A Phase 4 interventional study of rhDNAse and Placebo in Cystic Fibrosis, sponsored by The Hospital for Sick Children. Completed at 1 site in Canada. Open to participants aged 6 Years to 18 Years. Per ClinicalTrials.gov, last updated 2013-09-02.
Sponsored by The Hospital for Sick Children · Phase 4, Interventional, and Treatment
This study will assess whether rhDNase treatment improves ventilation inhomogeneity as assessed by lung clearance index (LCI) in patients with cystic fibrosis (CF).
Life expectancy in CF patients has greatly increased due to improved clinical care. While this is certainly beneficial to CF patients, it has made it more difficult to assess the effect of therapeutic interventions. Currently, FEV1 remains the primary outcome parameter for most clinical trials, but many CF patients have normal pulmonary function and the annual rate of decline is now less than 2 %. Therefore, additional parameters are needed that are more sensitive to define abnormalities in CF patients and that can be used in therapeutic trials.
Gas mixing techniques have been shown to be sensitive parameters to define abnormalities in patients with cystic fibrosis, but it is unclear how useful this technique is to detect changes after a therapeutic intervention. Abnormalities in gas clearance from the lung are largely due to retention of inhaled gases due to mucous obstruction in the lower airways and can be assessed with the lung clearance index (LCI). Interventions that improve mucous accumulation are expected to improve lung clearance as assessed by this technique. RhDNase has been demonstrated to improve lung function and reduce pulmonary exacerbations in patients with cystic fibrosis due to improved mucus clearance.
Lung clearance index (LCI) has been shown to be more sensitive than spirometry in detecting abnormalities in CF patients. Clear cut-offs have been found which can differentiate normal patients from even newly diagnosed CF patients. However, little is known about how LCI may change with treatment.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 17 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →The Hospital for Sick Children is the lead sponsor of 568 studies on the registry; 81 are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Respiratory culture positive for:
This arm will receive the active treatment for 28 days, followed by a 28 day washout period and then the placebo treatment for 28 days.
Drug: rhDNAse · Other: Placebo
This arm will receive the placebo treatment for 28 days, followed by a 28 day washout period and then the active treatment for 28 days.
Drug: rhDNAse · Other: Placebo
2.5 mg rhDNase will be dispensed in 2.5 ml vials and administered once a day for 28 days. Treatment will be administered by inhalation.
Also known as: Brand Name: Pulmozyme
2.5 mg of the placebo will be dispensed in 2.5 ml vials and administered once a day over 28 days. Treatment will be administered by inhalation.
The change in LCI from baseline to end of treatment in rhDnase treated patients versus patients receiving placebo
Time frame: The duration of the patient's involvement in the study (approximately 3 months)
Change in FEV1 % predicted
Time frame: The duration of the patient's involvement in the study (approximately 3 months)
Change in FVC (in litres)
Time frame: The duration of the patient's involvement in the study (approximately 3 months)
Change in FVC % predicted
Time frame: The duration of the patient's involvement in the study (approximately 3 months)
Change in FEF25-75 (liters/sec)
Time frame: The duration of the patient's involvement in the study (approximately 3 months)
Change in FEF25-75 % predicted
Time frame: The duration of the patient's involvement in the study (approximately 3 months)
Change in exhaled nitric oxide concentrations
Time frame: The duration of the patient's involvement in the study (approximately 3 months)
Incidence of adverse events and serious adverse events
Time frame: Duration of the study (approximately 1 year)
This study is completed, as verified in Aug 2013. You cannot join it, but the record below documents what was studied.
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