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

NCT00557089 | Completed Phase 4

• 1 other identifier: 1000010903
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

Brief Summary

This study will assess whether rhDNase treatment improves ventilation inhomogeneity as assessed by lung clearance index (LCI) in patients with cystic fibrosis (CF).

Conditions

Cystic Fibrosis

Keywords

Pediatrics; Cystic Fibrosis; Lung Clearance Index; rhDNAse

Outcome Measures

Primary Outcomes (1)

• The change in LCI from baseline to end of treatment in rhDnase treated patients versus patients receiving placebo

  The duration of the patient's involvement in the study (approximately 3 months)

Secondary Outcomes (7)

• Change in FEV1 % predicted

  The duration of the patient's involvement in the study (approximately 3 months)

• Change in FVC (in litres)

  The duration of the patient's involvement in the study (approximately 3 months)

• Change in FVC % predicted

  The duration of the patient's involvement in the study (approximately 3 months)

• Change in FEF25-75 (liters/sec)

  The duration of the patient's involvement in the study (approximately 3 months)

• Change in FEF25-75 % predicted

  The duration of the patient's involvement in the study (approximately 3 months)

Study Arms (2)

1

OTHER

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

2

OTHER

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

Interventions

rhDNAse DRUG

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

1; 2

Placebo OTHER

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.

1; 2

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• The Hospital for Sick Children: lead

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Related Publications (2)

• Amin R, Subbarao P, Lou W, Jabar A, Balkovec S, Jensen R, Kerrigan S, Gustafsson P, Ratjen F. The effect of dornase alfa on ventilation inhomogeneity in patients with cystic fibrosis. Eur Respir J. 2011 Apr;37(4):806-12. doi: 10.1183/09031936.00072510. Epub 2010 Aug 6.

  PMID: 20693248 RESULT

• Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.

  PMID: 33735508 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

dornase alfa

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Felix Ratjen, MD

  The Hospital for Sick Children, Toronto Canada

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Division Head, Respiratory Medicine

Study Record Dates

• First Submitted: November 9, 2007
• First Posted: November 12, 2007
• Study Start: January 1, 2008
• Primary Completion: June 1, 2009
• Study Completion: June 1, 2009
• Last Updated: September 2, 2013

Record last verified: 2013-08

Locations

CA (1)