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Inhaled Mannitol as a Mucoactive Therapy for Bronchiectasis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pharmaxis
ClinicalTrials.gov Identifier:
NCT00669331
First received: April 28, 2008
Last updated: March 29, 2016
Last verified: March 2016
Results First Received: November 22, 2015  
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
Condition: Bronchiectasis
Interventions: Drug: Inhaled mannitol
Drug: Matched control

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Prior to randomisation, subjects underwent a Mannitol Tolerance Test (MTT) - only those who passed the MTT were eligible to be randomised. 581 patients in total underwent the MTT

Reporting Groups
  Description
Mannitol

Inhaled mannitol

Inhaled mannitol: 400mg BD for 52 weeks

Control Matched control: Inhaled mannitol 50mg BD for 52 weeks

Participant Flow for 2 periods

Period 1:   Randomisation to First Dose
    Mannitol     Control  
STARTED     244     241  
COMPLETED     233     228  
NOT COMPLETED     11     13  
Adverse Event                 1                 0  
Protocol Violation                 1                 0  
Withdrawal by Subject                 3                 2  
Lost to Follow-up                 2                 0  
Death                 1                 0  
Sponsor decision                 0                 1  
Randomised in error                 3                 10  

Period 2:   Blinded Treatment Period
    Mannitol     Control  
STARTED     233     228  
COMPLETED     191     189  
NOT COMPLETED     42     39  
Adverse Event                 16                 10  
Protocol Violation                 1                 1  
Withdrawal by Subject                 17                 19  
Lost to Follow-up                 3                 2  
Death                 0                 2  
Physician Decision                 3                 3  
Unable/unwilling to comply w trial reqs                 2                 2  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Patients randomised and treated with at least one dose of study medication

Reporting Groups
  Description
Mannitol

Inhaled mannitol

Inhaled mannitol: 400mg BD for 52 weeks

Control Matched control: Inhaled mannitol 50mg BD for 52 weeks
Total Total of all reporting groups

Baseline Measures
    Mannitol     Control     Total  
Number of Participants  
[units: participants]
  233     228     461  
Age  
[units: years]
Mean (Standard Deviation)
  59.30  (14.06)     60.26  (13.02)     59.77  (13.55)  
Gender  
[units: participants]
     
Female     147     142     289  
Male     86     86     172  
Baseline Pulmonary Exacerbation rate  
[units: events/year]
Mean (Standard Deviation)
  3.20  (1.38)     3.25  (1.40)     3.22  (1.39)  
Baseline % of Predicted FEV1  
[units: participants]
     
<60%     101     107     208  
>=60%     132     121     253  
Extent of Bronchiectasis  
[units: participants]
     
Diffuse     133     118     251  
Focal     77     73     150  
Both (Diffuse and Focal)     23     37     60  



  Outcome Measures
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1.  Primary:   Rate of Graded Pulmonary Exacerbations   [ Time Frame: 52 weeks ]

2.  Secondary:   Quality of Life as Measured by the St. Georges Respiratory Questionnaire (SGRQ) Total Score   [ Time Frame: 52 weeks ]

3.  Secondary:   Antibiotic Use Prescribed for Treated Pulmonary Exacerbations   [ Time Frame: 52 weeks ]

4.  Secondary:   Time to First Graded Exacerbation   [ Time Frame: 52 weeks ]

5.  Secondary:   Duration of Graded Exacerbations   [ Time Frame: 52 weeks ]

6.  Secondary:   Sputum Volume   [ Time Frame: 52 weeks ]

7.  Secondary:   Daytime Sleepiness Scores   [ Time Frame: 52 weeks ]

8.  Secondary:   Lung Function - Change in FEV1 (Forced Expiratory Volume in One Second)   [ Time Frame: 52 weeks ]

9.  Secondary:   Lung Function - Change in FVC (Forced Vital Capacity)   [ Time Frame: 52 weeks ]

10.  Secondary:   Lung Function - Change in FEV1/FVC   [ Time Frame: 52 weeks ]

11.  Secondary:   Lung Function - Change in FEF25-75 (Forced Expiratory Flow Rate Averaged Over 25th -75th Percentile of FVC)   [ Time Frame: 52 weeks ]

12.  Secondary:   Safety Profile - Sputum Microbiology   [ Time Frame: 52 weeks ]

13.  Secondary:   Safety Profile - Clinical Chemistry   [ Time Frame: 52 weeks ]

14.  Secondary:   Safety Profile - Hematology   [ Time Frame: 52 weeks ]

15.  Secondary:   • (Exploratory) Number of Hospitalizations Due to Pulmonary Exacerbations   [ Time Frame: 52 weeks ]

16.  Other Pre-specified:   Health Related Costs of Treating Patients With Bronchiectasis   [ Time Frame: 52 weeks ]

17.  Other Pre-specified:   Health Status and Utility Scores   [ Time Frame: 52 weeks ]

18.  Other Pre-specified:   Health Related Quality of Life (HRQL) and Quality Adjusted Life Years (QALYs) by Treatment Group Using Utility Scores From the Health Utilities Index Questionnaire   [ Time Frame: 52 weeks ]

19.  Other Pre-specified:   Cost Effectiveness of Treating Patients With Bronchiectasis With Inhaled Mannitol   [ Time Frame: 52 weeks ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Relatively few studies in this area -ideal choice of endpoints not well-defined. No widely accepted exacerbation definition. Relies on patients’ accurate & timely symptom reporting.Unclear event end-dates may impact on counting of subsequent events.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr Brett Charlton, Medical Director
Organization: Pharmaxis Ltd
phone: +61 2 94547210
e-mail: brett.charlton@pharmaxis.com.au


Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Pharmaxis
ClinicalTrials.gov Identifier: NCT00669331     History of Changes
Other Study ID Numbers: DPM-B-305
Study First Received: April 28, 2008
Results First Received: November 22, 2015
Last Updated: March 29, 2016
Health Authority: United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Australia: Department of Health and Ageing Therapeutic Goods Administration
Germany: Federal Institute for Drugs and Medical Devices
United Kingdom: Medicines and Healthcare Products Regulatory Agency
New Zealand: Medsafe
Belgium: Federal Agency for Medicinal Products and Health Products
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Chile: Instituto de Salud Pública de Chile