E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Duchenne Muscular Dystrophy (DMD) |
Distrofia Muscolare di Duchenne (DMD) |
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E.1.1.1 | Medical condition in easily understood language |
Muscular Distrophy |
Distrofia Muscolare |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10013801 |
E.1.2 | Term | Duchenne muscular dystrophy |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main 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 |
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E.2.2 | Secondary 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 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
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E.4 | Principal 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. |
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E.5 End points |
E.5.1 | Primary 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) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At 96 weeks treatment |
A 96 settimane di trattamento |
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E.5.2 | Secondary 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 |
Secondari: • 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)
Esplorativi: • 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 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At 96 weeks treatment |
A 96 settimane di trattamento |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
Chile |
China |
Hong Kong |
Japan |
Korea, Republic of |
Mexico |
New Zealand |
Russian Federation |
Taiwan |
Turkey |
Ukraine |
United States |
Greece |
Italy |
Netherlands |
Norway |
Spain |
United Kingdom |
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E.8.7 | Trial 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). |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |