Novel outcome measures for clinical trials in cystic fibrosis
Summary Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measur...
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| Veröffentlicht in: | Pediatric pulmonology 2015-03, Vol.50 (3), p.302-315 |
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| container_title | Pediatric pulmonology |
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| creator | Tiddens, Harm A.W.M. Puderbach, Michael Venegas, Jose G. Ratjen, Felix Donaldson, Scott H. Davis, Stephanie D. Rowe, Steven M. Sagel, Scott D. Higgins, Mark Waltz, David A. |
| description | Summary
Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc. |
| doi_str_mv | 10.1002/ppul.23146 |
| format | Article |
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Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.</description><identifier>ISSN: 8755-6863</identifier><identifier>EISSN: 1099-0496</identifier><identifier>DOI: 10.1002/ppul.23146</identifier><identifier>PMID: 25641878</identifier><language>eng</language><publisher>United States: Blackwell Publishing Ltd</publisher><subject>CFTR activity ; cystic fibrosis ; endpoints ; imaging ; outcome measures ; Reviews ; sputum biomarkers</subject><ispartof>Pediatric pulmonology, 2015-03, Vol.50 (3), p.302-315</ispartof><rights>2014 The Authors. published by Wiley Periodicals, Inc.</rights><rights>2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.</rights><rights>2015 Wiley Periodicals, Inc.</rights><rights>2014 The Authors. published by Wiley Periodicals, Inc. 2014</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c5566-31d946e90d810d95e69fb3d3a71a89e54556e172aaf5260ef8dc21fbe39d56303</citedby><cites>FETCH-LOGICAL-c5566-31d946e90d810d95e69fb3d3a71a89e54556e172aaf5260ef8dc21fbe39d56303</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fppul.23146$$EPDF$$P50$$Gwiley$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fppul.23146$$EHTML$$P50$$Gwiley$$Hfree_for_read</linktohtml><link.rule.ids>230,314,780,784,885,1417,27924,27925,45574,45575</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/25641878$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Tiddens, Harm A.W.M.</creatorcontrib><creatorcontrib>Puderbach, Michael</creatorcontrib><creatorcontrib>Venegas, Jose G.</creatorcontrib><creatorcontrib>Ratjen, Felix</creatorcontrib><creatorcontrib>Donaldson, Scott H.</creatorcontrib><creatorcontrib>Davis, Stephanie D.</creatorcontrib><creatorcontrib>Rowe, Steven M.</creatorcontrib><creatorcontrib>Sagel, Scott D.</creatorcontrib><creatorcontrib>Higgins, Mark</creatorcontrib><creatorcontrib>Waltz, David A.</creatorcontrib><title>Novel outcome measures for clinical trials in cystic fibrosis</title><title>Pediatric pulmonology</title><addtitle>Pediatr Pulmonol</addtitle><description>Summary
Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.</description><subject>CFTR activity</subject><subject>cystic fibrosis</subject><subject>endpoints</subject><subject>imaging</subject><subject>outcome measures</subject><subject>Reviews</subject><subject>sputum biomarkers</subject><issn>8755-6863</issn><issn>1099-0496</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2015</creationdate><recordtype>article</recordtype><sourceid>24P</sourceid><sourceid>WIN</sourceid><recordid>eNp9kV1rFDEUhoModq3e-ANkwBsRpub740JBVrsKS61g8TJkM2c0NTNZk5nq_ntTt13UC69y8T7n4eS8CD0m-IRgTF9st3M8oYxweQctCDamxdzIu2ihlRCt1JIdoQelXGJcM0PuoyMqJCda6QV6eZauIDZpnnwaoBnAlTlDafqUGx_DGLyLzZSDi6UJY-N3ZQq-6cMmpxLKQ3Svrwk8unmP0cXp20_Ld-36w-r98vW69UJI2TLSGS7B4E4T3BkB0vQb1jGniNMGBK8UEEWd6wWVGHrdeUr6DTDTCckwO0av9t7tvBmg8zBO2UW7zWFweWeTC_bvZAxf7Zd0ZTmTQlFZBc9uBDl9n6FMdgjFQ4xuhDQXSzSVEitCeEWf_oNepjmP9XuWSMEN18rQSj3fU74eomToD8sQbK9bsdet2N-tVPjJn-sf0NsaKkD2wI8QYfcflT0_v1jfStv9TCgT_DzMuPzNSsWUsJ_PVvbjUq1Xp2-IVewXPlim_g</recordid><startdate>201503</startdate><enddate>201503</enddate><creator>Tiddens, Harm A.W.M.