Novel end points for clinical trials in young children with cystic fibrosis
Cystic fibrosis (CF) lung disease commences early in the disease progression and is the most common cause of mortality. While new CF disease-modifying agents are currently undergoing clinical trial evaluation, the implementation of such trials in young children is limited by the lack of age-appropri...
| Main Authors: | , , , , |
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| Format: | Journal Article |
| Published: |
Expert Reviews Ltd
2013
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| Online Access: | http://hdl.handle.net/20.500.11937/54974 |
| _version_ | 1848759512106795008 |
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| author | Simpson, S. Mott, L. Esther, C. Stick, S. Hall, Graham |
| author_facet | Simpson, S. Mott, L. Esther, C. Stick, S. Hall, Graham |
| author_sort | Simpson, S. |
| building | Curtin Institutional Repository |
| collection | Online Access |
| description | Cystic fibrosis (CF) lung disease commences early in the disease progression and is the most common cause of mortality. While new CF disease-modifying agents are currently undergoing clinical trial evaluation, the implementation of such trials in young children is limited by the lack of age-appropriate clinical trial end points. Advances in infant and preschool lung function testing, imaging of the chest and the development of biochemical biomarkers have led to increased possibility of quantifying mild lung disease in young children with CF and objectively monitoring disease progression over the course of an intervention. Despite this, further standardization and development of these techniques is required to provide robust objective measures for clinical trials in this age group. |
| first_indexed | 2025-11-14T10:01:03Z |
| format | Journal Article |
| id | curtin-20.500.11937-54974 |
| institution | Curtin University Malaysia |
| institution_category | Local University |
| last_indexed | 2025-11-14T10:01:03Z |
| publishDate | 2013 |
| publisher | Expert Reviews Ltd |
| recordtype | eprints |
| repository_type | Digital Repository |
| spelling | curtin-20.500.11937-549742018-03-29T09:09:37Z Novel end points for clinical trials in young children with cystic fibrosis Simpson, S. Mott, L. Esther, C. Stick, S. Hall, Graham Cystic fibrosis (CF) lung disease commences early in the disease progression and is the most common cause of mortality. While new CF disease-modifying agents are currently undergoing clinical trial evaluation, the implementation of such trials in young children is limited by the lack of age-appropriate clinical trial end points. Advances in infant and preschool lung function testing, imaging of the chest and the development of biochemical biomarkers have led to increased possibility of quantifying mild lung disease in young children with CF and objectively monitoring disease progression over the course of an intervention. Despite this, further standardization and development of these techniques is required to provide robust objective measures for clinical trials in this age group. 2013 Journal Article http://hdl.handle.net/20.500.11937/54974 10.1586/ers.13.25 Expert Reviews Ltd restricted |
| spellingShingle | Simpson, S. Mott, L. Esther, C. Stick, S. Hall, Graham Novel end points for clinical trials in young children with cystic fibrosis |
| title | Novel end points for clinical trials in young children with cystic fibrosis |
| title_full | Novel end points for clinical trials in young children with cystic fibrosis |
| title_fullStr | Novel end points for clinical trials in young children with cystic fibrosis |
| title_full_unstemmed | Novel end points for clinical trials in young children with cystic fibrosis |
| title_short | Novel end points for clinical trials in young children with cystic fibrosis |
| title_sort | novel end points for clinical trials in young children with cystic fibrosis |
| url | http://hdl.handle.net/20.500.11937/54974 |