E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hypersalivation (chronic sialorrhea)in patients with cerebral palsy |
Hipersecreción salival (sialorrea crónica). en pacientes con parálisis cerebral. |
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E.1.1.1 | Medical condition in easily understood language |
Chronic drooling in patients with cerebral palsy. |
Babeo crónico en pacientes con parálisis cerebral. |
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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 | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10039424 |
E.1.2 | Term | Salivary hypersecretion |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
EVALUATE THE SAFETY AND EFFICAY OF BOTULINUM TOXIN TYPE -A AS A TREATMENT FOR DECREASE DROOLING IN PATIENTS WITH CEREBRAL PASY AND DETERMINATE THE SALIVARY FLOW ML/MIN. |
EVALUAR LA SEGURIDAD Y EFICACIA DE LA TOXINA BOTULÍNICA TIPO-A INFILTRADA EN GLÁNDULAS SALIVALES EN EL TRATAMIENTO DE LA SIALORREA CRÓNICA EN PACIENTES CON PARÁLISIS CEREBRAL. DETERMINAR EL FLUJO SALIVAL ML/MIN |
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E.2.2 | Secondary objectives of the trial |
ASSESS AT WHAT PERIOD OF TIME AFTER INFILTRATION OCCURS THE MAXIMUM RESPONSE OF BOTULINUM NEUROTOXIN TYPE-A IN DECREASED DROOLING. EVALUATE THE LONG-TERM EFFECTS OF BOTULINUM NEUROTOXIN TYPE-A IN SALIVARY GLANDS. ASSESS WHETER THE BOTULINUM TOXIN TYPE-A MAY REDUCE THE VOLUME OF DROOLING WITHOUT ALTERING THE SWALLOWING FUNCTION. |
EVALUAR EN QUE PERIODO POST-INFILTRACIÓN SE PRODUCE LA MÁXIMA RESPUESTA EN DISMINUIR EL BABEO. EVALUAR EL TIEMPO MEDIO DE DURACIÓN APLICADA EN LAS GLÁNDULAS SALIVALES. EVALUAR LA DISMINUCIÓN DEL VOLUMEN SALIVAL POST-INFILTRACIÓN SIN ALTERAR LA FUNCIÓN DE LA DEGLUCIÓN. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Chronic sialorrhea,score of > on drooling severity and frecuency scale. - Patients aged > 18 years. - Confirmed diagnosis of cerebral palsy. - Patients with disorders for eating, drinking, chronic irritation of the skin, pneumonia, social exclusion. - Informed consent from parents, caregivers or self patient. |
-Sialorrea Crónica, con un marcaje >3 en la valoración de la severidad y frecuencia de babeo. -Edad de los sujetos >18 años y < de 65. -Diagnóstico confirmado de parálisis cerebral. _Pacientes que presenten dificultades para comer, beber,irritación crónica de la piel,neumonías de repetición, baja autoestima que les impida la exclusión social. -consentimiento informado sobre participación de estudio. |
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E.4 | Principal exclusion criteria |
Patients < 18 years. Swallowing disorders,dysphagia. -Diagnoses of Eaton-Lambert Syndrome. Amyothropic lateral Sclerosis or diseases that interfere with the function neuroglandular. -Presence of significant lung problems. - Pregnant or lactating period -Enrolment in another medical study or participation 30 days before. -Use of drugs that interfere with salival secretion. |
-Menores de 18 años -Presencia de disfagia por oclusión traumática,evaluación y determinación de alimentación oral. -Diagnóstico de miastenia gravis, síndrome de Eaton Lambert, esclerosis lateral aminotrófica o cualquier otra enfermedad que interfiera con la función neuromuscular o neuroglandular. -Pacientes embarazadas o lactantes. -Pacientes con profunda atrofía o excesiva debilidad de los músculos en el área donde se aplicará la inyección. - Participación recurrente en otro estudio clínico con medicación o participación 30 dias previos al comenzar el estudio clínico. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy Evaluate: Decrease of salivary flow expressed in ml/min before and after infiltration,will be considered the proportion of patients showing a decrease of 25% of salivary secretion. Safety Evaluate: The proportion of patients with a good response according to the criteria defined response during treatment or follow-up. Good response: If the patients register a proportion of adeverse events not more than usual due to underlying pathology. - Low/ non response. Register a high proportion of adverse events more than usual. |
Evaluación de la Eficacia: Es el descenso del flujo salival, expresado ml/min, antes y después de la infiltración se considerará la proporción de pacientes que manifiesten un descenso del 25% de secreción salival. Evaluación de la Seguridad. Buena respuesta: si se registra una proporción de aconecimientos adversos no superior a lo habitual debido a su patología de base. -Baja/ Nula respuesta: Aparición de acontecimientos adversos en un porcentaje superior a lo habitual debido a su patología de base |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Average time of evaluation every 4 weeks after first infiltration, allowed a washout period. The second infiltration evaluation every 4 weeks untill complete one year of follow-up. |
Tiempo medio de evaluación de cada variable cada 4 semanas después de la primera infiltración, ddejando un período de lavado. En la segunda infiltración la evaluación es cada cuatro semanas hasta completar un año de seguimiento. |
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E.5.2 | Secondary end point(s) |
Salivary flow expressed in ml/min evaluated in different subtypes of cerebral palsy compared with healthy volunteers salivary flow. To assess wheter patients with cerebral palsy have hypersalivation or oral motor dysfunction. Assess wheter the Botulinum Toxin Type A reduce the volume of drooling without altering the swallowing function |
- Evaluar el flujo salival expresado en ml/min en los diferentes subtipos pacientes con parálisis cerebral, comparandolo con le flujo salival de los voluntarios sanos. - Evaluar si los pacientes con parálisis cerebral presentan hipersalivación o es debido a la disfunción motora oral. - Evaluar si la utilización de la toxina botulínica tipo-A reduce el volumen del babeo sin alterar la función de deglución. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Average time of evaluation every 4 weeks after first infiltration, allowed a washout period. The second infiltration evaluation every 4 weeks untill complete one year of follow-up. |
Tiempo medio de evaluación de cada variable cada 4 semanas después de la primera infiltración, ddejando un período de lavado. En la segunda infiltración la evaluación es cada cuatro semanas hasta completar un año de seguimiento. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
E.8.1.6 | Cross over | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
1 grupo control sin tratamiento |
one control group without treatment |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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
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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 | 1 |
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 | 1 |
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 |