</creator><creator>Puderbach, Michael</creator><creator>Venegas, Jose G.</creator><creator>Ratjen, Felix</creator><creator>Donaldson, Scott H.</creator><creator>Davis, Stephanie D.</creator><creator>Rowe, Steven M.</creator><creator>Sagel, Scott D.</creator><creator>Higgins, Mark</creator><creator>Waltz, David A.</creator><general>Blackwell Publishing Ltd</general><general>Wiley Subscription Services, Inc</general><general>BlackWell Publishing Ltd</general><scope>BSCLL</scope><scope>24P</scope><scope>WIN</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>K9.</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>201503</creationdate><title>Novel outcome measures for clinical trials in cystic fibrosis</title><author>Tiddens, Harm A.W.M. ; Puderbach, Michael ; Venegas, Jose G. ; Ratjen, Felix ; Donaldson, Scott H. ; Davis, Stephanie D. ; Rowe, Steven M. ; Sagel, Scott D. ; Higgins, Mark ; Waltz, David A.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c5566-31d946e90d810d95e69fb3d3a71a89e54556e172aaf5260ef8dc21fbe39d56303</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2015</creationdate><topic>CFTR activity</topic><topic>cystic fibrosis</topic><topic>endpoints</topic><topic>imaging</topic><topic>outcome measures</topic><topic>Reviews</topic><topic>sputum biomarkers</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Tiddens, Harm A.W.M.</creatorcontrib><creatorcontrib>Puderbach, Michael</creatorcontrib><creatorcontrib>Venegas, Jose G.</creatorcontrib><creatorcontrib>Ratjen, Felix</creatorcontrib><creatorcontrib>Donaldson, Scott H.</creatorcontrib><creatorcontrib>Davis, Stephanie D.</creatorcontrib><creatorcontrib>Rowe, Steven M.</creatorcontrib><creatorcontrib>Sagel, Scott D.</creatorcontrib><creatorcontrib>Higgins, Mark</creatorcontrib><creatorcontrib>Waltz, David A.</creatorcontrib><collection>Istex</collection><collection>Wiley Online Library (Open Access Collection)</collection><collection>Wiley Online Library (Open Access Collection)</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Pediatric pulmonology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Tiddens, Harm A.W.M.</au><au>Puderbach, Michael</au><au>Venegas, Jose G.</au><au>Ratjen, Felix</au><au>Donaldson, Scott H.</au><au>Davis, Stephanie D.</au><au>Rowe, Steven M.</au><au>Sagel, Scott D.</au><au>Higgins, Mark</au><au>Waltz, David A.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Novel outcome measures for clinical trials in cystic fibrosis</atitle><jtitle>Pediatric pulmonology</jtitle><addtitle>Pediatr Pulmonol</addtitle><date>2015-03</date><risdate>2015</risdate><volume>50</volume><issue>3</issue><spage>302</spage><epage>315</epage><pages>302-315</pages><issn>8755-6863</issn><eissn>1099-0496</eissn><abstract>Summary
Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.</abstract><cop>United States</cop><pub>Blackwell Publishing Ltd</pub><pmid>25641878</pmid><doi>10.1002/ppul.23146</doi><tpages>14</tpages><oa>free_for_read</oa></addata></record> |
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| language | eng |
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| subjects | CFTR activity cystic fibrosis endpoints imaging outcome measures Reviews sputum biomarkers |
| title | Novel outcome measures for clinical trials in cystic fibrosis |
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