The Effect of rhDNase on Ventilation Inhomogeneity in Patients With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00557089
• Date of registration: 09/11/2007
• Prospective Registration: Yes
• Primary sponsor: The Hospital for Sick Children
• Public title: The Effect of rhDNase on Ventilation Inhomogeneity in Patients With Cystic Fibrosis
• Scientific title: Randomized, Placebo-controlled Trial on the Effect of rhDNase on Ventilation Inhomogeneity in Patients With Cystic Fibrosis
• Date of first enrolment: January 2008
• Target sample size: 17
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00557089
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 4

Countries of recruitment

Canada

Contacts

• Name: Felix Ratjen, MD
• Affiliation: The Hospital for Sick Children, Toronto Canada

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of CF as defined by clinical features of CF and a documented sweat chloride
> 60 mEq/L by quantitative pilocarpine iontophoresis test or a genotype showing two
well characterized disease causing mutations

- Informed consent and verbal assent (as appropriate) has been provided by the
subject's parent or legal guardian and the subject

- 6-18 years of age at enrolment

- Able to perform reproducible spirometry

- Clinically stable at enrolment

- Ability to comply with medication use, study visits and study procedures as judged by
the site investigator

- FEV1 % predicted > 70 % as calculated by the Wang reference equations

Exclusion Criteria:

- Respiratory culture positive for:

- NTM within past year or AFB positive at screening (sputum only)

- B. cepacia complex within past year or at screening

- Use of intravenous antibiotics or oral quinolones within 14 days of screening

- Investigational drug use within 30 days of screening

- History of alcohol, illicit drug or medication abuse within 1 year of screening

- Other major organ dysfunction excluding pancreatic dysfunction

- History of lung transplantation or currently on lung transplant list

- Physical findings at screening that would compromise the safety of the participant or
the quality of the study data



Age minimum: 6 Years
Age maximum: 18 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Other: Placebo

Drug: rhDNAse

Primary Outcome(s)

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)]

Secondary Outcome(s)

Change in FEF25-75 (liters/sec) [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 % predicted [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)]

Change in exhaled nitric oxide concentrations [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 FVC (in litres) [Time Frame: The duration of the patient's involvement in the study (approximately 3 months)]

Secondary ID(s)

1000010903

Source(s) of Monetary Support

Please refer to primary and secondary sponsors