We hypothesize that hypertonic saline nebulizations improve respiratory symptoms and quality of life in PCD patients.
ID
Source
Brief title
Condition
- Respiratory disorders congenital
- Bacterial infectious disorders
- Respiratory tract infections
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Change in St. George's Respiratory Questionnaire score.
Secondary outcome
Change in Quality of Life questionnaire for bronchiectasis patients score, lung
function, lung clearance index, sputum culture results, inflammatory markers in
sputum and blood, exacerbation frequency.
Background summary
Primary ciliary dyskinesia (PCD) is an autosomal recessive disorder,
characterized by dysfunction of the cilia. Cilia line the respiratory
epithelium and form the basis of effective mucociliary clearance. Patients
experience recurrent respiratory tract infections leading to permanent lung
damage. At present, there are no randomized controlled trials on the treatment
of patients with primary ciliary dyskinesia. Treatment is largely based on
expert opinions and is focused on the control of pulmonary exacerbations.
Protocols are derived from cystic fibrosis (CF) guidelines. However, CF has a
different pathophysiology and therefore responses to treatments may be
different. Hypertonic saline has been shown to improve mucociliary clearance in
CF patients, but also in asthma and healthy controls. In CF patients bi-daily
nebulizations wit hypertonic saline improves lung function and reduces
respiratory symptoms and exacerbations. However, studies in non-CF
bronchiectasis patients are limited and the results are contradicting
Study objective
We hypothesize that hypertonic saline nebulizations improve respiratory
symptoms and quality of life in PCD patients.
Study design
This is a randomized placebo-controlled double-blind crossover pilot study,
conducted at the VU University Medical Center (VUmc), Amsterdam and the
Academic Medical Center (AMC), Amsterdam. The study has an AB/BA crossover
design with two interventions.
Intervention
A Hypertonic saline (7%) inhalation bi-daily during 3 months
B Placebo: Isotonic saline (0.9%) inhalation bi-daily during 3 months
Study burden and risks
Safety and efficacy of hypertonic saline nebulizations have been observed in
children and adults with CF and non-CF bronchiectasis. This study consists of 6
study visits of which half can be combined with standard 3-monthly outpatient
clinic visits. During these visits patients will have their medical history
taken and will undergo a physical examination, lung function tests, sputum
induction and blood withdrawal. There are only minor risks associated with this
study. Patients could directly benefit from the effect of the hypertonic saline
during the study.
De Boelelaan 1117
Amsterdam 1081 HV
NL
De Boelelaan 1117
Amsterdam 1081 HV
NL
Listed location countries
Age
Inclusion criteria
o Diagnosis of primary ciliary dyskinesia
o * 18 years of age
o Capable of performing lung function tests.
o The forced expiratory volume in one second (FEV1), measured at screening, has to be within 10 % of the best value obtained during the previous six months and at least 40% of the predicted value for height, age and sex.
Exclusion criteria
o Smoking
o FEV1 < 40 %.
o Use of Pulmozyme or other mucolytics or non-routine antibiotics in the previous 30 days.
o A decline in lung function of more than 15 % or oxyhemoglobin of < 90% after test nebulisation with hypertonic saline at screening visit17.
o Women with a current or intended pregnancy during the trial
o Diagnosis of quinine sulphate allergy
o Myasthenia Gravis
o Lambert-Eaton syndrome
o Optic neuritis
o Tinnitus
o Atrium fibrillation and other currently severe cardiac disease
o Epilepsy
o Glucose 6PD deficiency
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2013-001020-19-NL |
| CCMO | NL42765.029.13 |
| Other | NTR candidate nr 14470 |