Determination of efficacy and safety of azithromycin maintenance therapy for 6 months in subjects with primary ciliary dyskinesia
- Conditions
- Primary ciliary dyskinesia (PCD)MedDRA version: 18.0Level: PTClassification code 10069713Term: Primary ciliary dyskinesiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
- Registration Number
- EUCTR2013-004664-58-DE
- Lead Sponsor
- Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 125
• 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 < 77 nl/min;
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 or 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.
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
• 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method