A cross-over comparative study of inhaled mannitol, alone and in combination with daily rhDNase, in children with cystic fibrosis - Inhaled Mannitol in Cystic Fibrosis

Phase 1

• Conditions: Cystic fibrosis (CF)

• Registration Number: EUCTR2004-001888-21-GB
• Lead Sponsor: Pharmaxis Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2005-02-22
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 42

Inclusion Criteria

1. Confirmed diagnosis of cystic fibrosis (sweat test or genotype)
2. Aged between 5 and 18 years
3. Have Forced Expiratory Volume in one second (FEV1) <70% of predicted for height, age and gender OR currently taking rhDNase
4. Able to perform acceptable-quality spirometry
5. Likely to co-operate, take medication as prescribed, and attend the clinic at the appointed times during the study
6. Are capable of and have given informed consent to participate in this study in accordance with local regulations
7. Clinically stable in the week up to study entry
8. No additional antibiotics for a period of 14 days before study entry (routine antibiotics permitted)

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Investigators, site personnel directly affiliated with this study, and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted.
2. Subjects with currently active asthma
3. Colonised with Burkholderia cepacia
4. Considered terminally ill” or listed for transplantation
5. Concurrent illnesses - Cor pulmonale, portal hypertension, varices
6. Requiring home oxygen or assisted ventilation
7. Significant episode of hemoptysis (>60 mL) in the previous 12 months
8. Heart attack or stroke in last 3 months
9. Known aortic aneurysm
10. Subjects who are breast feeding or pregnant.
11. At risk females unwilling to use appropriate contraception to prevent pregnancy during the course of the study
12. Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
13. Known intolerance to mannitol, rhDNase or ß agonists.
14. Uncontrolled hypertension, systolic BP > 200, or diastolic BP> than 100

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> Main Objective: - To compare and contrast the effect on Forced Expiratory Volume in one second(FEV1) using a 12 week treatment regimen of:<br> 1) mannitol to rhDNase, and<br> 2) mannitol + rhDNase to rhDNase<br><br> ;<br> Secondary Objective: - To compare mannitol to rhDNase on Forced Vital Capacity (FVC)<br> - To demonstrate that the effects of mannitol are additive to rhDNase<br> - To demonstrate that mannitol does not cause deterioration in bronchial hyper-reactvity (BHR) or airway inflammation<br> - To demonstrate that mannitol does not increase the bacterial load in the lung<br> - To demonstrate that the effects of mannitol are beneficial to quality of life<br> - To demonstrate that mannitol is a cost effective alternative to rhDNase<br> ;Primary end point(s): Forced Expiratory Volume in one second (FEV1)