E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping |
Pacientes con distrofia muscular de Duchenne susceptibles de omisión de los exones 45 o 53 |
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E.1.1.1 | Medical condition in easily understood language |
Duchenne muscular dystrophy |
Distrofia muscular de Duchenne |
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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 |
The primary objective of the study is to evaluate the safety and tolerability of long-term treatment with 30 mg/kg of casimersen or golodirsen. |
El objetivo principal del estudio es evaluar la seguridad y la tolerabilidad del tratamiento a largo plazo con 30 mg/kg de casimersén o golodirsén. |
|
E.2.2 | Secondary objectives of the trial |
• To evaluate changes in physical functioning with long-term treatment with 30 mg/kg of casimersen or golodirsen. • To evaluate changes in pulmonary function with long-term treatment with 30 mg/kg of casimersen or golodirsen. |
• Evaluar las variaciones de la función física con el tratamiento a largo plazo con 30 mg/kg de casimersén o golodirsén. • Evaluar las variaciones de la función pulmonar con el tratamiento a largo plazo con 30 mg/kg de casimersén o golodirsén. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Completed a clinical trial evaluating casimersen or golodirsen, per protocol. 2. If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a medically acceptable form of contraceptive (eg, female oral contraceptives) during this time frame. 3. Is able to understand and comply with all the study requirements and, if under 18 years of age, had as (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements. 4. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study. 5. Is between 7 and 23 years of age, inclusive, at randomization |
1. Completó un ensayo clínico de evaluación de casimersén o golodirsén, conforme al protocolo. 2. Si es sexualmente activo, se compromete a utilizar preservativo masculino cuando mantenga relaciones sexuales durante todo el estudio y durante 90 días después de la última dosis del fármaco del estudio. La pareja sexual también deberá utilizar un método anticonceptivo médicamente aceptable (por ejemplo, anticonceptivos orales femeninos) durante este período. 3. Es capaz de comprender y cumplir todos los requisitos del estudio y, si tiene menos de 18 años, los padres o tutores legales son capaces de comprender y cumplir todos los requisitos del estudio. 4. Está dispuesto a dar su asentimiento informado (si corresponde) y sus padres o tutores están dispuestos a dar su consentimiento informado por escrito para que el paciente participe en el studio. |
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E.4 | Principal exclusion criteria |
1. A medical condition that could, in the Investigator’sopinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results. 2. Any patient who, in the Investigator’s opinion, seems unable/unwilling to comply with the study procedures. |
1. Enfermedad que pueda, a criterio del investigador, afectar de forma adversa a la seguridad del paciente, reducir las probabilidades de completar el tratamiento o afectar a la evaluación de los resultados del estudio. 2. Pacientes que, en opinión del investigador, parezcan incapaces o no estén dispuestos a cumplir los procedimientos del estudio. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Patient incidence of serious adverse events (SAEs) |
Incidencia de acontecimientos adversos graves (AAG) por paciente |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• 6-minute walk test (6MWT) • Performance of the Upper Limb (PUL) • North Star Ambulatory Assessment (NSAA) • Loss of ambulation (LOA) • Pulmonary function tests (PFTs) • Need for assisted ventilation • Percentage of patients who develop anti-dystrophin, anti-casimersen, or anti-golodirsen antibodies |
• Prueba de la marcha de 6 minutos (6MWT). • Rendimiento de las extremidades superiores (PUL). • Evaluación ambulatoria North Star (NSAA). • Pérdida de la deambulación (PD). • Pruebas de función respiratoria (PFR). • Necesidad de ventilación asistida. • Porcentaje de pacientes que desarrollan anticuerpos anti-distrofina, anti-casimersén o anti-golodirsén. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
6MWT, PUL, NSAA, PFTs: Screening, Week 24, 48, 72, 96, 120, 144 LOA: Week 60, 72, 84, 96, 108, 120, 132, 144 Need for assisted ventilation: continuous Immunogenicity*: Screening, Week 1, 4, 8, 12, 24, 36, 48, 72, 96, 120, 144
*Patients who previously participated in 4045-101 or 4053-101 do not need to have immunogenicity testing at Weeks 1, 4 and 8 |
6MWT, PUL, NSAA, PFR: Selección, semanas 24, 48, 72, 96, 120, 144 PD: semanas 60, 72, 84, 96, 108, 120, 132, 144 Necesidad de ventilación asistida: continua Inmunogenicidad*: Selección, semanas 1, 4, 8, 12, 24, 36, 48, 72, 96, 120, 144
* Los pacientes que participaron previamente en 4045-101 o 4053-101 no necesitan pruebas de inmunogenicidad en las semanas 1, 4 y 8. |
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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 | No |
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 | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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 | No |
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 |
Belgium |
Canada |
Czech Republic |
Finland |
France |
Germany |
Ireland |
Israel |
Italy |
Poland |
Spain |
Sweden |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
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
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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 | 3 |
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 | 3 |
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 |