Primary ciliary dyskinesia (PCD) MedDRA version: 18.0 Level: PT Classification code 10069713 Term: Primary ciliary dyskinesia System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: AZITROMYCINE CF 250 mg
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: azithromycin monohydrate
CAS Number: 121470-24-4
Other descriptive name: AZITHROMYCIN MONOHYDRATE
Conce
Sponsors
Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • A confirmed diagnosis of PCD: - Characteristic clinical symptoms; and - High speed video microscopic recordings of abnormal ciliary beat pattern and/or frequency; and: either - Abnormally low nasal NO production 40 % at screening (Visit 1); • Ability to perform spirometry and Multiple Breath Washout (MBW); • Personally provide, or have a legal guardian provide written informed consent to participate in the trial, according to local regulations. Are the trial subjects under 18? yes Number of subjects for this age range: 65 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 60 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Known infection with Nontuberculous Mycobacteria (NTM) (found in sputum in the past 6 months prior to screening (Visit 1), Achromobacter xylosoxidans, Burkholderia cepacia or chronic infection with Pseudomonas aeruginosa; • Be currently participating in, or have participated in another investigative drug trial within four weeks prior of screening (Visit 1); • A history of allergic reaction to macrolide antibiotics incl. ketolide antibiotics, peanut, or to any of the excipients of ‘Azithromycine CF 250 mg’ or to any of the ingredients of the placebo (Carboxymethylstarch sodium, Cellulose microcryst. (PH 102), Colloidal anhydrous silica, Gelatin, Glycerol distearate, Lactose monohydrate, Magnesium stearate, Microcrystalline cellulose, Polyvinyl alcohol, Potato starch, Pregelatinised maize starch, Sodium laurilsulfate, Sodium starch glycolate, Soya lecithin, Talc, Titanium dioxide (E171) and Xanthan gum); • Liver disease with Alanine transaminase (ALT) twice or more the upper limits of normal or history of portal hypertension ; • Known kidney disease with serum creatinine > 150 µmol/l and/or Glomerular Filtration Rate (GFR) < 50 ml/min; • Known congenital or documented acquired prolonged QT-interval, cardiac arrhythmia, clinical relevant bradycardia, severe heart failure, or electrolyte disturbances. • Known myasthenia gravis. • Current treatment with ciclosporin, coumarin-like oral anticoagulants (e.g. warfarin), digoxin, ergotamine derivatives (e.g. methylergometrine), nelfinavir, rifabutin and active substances known to prolong QT interval such as amiodarone and other class ?A and class ??? antiarrhythmics, cisapride, terfenadin, antipsychotic agents such as pimozide, antidepressants such as citalopram and fluoroquinolones such as moxifloxacin and levofloxacin; • Be pregnant or breastfeeding; plan to become pregnant whilst in the trial; or be female of childbearing potential (at the discretion of the investigator) using an unreliable form of contraception; • Requirement of home oxygen (not incl. supplemental oxygen for use only when exercising, mountaineering or travelling by air) or assisted ventilation; or • Have any concomitant medical, psychiatric, or social condition that, in the Investigator’s opinion, would put the subject at significant risk, may confound the results or may significantly interfere with the subject’s participation in the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: •To determine the efficacy of azithromycin on FEV1, FVC and FEF25-75; •To determine the efficacy of azithromycin on Intra Thoracic Gas Volume (ITGV) and airways resistance; •To determine the efficacy of azithromycin on the new outcome measure: Lung Clearance Index (LCI); •To determine the efficacy of azithromycin on Respiratory symptoms, Sinus symptoms and Ear & Hearing symptoms on the Quality Of Life – Primary ciliary dyskinesia measure (QOL-PCD); •To determine the efficacy of azithromycin on hearing impairment; •To determine the efficacy of azithromycin on sputum microbiology and inflammatory markers; •To assess the safety of azithromycin. ;Primary end point(s): Difference in the number of respiratory system exacerbations between treatments in the pre- to post-intervention period. ;Timepoint(s) of evaluation of this end point: End of trial.;Main Objective: To determine the efficacy of 6 months of maintenance treatment with azithromycin on respiratory system exacerbations in subjects with Primary ciliary dyskinesia, 7-50 years of age. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Difference in FEV1 % predicted, FVC % predicted and FEF25-75 % predicted between treatments in the pre- to post-intervention period. • Difference in RV % predicted, RV/TLC % predicted and Raw % predicted between treatments in the pre- to post-intervention period. • Difference in Lung Clearance Index (LCI), Sacin and Scond between treatments in the pre- to post-intervention period. • Difference in Respiratory symptoms, Sinus symptoms and Ear & Hearing symptoms on the QOL-PCD between treatments in the pre- to post-intervention period. • Difference in hearing threshold between treatments in the pre- to post-intervention period. • Difference in tympanometry between treatments in the pre- to post-intervention period. • Difference in inflammatory markers between treatments in the pre- to post-intervention period. • Difference in sputum microbiology between treatments in the pre- to post-intervention period. • Adverse Events (AE) and Serious Adverse Events (SAE).;Timepoint(s) of evaluation of this end point: End of trial. | — |
Countries
Denmark, Germany, Netherlands, Switzerland, United Kingdom
Contacts
Public ContactPaediatric Pulmonary Service
Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
Outcome results
None listed