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    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).

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    EudraCT Number:2021-000122-10
    Sponsor's Protocol Code Number:NS-065/NCNP-01-302
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-08
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-000122-10
    A.3Full title of the trial
    A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
    Studio di estensione in aperto, multicentrico, di fase 3, volto a valutare la sicurezza e l’efficacia di viltolarsen in ragazzi deambulanti affetti da distrofia muscolare di Duchenne (DMD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)
    Uno studio di fase 3 su viltolarsen in ragazzi con distrofia muscolare di duchenne (DMD)
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberNS-065/NCNP-01-302
    A.5.4Other Identifiers
    Name:US INDNumber:127474
    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 SponsorNS Pharma, 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 supportNS Pharma, Inc.
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportNippon Shinyaku Co., Ltd
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace
    B.5.2Functional name of contact pointKyle Haas
    B.5.3 Address:
    B.5.3.1Street AddressMedpace Way
    B.5.3.2Town/ cityCincinnati
    B.5.3.3Post code5375
    B.5.3.4CountryUnited States
    B.5.4Telephone number+151357999111270
    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/3/20/2282
    D.3 Description of the IMP
    D.3.1Product nameViltolarsen
    D.3.2Product code [NS-065/NCNP-01]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation Yes
    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 INNVILTOLARSEN
    D.3.9.1CAS number 2055732-84-6
    D.3.9.2Current sponsor codeNS-065/NCNP-01
    D.3.9.4EV Substance CodeSUB195543
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy (DMD)
    Distrofia Muscolare di Duchenne (DMD)
    E.1.1.1Medical condition in easily understood language
    Muscular Distrophy
    Distrofia Muscolare
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    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 safety and tolerability of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg in boys who have completed the NS-065/NCNP-01-301 study
    Valutare la sicurezza e la tollerabilità di viltolarsen somministrato per via endovenosa (EV) a dosi settimanali di 80 mg/kg in ragazzi che hanno completato lo studio NS-065/NCNP-01-301
    E.2.2Secondary objectives of the trial
    Secondary Objective:
    To compare the efficacy of viltolarsen administered IV at weekly doses of 80 mg/kg in boys who have completed the NS-065/NCNP-01-301 study over a 96-week treatment period versus natural history controls using strength and endurance outcomes.

    Exploratory Objectives:
    - To evaluate health-related quality of life impact of viltolarsen treatment on patient's DMD
    - To evaluate preservation of ambulation of patients with DMD
    Obiettivo Secondario:
    Confrontare l’efficacia di viltolarsen somministrato per EV a dosi settimanali di 80 mg/kg in ragazzi che hanno completato lo studio NS-065/NCNP-01-301 nell’arco di un periodo di trattamento di 96 settimane rispetto ai controlli dell’anamnesi naturale
    utilizzando gli esiti di forza e resistenza

