Long Term Administration of Inhaled Mannitol in Cystic Fibrosis
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00630812 |
Recruitment Status :
Completed
First Posted : March 7, 2008
Results First Posted : October 9, 2020
Last Update Posted : October 9, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cystic Fibrosis | Drug: inhaled mannitol Drug: Placebo comparator | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 318 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Long Term Administration of Inhaled Mannitol in Cystic Fibrosis- A Safety and Efficacy Study |
Study Start Date : | September 2008 |
Actual Primary Completion Date : | April 2010 |
Actual Study Completion Date : | November 2010 |

Arm | Intervention/treatment |
---|---|
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 |
- 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.
- 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.

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Ages Eligible for Study: | 6 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Have given written informed consent to participate in this study in accordance with local regulations
- 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)
- Be aged > 6 years old
- Have FEV1 >40 % and < 90% predicted
- Be able to perform all the techniques necessary to measure lung function
Exclusion Criteria:
- 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.
- Be considered "terminally ill" or eligible for lung transplantation
- Have had a lung transplant
- Be using nebulized hypertonic saline in the 4 weeks prior to visit 1
- Have had a significant episode of hemoptysis (>60 mL) in the three months prior to enrolment
- Have had a myocardial infarction in the three months prior to enrolment
- Have had a cerebral vascular accident in the three months prior to enrolment
- Have had major ocular surgery in the three months prior to enrolment
- Have had major abdominal, chest or brain surgery in the three months prior to enrolment
- Have a known cerebral, aortic or abdominal aneurysm
- Be breast feeding or pregnant, or plan to become pregnant while in the study
- Be using an unreliable form of contraception (female subjects at risk of pregnancy only)
- Be participating in another investigative drug study, parallel to, or within 4 weeks of visit 0
- Have a known allergy to mannitol
- Be using beta blockers
- Have uncontrolled hypertension - systolic BP > 190 and / or diastolic BP > 100
- 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
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Be 'Mannitol Tolerance Test positive'
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00630812

Principal Investigator: | Moira L Aitken, MD | University of Washington Medical Centre, Seattle WA |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Pharmaxis |
ClinicalTrials.gov Identifier: | NCT00630812 |
Other Study ID Numbers: |
DPM-CF-302 |
First Posted: | March 7, 2008 Key Record Dates |
Results First Posted: | October 9, 2020 |
Last Update Posted: | October 9, 2020 |
Last Verified: | October 2020 |
cystic fibrosis mannitol mucoactive |
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases |
Genetic Diseases, Inborn Infant, Newborn, Diseases Mannitol Diuretics, Osmotic Diuretics Natriuretic Agents Physiological Effects of Drugs |