RANDOMIZED CONTROLLED TRIAL (RCT) TO DETERMINE THE EFFICACY AND SAFETY OF AZITHROMYCIN (AZN) MAINTENANCE THERAPY FOR 6 MONTHS IN SUBJECTS WITH PCD - A DOUBLE-BLIND, PARALLEL GROUP STUDY

Phase 4

Completed

• Conditions: immotile cilia syndrome; Primairy Ciliary Dyskinesia; 10019243; 10024970

• Registration Number: NL-OMON42237
• Lead Sponsor: Vrije Universiteit Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 20

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, and/or abnormal ciliary ultra-structure demonstrated by transmission electron microscopy analysis or high resolution immunofluorescence (not incl. isolated IDA) and/or an unequivocal genetic mutation recognized to cause PCD.
Age * 7 years and * 50 years at inclusion;
Ability to swallow tablets;
At least 30 days treatment with antibiotics prescribed against respiratory tract infections/pulmonary exacerbations in the previous 2 years;
No AZN treatment within 1 month prior to screening (visit 1);
No current therapy with systemic, inhaled maintenance antibiotics
FEV1 % predicted > 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.

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.
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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>To determine the efficacy of 6 months of maintenance treatment with AZN on<br /><br>respiratory system exacerbations in subjects with PCD, 7-40 years of age.<br /><br>An exacerbation is defined as:<br /><br>1. Respiratory symptoms leading to start of systemic antibiotic treatment,<br /><br>irrespective of results of bacterial culture.<br /><br>2. A decline in FEV1predicted of *10% relative to the avarage of FEV1 %<br /><br>predicted at Visit 1 and Visit 2, whether or not antibiotics are prescribed</p><br>