Mannitol Dose Response Study in Cystic Fibrosis

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00251056
• Other study ID #: DPM-CF-202
• Status: Completed
• Phase: Phase 2
• First received: June 30, 2005
• Last updated: August 27, 2008
• Start date: October 2005
• Est. completion date: August 2008

Study information

• Verified date: August 2008
• Source: Pharmaxis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health CanadaArgentina: Human Research Bioethics Committee
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 48
• Est. completion date: August 2008
• Est. primary completion date: August 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 7 Years and older

Eligibility

Inclusion Criteria:

• Diagnosis of cystic fibrosis (sweat test/genotype)

• 7 years or older

• FEV1 between 40% and 90% of predicted for height, age and gender.

• Able to perform acceptable-quality spirometry

• 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

• Currently active asthma

• Subjects colonized 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 (e.g. 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

Canada:

• Concurrent use of other pharmacological mucolytic agents other than Pulmozyme

Argentina:

• Concurrent use of other pharmacological mucolytic agents including Pulmozyme

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• mannitol
120mg BD
• mannitol
40 mg BD
• mannitol
240mg BD
• mannitol
400mg BD

Locations

Country	Name	City	State
Argentina	Hospital Interzonal Especializado Materno Infantil (HIEMI)	Buenos Aires
Argentina	Hospital General de Niños	Caba
Argentina	Hospital de Niños Superiora Sor María Ludovica	La Plata	Buenos Aires
Argentina	Hospital Pediatrico Dr Humberto J Notti	Mendoza
Argentina	Hospital Pediatrico	Resistencia	Chaco
Canada	Queen Elizabeth II Health Sciences Centre	Halifax	Nova Scotia
Canada	Hamilton Health Sciences Corporation	Hamilton	Ontario
Canada	Janeway Children's Health and Rehabilitation Center	St Johns	Newfoundland and Labrador
Canada	St Michaels Hospital	Toronto	Ontario
Canada	The Hospital for Sick Children	Toronto	Ontario
Canada	BC Children's Hospital	Vancouver	British Columbia
Canada	St Pauls Hospital	Vancouver	British Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Pharmaxis

Countries where clinical trial is conducted

Argentina,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	FEV1		2 weeks	No
Primary	FVC		2 weeks	No
Secondary	other measures of lung function		various	No
Secondary	QOL		2 weeks	No
Secondary	sputum microbiology		2 weeks	Yes
Secondary	safety		2 weeks	Yes
Secondary	sputum clearance and cough		2 weeks	No
Secondary	respiratory symptoms		2 weeks	No