number of patients who withdraw due to AEs.
    L'endpoint primario è valutare la sicurezza e la tollerabilità di WVE-210201, rispetto al placebo, in base al numero di pazienti con eventi avversi (AE), alla gravità degli AE, al numero di pazienti con AE seri (SAE), e al numero di pazienti che si ritirano a causa di AE.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary safety endpoints will be assessed as incidence of events from baseline through end of study.
    Saranno valutati gli endpoint primari di sicurezza come l’incidenza di eventi dalla baseline fino alla fine dello studio.
    E.5.2Secondary end point(s)
    The secondary endpoint is the assessment of PK parameters of WVE-210201 following single dose administration.
    L'endpoint secondario è la valutazione dei parametri di PK di WVE-210201 dopo la somministrazione di una dose singola.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 1, 2 and 8
    Giorno1,2 e 8
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    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 trial2
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Belgium
    Canada
    France
    Italy
    Netherlands
    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
    The end of the study is defined as the date on which the last patient completes the last visit (Day 85 ±3 days).
    La fine dello studio è definita come la data nella quale l'ultimo paziente completa l’ultima visita (Giorno 85 ±3 giorni).
    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 months0
    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 months7
    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.1.1.5.1Number of subjects for this age range: 19
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 13
    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.3.3.6.1Details of subjects incapable of giving consent
    Children
    Bambini
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 32
    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)
    Patients may be able to participate in an open-label extension study.
    I pazienti possono essere in grado di partecipare ad uno studio di estensione in aperto.
    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 Decision2018-05-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-19
    P. End of Trial
    P.End of Trial StatusCompleted
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