A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder 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: NCT00455130 |
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Recruitment Status :
Completed
First Posted : April 3, 2007
Last Update Posted : February 2, 2010
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Cystic fibrosis is the most frequent lethal genetic disease of childhood. Causes disruption of glandular function of the pancreas, intestine, liver, lungs (causing chronic lung infection with emphysema), sweat glands and reproductive organs. We know that many CF patients die of lung failure, brought about in part by repeated lung infections caused by thick, sticky mucus that cannot be readily cleared from the lung.
Inhaled mannitol is an osmotic agent that has been investigated in a number of small studies that have examined mucociliary clearance, quality of life and lung function in CF and bronchiectasis. The promising results of these studies warrant futher investigation. The aim of this study is to assess the safety and efficacy of inhaled mannitol when administered twice a day over two weeks in CF.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: Inhaled mannitol | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Allocation: | Randomized |
| Intervention Model: | Crossover Assignment |
| Masking: | Double |
| Primary Purpose: | Treatment |
| Study Start Date : | March 2004 |
| Actual Study Completion Date : | August 2005 |
- FEV1
- Other measures of lung function
- Quality of life
- Sputum microbiology
- Sputum rheology
- Safety
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, Learn About Clinical Studies.
| Ages Eligible for Study: | 8 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Confirmed diagnosis of cystic fibrosis (sweat test/genotype)
- Aged 8 years or older
- Have FEV1 between 40% and 80% of predicted for height, age and gender OR a decrease in FEV1 of 20% or more than that recorded 6-12 months previously.
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As determined by the investigator, are capable and willing to
- Use the study diary as required for this protocol
- Able to perform all of the techniques necessary to measure lung function
- Able to administer the dry powder mannitol
- Are capable of and have given informed consent
- Clinically stable at study entry
Exclusion Criteria:
- Investigators, site personnel directly affiliated with this study, and their immediate families.
- Subjects under the age of 8 years.
- Subjects with currently active asthma
- Subjects using hypertonic saline treatment in the last 2 weeks
- Considered "terminally ill" or listed for transplantation
- Requiring home oxygen or assisted ventilation
- Colonisation with Burkholderia cepacia
- Significant episode of hemoptysis (>60 mls) in the previous 12 months
- Myocardial Infarction in the six months prior to enrolment.
- Cerebral Vascular Accident in the six months prior to enrolment.
- Ocular surgery in the three months prior to enrolment.
- Abdominal surgery in the three months prior to enrolment.
- Subjects who are breast feeding or pregnant.
- Female subjects of reproductive capability, not using a reliable form of contraception
- Inability to obtain informed consent from the subject or subject's authorised representative.
- Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
- Known intolerance to mannitol or beta2 agonists.
- Uncontrolled hypertension - systolic BP > 160 and or diastoli
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): NCT00455130
| Australia, New South Wales | |
| Royal Prince Alfred Hospital | |
| Sydney, New South Wales, Australia, 2050 | |
| Childrens Hospital at Westmead | |
| Sydney, New South Wales, Australia, 2145 | |
| Australia, Queensland | |
| Prince Charles Hospital | |
| Brisbane, Queensland, Australia | |
| Australia, Victoria | |
| Royal Children's Hospital | |
| Melbourne, Victoria, Australia, 3052 | |
| The Alfred Hospital | |
| Melbourne, Victoria, Australia, 3181 | |
| Australia, Western Australia | |
| Princess Margaret Hospital for Children | |
| Perth, Western Australia, Australia, 6840 | |
| Sir Charles Gairdner | |
| Perth, Western Australia, Australia | |
| New Zealand | |
| Greenlane Hospital | |
| Auckland, North Island, New Zealand | |
| Study Director: | Brett Charlton | Pharmaxis |
| ClinicalTrials.gov Identifier: | NCT00455130 |
| Other Study ID Numbers: |
DPM-CF-201 |
| First Posted: | April 3, 2007 Key Record Dates |
| Last Update Posted: | February 2, 2010 |
| Last Verified: | January 2010 |
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mannitol cystic fibrosis mucolytic mucoactive |
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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 |