    Obiettivi Esplorativi:
    - Valutare l’impatto in termini di qualità della vita correlata alla salute del trattamento con viltolarsen sulla DMD del paziente
    - Valutare la conservazione della deambulazione dei pazienti con DMD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient has completed the NS-065/NCNP-01-301 study;
    2. Patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients will be asked to give written or verbal assent according to local requirements;
    3. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.
    1. Il paziente ha completato lo studio NS-065/NCNP-01-301.
    2. Il/I genitore/i o tutore/i legale/i del paziente ha/hanno fornito il consenso informato per iscritto e l’autorizzazione secondo la legge sulla portabilità e responsabilità delle polizze di assicurazione sanitaria (Health Insurance Portability and Accountability Act, HIPAA), ove applicabile, prima di iniziare qualsiasi procedura correlata allo studio; ai pazienti sarà chiesto di fornire il proprio assenso per iscritto o verbale secondo i requisiti locali.
    3. Il paziente e il/i genitore/i o tutore/i legale/i sono disposti, oltreché esserne in grado, ad attenersi alle visite programmate, al piano di somministrazione del farmaco dello studio e alle procedure dello studio.
    E.4Principal exclusion criteria
    1. Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the investigator and/or the sponsor, precludes safe use of viltolarsen for the patient in this study;
    2. Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NS-065/NCNP-01-301;
    3. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
    4. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.
    1. Il paziente ha manifestato un evento avverso nello studio NS-065/NCNP-01-301 che, a giudizio dello sperimentatore e/o dello Sponsor, preclude l’utilizzo sicuro di viltolarsen per il paziente in questo studio.
    2. Il paziente è stato sottoposto a un trattamento che è stato effettuato per finalità di induzione della distrofina o della proteina correlata alla distrofina dopo il completamento dello studio NS-065/NCNP-01-301.
    3. Il paziente ha assunto qualsiasi altro farmaco sperimentale durante o dopo il completamento dello studio NS-065/NCNP-01-301.
    4. Lo sperimentatore e/o lo Sponsor ritengono che il paziente non sia appropriato per partecipare allo studio di estensione per qualsiasi motivo.
    E.5 End points
    E.5.1Primary end point(s)
    - Vital signs
    - Physical examination
    - Clinical laboratory tests
    o Hematology and clinical chemistry
    o Urinalysis
    o Urine cytology
    - Antibodies to dystrophin and viltolarsen
    - 12-lead electrocardiogram (ECG)
    - Renal ultrasound
    - Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
    - Segni vitali
    - Esame obiettivo
    - Esami clinici di laboratorio
    o Ematologia e chimica clinica
    o Esame delle urine
    o Citologia urinaria
    - Anticorpi anti-distrofina e anti-viltolarsen
    - Elettrocardiogramma (ECG) a 12 derivazioni
    - Ecografia renale
    - Eventi avversi emergenti dal trattamento (TEAE) ed eventi avversi seri (SAE)
    E.5.1.1Timepoint(s) of evaluation of this end point
    At 96 weeks treatment
    A 96 settimane di trattamento
    E.5.2Secondary end point(s)
    - Time to Stand Test (TTSTAND)
    - Time to Run/Walk 10 Meters Test (TTRW)
    - Six-minute Walk Test (6MWT)
    - North Star Ambulatory Assessment (NSAA)
    - Time to Climb 4 Stairs Test (TTCLIMB)
    - Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only)

    - Pediatric Outcome Data Collection Instrument (PODCI)
    - Personal Adjustment and Role Skills Scale, 3rd edition (PARS III) Questionnaire
    - Loss of ambulation
    • Test del tempo impiegato per passare dalla posizione supina a quella eretta (TTSTAND)
    • Test del tempo impiegato per correre/camminare (TTRW) per 10 metri
    • Test del cammino dei 6 minuti (6MWT)
    • Valutazione della mobilità North Star (NSAA)
    • Test del tempo impiegato per salire 4 gradini (TTCLIMB)
    • Forza muscolare quantitativa misurata mediante dinamometro palmare (estensione e flessione del gomito, estensione e flessione del ginocchio solo sul lato dominante)

    • Strumento di raccolta dei dati sugli esiti pediatrici (PODCI)
    • Questionario della scala di adattamento personale e competenze del ruolo, 3a edizione (PARS III)
    • Perdita di deambulazione
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 96 weeks treatment
    A 96 settimane di trattamento
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    In aperto
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Hong Kong
    Korea, Republic of
    New Zealand
    Russian Federation
    United States
    United Kingdom
    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 study date is defined as the date when the last patient at the site is assessed or receives an intervention for evaluation in the study (i.e., last patient last visit).
    La data di fine dello studio è definita come la data in cui l'ultimo paziente nel centro viene valutato o riceve un intervento per la valutazione nello studio (cioè, l'ultima visita dell'ultimo paziente).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    E.8.9.2In all countries concerned by the trial months0
    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: 74
    F.1.1.6Adolescents (12-17 years) No
    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
    Assent only to be provided, where appropriate, due to patient age
    A causa dell'età del paziente sarà richiesto il solo assenso
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    F.4.2.2In the whole clinical trial 74
    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)
    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 Decision2021-04-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-25
    P. End of Trial
    P.End of Trial StatusOngoing
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    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
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