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    Summary
    EudraCT Number:2019-002076-13
    Sponsor's Protocol Code Number:NS-065/NCNP-01-301
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-11-06
    Trial 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 ConcernedSpain - AEMPS
    A.2EudraCT number2019-002076-13
    A.3Full title of the trial
    A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
    Estudio de fase 3, aleatorizado, doble ciego, controlado con placebo y multicéntrico para evaluar la eficacia y la seguridad de viltolarsén en niños con distrofia muscular de Duchenne (DMD) capaces de caminar
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
    Es un estudio aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia y seguridad de Viltolarsen en niños con distrofia muscular de Duchene (DMD) capaces de caminar
    A.3.2Name or abbreviated title of the trial where available
    RACER-53
    RACER-53
    A.4.1Sponsor's protocol code numberNS-065/NCNP-01-301
    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.4.2CountryJapan
    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 pointWendy Van den Branden
    B.5.3 Address:
    B.5.3.1Street AddressTechnologielaan 11
    B.5.3.2Town/ cityLeuven
    B.5.3.3Post codeB-3001
    B.5.3.4CountryBelgium
    B.5.4Telephone number+3216 39 18 702703
    B.5.5Fax number+3278 48 03 51
    B.5.6E-mailregsubmissions@medpace.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 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.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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 placeboInfusion
    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 (DMD)
    Distrofia muscular de Duchenne (DMD)
    E.1.1.1Medical condition in easily understood language
    Muscular Dystrophy
    Distrofia muscular
    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 compare the efficacy of viltolarsen administered intravenously at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in
    ambulant boys ages 4 to <8 years with DMD using Time to Stand Test (TTSTAND) as a measure of strength and function.
    Comparar la eficacia de viltolarsén administrado por vía intravenosa en dosis semanales de 80 mg/kg durante un período de tratamiento de 48 semanas con la de un placebo en niños de 4 a menos de 8 años con DMD capaces de caminar, utilizando para ello la prueba TTSTAND (Time to Stand Test, Tiempo en ponerse de pie) como medida de la fuerza y función.
    E.2.2Secondary objectives of the trial
    -To compare the efficacy of viltolarsen administered intravenously at weekly doses of 80 mg/kg in ambulant boys ages 4 to <8 years with DMD over a 48-week treatment period vs. placebo controls using hierarchical strength and endurance outcomes
    -To evaluate the safety and tolerability of viltolarsen administered intravenously at weekly doses of 80 mg/kg in ambulant boys ages 4 to <8 years with DMD
    - Comparar la eficacia de viltolarsén administrado por vía intravenosa en dosis semanales de 80 mg/kg durante un período de tratamiento de 48 semanas con la de un placebo en niños de 4 a menos de 8 años con DMD capaces de caminar, utilizando para ello medidas jerárquicas de fuerza y resistencia

    -Evaluar la seguridad y tolerabilidad de viltolarsén administrado por vía intravenosa en dosis semanales de 80 mg/kg en niños de 4 a menos de 8 años con DMD capaces de caminar
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participant’s parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act
    HIPAA) authorization, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to local requirements;
    2. Participant has a confirmed diagnosis of DMD defined as:
    a. Participant is male with clinical signs compatible with DMD; and
    b. Participant has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame including determination of unambiguously defined exon boundaries (using techniques such as multiplex ligation-dependent probe amplification [MLPA], comparative genomic hybridization [CGH] array or other techniques with similar capability);
    3. Participant is ≥ 4 years and <8 years of age at time of first infusion in the study;
    4. Participant is able to walk independently without assistive devices;
    5. Participant is able to complete the TTSTAND without assistance in <10 seconds, as assessed at the Screening Visit;
    6. Participant and parent(s)/guardian(s) are willing and able to comply with scheduled visits, study drug administration plan, and study procedures;
    7. Participant must be on a stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study.
    1.Los padres o el tutor legal del participante han otorgado su consentimiento informado por escrito y la autorización HIPAA (Health Insurance Portability and Accountability Act, Ley de Responsabilidad y Portabilidad del Seguro de Salud), dónde aplique, antes de realizar ningún procedimiento relacionado con el estudio; se pedirá a los participantes que otorguen su asentimiento verbal o por escrito de conformidad con los requisitos locales.
    2.El participante tiene un diagnóstico confirmado de DMD, definida como:
    a.El participante es un varón con signos clínicos compatibles con DMD y
    b.El participante tiene una mutación de DMD confirmada en el gen de la distrofina que es susceptible de omisión del exón 53 para restablecer el marco de lectura del ARNm de la distrofina, incluida una determinación de los límites del exón definidos de manera inequívoca (mediante técnicas tales como amplificación de sondas dependiente de ligandos múltiples [MLPA], matriz de hibridación genómica comparativa [CGH] u otras técnicas con capacidad similar).
    3.El participante tiene 4 años o más y menos de 8 años de edad en el momento de recibir la primera infusión en el estudio.
    4.El participante es capaz de caminar de forma independiente sin dispositivos de ayuda.
    5.El participante es capaz de completar la prueba TTSTAND sin ayuda en menos de 10 segundos, evaluada en la visita de selección.
    6.El participante y sus padres o tutor están dispuestos y son capaces de cumplir las visitas programadas, el plan de administración del fármaco del estudio y los procedimientos del estudio.
    7.El participante debe haber recibido una dosis estable de glucocorticoides (GC) durante al menos tres meses antes de la primera dosis de medicación del estudio y se espera que siga con una dosis estable de GC durante todo el estudio.
    E.4Principal exclusion criteria
    1. Participant has current or history of chronic systemic fungal or viral infections;
    2. Participant has had an acute illness within 4 weeks prior to the first dose of study drug based on the Principal Investigator’s judgment/discretion;
    3. Participant has evidence of symptomatic cardiomyopathy (Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary);
    4. Participant has an allergy or hypersensitivity to the study drug or to any of its constituents;
    5. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator;
    6. Participant has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator;
    7. Participant has had surgery within the 3 months prior to the first anticipated administration of study drug or surgery is planned for anytime during the duration of the study;
    8. Participant has positive test results for hepatitis B antigen, hepatitis C antibody or human immunodeficiency virus (HIV) antibody at screening;
    9. Participant is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the halflife of a medication, whichever is longer;
    10. Participant was previously enrolled in an interventional study of viltolarsen.
    11. Participant is currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of study drug.
    12. Participant has taken any gene therapy.
    1. El participante tiene o ha tenido infecciones micóticas o víricas sistémicas crónicas.
    2. El participante ha tenido una enfermedad aguda en las cuatro semanas previas a la primera dosis del fármaco del estudio a criterio del investigador principal.
    3. El participante tiene signos de una miocardiopatía sintomática. (Nota: Una anomalía cardíaca asintomática en investigación no sería motivo de exclusión).
    4. El participante tiene alergia o hipersensibilidad al fármaco del estudio o a cualquiera de sus componentes.
    5. El participante tiene problemas conductuales o cognitivos graves que, en opinión del investigador, le impiden participar en el estudio.
    6. El participante ha tenido previamente o tiene una enfermedad, antecedentes médicos, hallazgos físicos o anomalías analíticas que, en opinión del investigador, podrían afectar a la seguridad, hacer que sea poco probable que se completen correctamente el tratamiento y seguimiento o alterar la evaluación de los resultados del estudio.
    7. El participante se ha sometido a una intervención quirúrgica en los tres meses previos a la primera administración prevista del fármaco del estudio o hay prevista una intervención quirúrgica en cualquier momento del estudio.
    8. El participante tiene resultados positivos en las determinaciones de antígeno del virus de la hepatitis B, anticuerpos contra el virus de la hepatitis C o anticuerpos contra el virus de la inmunodeficiencia humana (VIH) realizadas en la fase de selección.
    9. El participante está recibiendo otro fármaco experimental o ha recibido otro fármaco experimental en los tres meses previos a la primera dosis del fármaco del estudio o durante el tiempo equivalente a cinco veces la semivida del fármaco, lo que suponga más tiempo.
    10. El participante ha sido incluido previamente en un estudio intervencionista de viltolarsén.
    11. El participante está recibiendo otro fármaco que causa omisión de exones o ha recibido cualquier otro fármaco que causa omisión de exones en los tres meses previos a la primera dosis del fármaco del estudio.
    12. El participante ha recibido cualquier tipo de terapia génica.
    E.5 End points
    E.5.1Primary end point(s)
    TTSTAND
    Prueba TTSTAND
    E.5.1.1Timepoint(s) of evaluation of this end point
    At 48 weeks treatment
    A las 48 semanas de tratamiento
    E.5.2Secondary end point(s)
    -Hierarchical analyses of the following strength and endurance measures:
    - Time to Run/Walk 10 Meters Test (TTRW)
    - Six-minute Walk Test (6MWT)
    - North Star Ambulatory Assessment (NSAA)
    - Time to Climb 4 Steps Test (TTCLIMB)
    - Hand-held dynamometer (elbow extension, elbow flexion, knee extension and knee flexion on the dominant side only)
    Análisis jerárquicos de las siguientes medidas de fuerza y resistencia:
    - Tiempo en recorrer corriendo o caminando 10 metros ( TTRW)
    - Prueba de marcha durante seis minutos ( 6 MWT)
    - Evaluación de la capacidad de caminar de North Star ( NSAA)
    - Tiempo en subir 4 escalones ( TTCLIMB)
    - Dinamómetro manual (extensión del codo, flexión del codo, extensión de la rodilla y flexión de la rodilla en el lado dominante únicamente)
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 48 weeks treatment (first 5 bullets)

    At each study visit (last 10 bullets)
    A las 48 semanas de tratamiento (primeras 5 viñetas)

    En cada visita del estudio (últimas 10 viñetas)
    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.7.1.3.1Other 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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Brazil
    Canada
    Chile
    France
    Italy
    Japan
    Korea, Republic of
    Netherlands
    Poland
    Russian Federation
    Spain
    Taiwan
    Turkey
    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 study date is defined as the date when the last participant at the site is assessed or receives an intervention for evaluation in the study (i.e., last participant last visit).
    La fecha de finalización del estudio se define como la fecha en la que el último participante de un centro es evaluado o recibe una intervención para su evaluación en el estudio (ej. último participante última visita)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 74
    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: 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.3.3.6.1Details of subjects incapable of giving consent
    Assent only to be provided, where appropriate, due to patient age
    El asentimiento solo se proporciona cuando corresponda, debido a la edad del paciente
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    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)
    At the completion of the study, an open-label extension study may be available to patients (who successfully complete the double-blind study) in countries where Viltolarsen is not commercially available.
    Al finalizar el estudio, un estudio de extensión abierta puede estar disponible para pacientes (que han completado el estudio doble ciego satisfactoriamente) en países donde Viltolarsen no está comercialmente disponible.
    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 Decision2019-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-24
    P. End of Trial
    P.End of Trial StatusOngoing
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