E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Spinal Muscular Atrophy |
atrofia muscular espinal |
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E.1.1.1 | Medical condition in easily understood language |
Spinal Muscular Atrophy |
atrofia muscular espinal |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10041582 |
E.1.2 | Term | Spinal muscular atrophy |
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 this study is to assess long-term safety in terms of treatment-emergent serious adverse events (SAEs) and treatment-emergent adverse events of special interest (AESIs) |
El objetivo principal de este estudio es evaluar la seguridad a largo plazo en términos de acontecimientos adversos graves (serious adverse events [SAEs]) surgidos durante el tratamiento y de acontecimientos adversos de especial interés (adverse events of special interest [AESIs]) surgidos durante el tratamiento. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of this study are; (1) assess long-term efficacy of OAV101 treatment. (2) assess long-term safety for measures other than adverse events . |
Los objetivos secundarios de este estudio son: (1) Evaluar la eficacia a largo plazo del tratamiento con OAV101 (2) Evaluar la seguridad a largo plazo mediante medidas distintas de los acontecimientos adversos. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Participants eligible for inclusion in this study must meet all of the following criteria: 1. Participated in an OAV101 clinical trial. 2. Written informed consent must be obtained before any assessment is performed. 3. Patient/Parent/legal guardian willing and able to comply with study procedures. |
Los participantes elegibles para su inclusión en este estudio deben cumplir todos los criterios siguientes: 1. Haber participado en un ensayo clínico con OAV101. 2. Obtención del consentimiento informado por escrito antes de la práctica de cualquier evaluación del estudio 3. Voluntariedad y capacidad del paciente/progenitores/tutor legal para cumplir con los procedimientos del estudio. |
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E.4 | Principal exclusion criteria |
There are no specific exclusion criteria for this study. |
El estudio carece de criterios de exclusión específicos. |
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E.5 End points |
E.5.1 | Primary end point(s) |
● Number and proportion of patients reporting treatment-emergent serious adverse events (SAEs) by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class (SOC) and Preferred Term (PT) for the entire duration of the study (i.e., up to 15 years). ● Number and proportion of patients reporting treatment-emergent adverse events of special interest (AESIs) by AESI category and MedDRA SOC and PT within each AESI category for the entire duration of the study (i.e., up to 15 years). |
- Número y el porcentaje de participantes con SAEs surgidos durante el tratamiento, utilizando para ello la clasificación System Organ Class (SOC) y el término preferido (preferred term [PT]) del Medical dictionary for regulatory activities (MedDRA), a lo largo de toda la duración del estudio (es decir, 15 años). - Número y el porcentaje de participantes con AESIs surgidos durante el tratamiento, por categoría de AESI y por SOC y PT según el MedDRA, a lo largo de toda la duración del estudio (es decir, 15 años). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
up to 15 years |
hasta 15 años |
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E.5.2 | Secondary end point(s) |
● The number and proportion of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist ● The number and proportion of participants demonstrating maintenance of each developmental milestone ● Change from Baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score ● Change from Baseline in the Revised Upper Limb Module (RULM) total score ● Number and proportion of patients with potentially clinically significant vital sign findings, summarized individually for each vital sign parameter ● Number and proportion of patients with potentially clinically significant laboratory values, summarized individually for each laboratory parameter |
● Número y porcentaje de participantes que alcancen cada developmental milestone según el Developmental Milestone Checklist ● Número y porcentaje de participantes que muestren mantenimiento de cada developmental milestone ● Cambio frente al basal en la puntuación total de la HFMSE ● Cambio frente al basal en la puntuación total del RULM ●Número y porcentaje de pacientes con hallazgos de signos vitales potencialmente significativos desde el punto de vista clínico, resumidos individualmente para cada parámetro de signo vital ●Número y porcentaje de pacientes con valores de laboratorio potencialmente significativos clínicamente, resumidos individualmente para cada parámetro de laboratorio |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Exploratory ● To explore other potential effects of OAV101 on exploratory efficacy and safety biomarkers ● To assess long-term safety in terms of adverse events ● To describe the time-course of the elevation of anti-AAV9 antibodies after administration of OAV101 ● To assess long-term Caregiver experience |
Exploratorios ● Explorar otros efectos potenciales de OAV101 en biomarcadores exploratorios de eficacia y seguridad ● Evaluar la seguridad a largo plazo en términos de acontecimientos adversos ● Describir el curso temporal de la elevación de anticuerpos anti-AAV9 después de la administración de OAV101 ● Evaluar la experiencia del cuidador a largo plazo |
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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 | Yes |
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 |
Segmto a largo plazo de ptes con AME tratados con OAV101 IT o administración IV. No hay fármaco |
Long-term follow-up of patients with SMA Treated with OAV101 IT or IV administration. No study drug. |
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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.8.3 |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 21 |
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 |
Brazil |
Canada |
China |
Colombia |
Egypt |
India |
Japan |
Malaysia |
Mexico |
Saudi Arabia |
Singapore |
South Africa |
Taiwan |
Thailand |
United States |
Viet Nam |
France |
Netherlands |
Spain |
Switzerland |
Germany |
Greece |
Italy |
Belgium |
Denmark |
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
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The end of the LTFU study for each individual Spinal Muscular Atrophy (SMA) patient will be 15 years after treated with OAV101 by intravenous (IV) or intrathecal (IT) administration received from a parent trial. The end of study as a whole is defined as the last patient's last visit (LPLV), which is the last patient's last evaluation (visit Year 15), or the time of premature withdrawal. |
El final del estudio LTFU para cada paciente individual con Atrofia Muscular Espinal (AME) será 15 años después del tratamiento con OAV101 por administración intravenosa (IV) o intratecal (IT) recibido de un ensayo principal. El final del estudio en su conjunto se define como la última visita del último paciente (LPLV), que es la última evaluación del último paciente (visita Año 15), o el momento de la retirada prematura. |
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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 | 15 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | 1 |
E.8.9.2 | In all countries concerned by the trial years | 17 |
E.8.9.2 | In all countries concerned by the trial months | 5 |