E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Angelman Syndrome |
Síndrome de Angelman |
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E.1.1.1 | Medical condition in easily understood language |
Angelman Syndrome |
Síndrome de Angelman |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10049004 |
E.1.2 | Term | Angelman's syndrome |
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 explore the efficacy of Mynocicline in the treatment of patients affected of Angelman Syndrome, in terms of increase in the age of development |
Explorar la eficacia de minociclina en el tratamiento de pacientes con Síndrome de Angelman, en términos de incremento en la edad de desarrollo equivalente obtenida a través de la Escala de Desarrollo Merrill-Palmer R (MP-R), tras 8 y/o 16 semanas de tratamiento. |
|
E.2.2 | Secondary objectives of the trial |
1) To compare mynocicline efficay vs placebo in terms of: -specific improvement of cognitive development, language and comunication, motor development, social-emotional development and adaptative conduct. - improvement of adaptive behaviour, , the ability to adapt to changes in their environment, learn new skills everyday and the level of independence 2) To evaluate the safety of mynociclne |
1) Comparar la eficacia de minociclina frente a placebo en términos de: - Mejoría especifica del desarrollo cognitivo, lenguaje y comunicación, desarrollo motor, desarrollo socio-emocional y conducta adaptativa obtenidos a través de la Escala de Desarrollo Merrill-Palmer R (MP-R), en la 8, 16 y 24 semanas. - Mejora en los comportamientos de adaptación, la capacidad de adaptarse a los cambios en su entorno, aprender nuevas habilidades cotidianas y el nivel de independencia en la 8, 16 y 24 semanas obtenidos a través de la Escala de Comportamiento Vineland Adaptive, cuarta edición (Vin-II). - Mejora de los EEG. Medida en base a los cambios en la actividad de fondo, tipo, número y duración de las crisis, tendencia a la generalización de las crisis, tipos de anomalías paroxísticas registradas y la evaluación global del neurofisiologo clínico, en la 8, 16 y 24 semanas 2) Evaluar la seguridad de minociclina |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
(1) Male or female between 6 and 30 years old. (2) Clinical diagnosis of Angelman Syndrome and molecular confirmation of diagnosis. (3) The participant has an acceptable guardian can give consent on behalf of the participant. |
(1) Hombre o mujer entre 6 y 30 años de edad. (2) Diagnostico clínico de Sindrome de Angelman y confirmación molecular del diagnóstico. (3) El participante tiene un tutor aceptable capaz de dar su consentimiento en nombre del participante, |
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E.4 | Principal exclusion criteria |
(1) Patients with hypersensitivity to tetracyclines. (2) Patients with impaired hepatic or renal function and in those with mainly drug allergy history. (3) Any other condition that in the opinion of the investigator is considered clinically relevant and that administration of minocycline contraindicated .. |
(1) Pacientes con hipersensibilidad a las tetraciclinas. (2) Pacientes con las funciones hepática o renal alteradas y en aquellos con historial alérgico fundamentalmente medicamentoso. (3) Cualquier otra patología que a criterio del investigador se considere clínicamente relevante y que contraindique la administración de minociclina.. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R) |
Incremento en la edad de desarrollo equivalente, obtenida a través de la Escala de Desarrollo Merrill-Palmer R (MP-R) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
8, 16 and 24 weeks |
SEMANA 8, 16 y 24 |
|
E.5.2 | Secondary end point(s) |
- Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior obtained through Development Scale R Merrill-Palmer (MP-R), at 8, 16 and 24 weeks. - Improved adaptive behaviors, the ability to adapt to changes in their environment, learn new life skills and level of independence in the 8, 16 and 24 weeks obtained through the Vineland Adaptive Behavior Scale, Fourth Edition ( Vin-II). - Improvement of EEG. Measure based on changes in the background activity, type, number and duration of crises, widespread tendency to crises, paroxysmal abnormalities recorded types and the overall evaluation of clinical neurophysiologist, at 8, 16 and 24 weeks - Safety and tolerability. a) Physical Examination b) Vital signs c) Laboratory Tests d) Adverse effects (AEs) list for treatment, laboratory values, values ??outside the reference range and descriptive statistics. |
Variables secundarias: - Mejoría especifica del desarrollo cognitivo, lenguaje y comunicación, desarrollo motor, desarrollo socio-emocional y conducta adaptativa obtenidos a través de la Escala de Desarrollo Merrill-Palmer R (MP-R), en la 8, 16 y 24 semanas. - Mejora en los comportamientos de adaptación, la capacidad de adaptarse a los cambios en su entorno, aprender nuevas habilidades cotidianas y el nivel de independencia en la 8, 16 y 24 semanas obtenidos a través de la Escala de Comportamiento Vineland Adaptive, cuarta edición (Vin-II). - Mejora de los EEG. Medida en base a los cambios en la actividad de fondo, tipo, número y duración de las crisis, tendencia a la generalización de las crisis, tipos de anomalías paroxísticas registradas y la evaluación global del neurofisiologo clínico, en la 8, 16 y 24 semanas - Seguridad y tolerancia. a) Examen físico b) Signos vitales c) Pruebas de laboratorio d) Efectos adversos (EA): listado por tratamiento, valores de laboratorio, valores fuera del rango de referencia y estadísticas descriptivas. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
8, 16 and 24 weeks |
SEMANA 8, 16 y 24 |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
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 | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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
|
Patients may stop participating in the trial at the time they wish. Also, in his judgment and discretion, the researcher may decide to withdraw the patient from the trial, does not meet the rules of the protocol. |
Los pacientes podrán suspender su participación en el ensayo en el momento en que lo deseen. Asimismo, a su juicio y criterio, el investigador podrá decidir la retirada del paciente del ensayo, si no cumple las normas del protocolo. |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |