|- candidate number||18375|
|- NTR Number||NTR4674|
|- ISRCTN||ISRCTN no longer applicable|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||15-jul-2014|
|- Secondary IDs||2013_198 METC |
|- Public Title||Hypertonic saline in Primary Ciliary Dyskinesia: a pilot study|
|- Scientific Title||Hypertonic saline in Primary Ciliary Dyskinesia: a pilot study|
|- ACRONYM||HS study|
|- hypothesis||We hypothesize that bi-daily 5ml NaCl 7% nebulizations improve respiratory symptoms and quality of life in PCD patients compared to 5ml NaCl 0,9% nebulizations.|
|- Healt Condition(s) or Problem(s) studied||Primary ciliary dyskinesia|
|- Inclusion criteria||* Diagnosis of primary ciliary dyskinesia |
* ≥ 18 years of age
* Capable of performing lung function tests.
* 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||* Smoking|
* FEV1 < 40 %.
* Use of Pulmozyme or other mucolytics or non-routine antibiotics in the previous 30 days.
* A decline in lung function of more than 15 % or oxyhemoglobin of < 90% after test nebulization with hypertonic saline at screening visit
* Women with a current or intended pregnancy during the trial
* Diagnosis of quinine sulphate allergy
* Myasthenia Gravis
* Lambert-Eaton syndrome
* Optic neuritis
* Atrium fibrillation and other severe cardiac heart disease
* Glucose 6PD deficiency
|- mec approval received||yes|
|- multicenter trial||yes|
|- Type||2 or more arms, randomized|
|- planned startdate ||15-sep-2013|
|- planned closingdate||1-jun-2015|
|- Target number of participants||20|
|- Interventions||A: NaCl 7% nebulization with 0,25mg/ml quinine sulphate as taste masking agent|
B: NaCl 0,9% nebulization with 0,25mg/ml quinine sulphate as taste masking agent
|- Primary outcome||Primary research question:|
- Is there a significant difference in change in the St. Georgeís Respiratory Questionnaire total and sub-scores (SGRQ) within intervention periods?
|- Secondary outcome||Secondary research questions:|
- Are there significant differences in change in the Quality of Life score for bronchiectasis patients score (QoL-B) , lung function, lung clearance index, respiratory culture results, inflammatory parameters in sputum and blood and exacerbation frequency?
- What are the effect sizes of these differences in PCD patients?
|- Timepoints||6 visits: baseline, 6 weeks, 12 weeks, 16 weeks, 22 weeks, 28 weeks.|
|- Trial web site|
|- status||open: patient inclusion|
|- CONTACT FOR PUBLIC QUERIES||MD. T. Paff|
|- CONTACT for SCIENTIFIC QUERIES||MD. PhD. E.G. Haarman|
|- Sponsor/Initiator ||VU University Medical Center|
(Source(s) of Monetary or Material Support)
|- Brief summary|
|- Main changes (audit trail)|
|- RECORD||15-jul-2014 - 10-aug-2014|