primary ciliary dyskinesia. Diseases of bronchus, not elsewhere classified
Conditions
Interventions
Intervention 1: Intervention group: In this clinical trial, the primary intervention is administered orally as the drug Azithromycin dihydrate. Each film-coated tablet contains 250 mg of azithromycin
Children weighing less than 40 kg receive 250 mg (1 tablet) on even days of the week (three times a week: two, four, and six) each time, and children weighing 40 kg or more receive the same schedule w
Sponsors
Mashhad University of Medical Sciences
Eligibility
Sex/Gender
All
Age
6 Years to 18 Years
Inclusion criteria
Inclusion criteria: Children with primary ciliary dyskinesia (PCD) whose diagnosis has been confirmed by nasal nitric oxide testing or genetic testing and examination of relevant mutations. The age range of participants is between 6 and 18 years. At least three pulmonary exacerbations requiring hospitalization and intravenous treatment within the past year. The child's pulmonary status is at a level that meets the definition of pulmonary exacerbation. Absence of QT interval prolongation on standard electrocardiogram (long QT interval). Written informed consent of the parents for the child's participation in the study. Continuation of previous treatments other than maintenance antibiotics and prohibited drugs, without changes during the study.
Exclusion criteria
Exclusion criteria: A history of severe allergic reaction or intolerance to azithromycin or any macrolide antibiotic. Presence of any severe chronic underlying disease or complex medical condition that, in the opinion of the treating physician, could compromise participant safety or the validity of the study results.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of pulmonary exacerbations requiring hospitalization. Timepoint: Before the start of the intervention, three months after the intervention, and then 6 months after the intervention. Method of measurement: Patient's hospital admission records and bimonthly visit reports.;Forced expiratory volume in one second (FEV1). Timepoint: Before the start of the intervention, three months after the intervention, and then 6 months after the intervention. Method of measurement: Spirometry will be performed using a calibrated spirometer (e.g. MIR Spirolab III, Italy) according to ATS/ERS guidelines.;Forced vital capacity (FVC). Timepoint: Before the start of the intervention, three months after the intervention, and then 6 months after the intervention. Method of measurement: Spirometry will be performed using a calibrated spirometer (e.g. MIR Spirolab III, Italy) according to ATS/ERS guidelines.;FEV1 to FVC ratio. Timepoint: Before the start of the intervention, three months after the intervention, and then 6 months after the intervention. Method of measurement: Spirometry will be performed using a calibrated spirometer (e.g. MIR Spirolab III, Italy) according to ATS/ERS guidelines. | — |
Secondary
| Measure | Time frame |
|---|---|
| Respiratory symptoms (cough and phlegm). Timepoint: Before the start of the intervention, three and six months after the start of the intervention. Method of measurement: Using the Symptom Checklist at each visit.;Sputum Culture Results. Timepoint: Before the start of the intervention, three and six months after the start of the intervention. Method of measurement: Culture is performed on specific media (such as Blood agar, MacConkey agar) and growth of common bacteria (such as Pseudomonas aeruginosa, Haemophilus influenzae, Staphylococcus aureus) is reported.;Weight-Gain Trend. Timepoint: Before the start of the intervention, three and six months after the start of the intervention. Method of measurement: Standard digital scale. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactFatemeh Javaheri
Mashhad University of Medical Sciences
Outcome results
None listed