Mannitol Dose Response Study in Cystic Fibrosis

Many cystic fibrosis patients die of lung failure caused by repeated lung infections from thick, sticky mucus. Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology and replenishing the airway surface liquid layer in these patients, thereby enhancing the shift of stagnant mucus from the lungs. The study aim is to determine the optimal dose of mannitol to generate clinical improvement in patients with cystic fibrosis.

Official Title

A Phase IIa Randomised, Open Label, Dose Response Study to Determine the Optimum Dose of Dry Powder Mannitol Required to Generate Clinical Improvement In Patients With Cystic Fibrosis

Conditions

• Cystic Fibrosis

Study Type

Interventional

Study Design

Treatment, Randomized, Open Label, Dose Comparison, Crossover Assignment, Safety/Efficacy Study

Further Details

• Primary Outcomes: FEV1; FVC
• Secondary Outcomes: other measures of lung function; QOL; sputum microbiology; safety; sputum clearance and cough; respiratory symptoms

Study Start

October 2005; Expected completion: July 2007

Eligibility & Criteria

• Ages Eligible for Study: 7 Years and above
• Genders Eligible for Study: Both

Inclusion Criteria:

• Confirmed diagnosis of cystic fibrosis
• Aged 7 years or older
• Have FEV1 between 40% and 80% of predicted OR a deterioration in FEV1 of 20% or greater over the previous 12 months, if >80% predicted.
• Able to perform acceptable-quality spirometry
• Likely to co-operate, take medication as prescribed, and attend the clinic at the appointed times during the study
• Has given consent, and child age-appropriate assent (for minors) to participate in this study in accordance with local regulations
• Clinically stable in the week up to study entry
• No additional antibiotics or additional oral steroids for a period of 14 days before study entry (routine antibiotics permitted)

Exclusion Criteria:

• Investigators, site personnel directly affiliated with this study, and their immediate families.
• Subjects with currently active asthma
• Subjects colonised with Burkholderia cepacia or MRSA
• Considered “terminally ill” or listed for transplantation
• Requiring home oxygen or assisted ventilation
• Concurrent illness that in the investigators opinion may contribute to an increased and unacceptable risk if the subject was enrolled in the study (eg. significant varicies, portal hypertension, cor pulmonale)
• Significant episode of haemoptysis (>60 mLs) in the previous 12 months
• Heart attack or stroke in last 3 months
• Known aortic or cerebral aneurysm
• Subjects who are breast feeding or pregnant.
• At risk females unwilling to use appropriate contraception to prevent pregnancy during the course of the study
• Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
• Known intolerance to mannitol or unable to take any form of bronchodilator medications.
• Uncontrolled hypertension, systolic BP > 200 or diastolic BP> than 100
• Concurrent use of beta blocker medication
• Concurrent use of hypertonic saline
• Concurrent use of other pharmacological mucolytic agents
• Concurrent use of rhDNase [pulmozyme]

Total Enrolment

42 (expected)

Contact Details

• Tara Vouriot, BS 514.421.0442 Ext. 210 tvouriot@ethicaclinical.ca
• Lorena Figueroa lorena.figueroa@pharmaxis.com.au
  

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