Long Term Administration of Inhaled Mannitol in Cystic Fibrosis

This study has been completed.
Sponsor:
Collaborators:
ethica Clinical Research Inc.
Europe: KasaConsult bvba, Hoegaarden, Belgium
Argentina: Resolution Latin America; Buenos Aires, Argentina
Information provided by (Responsible Party):
Pharmaxis
ClinicalTrials.gov Identifier:
NCT00630812
First received: February 27, 2008
Last updated: August 6, 2012
Last verified: August 2012
  Purpose

The purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis. Previous studies have demonstrated improvements in lung function, mucociliary clearance, changes in physical properties of mucus, 24 hour sputum weight and quality of life. The results of this study are to further investigate and confirm these findings in addition to examine the effect on antibiotic use and chest infections. It is hypothesised that inhaled mannitol will have beneficial effects compared to a control treatment. An open label phase of 26 weeks duration will follow the blinded 26 week phase. During the open label phase all subjects will receive active treatment.


Condition Intervention Phase
Cystic Fibrosis
Drug: inhaled mannitol
Drug: Placebo comparator
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Long Term Administration of Inhaled Mannitol in Cystic Fibrosis- A Safety and Efficacy Study

Resource links provided by NLM:


Further study details as provided by Pharmaxis:

Primary Outcome Measures:
  • Change in absolute FEV1 [ Time Frame: 26 weeks ] [ Designated as safety issue: No ]

Enrollment: 318
Study Start Date: September 2008
Study Completion Date: November 2010
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A
active treatment
Drug: inhaled mannitol
400 mg BD for 26 + 26 weeks
Placebo Comparator: B Drug: Placebo comparator
BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Have given written informed consent to participate in this study in accordance with local regulations
  2. Have a confirmed diagnosis of cystic fibrosis (positive sweat chloride value ≥ 60 mEq/L) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype)
  3. Be aged > 6 years old
  4. Have FEV1 >40 % and < 90% predicted
  5. Be able to perform all the techniques necessary to measure lung function

Exclusion Criteria:

  1. Investigators, site personnel directly affiliated with this study, or their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted.
  2. Be considered "terminally ill" or eligible for lung transplantation
  3. Have had a lung transplant
  4. Be using nebulized hypertonic saline in the 4 weeks prior to visit 1
  5. Have had a significant episode of hemoptysis (>60 mL) in the three months prior to enrolment
  6. Have had a myocardial infarction in the three months prior to enrolment
  7. Have had a cerebral vascular accident in the three months prior to enrolment
  8. Have had major ocular surgery in the three months prior to enrolment
  9. Have had major abdominal, chest or brain surgery in the three months prior to enrolment
  10. Have a known cerebral, aortic or abdominal aneurysm
  11. Be breast feeding or pregnant, or plan to become pregnant while in the study
  12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only)
  13. Be participating in another investigative drug study, parallel to, or within 4 weeks of visit 0
  14. Have a known allergy to mannitol
  15. Be using beta blockers
  16. Have uncontrolled hypertension - systolic BP > 190 and / or diastolic BP > 100
  17. Have a condition or be in a situation which in the Investigator's opinion may put the subject at significant risk, may confound results or may interfere significantly with the patient's participation in the study
  18. Be 'Mannitol Tolerance Test positive'

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  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: NCT00630812

  Show 53 Study Locations
Sponsors and Collaborators
Pharmaxis
ethica Clinical Research Inc.
Europe: KasaConsult bvba, Hoegaarden, Belgium
Argentina: Resolution Latin America; Buenos Aires, Argentina
Investigators
Principal Investigator: Moira L Aitken, MD University of Washington Medical Centre, Seattle WA
  More Information

Publications:

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Pharmaxis
ClinicalTrials.gov Identifier: NCT00630812     History of Changes
Other Study ID Numbers: DPM-CF-302
Study First Received: February 27, 2008
Last Updated: August 6, 2012
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Germany: Federal Institute for Drugs and Medical Devices
Belgium: Federal Agency for Medicinal Products and Health Products
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Keywords provided by Pharmaxis:
cystic fibrosis
mannitol
mucoactive

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 July 31, 2014