Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental |
| |
| 2 | Active Comparator | DNase daily for 12 weeks |
|
| 3 | Other | combination |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mannitol | Drug | 400mg BD for 12 weeks |
| |
| mannitol + pulmozyme |
| Measure | Description | Time Frame |
|---|---|---|
| FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase | 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| to compare mannitol to rhDNase on FVC | 12 weeks | |
| to assess whether the effects of mannitol are additive to rhDNase | 12 weeks | |
| to demonstrate that mannitol does not cause deterioration in airway inflammation |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Andrew Bush, FRCPCH | Royal Brompton and Harefiled NHS Trust | Principal Investigator |
| Colin Wallis, FRCPCH | Great Ormond Street Hospital for Children NHS Foundation Trust | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Great Ormond Hospital for Children | London | United Kingdom | ||||
| Royal Brompton Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 11684212 | Background | Suri R, Metcalfe C, Lees B, Grieve R, Flather M, Normand C, Thompson S, Bush A, Wallis C. Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet. 2001 Oct 20;358(9290):1316-21. doi: 10.1016/S0140-6736(01)06412-1. | |
| 10543292 | Background |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D008353 | Mannitol |
| C568813 | dornase alfa |
| ID | Term |
|---|---|
| D013402 | Sugar Alcohols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D002241 | Carbohydrates |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Drug |
combination |
|
| Dornase alpha | Drug | 2.5mg daily for 2 weeks |
|
|
| 12 weeks |
| to assess whether mannitol reduces the bacterial load in the lung | 12 weeks |
| to assess whether the effects of mannitol are beneficial to quality of life | 12 weeks |
| to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone | 12 weeks |
| London |
| United Kingdom |
| Robinson M, Daviskas E, Eberl S, Baker J, Chan HK, Anderson SD, Bye PT. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J. 1999 Sep;14(3):678-85. doi: 10.1034/j.1399-3003.1999.14c30.x. |
| 9426077 | Background | Daviskas E, Anderson SD, Brannan JD, Chan HK, Eberl S, Bautovich G. Inhalation of dry-powder mannitol increases mucociliary clearance. Eur Respir J. 1997 Nov;10(11):2449-54. doi: 10.1183/09031936.97.10112449. |
| 10351929 | Background | Daviskas E, Anderson SD, Eberl S, Chan HK, Bautovich G. Inhalation of dry powder mannitol improves clearance of mucus in patients with bronchiectasis. Am J Respir Crit Care Med. 1999 Jun;159(6):1843-8. doi: 10.1164/ajrccm.159.6.9809074. |
| 11171717 | Background | Daviskas E, Anderson SD, Eberl S, Chan HK, Young IH. The 24-h effect of mannitol on the clearance of mucus in patients with bronchiectasis. Chest. 2001 Feb;119(2):414-21. doi: 10.1378/chest.119.2.414. |
| 15691238 | Background | Daviskas E, Anderson SD, Gomes K, Briffa P, Cochrane B, Chan HK, Young IH, Rubin BK. Inhaled mannitol for the treatment of mucociliary dysfunction in patients with bronchiectasis: effect on lung function, health status and sputum. Respirology. 2005 Jan;10(1):46-56. doi: 10.1111/j.1440-1843.2005.00659.x. |
| 19996349 | Result | Minasian C, Wallis C, Metcalfe C, Bush A. Comparison of inhaled mannitol, daily rhDNase and a combination of both in children with cystic fibrosis: a randomised trial. Thorax. 2010 Jan;65(1):51-6. doi: 10.1136/thx.2009.116970. Epub 2009 Dec 8. |
| 18972410 | Result | Minasian C, Wallis C, Metcalfe C, Bush A. Bronchial provocation testing with dry powder mannitol in children with cystic fibrosis. Pediatr Pulmonol. 2008 Nov;43(11):1078-1084. doi: 10.1002/ppul.20903. |
| 33735508 | Derived | Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5. |
| 17337677 | Derived | Enderby B, Doull I. Hypertonic saline inhalation in cystic fibrosis--salt in the wound, or sweet success? Arch Dis Child. 2007 Mar;92(3):195-6. doi: 10.1136/adc.2006.094979. |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |