Stránka klinických studií Nct

Summary
EudraCT Number:2021-000122-10
Sponsor's Protocol Code Number:NS-065/NCNP-01-302
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:2021-07-30
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
A. Protocol Information
A.1Member State ConcernedSpain - AEMPS
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)
Estudio de extensión de fase 3, multicéntrico y abierto, 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
A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)
Estudio de fase 3 de viltolarsén en niños con distrofia muscular de Duchenne (DMD)
A.3.2Name or abbreviated title of the trial where available
RACER-53X
RACER-53X
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.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 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
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 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.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
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 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
Evaluar la seguridad y tolerabilidad de viltolarsén administrado por vía intravenosa (IV) en dosis semanales de 80 mg/kg en niños que hayan completado el estudio 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
Objetivo secundario:
Comparar la eficacia de viltolarsén administrado IV en dosis semanales de 80 mg/kg a niños que hayan completado el estudio NS 065/NCNP-01-301 durante un período de tratamiento de 96 semanas con controles con evolución natural de la enfermedad, utilizando para ello medidas de fuerza y Resistencia

Objetivos exploratorios
Evaluar el efecto del tratamiento con viltolarsén sobre la calidad de vida relacionada con la salud de la DMD del paciente
Evaluar la preservación de la deambulación de los pacientes 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.El pacientes ha completado el estudio NS-065/NCNP-01-301.
2.Los padres o el tutor legal del paciente 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), cuando sea aplicable, 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.
3.El paciente y sus padres o tutor legal están dispuestos a cumplir las visitas programadas, el plan de administración del producto en investigación (PEI) y los procedimientos del estudio, y quieren hacerlo.
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.El paciente ha sufrido un acontecimiento adverso en el estudio NS 065/NCNP 01 301 que, en opinión del investigador y/o del promotor, impide el uso seguro de viltolarsén en el paciente en este estudio.
2.El paciente ha recibido un tratamiento cuyo objetivo era la inducción de la distrofina o una proteína relacionada con la distrofina después de completar el estudio NS-065/NCNP-01-301.
3.El paciente recibió otro u otros medicamentos en investigación durante el estudio NS-065/NCNP-01-301 o después de completarlo.
4.El investigador y/o el promotor no consideran apropiado que el paciente participe en el estudio de extensión por algún 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)
•Constantes vitales
•Exploración física
•Análisis clínicos
oHematología y bioquímica clínica
oAnálisis de orina
oCitología urinaria
•Anticuerpos contra la distrofina y viltolarsén
•ECG de 12 derivaciones
•Ecografía renal
•Eventos adversos emergentes del tratamiento (AAT) y eventos adversos graves (AAG)
E.5.1.1Timepoint(s) of evaluation of this end point
At 96 weeks treatment
A las 96 semanas de tratamiento
E.5.2Secondary end point(s)
Secondary:
- 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)

Exploratory:
- Pediatric Outcome Data Collection Instrument (PODCI)
- Personal Adjustment and Role Skills Scale, 3rd edition (PARS III) Questionnaire
-Loss of ambulation
Secundario:
- Prueba de tiempo en reposo (TTSTAND)
- Tiempo de recorrer corriendo o camninando 10 metros (TTRW)
- Prueba de marcha durante 6 minutos (6 MWT)
- Evaluación de la capacidad de caminar de North Star (NSAA)
- Tiempo en subir 4 escalones (TTCLIMB)
-Fuerza muscular cuantitativa medida con un Dinamómetro manual( extension del codo, flexion del codo, extensión de la rodilla y flexion de la rodilla en el lado dominante únicamente)

Exploratorias:
•Escala PODCI
•Cuestionario PARS III
•Pérdida de la capacidad de caminar
E.5.2.1Timepoint(s) of evaluation of this end point
At 96 weeks treatment
A las 96 semanas de tratamiento
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 No
E.8.1.1Randomised No
E.8.1.2Open Yes
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 No
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.8.3 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 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 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
Canada
Chile
China
Greece
Hong Kong
Italy
Japan
Korea, Republic of
Mexico
Netherlands
New Zealand
Norway
Russian Federation
Spain
Taiwan
Turkey
Ukraine
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 patient at the site is assessed or receives an intervention for evaluation in the study (i.e., last patient last visit).
La fecha de finalización del estudio se define como la fecha en que el ultimo paciente del centro es evaluado o recibe una intervención para evaluación en el estudio (e.j. ultimo paciente última visita)
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.1.1Number 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
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 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)
None
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 Decision2021-06-15
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2021-06-09
P. End of Trial
P.End of Trial StatusOngoing

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