E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pediatric patients with recurrent pneumonia and MBL deficiency. |
Pacientes pediátricos con neumonías recurrentes y déficit de MBL |
|
E.1.1.1 | Medical condition in easily understood language |
Children with recurrent pneumonia and immune system disorders (MBL deficiency) |
Niños con neumonías de repetición y con alteraciones del sistema inmunitario (déficit de MBL) |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061598 |
E.1.2 | Term | Immunodeficiency |
E.1.2 | System Organ Class | 10021428 - Immune system disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10010120 |
E.1.2 | Term | Community acquired pneumonia |
E.1.2 | System Organ Class | 100000004862 |
|
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 |
To evaluate the role of azithromycin as an immunomodulator of the inflammatory response in pediatric patients with recurrent pneumonia and MBL deficiency, analyzing the role of the antibiotic in the expression of MBL receptor (CDw93) and evaluate the significance of their use in practice daily clinical practice.
Quantify plasma inflammatory markers (IL-6, IL-8, TNF-?) before the start of treatment (azithromycin vs. placebo) and 3 months of it. |
Valorar el papel de la azitromicina como inmunomodulador de la respuesta inflamatoria en pacientes pediátricos con neumonías recurrentes y déficit de MBL, analizar el papel de dicho antibiótico en la expresión de los receptores de MBL (CDw93) y evaluar la trascendencia de su uso en la práctica clínica diaria.
Cuantificar los marcadores inflamatorios en plasma (IL-6, IL-8, TNF-?) antes del inicio de tratamiento (azitromicina vs placebo) y a los 3 meses del mismo. |
|
E.2.2 | Secondary objectives of the trial |
To assess the rate of pneumonia presenting our patients during the 3 months of treatment (azithromycin vs. placebo) and 6 months of the suspension.
Analyze the days of hospitalization, days of consultations in emergency services, school absenteeism (days per month does not go to school) and labor (days per month that parents do not go to work) during the 3 months of treatment (azithromycin vs placebo) and 6 months of suspension. |
Valorar la tasa de neumonías que presentan nuestros pacientes durante los 3 meses del tratamiento (azitromicina vs placebo) y a los 6 meses de la suspensión del mismo.
Analizar los días de ingresos hospitalarios, días de consultas en servicios de urgencias, absentismo escolar (días al mes que no acude al colegio) y laboral (días al mes que no asisten los padres al trabajo) durante los 3 meses de tratamiento (azitromicina vs placebo) y a los 6 meses de suspensión del mismo. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
A. - Patient greater than six months and less than 14 years. 2. - Patients with NR, ie, at least two pneumonic episodes observed radiologically in an interval of one year or three episodes at any time in his life, whenever there is radiological normalization between episodes. 3. - Patient with MBL deficiency, ie serum MBL levels <500 ng / ml. 4. - Patients whose parents or guardians accept their participation in the study by signing an informed consent. |
1.- Paciente mayor de seis meses y menores de 14 años. 2.- Pacientes con NR, es decir, al menos dos episodios neumónicos constatados radiológicamente en un intervalo de un año, o bien tres episodios en cualquier momento de su vida, siempre que exista normalización radiológica entre los episodios. 3.- Paciente con déficit de MBL, esto es, niveles de MBL en suero < 500 ng/ml. 4.- Pacientes cuyos padres o tutores acepten su participación en el estudio mediante la firma de un consentimiento informado. |
|
E.4 | Principal exclusion criteria |
1. - Patients with other associated immunodeficiencies. 2. - Patients with other lung diseases, heart disease or cancer processes. 3. - Patients with allergies to azitromicine, other macrolides or any excipients of the formulation. 4. - Patients treated with other drugs that may interact with macrolides. 5. - Patients with long QT for age. |
1.- Pacientes con otras inmunodeficiencias asociadas. 2.- Pacientes con otras neumopatías, cardiopatías o procesos oncológicos. 3.- Pacientes con alergias a azitromicina, a otros antibióticos macrólidos, o a cualquiera de los excipientes de la formulación. 4.- Pacientes en tratamiento con otros fármacos que puedan interaccionar con los macrólidos. 5.- Pacientes con QT largo para su edad. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Quantification of inflammatory markers (IL-6, IL-8, TNF-?). |
Cuantificación de los marcadores inflamatorio (IL-6, IL-8, TNF-?). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
The inflammatory markers were quantified at baseline (azithromycin vs. placebo) and 3 months of it. |
Los marcadores inflamatorios se cuantificarán al inicio del tratamiento (azitromicina vs placebo) y a los 3 meses del mismo. |
|
E.5.2 | Secondary end point(s) |
To assess the rate of pneumonia and Analyze the days of hospitalization, days of consultations in emergency services, school absenteeism (days per month does not go to school) and labor (days per month that parents do not go to work) during the 3 months of treatment (azithromycin vs placebo) and 6 months of suspension. |
Valorar la tasa de neumonías y analizar los días de ingresos hospitalarios, días de consultas en servicios de urgencias, absentismo escolar (días al mes que no acude al colegio) y laboral (días al mes que no asisten los padres al trabajo). |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
3 months of treatment (azithromycin vs. placebo) and 6 months of the suspension. |
Valorar la tasa de neumonías durante los 3 meses del tratamiento (azitromicina vs placebo) y a los 6 meses de la suspensión del mismo.
Analizar los días de ingresos hospitalarios, días de consultas en servicios de urgencias, absentismo escolar (días al mes que no acude al colegio) y laboral (días al mes que no asisten los padres al trabajo) durante los 3 meses de tratamiento (azitromicina vs placebo) y a los 6 meses de suspensión del mismo. |
|
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 | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | 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 | Yes |
E.8.2.3 | Other | No |
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
|
The study will end after 3 months of treatment (azithromycin vs. placebo) and 6 months follow-up after completion of treatment. |
El estudio finalizará tras 3 meses de tratamiento (azitromicina vs placebo) y 6 meses de seguimiento posteriores a la finalización del tratamiento. |
|
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 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
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 |