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Trial record 1 of 1 for:    NCT00630812
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Long Term Administration of Inhaled Mannitol in Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT00630812
Recruitment Status : Completed
First Posted : March 7, 2008
Results First Posted : October 9, 2020
Last Update Posted : October 9, 2020
Sponsor:
Collaborators:
ethica Clinical Research Inc.
Europe: KasaConsult bvba, Hoegaarden, Belgium
Argentina: Resolution Latin America; Buenos Aires, Argentina
Information provided by (Responsible Party):
Pharmaxis

Tracking Information
First Submitted Date  ICMJE February 27, 2008
First Posted Date  ICMJE March 7, 2008
Results First Submitted Date  ICMJE August 11, 2020
Results First Posted Date  ICMJE October 9, 2020
Last Update Posted Date October 9, 2020
Study Start Date  ICMJE September 2008
Actual Primary Completion Date April 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 6, 2020)
Change in Absolute FEV1 From Baseline Over 26 Weeks [ Time Frame: 26 weeks ]
Change from baseline in forced expiratory volume at one second (FEV1) averaged over 26 weeks (measured at 6,14 and 26 weeks) The mean absolute change from baseline FEV1 (mL) over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach.Least square means presented are for the average change over the 6, 14, and 26 week visits.
Original Primary Outcome Measures  ICMJE
 (submitted: February 27, 2008)
FEV1 [ Time Frame: 26 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 6, 2020)
  • Change in FEV1 From Baseline Over 26 Weeks - Dornase Users [ Time Frame: 26 weeks ]
    In the subset of dornase users, the mean absolute change from baseline FEV1 (mL) averaged over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits. Change from baseline over 26 weeks (measured at 6,14, 26 weeks) in subset of dornase users
  • Rate of Protocol Defined Pulmonary Exacerbations (PDPE) [ Time Frame: 26 weeks ]
    Exacerbations treated with IV antibiotics and with at least 4 signs and symptoms according to Fuchs criteria (1994). Summary table presents the number with 0, 1,2 and 3 PDPEs during the 26 week treatment period.
  • Hospitalisations Associated With Protocol Defined Pulmonary Exacerbations (PDPEs) [ Time Frame: 26 weeks ]
    The number of hospitalisations is summarised and then the rate per person is analysed.
  • Antibiotic Use Associated With PDPEs [ Time Frame: 26 weeks ]
    Number of courses per person in the 26 week period is summarised and then the rate per person analysed.
  • Absolute Change in FEV1 Percent Predicted at 26 Weeks [ Time Frame: 26 weeks ]
    Change from baseline at 26 weeks in FEV1 percent predicted with BOCF for those with missing values at week 26
  • Change in FVC (mL) Across 26 Weeks [ Time Frame: 26 weeks ]
    Change from baseline in forced vital capacity (FVC) across 26 weeks (measured at 6,14 and 26 weeks)
  • Change From Baseline FEF25-75 (mL/s) Over 26 Weeks [ Time Frame: 26 weeks ]
    Change from baseline in forced expiratory flow at 25-75% of forced vital capacity (FEF25-75) (mL/s) averaged over 26 weeks (measured at 6,14 and 26 weeks) The mean absolute change from baseline over 26 weeks (measured at week 6, 14 and 26) was compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach. Least square means presented are for the average change over the 6, 14, and 26 week visits.
  • Sputum Weight at Baseline in Response to First Dose of Treatment [ Time Frame: up to 30 mins after first dose of trial treatment ]
    Sputum was collected during and for 30 minutes following the administration of the first dose of study treatment.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long Term Administration of Inhaled Mannitol in Cystic Fibrosis
Official Title  ICMJE Long Term Administration of Inhaled Mannitol in Cystic Fibrosis- A Safety and Efficacy Study
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: inhaled mannitol
    400 mg BD for 26 + 26 weeks
  • Drug: Placebo comparator
    BD for 26 weeks followed by 26 weeks of inhaled mannitol in the open label phase
Study Arms  ICMJE
  • Experimental: A
    active treatment
    Intervention: Drug: inhaled mannitol
  • Placebo Comparator: B
    Intervention: Drug: Placebo comparator
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 23, 2010)
318
Original Estimated Enrollment  ICMJE
 (submitted: February 27, 2008)
250
Actual Study Completion Date  ICMJE November 2010
Actual Primary Completion Date April 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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'

    -

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Belgium,   Canada,   France,   Germany,   Netherlands,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00630812
Other Study ID Numbers  ICMJE DPM-CF-302
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pharmaxis
Study Sponsor  ICMJE Pharmaxis
Collaborators  ICMJE
  • ethica Clinical Research Inc.
  • Europe: KasaConsult bvba, Hoegaarden, Belgium
  • Argentina: Resolution Latin America; Buenos Aires, Argentina
Investigators  ICMJE
Principal Investigator: Moira L Aitken, MD University of Washington Medical Centre, Seattle WA
PRS Account Pharmaxis
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP