Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

This study has been completed.
Sponsor:
Information provided by:
Pharmaxis
ClinicalTrials.gov Identifier:
NCT00117208
First received: June 30, 2005
Last updated: January 31, 2010
Last verified: January 2010
  Purpose

The purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.


Condition Intervention Phase
Cystic Fibrosis
Drug: mannitol
Drug: mannitol + pulmozyme
Drug: Dornase alpha
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Cross-Over Comparative Study of Inhaled Mannitol, Alone and in Combination With Daily rhDNase, in Children With Cystic Fibrosis

Resource links provided by NLM:


Further study details as provided by Pharmaxis:

Primary Outcome Measures:
  • FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase [ Time Frame: 12 weeks ]

Secondary Outcome Measures:
  • to compare mannitol to rhDNase on FVC [ Time Frame: 12 weeks ]
  • to assess whether the effects of mannitol are additive to rhDNase [ Time Frame: 12 weeks ]
  • to demonstrate that mannitol does not cause deterioration in airway inflammation [ Time Frame: 12 weeks ]
  • to assess whether mannitol reduces the bacterial load in the lung [ Time Frame: 12 weeks ]
  • to assess whether the effects of mannitol are beneficial to quality of life [ Time Frame: 12 weeks ]
  • to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone [ Time Frame: 12 weeks ]

Enrollment: 20
Study Start Date: November 2005
Study Completion Date: February 2008
Primary Completion Date: February 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: mannitol
400mg BD for 12 weeks
Active Comparator: 2
DNase daily for 12 weeks
Drug: Dornase alpha
2.5mg daily for 2 weeks
Other Name: rhDNase, pulmozyme
3
combination
Drug: mannitol + pulmozyme
combination

  Eligibility

Ages Eligible for Study:   8 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Known diagnosis of cystic fibrosis (sweat test or genotype)
  • Of either gender
  • Aged between 8 and 18 years
  • Have a baseline FEV1 of <70% of the predicted normal value
  • Currently taking rhDNase for at least 4 weeks

Exclusion Criteria:

  • Currently active asthma, uncontrolled hypertension, colonised with Burkholderia cepacia or MRSA
  • Listed for transplantation
  • Known intolerance to mannitol, rhDNase or bronchodilators
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00117208

Locations
United Kingdom
Royal Brompton Hospital
London, United Kingdom
Great Ormond Hospital for Children
London, United Kingdom
Sponsors and Collaborators
Pharmaxis
Investigators
Principal Investigator: Andrew Bush, FRCPCH Royal Brompton and Harefiled NHS Trust
Principal Investigator: Colin Wallis, FRCPCH Great Ormond Street Hospital for Children NHS Foundation Trust
  More Information

Additional Information:
Publications:

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
ClinicalTrials.gov Identifier: NCT00117208     History of Changes
Other Study ID Numbers: DPM-CF-203
Study First Received: June 30, 2005
Last Updated: January 31, 2010
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Pharmaxis:
cystic fibrosis

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes
Mannitol
Diuretics, Osmotic
Diuretics
Natriuretic Agents
Physiological Effects of Drugs
Pharmacologic Actions
Cardiovascular Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on August 20, 2014